Pediatrics

I think the question was intended as polite, dinner party chit chat ... maybe an attempt by a gracious hostess to make sure everyone was engaged in conversation.

“So what pediatric infectious disease should parents be most worried about?” she asked me.

I’ll admit that a couple of perfectly respectable and noncontroversial possibilities crossed my mind before I answered.

Acute flaccid myelitis? Measles?

When I replied, “gonorrhea,” conversation at the table pretty much stopped.

Let me explain. Acute flaccid myelitis is a polio-like neurologic condition that has been grabbing headlines. Yes, it is concerning that most cases have occurred in children and some affected children are left with long-term deficits. Technically though, AFM is a neurologic rather than an infectious disease. When cases occur, we suspect a viral infection but according to the Centers for Disease Control and Prevention, no pathogen has been consistently identified from the spinal fluid of infected patients. From August 2014 to September 2018, the CDC received information on 368 confirmed cases, so AFM fortunately is still rare.

News reports describe measles outbreaks raging in Europe – more than 41,000 cases so far this year, and 40 deaths – and warn that the United States could be next. But let’s be honest: We have a safe and effective vaccine for measles and outbreaks like this don’t happen when individuals are appropriately immunized. Parents, immunize your children. If you are lucky enough to be traveling to Europe with your baby, remember that MMR vaccine is indicated for 6- to 11-month olds, but it doesn’t count in the 2-dose series.

But gonorrhea?

In 2017, the World Health Organization included Neisseria gonorrhoeae on its list of bacteria that pose the greatest threat to human health and for which new antibiotics are urgently needed. The popular media are calling N. gonorrhoeae one of the new “superbugs.” Globally, patients are being diagnosed with strains of gonorrhea that are resistant to all commonly used antibiotics. As reported during IDWeek 2018 this October, patients also are being diagnosed in the United States.

CDC

Sancta St. Cyr, MD, of the Centers for Disease Control and Prevention, and her colleagues reported data from the Gonococcal Isolate Surveillance Project (GISP) and trends in multidrug resistant (MDR) and extensively-drug resistant (XDR) gonorrhea in the United States. A gonococcal isolate with resistance or elevated minimum inhibitory concentrations (MIC) to greater than or equal to two classes of antimicrobials is classified as MDR and an isolate with elevated MICs to greater than or equal to three classes of antimicrobials is classified as XDR. The MIC is the lowest antimicrobial concentration that inhibits growth of bacteria in the laboratory and rising MICs – evidence that higher levels of an antibiotic are needed to stop bacterial growth – can be an early indicator that resistance is emerging.

More than 150,000 gonococcal isolates were tested between 1987 and 2016. The first isolates with elevated MICs to cephalosporins and macrolides were identified in 1998, and since 2011, MDR resistance rates have hovered around 1%. In 2016, the rate was 1.1%, down from 1.3% in 2011. A single XDR isolate with resistance to fluoroquinolones with elevated MICs to both cephalosporins and macrolides was identified in 2011.

One could look at these data and ask if this is a “glass half full or half empty” situation, but I propose that clinicians and public health officials should not look at these data and be reassured that rates of MDR-gonorrhea remained stable between 2010 and 2016. According to a recent surveillance report released by the CDC, the absolute number of cases of gonorrhea has continued to rise. In 2017, there were 555,608 cases reported in the United States, a 67% increase since 2013. If we assume that rates of resistance in 2017 were similar to those in 2016, that’s more than 5,000 cases of MDR-gonorrhea in a single year.

“That’s bad,” one of my dining companions agreed. “But is gonorrhea really a pediatric issue?”

To answer that question, we just have to look at the numbers. According to the 2017 Youth Risk Behavior Survey, the percentage of high school students who had ever had sex was approximately 40% and about 10% of students had four or more lifetime partners. More than 45% of sexually active students denied the use of a condom during the last sexual intercourse. Certainly, that puts many teenagers at risk for sexually transmitted infections (STIs). Perhaps it shouldn’t be surprising that public health authorities report that half of all new STIs occur in individuals aged 15-24 years. Moreover, 25% of sexually active adolescent girls contract at least one STI.

Gonorrhea is the second most commonly reported notifiable disease in the United States, and according to the CDC, rates of disease in 2017 were highest among adolescents and young adults. In females specifically, the highest rates of gonorrhea were observed among those aged 20-24 years (684.8 cases per 100,000 females) and 15-19 years (557.4 cases per 100,000 females).

It makes sense that pediatricians and parents advocate for making the reduction of gonorrhea transmission rates a public health priority. We also need to recognize that prompt diagnosis and appropriate treatment are critical. Since 2015, dual therapy with ceftriaxone and azithromycin is the only CDC-recommended treatment for gonorrhea.

At that dinner party, my closest friend, who also happens to be a pediatrician, rolled her eyes and shot me look that I’m sure meant, “Nobody really wants to talk about gonorrhea over dessert.” Still, because she is a good friend she said, “So basically you’re saying that millions of teenagers are getting infected with a form of gonorrhea that takes at least two antibiotics to treat, and if this keeps up, we may see kids with untreatable infection. Now that is scary.”

I kept quiet after that but I wanted to mention that in 2017, less than 85% of patients diagnosed with gonorrhea at selected surveillance sites received the recommended treatment with two antibiotics. Patients with inadequately treated gonorrhea are at risk for a host of sequelae. Women can develop pelvic inflammatory disease, abscesses, chronic pelvic pain, and damage of the fallopian tubes that can lead to infertility. Men can develop epididymitis, which occasionally results in infertility. Rarely, N. gonorrhoeae can spread to the blood and cause life-threatening infection. Of course, patients who aren’t treated appropriately may continue to spread the bacteria. Scary? You bet.

For pediatricians who need a refresher course in the treatment of STIs, there are free resources available. The CDC’s 2015 STD Treatment Guidelines are available in a free app; the app contains a nice refresher on taking a sexual history. There also is a print version, wall chart, and pocket guide. Providers also may want to check out the National STD Curriculum offered by the University of Washington STD Prevention Training Center and the University of Washington. Visit https://www.std.uw.edu/.
 

Dr. Bryant is a pediatrician specializing in infectious diseases at the University of Louisville (Ky.) and Norton Children’s Hospital, also in Louisville. She said she had no relevant financial disclosures. Email her at pdnews@mdedge.com.

I think the question was intended as polite, dinner party chit chat ... maybe an attempt by a gracious hostess to make sure everyone was engaged in conversation.

“So what pediatric infectious disease should parents be most worried about?” she asked me.

I’ll admit that a couple of perfectly respectable and noncontroversial possibilities crossed my mind before I answered.

Acute flaccid myelitis? Measles?

When I replied, “gonorrhea,” conversation at the table pretty much stopped.

Let me explain. Acute flaccid myelitis is a polio-like neurologic condition that has been grabbing headlines. Yes, it is concerning that most cases have occurred in children and some affected children are left with long-term deficits. Technically though, AFM is a neurologic rather than an infectious disease. When cases occur, we suspect a viral infection but according to the Centers for Disease Control and Prevention, no pathogen has been consistently identified from the spinal fluid of infected patients. From August 2014 to September 2018, the CDC received information on 368 confirmed cases, so AFM fortunately is still rare.

News reports describe measles outbreaks raging in Europe – more than 41,000 cases so far this year, and 40 deaths – and warn that the United States could be next. But let’s be honest: We have a safe and effective vaccine for measles and outbreaks like this don’t happen when individuals are appropriately immunized. Parents, immunize your children. If you are lucky enough to be traveling to Europe with your baby, remember that MMR vaccine is indicated for 6- to 11-month olds, but it doesn’t count in the 2-dose series.

But gonorrhea?

In 2017, the World Health Organization included Neisseria gonorrhoeae on its list of bacteria that pose the greatest threat to human health and for which new antibiotics are urgently needed. The popular media are calling N. gonorrhoeae one of the new “superbugs.” Globally, patients are being diagnosed with strains of gonorrhea that are resistant to all commonly used antibiotics. As reported during IDWeek 2018 this October, patients also are being diagnosed in the United States.

CDC

Sancta St. Cyr, MD, of the Centers for Disease Control and Prevention, and her colleagues reported data from the Gonococcal Isolate Surveillance Project (GISP) and trends in multidrug resistant (MDR) and extensively-drug resistant (XDR) gonorrhea in the United States. A gonococcal isolate with resistance or elevated minimum inhibitory concentrations (MIC) to greater than or equal to two classes of antimicrobials is classified as MDR and an isolate with elevated MICs to greater than or equal to three classes of antimicrobials is classified as XDR. The MIC is the lowest antimicrobial concentration that inhibits growth of bacteria in the laboratory and rising MICs – evidence that higher levels of an antibiotic are needed to stop bacterial growth – can be an early indicator that resistance is emerging.

More than 150,000 gonococcal isolates were tested between 1987 and 2016. The first isolates with elevated MICs to cephalosporins and macrolides were identified in 1998, and since 2011, MDR resistance rates have hovered around 1%. In 2016, the rate was 1.1%, down from 1.3% in 2011. A single XDR isolate with resistance to fluoroquinolones with elevated MICs to both cephalosporins and macrolides was identified in 2011.

One could look at these data and ask if this is a “glass half full or half empty” situation, but I propose that clinicians and public health officials should not look at these data and be reassured that rates of MDR-gonorrhea remained stable between 2010 and 2016. According to a recent surveillance report released by the CDC, the absolute number of cases of gonorrhea has continued to rise. In 2017, there were 555,608 cases reported in the United States, a 67% increase since 2013. If we assume that rates of resistance in 2017 were similar to those in 2016, that’s more than 5,000 cases of MDR-gonorrhea in a single year.

“That’s bad,” one of my dining companions agreed. “But is gonorrhea really a pediatric issue?”

To answer that question, we just have to look at the numbers. According to the 2017 Youth Risk Behavior Survey, the percentage of high school students who had ever had sex was approximately 40% and about 10% of students had four or more lifetime partners. More than 45% of sexually active students denied the use of a condom during the last sexual intercourse. Certainly, that puts many teenagers at risk for sexually transmitted infections (STIs). Perhaps it shouldn’t be surprising that public health authorities report that half of all new STIs occur in individuals aged 15-24 years. Moreover, 25% of sexually active adolescent girls contract at least one STI.

Gonorrhea is the second most commonly reported notifiable disease in the United States, and according to the CDC, rates of disease in 2017 were highest among adolescents and young adults. In females specifically, the highest rates of gonorrhea were observed among those aged 20-24 years (684.8 cases per 100,000 females) and 15-19 years (557.4 cases per 100,000 females).

It makes sense that pediatricians and parents advocate for making the reduction of gonorrhea transmission rates a public health priority. We also need to recognize that prompt diagnosis and appropriate treatment are critical. Since 2015, dual therapy with ceftriaxone and azithromycin is the only CDC-recommended treatment for gonorrhea.

At that dinner party, my closest friend, who also happens to be a pediatrician, rolled her eyes and shot me look that I’m sure meant, “Nobody really wants to talk about gonorrhea over dessert.” Still, because she is a good friend she said, “So basically you’re saying that millions of teenagers are getting infected with a form of gonorrhea that takes at least two antibiotics to treat, and if this keeps up, we may see kids with untreatable infection. Now that is scary.”

I kept quiet after that but I wanted to mention that in 2017, less than 85% of patients diagnosed with gonorrhea at selected surveillance sites received the recommended treatment with two antibiotics. Patients with inadequately treated gonorrhea are at risk for a host of sequelae. Women can develop pelvic inflammatory disease, abscesses, chronic pelvic pain, and damage of the fallopian tubes that can lead to infertility. Men can develop epididymitis, which occasionally results in infertility. Rarely, N. gonorrhoeae can spread to the blood and cause life-threatening infection. Of course, patients who aren’t treated appropriately may continue to spread the bacteria. Scary? You bet.

