CML

Key clinical point: Bosutinib was effective and safe as a second or subsequent line of treatment in a real-life cohort of elderly patients with chronic-phase chronic myeloid leukemia (CML-CP) and multiple baseline comorbidities who were resistant or intolerant to previous tyrosine kinase inhibitors (TKIs).

Major finding: Overall, rates of cytogenic response, molecular response, 3-year event-free survival, and 3-year overall survival were 81.2%, 66.6%, 60.9%, and 86.4%, respectively. Grade 3/4 hematological and extra-hematological toxicities were reported in 6.9% and 18.8% of patients, respectively, with 11.9% of patients permanently discontinuing bosutinib because of toxicity.

Study details: Findings are from a retrospective analysis of 101 elderly (age, older than 65 years) patients with CML-CP treated with bosutinib in second or subsequent line. Patients switched to bosutinib because of intolerance (n=46) or resistance (n=55) to previous TKI therapies.

Disclosures: This study did not receive any type of funding. Some investigators including the lead author reported consulting for or receiving honoraria from various pharmaceutical companies.

Source: Latagliata R et al. Hematol Oncol. 2021 Feb 22. doi: 10.1002/hon.2851.

Key clinical point: Bosutinib was effective and safe as a second or subsequent line of treatment in a real-life cohort of elderly patients with chronic-phase chronic myeloid leukemia (CML-CP) and multiple baseline comorbidities who were resistant or intolerant to previous tyrosine kinase inhibitors (TKIs).

Major finding: Overall, rates of cytogenic response, molecular response, 3-year event-free survival, and 3-year overall survival were 81.2%, 66.6%, 60.9%, and 86.4%, respectively. Grade 3/4 hematological and extra-hematological toxicities were reported in 6.9% and 18.8% of patients, respectively, with 11.9% of patients permanently discontinuing bosutinib because of toxicity.

Study details: Findings are from a retrospective analysis of 101 elderly (age, older than 65 years) patients with CML-CP treated with bosutinib in second or subsequent line. Patients switched to bosutinib because of intolerance (n=46) or resistance (n=55) to previous TKI therapies.

Disclosures: This study did not receive any type of funding. Some investigators including the lead author reported consulting for or receiving honoraria from various pharmaceutical companies.

Source: Latagliata R et al. Hematol Oncol. 2021 Feb 22. doi: 10.1002/hon.2851.

Key clinical point: Bosutinib was effective and safe as a second or subsequent line of treatment in a real-life cohort of elderly patients with chronic-phase chronic myeloid leukemia (CML-CP) and multiple baseline comorbidities who were resistant or intolerant to previous tyrosine kinase inhibitors (TKIs).

Major finding: Overall, rates of cytogenic response, molecular response, 3-year event-free survival, and 3-year overall survival were 81.2%, 66.6%, 60.9%, and 86.4%, respectively. Grade 3/4 hematological and extra-hematological toxicities were reported in 6.9% and 18.8% of patients, respectively, with 11.9% of patients permanently discontinuing bosutinib because of toxicity.

Study details: Findings are from a retrospective analysis of 101 elderly (age, older than 65 years) patients with CML-CP treated with bosutinib in second or subsequent line. Patients switched to bosutinib because of intolerance (n=46) or resistance (n=55) to previous TKI therapies.

Disclosures: This study did not receive any type of funding. Some investigators including the lead author reported consulting for or receiving honoraria from various pharmaceutical companies.

Source: Latagliata R et al. Hematol Oncol. 2021 Feb 22. doi: 10.