For pediatricians who need a refresher course in the treatment of STIs, there are free resources available. The CDC’s 2015 STD Treatment Guidelines are available in a free app; the app contains a nice refresher on taking a sexual history. There also is a print version, wall chart, and pocket guide. Providers also may want to check out the National STD Curriculum offered by the University of Washington STD Prevention Training Center and the University of Washington. Visit https://www.std.uw.edu/.
 

Dr. Bryant is a pediatrician specializing in infectious diseases at the University of Louisville (Ky.) and Norton Children’s Hospital, also in Louisville. She said she had no relevant financial disclosures. Email her at pdnews@mdedge.com.

I think the question was intended as polite, dinner party chit chat ... maybe an attempt by a gracious hostess to make sure everyone was engaged in conversation.

“So what pediatric infectious disease should parents be most worried about?” she asked me.

I’ll admit that a couple of perfectly respectable and noncontroversial possibilities crossed my mind before I answered.

Acute flaccid myelitis? Measles?

When I replied, “gonorrhea,” conversation at the table pretty much stopped.

Let me explain. Acute flaccid myelitis is a polio-like neurologic condition that has been grabbing headlines. Yes, it is concerning that most cases have occurred in children and some affected children are left with long-term deficits. Technically though, AFM is a neurologic rather than an infectious disease. When cases occur, we suspect a viral infection but according to the Centers for Disease Control and Prevention, no pathogen has been consistently identified from the spinal fluid of infected patients. From August 2014 to September 2018, the CDC received information on 368 confirmed cases, so AFM fortunately is still rare.

News reports describe measles outbreaks raging in Europe – more than 41,000 cases so far this year, and 40 deaths – and warn that the United States could be next. But let’s be honest: We have a safe and effective vaccine for measles and outbreaks like this don’t happen when individuals are appropriately immunized. Parents, immunize your children. If you are lucky enough to be traveling to Europe with your baby, remember that MMR vaccine is indicated for 6- to 11-month olds, but it doesn’t count in the 2-dose series.

But gonorrhea?

In 2017, the World Health Organization included Neisseria gonorrhoeae on its list of bacteria that pose the greatest threat to human health and for which new antibiotics are urgently needed. The popular media are calling N. gonorrhoeae one of the new “superbugs.” Globally, patients are being diagnosed with strains of gonorrhea that are resistant to all commonly used antibiotics. As reported during IDWeek 2018 this October, patients also are being diagnosed in the United States.

CDC

Sancta St. Cyr, MD, of the Centers for Disease Control and Prevention, and her colleagues reported data from the Gonococcal Isolate Surveillance Project (GISP) and trends in multidrug resistant (MDR) and extensively-drug resistant (XDR) gonorrhea in the United States. A gonococcal isolate with resistance or elevated minimum inhibitory concentrations (MIC) to greater than or equal to two classes of antimicrobials is classified as MDR and an isolate with elevated MICs to greater than or equal to three classes of antimicrobials is classified as XDR. The MIC is the lowest antimicrobial concentration that inhibits growth of bacteria in the laboratory and rising MICs – evidence that higher levels of an antibiotic are needed to stop bacterial growth – can be an early indicator that resistance is emerging.

More than 150,000 gonococcal isolates were tested between 1987 and 2016. The first isolates with elevated MICs to cephalosporins and macrolides were identified in 1998, and since 2011, MDR resistance rates have hovered around 1%. In 2016, the rate was 1.1%, down from 1.3% in 2011. A single XDR isolate with resistance to fluoroquinolones with elevated MICs to both cephalosporins and macrolides was identified in 2011.

One could look at these data and ask if this is a “glass half full or half empty” situation, but I propose that clinicians and public health officials should not look at these data and be reassured that rates of MDR-gonorrhea remained stable between 2010 and 2016. According to a recent surveillance report released by the CDC, the absolute number of cases of gonorrhea has continued to rise. In 2017, there were 555,608 cases reported in the United States, a 67% increase since 2013. If we assume that rates of resistance in 2017 were similar to those in 2016, that’s more than 5,000 cases of MDR-gonorrhea in a single year.

“That’s bad,” one of my dining companions agreed. “But is gonorrhea really a pediatric issue?”

To answer that question, we just have to look at the numbers. According to the 2017 Youth Risk Behavior Survey, the percentage of high school students who had ever had sex was approximately 40% and about 10% of students had four or more lifetime partners. More than 45% of sexually active students denied the use of a condom during the last sexual intercourse. Certainly, that puts many teenagers at risk for sexually transmitted infections (STIs). Perhaps it shouldn’t be surprising that public health authorities report that half of all new STIs occur in individuals aged 15-24 years. Moreover, 25% of sexually active adolescent girls contract at least one STI.

Gonorrhea is the second most commonly reported notifiable disease in the United States, and according to the CDC, rates of disease in 2017 were highest among adolescents and young adults. In females specifically, the highest rates of gonorrhea were observed among those aged 20-24 years (684.8 cases per 100,000 females) and 15-19 years (557.4 cases per 100,000 females).

It makes sense that pediatricians and parents advocate for making the reduction of gonorrhea transmission rates a public health priority. We also need to recognize that prompt diagnosis and appropriate treatment are critical. Since 2015, dual therapy with ceftriaxone and azithromycin is the only CDC-recommended treatment for gonorrhea.

At that dinner party, my closest friend, who also happens to be a pediatrician, rolled her eyes and shot me look that I’m sure meant, “Nobody really wants to talk about gonorrhea over dessert.” Still, because she is a good friend she said, “So basically you’re saying that millions of teenagers are getting infected with a form of gonorrhea that takes at least two antibiotics to treat, and if this keeps up, we may see kids with untreatable infection. Now that is scary.”

I kept quiet after that but I wanted to mention that in 2017, less than 85% of patients diagnosed with gonorrhea at selected surveillance sites received the recommended treatment with two antibiotics. Patients with inadequately treated gonorrhea are at risk for a host of sequelae. Women can develop pelvic inflammatory disease, abscesses, chronic pelvic pain, and damage of the fallopian tubes that can lead to infertility. Men can develop epididymitis, which occasionally results in infertility. Rarely, N. gonorrhoeae can spread to the blood and cause life-threatening infection. Of course, patients who aren’t treated appropriately may continue to spread the bacteria. Scary? You bet.

For pediatricians who need a refresher course in the treatment of STIs, there are free resources available. The CDC’s 2015 STD Treatment Guidelines are available in a free app; the app contains a nice refresher on taking a sexual history. There also is a print version, wall chart, and pocket guide. Providers also may want to check out the National STD Curriculum offered by the University of Washington STD Prevention Training Center and the University of Washington. Visit https://www.std.uw.edu/.
 

Dr. Bryant is a pediatrician specializing in infectious diseases at the University of Louisville (Ky.) and Norton Children’s Hospital, also in Louisville. She said she had no relevant financial disclosures. Email her at pdnews@mdedge.com.

Clinicians consulting the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices vaccination schedules for children, adolescents, and adults in 2019 will find a simpler design and more useful product, according to David Kim, MD, of the Immunization Services Division of the Centers for Disease Control and Prevention, Atlanta.

In a single vote to cover both adult and child/adolescent schedules, the committee voted unanimously in favor of a redesign of the schedules and several clinical updates.

In 2016, the working group for vaccination schedules conducted an ad hoc evaluation of the adult schedule to assess its usability, Dr. Kim said at a meeting of the CDC’s ACIP.

The design of the adult schedule was fully evaluated in 2018 via a three-step process – interviews with 48 health care providers, a redesign of the schedule, and a survey after the redesign. Design changes to the child/adolescent schedule were harmonized with the adult schedule, Dr. Kim explained.

The adult vaccination schedule itself includes several updates in ACIP recommendations in addition to the aesthetic design changes.

The 2019 Adult Immunization Schedule includes the option of the live attenuated influenza vaccine (LAIV) for influenza, the addition of homelessness as an indication for hepatitis A vaccination, and the use of CpG-adjuvanted hepatitis B vaccine, Dr. Kim said.

The additions to the 2019 Child and Adolescent Immunization Schedule are the optional use of the LAIV for influenza, the addition of homelessness as an indication for hepatitis A vaccination, the use of CpG-adjuvanted hepatitis B vaccine (a cytosine phosphoguanosine oligodeoxynucleotide adjuvant), and the addition of the Tdap vaccination of individuals who received Tdap at age 7-10 years.

Some of the key design changes include the use of bright purple on the child/adolescent schedule to more easily distinguish it from the adult version, said Dr. Kim.

Other changes to both schedules include shorter titles, lists of vaccines and trade names, and compartmentalized information for easier reference. Figures have been replaced by tables, and footnotes are simply “Notes” at the end of the schedule, compartmentalized for easier reading, he said. In addition, the schedules include resources for vaccination in outbreak situations and a section on how to report vaccine preventable disease outbreaks.

The ACIP committee members had no relevant financial conflicts to disclose.

Clinicians consulting the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices vaccination schedules for children, adolescents, and adults in 2019 will find a simpler design and more useful product, according to David Kim, MD, of the Immunization Services Division of the Centers for Disease Control and Prevention, Atlanta.

In a single vote to cover both adult and child/adolescent schedules, the committee voted unanimously in favor of a redesign of the schedules and several clinical updates.

In 2016, the working group for vaccination schedules conducted an ad hoc evaluation of the adult schedule to assess its usability, Dr. Kim said at a meeting of the CDC’s ACIP.

The design of the adult schedule was fully evaluated in 2018 via a three-step process – interviews with 48 health care providers, a redesign of the schedule, and a survey after the redesign. Design changes to the child/adolescent schedule were harmonized with the adult schedule, Dr. Kim explained.

The adult vaccination schedule itself includes several updates in ACIP recommendations in addition to the aesthetic design changes.

The 2019 Adult Immunization Schedule includes the option of the live attenuated influenza vaccine (LAIV) for influenza, the addition of homelessness as an indication for hepatitis A vaccination, and the use of CpG-adjuvanted hepatitis B vaccine, Dr. Kim said.

The additions to the 2019 Child and Adolescent Immunization Schedule are the optional use of the LAIV for influenza, the addition of homelessness as an indication for hepatitis A vaccination, the use of CpG-adjuvanted hepatitis B vaccine (a cytosine phosphoguanosine oligodeoxynucleotide adjuvant), and the addition of the Tdap vaccination of individuals who received Tdap at age 7-10 years.

Some of the key design changes include the use of bright purple on the child/adolescent schedule to more easily distinguish it from the adult version, said Dr. Kim.

Other changes to both schedules include shorter titles, lists of vaccines and trade names, and compartmentalized information for easier reference. Figures have been replaced by tables, and footnotes are simply “Notes” at the end of the schedule, compartmentalized for easier reading, he said. In addition, the schedules include resources for vaccination in outbreak situations and a section on how to report vaccine preventable disease outbreaks.

The ACIP committee members had no relevant financial conflicts to disclose.

Clinicians consulting the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices vaccination schedules for children, adolescents, and adults in 2019 will find a simpler design and more useful product, according to David Kim, MD, of the Immunization Services Division of the Centers for Disease Control and Prevention, Atlanta.

In a single vote to cover both adult and child/adolescent schedules, the committee voted unanimously in favor of a redesign of the schedules and several clinical updates.

In 2016, the working group for vaccination schedules conducted an ad hoc evaluation of the adult schedule to assess its usability, Dr. Kim said at a meeting of the CDC’s ACIP.

The design of the adult schedule was fully evaluated in 2018 via a three-step process – interviews with 48 health care providers, a redesign of the schedule, and a survey after the redesign. Design changes to the child/adolescent schedule were harmonized with the adult schedule, Dr. Kim explained.

The adult vaccination schedule itself includes several updates in ACIP recommendations in addition to the aesthetic design changes.

The 2019 Adult Immunization Schedule includes the option of the live attenuated influenza vaccine (LAIV) for influenza, the addition of homelessness as an indication for hepatitis A vaccination, and the use of CpG-adjuvanted hepatitis B vaccine, Dr. Kim said.

The additions to the 2019 Child and Adolescent Immunization Schedule are the optional use of the LAIV for influenza, the addition of homelessness as an indication for hepatitis A vaccination, the use of CpG-adjuvanted hepatitis B vaccine (a cytosine phosphoguanosine oligodeoxynucleotide adjuvant), and the addition of the Tdap vaccination of individuals who received Tdap at age 7-10 years.

Some of the key design changes include the use of bright purple on the child/adolescent schedule to more easily distinguish it from the adult version, said Dr. Kim.

Other changes to both schedules include shorter titles, lists of vaccines and trade names, and compartmentalized information for easier reference. Figures have been replaced by tables, and footnotes are simply “Notes” at the end of the schedule, compartmentalized for easier reading, he said. In addition, the schedules include resources for vaccination in outbreak situations and a section on how to report vaccine preventable disease outbreaks.

The ACIP committee members had no relevant financial conflicts to disclose.

SAN ANTONIO – Obstructive sleep apnea (OSA) in children is associated with an abnormal metabolic profile, but not with body mass index (BMI), according to new research.

Tara Haelle/MDedge News

“Screening for metabolic dysfunction in obese children with obstructive sleep apnea can help identify those at risk for cardiovascular complications,” Kanika Mathur, MD, of the Albert Einstein College of Medicine and the Children’s Hospital at Montefiore, both in New York, told attendees at the annual meeting of the American College of Chest Physicians. Dr. Mathur explained that no consensus currently exists regarding routine cardiac evaluation of children with OSA.

“The American Academy of Pediatrics does not mention any sort of cardiac evaluation in children with OSA while the most recent guidelines from the American Heart Association and the American Thoracic Society recommend echocardiographic evaluation in children with severe obstructive sleep apnea, specifically to evaluate for pulmonary hypertension and right ventricular dysfunction,” Dr. Mathur told attendees.

OSA’s association with obesity, diabetes, and hypertension is well established in adults. It is an independent risk factor for coronary artery disease, heart failure, stroke and atrial fibrillation, and research has suggested OSA treatment can reduce cardiovascular risk in adults, Dr. Mathur explained, but little data on children exist. She and her colleagues set out to understand the relationship of OSA in children with various measures of cardiovascular and metabolic health.

“Despite similar degrees of obesity and systemic blood pressure, pediatric patients with OSA had significantly higher diastolic blood pressure, heart rate, and abnormal metabolic profile, including elevated alanine transaminase, aspartate transaminase, triglycerides and hemoglobin A_1c,” they found.

Their study included patients aged 3-21 years with a BMI of at least the 95th percentile who had undergone sleep study and an echocardiogram at the Children’s Hospital at Montefiore between November 2016 and November 2017.

They excluded those with comorbidities related to cardiovascular morbidity: heart disease, neuromuscular disease, sickle cell disease, rheumatologic diseases, significant cranial facial abnormalities, tracheostomy, and any lung disease. However, 7% of the patients had trisomy 21.

Among the 81 children who met their criteria, 37 were male and 44 were female, with an average age of 14 years old and a mean BMI of 39.4 kg/m² (mean BMI z score of 2.22). Most of the patients (53.1%) had severe OSA (apnea-hypopnea index of at least 10), 21% had moderate OSA (AHI 5-9.9), 12.3% had mild OSA (AHI 2-4.9), and 13.6% did not have OSA. The median AHI of the children was 10.3.

Among all the children, “about half had elevated systolic blood pressure, which is already a risk factor for cardiovascular morbidity,” Mathur reported.

BMI, BMI z score, systolic blood pressure z score, oxygen saturation and cholesterol (overall and both HDL and LDL cholesterol levels) did not significantly differ between children who had OSA and those who did not, but diastolic blood pressure and heart rate did. Those with OSA had a diastolic blood pressure of 65 mm Hg, compared with 58 mm Hg without OSA (P = .008). Heart rate was 89 bpm in the children with OSA, compared with 78 bpm in those without (P = .004).

The children with OSA also showed higher mean levels of several other metabolic biomarkers:

• Alanine transaminase: 26 U/L with OSA vs. 18 U/L without (P = .01).
• Aspartate transaminase: 23 U/L with OSA vs. 18 U/L without (P = .03).
• Triglycerides: 138 mg/dL with OSA vs 84 mg/dL without (P = .004).
• Hemoglobin A_1c: 6.2% with OSA vs. 5.4% without (P = .002).

Children with and without OSA did not have any significant differences in left atrial indexed volume, left ventricular volume, left ventricular ejection fraction, or left ventricular mass (measured by M-mode or 5/6 area length formula). Though research has shown these measures to differ in adults with and without OSA, evidence on echocardiographic changes in children has been conflicting, Dr. Mathur noted.

The researchers also conducted subanalyses according to OSA severity, but BMI, BMI Z-score, systolic or diastolic blood pressure Z-score, heart rate and oxygen saturation did not differ between those with mild OSA vs those with moderate or severe OSA. No differences in echocardiographic measurements existed between these subgroups, either.

However, children with moderate to severe OSA did have higher alanine transaminase (27 U/L with moderate to severe vs. 17 U/L with mild OSA; P = .005) and higher triglycerides (148 vs 74; P = .001).

“Certainly we need further evaluation to see the efficacy of obstructive sleep apnea therapies on metabolic dysfunction and whether weight loss needs to be an adjunct therapy for these patients,” Dr. Mathur told attendees. She also noted the need to define the role of echocardiography in managing children with OSA.

The study had several limitations, including its retrospective cross-sectional nature at a single center and its small sample size.

Andrew D. Bowser/MDedge News

“Additionally, we have a wide variety of ages, which could represent different pathophysiology of the associated metabolic dysfunction in these patients,” Mathur said. “There is an inherent difficulty to performing echocardiograms in a very obese population as well.”

Both the moderators of the pediatrics section, Christopher Carroll, MD, FCCP, of Connecticut Children’s Medical Center in Hartford, and Shahid Sheikh, MD, FCCP, of Nationwide Children’s Hospital in Columbus, Ohio, were impressed with the research. Dr. Carroll called it a “very elegant” study, and Dr. Sheikh noted the need for these studies in pediatrics “so that we don’t have to rely on grown-up data,” which may or may not generalize to children.

SOURCE: CHEST 2018. https://journal.chestnet.org/article/S0012-3692(18)31935-4/fulltext

SAN ANTONIO – Obstructive sleep apnea (OSA) in children is associated with an abnormal metabolic profile, but not with body mass index (BMI), according to new research.

Tara Haelle/MDedge News

“Screening for metabolic dysfunction in obese children with obstructive sleep apnea can help identify those at risk for cardiovascular complications,” Kanika Mathur, MD, of the Albert Einstein College of Medicine and the Children’s Hospital at Montefiore, both in New York, told attendees at the annual meeting of the American College of Chest Physicians. Dr. Mathur explained that no consensus currently exists regarding routine cardiac evaluation of children with OSA.

“The American Academy of Pediatrics does not mention any sort of cardiac evaluation in children with OSA while the most recent guidelines from the American Heart Association and the American Thoracic Society recommend echocardiographic evaluation in children with severe obstructive sleep apnea, specifically to evaluate for pulmonary hypertension and right ventricular dysfunction,” Dr. Mathur told attendees.

OSA’s association with obesity, diabetes, and hypertension is well established in adults. It is an independent risk factor for coronary artery disease, heart failure, stroke and atrial fibrillation, and research has suggested OSA treatment can reduce cardiovascular risk in adults, Dr. Mathur explained, but little data on children exist. She and her colleagues set out to understand the relationship of OSA in children with various measures of cardiovascular and metabolic health.

“Despite similar degrees of obesity and systemic blood pressure, pediatric patients with OSA had significantly higher diastolic blood pressure, heart rate, and abnormal metabolic profile, including elevated alanine transaminase, aspartate transaminase, triglycerides and hemoglobin A_1c,” they found.

Their study included patients aged 3-21 years with a BMI of at least the 95th percentile who had undergone sleep study and an echocardiogram at the Children’s Hospital at Montefiore between November 2016 and November 2017.

They excluded those with comorbidities related to cardiovascular morbidity: heart disease, neuromuscular disease, sickle cell disease, rheumatologic diseases, significant cranial facial abnormalities, tracheostomy, and any lung disease. However, 7% of the patients had trisomy 21.

Among the 81 children who met their criteria, 37 were male and 44 were female, with an average age of 14 years old and a mean BMI of 39.4 kg/m² (mean BMI z score of 2.22). Most of the patients (53.1%) had severe OSA (apnea-hypopnea index of at least 10), 21% had moderate OSA (AHI 5-9.9), 12.3% had mild OSA (AHI 2-4.9), and 13.6% did not have OSA. The median AHI of the children was 10.3.

Among all the children, “about half had elevated systolic blood pressure, which is already a risk factor for cardiovascular morbidity,” Mathur reported.

BMI, BMI z score, systolic blood pressure z score, oxygen saturation and cholesterol (overall and both HDL and LDL cholesterol levels) did not significantly differ between children who had OSA and those who did not, but diastolic blood pressure and heart rate did. Those with OSA had a diastolic blood pressure of 65 mm Hg, compared with 58 mm Hg without OSA (P = .008). Heart rate was 89 bpm in the children with OSA, compared with 78 bpm in those without (P = .004).

The children with OSA also showed higher mean levels of several other metabolic biomarkers:

• Alanine transaminase: 26 U/L with OSA vs. 18 U/L without (P = .01).
• Aspartate transaminase: 23 U/L with OSA vs. 18 U/L without (P = .03).
• Triglycerides: 138 mg/dL with OSA vs 84 mg/dL without (P = .004).
• Hemoglobin A_1c: 6.2% with OSA vs. 5.4% without (P = .002).

Children with and without OSA did not have any significant differences in left atrial indexed volume, left ventricular volume, left ventricular ejection fraction, or left ventricular mass (measured by M-mode or 5/6 area length formula). Though research has shown these measures to differ in adults with and without OSA, evidence on echocardiographic changes in children has been conflicting, Dr. Mathur noted.

The researchers also conducted subanalyses according to OSA severity, but BMI, BMI Z-score, systolic or diastolic blood pressure Z-score, heart rate and oxygen saturation did not differ between those with mild OSA vs those with moderate or severe OSA. No differences in echocardiographic measurements existed between these subgroups, either.

However, children with moderate to severe OSA did have higher alanine transaminase (27 U/L with moderate to severe vs. 17 U/L with mild OSA; P = .005) and higher triglycerides (148 vs 74; P = .001).

“Certainly we need further evaluation to see the efficacy of obstructive sleep apnea therapies on metabolic dysfunction and whether weight loss needs to be an adjunct therapy for these patients,” Dr. Mathur told attendees. She also noted the need to define the role of echocardiography in managing children with OSA.

The study had several limitations, including its retrospective cross-sectional nature at a single center and its small sample size.

Andrew D. Bowser/MDedge News

“Additionally, we have a wide variety of ages, which could represent different pathophysiology of the associated metabolic dysfunction in these patients,” Mathur said. “There is an inherent difficulty to performing echocardiograms in a very obese population as well.”

Both the moderators of the pediatrics section, Christopher Carroll, MD, FCCP, of Connecticut Children’s Medical Center in Hartford, and Shahid Sheikh, MD, FCCP, of Nationwide Children’s Hospital in Columbus, Ohio, were impressed with the research. Dr. Carroll called it a “very elegant” study, and Dr. Sheikh noted the need for these studies in pediatrics “so that we don’t have to rely on grown-up data,” which may or may not generalize to children.

SOURCE: CHEST 2018. https://journal.chestnet.org/article/S0012-3692(18)31935-4/fulltext

SAN ANTONIO – Obstructive sleep apnea (OSA) in children is associated with an abnormal metabolic profile, but not with body mass index (BMI), according to new research.

Tara Haelle/MDedge News

“Screening for metabolic dysfunction in obese children with obstructive sleep apnea can help identify those at risk for cardiovascular complications,” Kanika Mathur, MD, of the Albert Einstein College of Medicine and the Children’s Hospital at Montefiore, both in New York, told attendees at the annual meeting of the American College of Chest Physicians. Dr. Mathur explained that no consensus currently exists regarding routine cardiac evaluation of children with OSA.

“The American Academy of Pediatrics does not mention any sort of cardiac evaluation in children with OSA while the most recent guidelines from the American Heart Association and the American Thoracic Society recommend echocardiographic evaluation in children with severe obstructive sleep apnea, specifically to evaluate for pulmonary hypertension and right ventricular dysfunction,” Dr. Mathur told attendees.

OSA’s association with obesity, diabetes, and hypertension is well established in adults. It is an independent risk factor for coronary artery disease, heart failure, stroke and atrial fibrillation, and research has suggested OSA treatment can reduce cardiovascular risk in adults, Dr. Mathur explained, but little data on children exist. She and her colleagues set out to understand the relationship of OSA in children with various measures of cardiovascular and metabolic health.

“Despite similar degrees of obesity and systemic blood pressure, pediatric patients with OSA had significantly higher diastolic blood pressure, heart rate, and abnormal metabolic profile, including elevated alanine transaminase, aspartate transaminase, triglycerides and hemoglobin A_1c,” they found.

Their study included patients aged 3-21 years with a BMI of at least the 95th percentile who had undergone sleep study and an echocardiogram at the Children’s Hospital at Montefiore between November 2016 and November 2017.

They excluded those with comorbidities related to cardiovascular morbidity: heart disease, neuromuscular disease, sickle cell disease, rheumatologic diseases, significant cranial facial abnormalities, tracheostomy, and any lung disease. However, 7% of the patients had trisomy 21.

Among the 81 children who met their criteria, 37 were male and 44 were female, with an average age of 14 years old and a mean BMI of 39.4 kg/m² (mean BMI z score of 2.22). Most of the patients (53.1%) had severe OSA (apnea-hypopnea index of at least 10), 21% had moderate OSA (AHI 5-9.9), 12.3% had mild OSA (AHI 2-4.9), and 13.6% did not have OSA. The median AHI of the children was 10.3.

Among all the children, “about half had elevated systolic blood pressure, which is already a risk factor for cardiovascular morbidity,” Mathur reported.

BMI, BMI z score, systolic blood pressure z score, oxygen saturation and cholesterol (overall and both HDL and LDL cholesterol levels) did not significantly differ between children who had OSA and those who did not, but diastolic blood pressure and heart rate did. Those with OSA had a diastolic blood pressure of 65 mm Hg, compared with 58 mm Hg without OSA (P = .008). Heart rate was 89 bpm in the children with OSA, compared with 78 bpm in those without (P = .004).

The children with OSA also showed higher mean levels of several other metabolic biomarkers:

• Alanine transaminase: 26 U/L with OSA vs. 18 U/L without (P = .01).
• Aspartate transaminase: 23 U/L with OSA vs. 18 U/L without (P = .03).
• Triglycerides: 138 mg/dL with OSA vs 84 mg/dL without (P = .004).
• Hemoglobin A_1c: 6.2% with OSA vs. 5.4% without (P = .002).

Children with and without OSA did not have any significant differences in left atrial indexed volume, left ventricular volume, left ventricular ejection fraction, or left ventricular mass (measured by M-mode or 5/6 area length formula). Though research has shown these measures to differ in adults with and without OSA, evidence on echocardiographic changes in children has been conflicting, Dr. Mathur noted.

The researchers also conducted subanalyses according to OSA severity, but BMI, BMI Z-score, systolic or diastolic blood pressure Z-score, heart rate and oxygen saturation did not differ between those with mild OSA vs those with moderate or severe OSA. No differences in echocardiographic measurements existed between these subgroups, either.

However, children with moderate to severe OSA did have higher alanine transaminase (27 U/L with moderate to severe vs. 17 U/L with mild OSA; P = .005) and higher triglycerides (148 vs 74; P = .001).

“Certainly we need further evaluation to see the efficacy of obstructive sleep apnea therapies on metabolic dysfunction and whether weight loss needs to be an adjunct therapy for these patients,” Dr. Mathur told attendees. She also noted the need to define the role of echocardiography in managing children with OSA.

The study had several limitations, including its retrospective cross-sectional nature at a single center and its small sample size.

Andrew D. Bowser/MDedge News

“Additionally, we have a wide variety of ages, which could represent different pathophysiology of the associated metabolic dysfunction in these patients,” Mathur said. “There is an inherent difficulty to performing echocardiograms in a very obese population as well.”

Both the moderators of the pediatrics section, Christopher Carroll, MD, FCCP, of Connecticut Children’s Medical Center in Hartford, and Shahid Sheikh, MD, FCCP, of Nationwide Children’s Hospital in Columbus, Ohio, were impressed with the research. Dr. Carroll called it a “very elegant” study, and Dr. Sheikh noted the need for these studies in pediatrics “so that we don’t have to rely on grown-up data,” which may or may not generalize to children.

SOURCE: CHEST 2018. https://journal.chestnet.org/article/S0012-3692(18)31935-4/fulltext

SAN FRANCISCO – There’s no need to wait for western blot results to differentiate Lyme arthritis from septic arthritis in children, as long as your lab, like many, uses the Liaison Borrelia burgdorferi assay, according to investigators at the University of Minnesota, Minneapolis.

M. Alexander Otto/MDedge News

Acute, isolated monoarthritis presents with a single swollen joint and pain whether it’s due to Lyme disease or infection, so it’s hard to tell them apart. Current guidelines recommend a two-tier approach to diagnose Lyme arthritis, an initial blood screen followed by western blot confirmation. Screening results come back in a few hours, but western blot confirmation can take days.

In the meantime, children are treated presumptively for the more concerning diagnosis – septic arthritis – which means hospitalization, surgical drainage, and IV antibiotics. Those who turn out to have Lyme are exposed to the risks and costs of unnecessary treatment and delays to proper diagnosis and doxycycline.

When “kids come in with a swollen knee, maybe 10% or 15% end up in the hospital being treated for septic arthritis that they never had. I wanted to see if we can diagnose Lyme arthritis more quickly,” said lead investigator Bazak Sharon, MD, a pediatric infectious disease specialist at the university’s Masonic Children’s Hospital.

Masonic and its affiliated health system use the Liaison Borrelia burgdorferi assay (DiaSorin) to screen for Lyme, and a careful parsing of the results seems to solve the problem.

Liaison is a chemiluminescence immunoassay that uses light to measure IgM and IgG antibodies to a B. burgdorferi surface protein in serum samples. Results are reported as relative light units (RLUs); below 0.9 RLUs is negative; 0.9-1.1 is equivocal, and over 1.1 is positive.

It’s where patients fall in the range of positivity that matters when it comes to differentiating Lyme from septic arthritis, Dr. Sharon said at ID Week, an annual scientific meeting on infectious diseases (Clin Vaccine Immunol. 2008 Dec;15[12]:1796-804).

He and his team reviewed 60 cases of acute, isolated monoarthritis culled from more than 700 children who presented with joint complaints from 2011 to 2016; 47 had Lyme arthritis confirmed by western blot; 13 had septic arthritis.

It turned out that “every single patient with a” Liaison value of 9 RLUs or higher was confirmed on western blot for Lyme. “Under 9, there was not a single case of Lyme arthritis,” Dr. Sharon said. Three other patients with acute arthritis also tested positive on the screen, but their RLU values were below 4; two turned out to be trauma related and one was ultimately diagnosed with juvenile idiopathic arthritis. Western blots were negative in all three.

The RLU number reported on the screening test “appears to correlate very well with Lyme arthritis. In an otherwise healthy child presenting with acute joint swelling, utilizing this screening test can confirm clinical suspicion of Lyme arthritis within hours, and prevent the potential harmful interventions accompanying a misdiagnosis of septic arthritis. Just do the screening. If it comes up above 9, you’ve got Lyme arthritis,” and don’t need to wait for western blot results to treat, Dr. Sharon said.

In other words, above 9, treat for Lyme.

The investigators plan to delve further into their results with sensitivity/specificity and other analyses before publishing. Ultimately, “my goal is to have a better diagnosis algorithm for kids who present with acute, isolated monoarthritis,” Dr. Sharon said.

There was no industry funding for the work, and the investigators didn’t have any disclosures.
 

aotto@mdedge.com

SOURCE: Sharon B et al. 2018 ID Week abstract 286.

SAN FRANCISCO – There’s no need to wait for western blot results to differentiate Lyme arthritis from septic arthritis in children, as long as your lab, like many, uses the Liaison Borrelia burgdorferi assay, according to investigators at the University of Minnesota, Minneapolis.

M. Alexander Otto/MDedge News

Acute, isolated monoarthritis presents with a single swollen joint and pain whether it’s due to Lyme disease or infection, so it’s hard to tell them apart. Current guidelines recommend a two-tier approach to diagnose Lyme arthritis, an initial blood screen followed by western blot confirmation. Screening results come back in a few hours, but western blot confirmation can take days.

In the meantime, children are treated presumptively for the more concerning diagnosis – septic arthritis – which means hospitalization, surgical drainage, and IV antibiotics. Those who turn out to have Lyme are exposed to the risks and costs of unnecessary treatment and delays to proper diagnosis and doxycycline.

When “kids come in with a swollen knee, maybe 10% or 15% end up in the hospital being treated for septic arthritis that they never had. I wanted to see if we can diagnose Lyme arthritis more quickly,” said lead investigator Bazak Sharon, MD, a pediatric infectious disease specialist at the university’s Masonic Children’s Hospital.

Masonic and its affiliated health system use the Liaison Borrelia burgdorferi assay (DiaSorin) to screen for Lyme, and a careful parsing of the results seems to solve the problem.

Liaison is a chemiluminescence immunoassay that uses light to measure IgM and IgG antibodies to a B. burgdorferi surface protein in serum samples. Results are reported as relative light units (RLUs); below 0.9 RLUs is negative; 0.9-1.1 is equivocal, and over 1.1 is positive.

It’s where patients fall in the range of positivity that matters when it comes to differentiating Lyme from septic arthritis, Dr. Sharon said at ID Week, an annual scientific meeting on infectious diseases (Clin Vaccine Immunol. 2008 Dec;15[12]:1796-804).

He and his team reviewed 60 cases of acute, isolated monoarthritis culled from more than 700 children who presented with joint complaints from 2011 to 2016; 47 had Lyme arthritis confirmed by western blot; 13 had septic arthritis.

It turned out that “every single patient with a” Liaison value of 9 RLUs or higher was confirmed on western blot for Lyme. “Under 9, there was not a single case of Lyme arthritis,” Dr. Sharon said. Three other patients with acute arthritis also tested positive on the screen, but their RLU values were below 4; two turned out to be trauma related and one was ultimately diagnosed with juvenile idiopathic arthritis. Western blots were negative in all three.

The RLU number reported on the screening test “appears to correlate very well with Lyme arthritis. In an otherwise healthy child presenting with acute joint swelling, utilizing this screening test can confirm clinical suspicion of Lyme arthritis within hours, and prevent the potential harmful interventions accompanying a misdiagnosis of septic arthritis. Just do the screening. If it comes up above 9, you’ve got Lyme arthritis,” and don’t need to wait for western blot results to treat, Dr. Sharon said.

In other words, above 9, treat for Lyme.

The investigators plan to delve further into their results with sensitivity/specificity and other analyses before publishing. Ultimately, “my goal is to have a better diagnosis algorithm for kids who present with acute, isolated monoarthritis,” Dr. Sharon said.

There was no industry funding for the work, and the investigators didn’t have any disclosures.
 

aotto@mdedge.com

SOURCE: Sharon B et al. 2018 ID Week abstract 286.

SAN FRANCISCO – There’s no need to wait for western blot results to differentiate Lyme arthritis from septic arthritis in children, as long as your lab, like many, uses the Liaison Borrelia burgdorferi assay, according to investigators at the University of Minnesota, Minneapolis.

M. Alexander Otto/MDedge News

Acute, isolated monoarthritis presents with a single swollen joint and pain whether it’s due to Lyme disease or infection, so it’s hard to tell them apart. Current guidelines recommend a two-tier approach to diagnose Lyme arthritis, an initial blood screen followed by western blot confirmation. Screening results come back in a few hours, but western blot confirmation can take days.

In the meantime, children are treated presumptively for the more concerning diagnosis – septic arthritis – which means hospitalization, surgical drainage, and IV antibiotics. Those who turn out to have Lyme are exposed to the risks and costs of unnecessary treatment and delays to proper diagnosis and doxycycline.

When “kids come in with a swollen knee, maybe 10% or 15% end up in the hospital being treated for septic arthritis that they never had. I wanted to see if we can diagnose Lyme arthritis more quickly,” said lead investigator Bazak Sharon, MD, a pediatric infectious disease specialist at the university’s Masonic Children’s Hospital.

Masonic and its affiliated health system use the Liaison Borrelia burgdorferi assay (DiaSorin) to screen for Lyme, and a careful parsing of the results seems to solve the problem.

Liaison is a chemiluminescence immunoassay that uses light to measure IgM and IgG antibodies to a B. burgdorferi surface protein in serum samples. Results are reported as relative light units (RLUs); below 0.9 RLUs is negative; 0.9-1.1 is equivocal, and over 1.1 is positive.

It’s where patients fall in the range of positivity that matters when it comes to differentiating Lyme from septic arthritis, Dr. Sharon said at ID Week, an annual scientific meeting on infectious diseases (Clin Vaccine Immunol. 2008 Dec;15[12]:1796-804).

He and his team reviewed 60 cases of acute, isolated monoarthritis culled from more than 700 children who presented with joint complaints from 2011 to 2016; 47 had Lyme arthritis confirmed by western blot; 13 had septic arthritis.

It turned out that “every single patient with a” Liaison value of 9 RLUs or higher was confirmed on western blot for Lyme. “Under 9, there was not a single case of Lyme arthritis,” Dr. Sharon said. Three other patients with acute arthritis also tested positive on the screen, but their RLU values were below 4; two turned out to be trauma related and one was ultimately diagnosed with juvenile idiopathic arthritis. Western blots were negative in all three.

The RLU number reported on the screening test “appears to correlate very well with Lyme arthritis. In an otherwise healthy child presenting with acute joint swelling, utilizing this screening test can confirm clinical suspicion of Lyme arthritis within hours, and prevent the potential harmful interventions accompanying a misdiagnosis of septic arthritis. Just do the screening. If it comes up above 9, you’ve got Lyme arthritis,” and don’t need to wait for western blot results to treat, Dr. Sharon said.

In other words, above 9, treat for Lyme.

The investigators plan to delve further into their results with sensitivity/specificity and other analyses before publishing. Ultimately, “my goal is to have a better diagnosis algorithm for kids who present with acute, isolated monoarthritis,” Dr. Sharon said.

There was no industry funding for the work, and the investigators didn’t have any disclosures.
 

aotto@mdedge.com

SOURCE: Sharon B et al. 2018 ID Week abstract 286.

The Food and Drug Administration has approved Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in people aged 12 years or older who have been symptomatic for 48 hours or less.

The FDA approval is based on results from two randomized, clinical trials. In both trials, patients who received Xofluza experienced a shorter duration until alleviation of symptoms, compared with patients who received a placebo. In the second trial, patients who received Xofluza and patients who received another approved antiviral influenza medication experienced similar durations until symptom alleviation.

“When treatment is started within 48 hours of becoming sick with flu symptoms, antiviral drugs can lessen symptoms and shorten the time patients feel sick. Having more treatment options that work in different ways to attack the virus is important because flu viruses can become resistant to antiviral drugs,” Debra Birnkrant, MD, director of the Division of Antiviral Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.

The most common adverse events associated with Xofluza were diarrhea and bronchitis.

“This is the first new antiviral flu treatment with a novel mechanism of action approved by the FDA in nearly 20 years,” FDA Commissioner Scott Gottlieb, MD, added. “With thousands of people getting the flu every year, and many people becoming seriously ill, having safe and effective treatment alternatives is critical. This novel drug provides an important, additional treatment option.”

Find the full press release on the FDA website.

lfranki@mdedge.com

The Food and Drug Administration has approved Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in people aged 12 years or older who have been symptomatic for 48 hours or less.

The FDA approval is based on results from two randomized, clinical trials. In both trials, patients who received Xofluza experienced a shorter duration until alleviation of symptoms, compared with patients who received a placebo. In the second trial, patients who received Xofluza and patients who received another approved antiviral influenza medication experienced similar durations until symptom alleviation.

“When treatment is started within 48 hours of becoming sick with flu symptoms, antiviral drugs can lessen symptoms and shorten the time patients feel sick. Having more treatment options that work in different ways to attack the virus is important because flu viruses can become resistant to antiviral drugs,” Debra Birnkrant, MD, director of the Division of Antiviral Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.

The most common adverse events associated with Xofluza were diarrhea and bronchitis.

“This is the first new antiviral flu treatment with a novel mechanism of action approved by the FDA in nearly 20 years,” FDA Commissioner Scott Gottlieb, MD, added. “With thousands of people getting the flu every year, and many people becoming seriously ill, having safe and effective treatment alternatives is critical. This novel drug provides an important, additional treatment option.”

Find the full press release on the FDA website.

lfranki@mdedge.com

The Food and Drug Administration has approved Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in people aged 12 years or older who have been symptomatic for 48 hours or less.

The FDA approval is based on results from two randomized, clinical trials. In both trials, patients who received Xofluza experienced a shorter duration until alleviation of symptoms, compared with patients who received a placebo. In the second trial, patients who received Xofluza and patients who received another approved antiviral influenza medication experienced similar durations until symptom alleviation.

“When treatment is started within 48 hours of becoming sick with flu symptoms, antiviral drugs can lessen symptoms and shorten the time patients feel sick. Having more treatment options that work in different ways to attack the virus is important because flu viruses can become resistant to antiviral drugs,” Debra Birnkrant, MD, director of the Division of Antiviral Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.

The most common adverse events associated with Xofluza were diarrhea and bronchitis.

“This is the first new antiviral flu treatment with a novel mechanism of action approved by the FDA in nearly 20 years,” FDA Commissioner Scott Gottlieb, MD, added. “With thousands of people getting the flu every year, and many people becoming seriously ill, having safe and effective treatment alternatives is critical. This novel drug provides an important, additional treatment option.”

Find the full press release on the FDA website.

lfranki@mdedge.com

WASHINGTON – Ataxia is the most common symptom at disease onset in patients with opsoclonus-myoclonus syndrome (OMS), a rare disease affecting only 1 in 5,000,000 individuals, mostly aged 1-5 years, based on data from a new patient registry.

In partnership with the National Organization of Rare Disorders (NORD) the nonprofit OMSLife Foundation has created a patient registry to better understand the disease experience in patients, wrote Mike Michaelis, chairman of OMSLife, and his colleagues. Early data from 275 enrolled patients were presented in a poster at the NORD Rare Summit, held by the National Organization for Rare Disorders.

The registry patients were mainly born in the United States (86%) and white (74%); approximately half were female. Of 150 patients who indicated symptoms at onset, 87% reported ataxia. Additional symptoms at onset were myoclonus (61%), opsoclonus (59%), tremors (46%), sleep disturbances (45%), temper tantrums (38%), vomiting (27%), fever (13%), headache (9%) and other symptoms (13%).

The researchers reviewed associations of symptoms at onset to determine the frequency of other symptoms for each individual symptom. Ataxia was present with 89% or higher instances of the other reported symptoms. Of note, some symptoms occurred more frequently than expected, such as temper tantrums and tremors in approximately 70% of patients with sleep disturbances. Myoclonus and opsoclonus, as well as fever and vomiting, also were significantly associated with the presence of other symptoms.

Two-thirds of the registry patients (69%) were diagnosed within 3 months of symptom onset, and 83% of these were diagnosed by a neurologist. Based on the Mitchell-Pike OMS severity scale, 59% of the patients met criteria for severe disease, 34% were classified as moderate, and 7% were mild. The registry is ongoing, but the current data provide insight on the clinical picture and common symptoms of OMS, the researchers said.

OMS Life was established in 2012 to support patients, caregivers, and researchers in raising awareness of opsoclonus-myoclonus syndrome as well as funds for research.

The study was supported by the OMSLife Foundation, NORD, and Trio Health Analytics. The researchers are employed by these organizations.

WASHINGTON – Ataxia is the most common symptom at disease onset in patients with opsoclonus-myoclonus syndrome (OMS), a rare disease affecting only 1 in 5,000,000 individuals, mostly aged 1-5 years, based on data from a new patient registry.

In partnership with the National Organization of Rare Disorders (NORD) the nonprofit OMSLife Foundation has created a patient registry to better understand the disease experience in patients, wrote Mike Michaelis, chairman of OMSLife, and his colleagues. Early data from 275 enrolled patients were presented in a poster at the NORD Rare Summit, held by the National Organization for Rare Disorders.

The registry patients were mainly born in the United States (86%) and white (74%); approximately half were female. Of 150 patients who indicated symptoms at onset, 87% reported ataxia. Additional symptoms at onset were myoclonus (61%), opsoclonus (59%), tremors (46%), sleep disturbances (45%), temper tantrums (38%), vomiting (27%), fever (13%), headache (9%) and other symptoms (13%).

The researchers reviewed associations of symptoms at onset to determine the frequency of other symptoms for each individual symptom. Ataxia was present with 89% or higher instances of the other reported symptoms. Of note, some symptoms occurred more frequently than expected, such as temper tantrums and tremors in approximately 70% of patients with sleep disturbances. Myoclonus and opsoclonus, as well as fever and vomiting, also were significantly associated with the presence of other symptoms.

Two-thirds of the registry patients (69%) were diagnosed within 3 months of symptom onset, and 83% of these were diagnosed by a neurologist. Based on the Mitchell-Pike OMS severity scale, 59% of the patients met criteria for severe disease, 34% were classified as moderate, and 7% were mild. The registry is ongoing, but the current data provide insight on the clinical picture and common symptoms of OMS, the researchers said.

OMS Life was established in 2012 to support patients, caregivers, and researchers in raising awareness of opsoclonus-myoclonus syndrome as well as funds for research.

The study was supported by the OMSLife Foundation, NORD, and Trio Health Analytics. The researchers are employed by these organizations.

WASHINGTON – Ataxia is the most common symptom at disease onset in patients with opsoclonus-myoclonus syndrome (OMS), a rare disease affecting only 1 in 5,000,000 individuals, mostly aged 1-5 years, based on data from a new patient registry.

In partnership with the National Organization of Rare Disorders (NORD) the nonprofit OMSLife Foundation has created a patient registry to better understand the disease experience in patients, wrote Mike Michaelis, chairman of OMSLife, and his colleagues. Early data from 275 enrolled patients were presented in a poster at the NORD Rare Summit, held by the National Organization for Rare Disorders.

The registry patients were mainly born in the United States (86%) and white (74%); approximately half were female. Of 150 patients who indicated symptoms at onset, 87% reported ataxia. Additional symptoms at onset were myoclonus (61%), opsoclonus (59%), tremors (46%), sleep disturbances (45%), temper tantrums (38%), vomiting (27%), fever (13%), headache (9%) and other symptoms (13%).

The researchers reviewed associations of symptoms at onset to determine the frequency of other symptoms for each individual symptom. Ataxia was present with 89% or higher instances of the other reported symptoms. Of note, some symptoms occurred more frequently than expected, such as temper tantrums and tremors in approximately 70% of patients with sleep disturbances. Myoclonus and opsoclonus, as well as fever and vomiting, also were significantly associated with the presence of other symptoms.

Two-thirds of the registry patients (69%) were diagnosed within 3 months of symptom onset, and 83% of these were diagnosed by a neurologist. Based on the Mitchell-Pike OMS severity scale, 59% of the patients met criteria for severe disease, 34% were classified as moderate, and 7% were mild. The registry is ongoing, but the current data provide insight on the clinical picture and common symptoms of OMS, the researchers said.

OMS Life was established in 2012 to support patients, caregivers, and researchers in raising awareness of opsoclonus-myoclonus syndrome as well as funds for research.

The study was supported by the OMSLife Foundation, NORD, and Trio Health Analytics. The researchers are employed by these organizations.

The Food and Drug Administration has extended the indication for a quadrivalent influenza vaccine (Afluria, marketed by Seqirus) to include infants and younger children.

The expanded approval now includes persons aged 6-59 months; the quadrivalent vaccine had previously been approved for ages 5 years and up. A trivalent version of the Afluria influenza vaccine also now is indicated for people aged 6 months and up, according to an Oct. 4 communication from the FDA.

A total of 172 pediatric influenza-related deaths occurred in the United States during the 2017-2018 season, representing a new high in nonpandemic influenza seasons. About half of the pediatric influenza deaths occurred in otherwise healthy children, and about 22% of children who died were fully vaccinated, according to the Centers for Disease Control and Prevention, reporting U.S. data from 2010 to 2016.

“As we enter a new flu season, we are reminded of the enormous impact that influenza can have on public health,” Seqirus’ vice president of medical affairs Gregg Sylvester, MD, said in a press release announcing the extended indication. “Having another option to fight this disease can translate to saved lives and fewer flu-related hospitalizations this season and going forward.”

According to the CDC, the 2018-2019 influenza vaccine has been updated to provide a better match – and more protection against – viruses circulating in this influenza season. Specifically, says the CDC, the influenza B Victoria lineage and the influenza A(H3N2) components were updated.

In addition to providing protection against these two strains of influenza, trivalent vaccines for the 2018-2019 season are recommended to include protection against H1N1 influenza as well. Quadrivalent vaccines protect against a second influenza B lineage.

Most people will receive a quadrivalent vaccine this year, according to the CDC.

koakes@mdedge.com

The Food and Drug Administration has extended the indication for a quadrivalent influenza vaccine (Afluria, marketed by Seqirus) to include infants and younger children.

The expanded approval now includes persons aged 6-59 months; the quadrivalent vaccine had previously been approved for ages 5 years and up. A trivalent version of the Afluria influenza vaccine also now is indicated for people aged 6 months and up, according to an Oct. 4 communication from the FDA.

A total of 172 pediatric influenza-related deaths occurred in the United States during the 2017-2018 season, representing a new high in nonpandemic influenza seasons. About half of the pediatric influenza deaths occurred in otherwise healthy children, and about 22% of children who died were fully vaccinated, according to the Centers for Disease Control and Prevention, reporting U.S. data from 2010 to 2016.

“As we enter a new flu season, we are reminded of the enormous impact that influenza can have on public health,” Seqirus’ vice president of medical affairs Gregg Sylvester, MD, said in a press release announcing the extended indication. “Having another option to fight this disease can translate to saved lives and fewer flu-related hospitalizations this season and going forward.”

According to the CDC, the 2018-2019 influenza vaccine has been updated to provide a better match – and more protection against – viruses circulating in this influenza season. Specifically, says the CDC, the influenza B Victoria lineage and the influenza A(H3N2) components were updated.

In addition to providing protection against these two strains of influenza, trivalent vaccines for the 2018-2019 season are recommended to include protection against H1N1 influenza as well. Quadrivalent vaccines protect against a second influenza B lineage.

Most people will receive a quadrivalent vaccine this year, according to the CDC.

koakes@mdedge.com

The Food and Drug Administration has extended the indication for a quadrivalent influenza vaccine (Afluria, marketed by Seqirus) to include infants and younger children.

The expanded approval now includes persons aged 6-59 months; the quadrivalent vaccine had previously been approved for ages 5 years and up. A trivalent version of the Afluria influenza vaccine also now is indicated for people aged 6 months and up, according to an Oct. 4 communication from the FDA.

A total of 172 pediatric influenza-related deaths occurred in the United States during the 2017-2018 season, representing a new high in nonpandemic influenza seasons. About half of the pediatric influenza deaths occurred in otherwise healthy children, and about 22% of children who died were fully vaccinated, according to the Centers for Disease Control and Prevention, reporting U.S. data from 2010 to 2016.

“As we enter a new flu season, we are reminded of the enormous impact that influenza can have on public health,” Seqirus’ vice president of medical affairs Gregg Sylvester, MD, said in a press release announcing the extended indication. “Having another option to fight this disease can translate to saved lives and fewer flu-related hospitalizations this season and going forward.”

According to the CDC, the 2018-2019 influenza vaccine has been updated to provide a better match – and more protection against – viruses circulating in this influenza season. Specifically, says the CDC, the influenza B Victoria lineage and the influenza A(H3N2) components were updated.

In addition to providing protection against these two strains of influenza, trivalent vaccines for the 2018-2019 season are recommended to include protection against H1N1 influenza as well. Quadrivalent vaccines protect against a second influenza B lineage.

Most people will receive a quadrivalent vaccine this year, according to the CDC.

koakes@mdedge.com

Recent recommendations from an international task force advocating that patients with juvenile idiopathic arthritis (JIA) be treated to a target of clinical remission have been backed up by research that finds the goal is a feasible one.

Published online in the Annals of the Rheumatic Diseases, the investigator-initiated, multicenter, randomized BEST for Kids study found that, regardless of initial specific treatments, after 24 months of treatment to target aimed at drug-free inactive disease, 71% of recent-onset patients with JIA had inactive disease (at a median onset of 9 months), and 39% were drug free.

The investigators, led by Petra Hissink Muller, MD, department of pediatrics, Leiden (the Netherlands) University Medical Center said previous studies of JIA supported the “window of opportunity” hypothesis in which disease optimally responds to treatment.

“In JIA, continuous treatment-to-target therapy in a tight control setting, with treatment adjustments based on frequent evaluations of disease activity, has not yet been studied. Recent recommendations agree that treatment to target should be implemented in daily practice,” they wrote.

The aim of the BEST for Kids study was to investigate which of three frequently used treatment-to-target strategies was most effective and safe.

Overall, 94 children (67% girls) with a median age of 9.1 years with new-onset (oligoarticular, juvenile psoriatic arthritis, or rheumatoid factor–negative polyarticular) JIA, without previous disease-modifying antirheumatic drug (DMARD) therapy and symptom duration of less than 18 months were enrolled in the study.

Median symptom duration was 7.5 months and median duration between diagnosis and inclusion in the study was 6 weeks. The investigators noted that two patients left the study during follow-up because of a revised diagnosis.

All study participants were randomized into three strategy arms:

• Initial treatment with conventional synthetic DMARD monotherapy (methotrexate [MTX] or sulfasalazine if preferred by treating physician), n = 32.
• Initial treatment with MTX and 6 weeks of tapered prednisolone (‘bridging therapy), n = 32).
• Initial treatment with MTX and etanercept, n = 30.

In case of inactive disease for at least 3 (oligoarticular disease) or 6 (polyarticular disease) consecutive months, DMARDs were tapered and stopped in all three arms. For combination therapy, etanercept was tapered to once every 2 weeks, followed by a 50% dose reduction, then halted. The dose of MTX or sulfasalazine was reduced by 25% per week to zero.

In case of a disease flare, the last discontinued drug and/or the last effective dose was reintroduced. The protocol did not allow prednisolone to be restarted, whereas etanercept could be restarted but not discontinued for a second time.

The primary outcome of the trial was time to inactive disease and time to flare (defined as the time between first moment of drug-free inactive disease (DFID) and the first arthritis flare as judged by the treating physician) after tapering and stopping all DMARD therapy. Secondary outcomes were adapted ACR Pediatric (ACR Pedi) 30/50/70/90 scores, functional ability, and adverse events.

Results showed that after 24 months inactive disease was achieved by more than 70% of the patients (71%, arm 1; 70%, arm 2; and 72%, arm 3).

Furthermore, drug-free inactive disease (DFID) was achieved by 59% of the cohort (54 of 92 patients; 45% of arm 1, 31% of arm 2, and 41% of arm 3), although the authors noted that early flares did occur in 14 patients and were successfully retreated, resulting in 39% of the patients in DFID at the 2-year study endpoint.

In the treatment of JIA,“we showed that tapering and discontinuation of treatment is a realistic goal. On the other hand, treatment to target resulted in a relatively high use of [biologic] DMARDs, greater than 50% of patients in all arms,” the investigators wrote.

Median time to inactive disease was 9 months in all arms (P = .30), and time to flare was also not significantly different among the groups (overall 3 months [3.0-6.8]; P = .7). The investigators noted that while overall flare rates (26%) were lower than the 37%-60% mentioned in previous cohorts, this finding could be a result of the limited follow-up time.

After 3 months of treatment, more patients who started with MTX and etanercept (arm 3) had achieved rapid improvement as determined by ACR Pedi 70 scores. However, the investigators noted that, because of treatment adjustments in cases of active disease, which were needed more often in arms 1 and 2 than in arm 3, ACR Pedi improvement scores were met in similar percentages of patients over time across the arms.

Adverse events were similar across treatment arms and were generally mild, involving mostly gastrointestinal complaints, upper respiratory tract and other infections, and general malaise.

Overall, the investigators concluded that DFID was a “feasible goal” in the treatment of children with JIA.

Limitations of the study included breaches of treatment protocol by physicians in patients across the three arms.“The physicians did not follow protocol for various reasons, mainly reluctance to intensify therapy based on shared decision making,” they wrote.

Another limitation was the small sample size, which the investigators said could obscure differences between groups that in a larger population might have become clear.

“Long-term follow-up of the BEST for Kids cohort, including radiology results, is initiated to investigate possible lasting positive results of treatment to target in JIA,” they said.

SOURCE: Hissink Muller P et al. Ann Rheum Dis. Oct 12. doi: 10.1136/annrheumdis-2018-213902.

Recent recommendations from an international task force advocating that patients with juvenile idiopathic arthritis (JIA) be treated to a target of clinical remission have been backed up by research that finds the goal is a feasible one.

Published online in the Annals of the Rheumatic Diseases, the investigator-initiated, multicenter, randomized BEST for Kids study found that, regardless of initial specific treatments, after 24 months of treatment to target aimed at drug-free inactive disease, 71% of recent-onset patients with JIA had inactive disease (at a median onset of 9 months), and 39% were drug free.

The investigators, led by Petra Hissink Muller, MD, department of pediatrics, Leiden (the Netherlands) University Medical Center said previous studies of JIA supported the “window of opportunity” hypothesis in which disease optimally responds to treatment.

“In JIA, continuous treatment-to-target therapy in a tight control setting, with treatment adjustments based on frequent evaluations of disease activity, has not yet been studied. Recent recommendations agree that treatment to target should be implemented in daily practice,” they wrote.

The aim of the BEST for Kids study was to investigate which of three frequently used treatment-to-target strategies was most effective and safe.

Overall, 94 children (67% girls) with a median age of 9.1 years with new-onset (oligoarticular, juvenile psoriatic arthritis, or rheumatoid factor–negative polyarticular) JIA, without previous disease-modifying antirheumatic drug (DMARD) therapy and symptom duration of less than 18 months were enrolled in the study.

Median symptom duration was 7.5 months and median duration between diagnosis and inclusion in the study was 6 weeks. The investigators noted that two patients left the study during follow-up because of a revised diagnosis.

All study participants were randomized into three strategy arms:

• Initial treatment with conventional synthetic DMARD monotherapy (methotrexate [MTX] or sulfasalazine if preferred by treating physician), n = 32.
• Initial treatment with MTX and 6 weeks of tapered prednisolone (‘bridging therapy), n = 32).
• Initial treatment with MTX and etanercept, n = 30.

In case of inactive disease for at least 3 (oligoarticular disease) or 6 (polyarticular disease) consecutive months, DMARDs were tapered and stopped in all three arms. For combination therapy, etanercept was tapered to once every 2 weeks, followed by a 50% dose reduction, then halted. The dose of MTX or sulfasalazine was reduced by 25% per week to zero.

In case of a disease flare, the last discontinued drug and/or the last effective dose was reintroduced. The protocol did not allow prednisolone to be restarted, whereas etanercept could be restarted but not discontinued for a second time.

The primary outcome of the trial was time to inactive disease and time to flare (defined as the time between first moment of drug-free inactive disease (DFID) and the first arthritis flare as judged by the treating physician) after tapering and stopping all DMARD therapy. Secondary outcomes were adapted ACR Pediatric (ACR Pedi) 30/50/70/90 scores, functional ability, and adverse events.

Results showed that after 24 months inactive disease was achieved by more than 70% of the patients (71%, arm 1; 70%, arm 2; and 72%, arm 3).

Furthermore, drug-free inactive disease (DFID) was achieved by 59% of the cohort (54 of 92 patients; 45% of arm 1, 31% of arm 2, and 41% of arm 3), although the authors noted that early flares did occur in 14 patients and were successfully retreated, resulting in 39% of the patients in DFID at the 2-year study endpoint.

In the treatment of JIA,“we showed that tapering and discontinuation of treatment is a realistic goal. On the other hand, treatment to target resulted in a relatively high use of [biologic] DMARDs, greater than 50% of patients in all arms,” the investigators wrote.

Median time to inactive disease was 9 months in all arms (P = .30), and time to flare was also not significantly different among the groups (overall 3 months [3.0-6.8]; P = .7). The investigators noted that while overall flare rates (26%) were lower than the 37%-60% mentioned in previous cohorts, this finding could be a result of the limited follow-up time.

After 3 months of treatment, more patients who started with MTX and etanercept (arm 3) had achieved rapid improvement as determined by ACR Pedi 70 scores. However, the investigators noted that, because of treatment adjustments in cases of active disease, which were needed more often in arms 1 and 2 than in arm 3, ACR Pedi improvement scores were met in similar percentages of patients over time across the arms.

Adverse events were similar across treatment arms and were generally mild, involving mostly gastrointestinal complaints, upper respiratory tract and other infections, and general malaise.

Overall, the investigators concluded that DFID was a “feasible goal” in the treatment of children with JIA.

Limitations of the study included breaches of treatment protocol by physicians in patients across the three arms.“The physicians did not follow protocol for various reasons, mainly reluctance to intensify therapy based on shared decision making,” they wrote.

Another limitation was the small sample size, which the investigators said could obscure differences between groups that in a larger population might have become clear.

“Long-term follow-up of the BEST for Kids cohort, including radiology results, is initiated to investigate possible lasting positive results of treatment to target in JIA,” they said.

SOURCE: Hissink Muller P et al. Ann Rheum Dis. Oct 12. doi: 10.1136/annrheumdis-2018-213902.

Recent recommendations from an international task force advocating that patients with juvenile idiopathic arthritis (JIA) be treated to a target of clinical remission have been backed up by research that finds the goal is a feasible one.

Published online in the Annals of the Rheumatic Diseases, the investigator-initiated, multicenter, randomized BEST for Kids study found that, regardless of initial specific treatments, after 24 months of treatment to target aimed at drug-free inactive disease, 71% of recent-onset patients with JIA had inactive disease (at a median onset of 9 months), and 39% were drug free.

The investigators, led by Petra Hissink Muller, MD, department of pediatrics, Leiden (the Netherlands) University Medical Center said previous studies of JIA supported the “window of opportunity” hypothesis in which disease optimally responds to treatment.

“In JIA, continuous treatment-to-target therapy in a tight control setting, with treatment adjustments based on frequent evaluations of disease activity, has not yet been studied. Recent recommendations agree that treatment to target should be implemented in daily practice,” they wrote.

The aim of the BEST for Kids study was to investigate which of three frequently used treatment-to-target strategies was most effective and safe.

Overall, 94 children (67% girls) with a median age of 9.1 years with new-onset (oligoarticular, juvenile psoriatic arthritis, or rheumatoid factor–negative polyarticular) JIA, without previous disease-modifying antirheumatic drug (DMARD) therapy and symptom duration of less than 18 months were enrolled in the study.

Median symptom duration was 7.5 months and median duration between diagnosis and inclusion in the study was 6 weeks. The investigators noted that two patients left the study during follow-up because of a revised diagnosis.

All study participants were randomized into three strategy arms:

• Initial treatment with conventional synthetic DMARD monotherapy (methotrexate [MTX] or sulfasalazine if preferred by treating physician), n = 32.
• Initial treatment with MTX and 6 weeks of tapered prednisolone (‘bridging therapy), n = 32).
• Initial treatment with MTX and etanercept, n = 30.

In case of inactive disease for at least 3 (oligoarticular disease) or 6 (polyarticular disease) consecutive months, DMARDs were tapered and stopped in all three arms. For combination therapy, etanercept was tapered to once every 2 weeks, followed by a 50% dose reduction, then halted. The dose of MTX or sulfasalazine was reduced by 25% per week to zero.

In case of a disease flare, the last discontinued drug and/or the last effective dose was reintroduced. The protocol did not allow prednisolone to be restarted, whereas etanercept could be restarted but not discontinued for a second time.

The primary outcome of the trial was time to inactive disease and time to flare (defined as the time between first moment of drug-free inactive disease (DFID) and the first arthritis flare as judged by the treating physician) after tapering and stopping all DMARD therapy. Secondary outcomes were adapted ACR Pediatric (ACR Pedi) 30/50/70/90 scores, functional ability, and adverse events.

Results showed that after 24 months inactive disease was achieved by more than 70% of the patients (71%, arm 1; 70%, arm 2; and 72%, arm 3).

Furthermore, drug-free inactive disease (DFID) was achieved by 59% of the cohort (54 of 92 patients; 45% of arm 1, 31% of arm 2, and 41% of arm 3), although the authors noted that early flares did occur in 14 patients and were successfully retreated, resulting in 39% of the patients in DFID at the 2-year study endpoint.

In the treatment of JIA,“we showed that tapering and discontinuation of treatment is a realistic goal. On the other hand, treatment to target resulted in a relatively high use of [biologic] DMARDs, greater than 50% of patients in all arms,” the investigators wrote.

Median time to inactive disease was 9 months in all arms (P = .30), and time to flare was also not significantly different among the groups (overall 3 months [3.0-6.8]; P = .7). The investigators noted that while overall flare rates (26%) were lower than the 37%-60% mentioned in previous cohorts, this finding could be a result of the limited follow-up time.

After 3 months of treatment, more patients who started with MTX and etanercept (arm 3) had achieved rapid improvement as determined by ACR Pedi 70 scores. However, the investigators noted that, because of treatment adjustments in cases of active disease, which were needed more often in arms 1 and 2 than in arm 3, ACR Pedi improvement scores were met in similar percentages of patients over time across the arms.

Adverse events were similar across treatment arms and were generally mild, involving mostly gastrointestinal complaints, upper respiratory tract and other infections, and general malaise.

Overall, the investigators concluded that DFID was a “feasible goal” in the treatment of children with JIA.

Limitations of the study included breaches of treatment protocol by physicians in patients across the three arms.“The physicians did not follow protocol for various reasons, mainly reluctance to intensify therapy based on shared decision making,” they wrote.

Another limitation was the small sample size, which the investigators said could obscure differences between groups that in a larger population might have become clear.

“Long-term follow-up of the BEST for Kids cohort, including radiology results, is initiated to investigate possible lasting positive results of treatment to target in JIA,” they said.

SOURCE: Hissink Muller P et al. Ann Rheum Dis. Oct 12. doi: 10.1136/annrheumdis-2018-213902.

Both age and type of insurance are significantly associated with greater delays in treatment of febrile urinary tract infections (UTIs), even after adjustment for confounders, according to a report in the Journal of Pediatrics.

Zizulhalmi/Thinkstock

Stephanie W. Hum and Nader Shaikh, MD, MPH, of the University of Pittsburgh, drew from data provided by two studies, Randomized Intervention for Children With Vesicoureteral Reflux and Careful Urinary Tract Infection Evaluation. Specifically, they extracted data regarding patients’ age, sex, history of UTIs, ethnicity, race, insurance, household size, and duration of fever before initiation of antimicrobial therapy, as well as primary caregivers’ level of education and income. Some factors were analyzed because of associations seen in adult studies, and others because of concerns about access to care. In this analysis, the researchers defined “treatment delay” as the number of hours between onset of fever and initiation of antimicrobial treatment and, after exclusion of afebrile children and those with missing data, included 660 patients.

In univariate analysis, both older age and commercial insurance were found to be significantly associated with delays in treatment. Compared with time to treatment seen with younger children, treatment was delayed by an average of 26.2 hours in children aged 12 months and older (P less than .001). Patients with commercial insurance were treated a mean of 12.6 hours later than were those with noncommercial insurance (P = .002). These associations remained significant even after adjustment in a multivariable regression model for sex, history of UTIs, ethnicity, race, primary caregivers’ level of education, insurance, and income level.

The finding regarding age is consistent with a previous study, and Ms. Hum and Dr. Shaikh suggested it may reflect parents experiencing reduced urgency regarding febrile illnesses among older children. However, the researchers also noted that greater rates of renal scarring are seen in older children, so “it seems important to educate physicians, parents, and triage nurses about the importance of early evaluation of children with fever,” even those older than 12 months.

The finding regarding insurance status, however, is contrary to what studies in adult populations have found, as well as those in pediatric EDs. The researchers suggested that perhaps parents with noncommercial insurance are more likely to take their children to EDs, where testing can be done on-site 24 hours a day, rather than to private clinics, which often have to send out testing to off-site laboratories.

One of the strengths of the study is its relatively large sample size, they said. Among its weaknesses is that treatment delays were self-reported by parents and might be inaccurate and that information regarding location of initial evaluation was not gathered and could not be examined with other factors.

This study was supported by a T35 training grant from the National Institute of Diabetes and Digestive and Kidney Diseases, sponsored by Tom R. Kleyman, MD, chief of the renal-electrolyte division at the University of Pittsburgh. The authors declared no conflicts of interest.

cpalmer@mdedge.com

SOURCE: Hum SW et al. J Pediatr. 2018 Oct 16. doi: 10.1016/j.jpeds.2018.09.029.

This article was updated 10/24/18.

Both age and type of insurance are significantly associated with greater delays in treatment of febrile urinary tract infections (UTIs), even after adjustment for confounders, according to a report in the Journal of Pediatrics.

Zizulhalmi/Thinkstock

Stephanie W. Hum and Nader Shaikh, MD, MPH, of the University of Pittsburgh, drew from data provided by two studies, Randomized Intervention for Children With Vesicoureteral Reflux and Careful Urinary Tract Infection Evaluation. Specifically, they extracted data regarding patients’ age, sex, history of UTIs, ethnicity, race, insurance, household size, and duration of fever before initiation of antimicrobial therapy, as well as primary caregivers’ level of education and income. Some factors were analyzed because of associations seen in adult studies, and others because of concerns about access to care. In this analysis, the researchers defined “treatment delay” as the number of hours between onset of fever and initiation of antimicrobial treatment and, after exclusion of afebrile children and those with missing data, included 660 patients.

In univariate analysis, both older age and commercial insurance were found to be significantly associated with delays in treatment. Compared with time to treatment seen with younger children, treatment was delayed by an average of 26.2 hours in children aged 12 months and older (P less than .001). Patients with commercial insurance were treated a mean of 12.6 hours later than were those with noncommercial insurance (P = .002). These associations remained significant even after adjustment in a multivariable regression model for sex, history of UTIs, ethnicity, race, primary caregivers’ level of education, insurance, and income level.

The finding regarding age is consistent with a previous study, and Ms. Hum and Dr. Shaikh suggested it may reflect parents experiencing reduced urgency regarding febrile illnesses among older children. However, the researchers also noted that greater rates of renal scarring are seen in older children, so “it seems important to educate physicians, parents, and triage nurses about the importance of early evaluation of children with fever,” even those older than 12 months.

The finding regarding insurance status, however, is contrary to what studies in adult populations have found, as well as those in pediatric EDs. The researchers suggested that perhaps parents with noncommercial insurance are more likely to take their children to EDs, where testing can be done on-site 24 hours a day, rather than to private clinics, which often have to send out testing to off-site laboratories.

One of the strengths of the study is its relatively large sample size, they said. Among its weaknesses is that treatment delays were self-reported by parents and might be inaccurate and that information regarding location of initial evaluation was not gathered and could not be examined with other factors.

This study was supported by a T35 training grant from the National Institute of Diabetes and Digestive and Kidney Diseases, sponsored by Tom R. Kleyman, MD, chief of the renal-electrolyte division at the University of Pittsburgh. The authors declared no conflicts of interest.

cpalmer@mdedge.com

SOURCE: Hum SW et al. J Pediatr. 2018 Oct 16. doi: 10.1016/j.jpeds.2018.09.029.

This article was updated 10/24/18.

Both age and type of insurance are significantly associated with greater delays in treatment of febrile urinary tract infections (UTIs), even after adjustment for confounders, according to a report in the Journal of Pediatrics.

Zizulhalmi/Thinkstock

Stephanie W. Hum and Nader Shaikh, MD, MPH, of the University of Pittsburgh, drew from data provided by two studies, Randomized Intervention for Children With Vesicoureteral Reflux and Careful Urinary Tract Infection Evaluation. Specifically, they extracted data regarding patients’ age, sex, history of UTIs, ethnicity, race, insurance, household size, and duration of fever before initiation of antimicrobial therapy, as well as primary caregivers’ level of education and income. Some factors were analyzed because of associations seen in adult studies, and others because of concerns about access to care. In this analysis, the researchers defined “treatment delay” as the number of hours between onset of fever and initiation of antimicrobial treatment and, after exclusion of afebrile children and those with missing data, included 660 patients.

In univariate analysis, both older age and commercial insurance were found to be significantly associated with delays in treatment. Compared with time to treatment seen with younger children, treatment was delayed by an average of 26.2 hours in children aged 12 months and older (P less than .001). Patients with commercial insurance were treated a mean of 12.6 hours later than were those with noncommercial insurance (P = .002). These associations remained significant even after adjustment in a multivariable regression model for sex, history of UTIs, ethnicity, race, primary caregivers’ level of education, insurance, and income level.

The finding regarding age is consistent with a previous study, and Ms. Hum and Dr. Shaikh suggested it may reflect parents experiencing reduced urgency regarding febrile illnesses among older children. However, the researchers also noted that greater rates of renal scarring are seen in older children, so “it seems important to educate physicians, parents, and triage nurses about the importance of early evaluation of children with fever,” even those older than 12 months.

The finding regarding insurance status, however, is contrary to what studies in adult populations have found, as well as those in pediatric EDs. The researchers suggested that perhaps parents with noncommercial insurance are more likely to take their children to EDs, where testing can be done on-site 24 hours a day, rather than to private clinics, which often have to send out testing to off-site laboratories.

One of the strengths of the study is its relatively large sample size, they said. Among its weaknesses is that treatment delays were self-reported by parents and might be inaccurate and that information regarding location of initial evaluation was not gathered and could not be examined with other factors.

This study was supported by a T35 training grant from the National Institute of Diabetes and Digestive and Kidney Diseases, sponsored by Tom R. Kleyman, MD, chief of the renal-electrolyte division at the University of Pittsburgh. The authors declared no conflicts of interest.

cpalmer@mdedge.com

SOURCE: Hum SW et al. J Pediatr. 2018 Oct 16. doi: 10.1016/j.jpeds.2018.09.029.

This article was updated 10/24/18.

Photo from Novartis

Tisagenlecleucel has the potential to be cost-effective for pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients in the United States, according to researchers.

The group found evidence to suggest the chimeric antigen receptor (CAR) T-cell therapy—which has a list price of $475,000—may prove cost-effective if long-term survival benefits are realized.

An analysis indicated that the incremental cost-effectiveness ratio for tisagenlecleucel compared to clofarabine ranged from $37,000 to $78,000 per quality-adjusted life year (QALY) gained.

Melanie D. Whittington, PhD, of the University of Colorado at Denver, Aurora, and her colleagues described this work in JAMA Pediatrics.

For this study, the researchers used a decision analytic model that extrapolated the evidence from clinical trials over a patient’s lifetime to assess life-years gained, QALYs gained, and incremental costs per life-year and QALY gained. The researchers compared tisagenlecleucel to the antineoplastic agent clofarabine.

While tisagenlecleucel has a list price of $475,000, researchers discounted the price by 3% and added several additional costs, such as hospital administration, pretreatment, and potential adverse events, to get to a total discounted cost of about $667,000.

The team estimated that 42.6% of B-ALL patients would be long-term survivors with tisagenlecleucel, 10.34 life-years would be gained, and 9.28 QALYs would be gained.

In comparison, clofarabine had a total discounted cost of approximately $337,000, which included an initial discounted price of $164,000 plus additional treatment and administrative costs.

With clofarabine, 10.8% of B-ALL patients were long-term survivors, 2.43 life-years were gained, and 2.10 QALYs were gained in the model.

Overall, the mean incremental cost-effectiveness ratio was about $46,000 per QALY gained in this base-case model.

In analyses of different scenarios, such as a deeper discount, a different treatment start, or a different calculation of future treatment costs, the cost-effectiveness ratio varied from $37,000 to $78,000 per QALY gained.

The researchers noted that clinical trial evidence for tisagenlecleucel came from single-arm trials, which made the selection of a comparator challenging. Clofarabine was chosen because it had the most similar baseline population characteristics, but the researchers acknowledged that blinatumomab is also frequently used as a treatment for these patients.

“We suspect that tisagenlecleucel would remain cost-effective compared with blinatumomab,” the researchers wrote in JAMA Pediatrics. “A study conducted by other researchers found the incremental cost-effectiveness ratio of tisagenlecleucel versus blinatumomab was similar to the incremental cost-effectiveness ratio of tisagenlecleucel versus clofarabine [i.e., $3,000 more per QALY].”

The researchers suggested that uncertainties in the evidence should be considered as payers are negotiating coverage and payment for tisagenlecleucel.

This study was funded by the Institute for Clinical and Economic Review, which receives some funding from the pharmaceutical industry. Four study authors are employees of the Institute for Clinical and Economic Review.

Photo from Novartis

Tisagenlecleucel has the potential to be cost-effective for pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients in the United States, according to researchers.

The group found evidence to suggest the chimeric antigen receptor (CAR) T-cell therapy—which has a list price of $475,000—may prove cost-effective if long-term survival benefits are realized.

An analysis indicated that the incremental cost-effectiveness ratio for tisagenlecleucel compared to clofarabine ranged from $37,000 to $78,000 per quality-adjusted life year (QALY) gained.

Melanie D. Whittington, PhD, of the University of Colorado at Denver, Aurora, and her colleagues described this work in JAMA Pediatrics.

For this study, the researchers used a decision analytic model that extrapolated the evidence from clinical trials over a patient’s lifetime to assess life-years gained, QALYs gained, and incremental costs per life-year and QALY gained. The researchers compared tisagenlecleucel to the antineoplastic agent clofarabine.

While tisagenlecleucel has a list price of $475,000, researchers discounted the price by 3% and added several additional costs, such as hospital administration, pretreatment, and potential adverse events, to get to a total discounted cost of about $667,000.

The team estimated that 42.6% of B-ALL patients would be long-term survivors with tisagenlecleucel, 10.34 life-years would be gained, and 9.28 QALYs would be gained.

In comparison, clofarabine had a total discounted cost of approximately $337,000, which included an initial discounted price of $164,000 plus additional treatment and administrative costs.

With clofarabine, 10.8% of B-ALL patients were long-term survivors, 2.43 life-years were gained, and 2.10 QALYs were gained in the model.

Overall, the mean incremental cost-effectiveness ratio was about $46,000 per QALY gained in this base-case model.

In analyses of different scenarios, such as a deeper discount, a different treatment start, or a different calculation of future treatment costs, the cost-effectiveness ratio varied from $37,000 to $78,000 per QALY gained.

The researchers noted that clinical trial evidence for tisagenlecleucel came from single-arm trials, which made the selection of a comparator challenging. Clofarabine was chosen because it had the most similar baseline population characteristics, but the researchers acknowledged that blinatumomab is also frequently used as a treatment for these patients.

“We suspect that tisagenlecleucel would remain cost-effective compared with blinatumomab,” the researchers wrote in JAMA Pediatrics. “A study conducted by other researchers found the incremental cost-effectiveness ratio of tisagenlecleucel versus blinatumomab was similar to the incremental cost-effectiveness ratio of tisagenlecleucel versus clofarabine [i.e., $3,000 more per QALY].”

The researchers suggested that uncertainties in the evidence should be considered as payers are negotiating coverage and payment for tisagenlecleucel.

This study was funded by the Institute for Clinical and Economic Review, which receives some funding from the pharmaceutical industry. Four study authors are employees of the Institute for Clinical and Economic Review.

Photo from Novartis

Tisagenlecleucel has the potential to be cost-effective for pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients in the United States, according to researchers.

The group found evidence to suggest the chimeric antigen receptor (CAR) T-cell therapy—which has a list price of $475,000—may prove cost-effective if long-term survival benefits are realized.

An analysis indicated that the incremental cost-effectiveness ratio for tisagenlecleucel compared to clofarabine ranged from $37,000 to $78,000 per quality-adjusted life year (QALY) gained.

Melanie D. Whittington, PhD, of the University of Colorado at Denver, Aurora, and her colleagues described this work in JAMA Pediatrics.

For this study, the researchers used a decision analytic model that extrapolated the evidence from clinical trials over a patient’s lifetime to assess life-years gained, QALYs gained, and incremental costs per life-year and QALY gained. The researchers compared tisagenlecleucel to the antineoplastic agent clofarabine.

While tisagenlecleucel has a list price of $475,000, researchers discounted the price by 3% and added several additional costs, such as hospital administration, pretreatment, and potential adverse events, to get to a total discounted cost of about $667,000.

The team estimated that 42.6% of B-ALL patients would be long-term survivors with tisagenlecleucel, 10.34 life-years would be gained, and 9.28 QALYs would be gained.

In comparison, clofarabine had a total discounted cost of approximately $337,000, which included an initial discounted price of $164,000 plus additional treatment and administrative costs.

With clofarabine, 10.8% of B-ALL patients were long-term survivors, 2.43 life-years were gained, and 2.10 QALYs were gained in the model.

Overall, the mean incremental cost-effectiveness ratio was about $46,000 per QALY gained in this base-case model.

In analyses of different scenarios, such as a deeper discount, a different treatment start, or a different calculation of future treatment costs, the cost-effectiveness ratio varied from $37,000 to $78,000 per QALY gained.

The researchers noted that clinical trial evidence for tisagenlecleucel came from single-arm trials, which made the selection of a comparator challenging. Clofarabine was chosen because it had the most similar baseline population characteristics, but the researchers acknowledged that blinatumomab is also frequently used as a treatment for these patients.

“We suspect that tisagenlecleucel would remain cost-effective compared with blinatumomab,” the researchers wrote in JAMA Pediatrics. “A study conducted by other researchers found the incremental cost-effectiveness ratio of tisagenlecleucel versus blinatumomab was similar to the incremental cost-effectiveness ratio of tisagenlecleucel versus clofarabine [i.e., $3,000 more per QALY].”

The researchers suggested that uncertainties in the evidence should be considered as payers are negotiating coverage and payment for tisagenlecleucel.

This study was funded by the Institute for Clinical and Economic Review, which receives some funding from the pharmaceutical industry. Four study authors are employees of the Institute for Clinical and Economic Review.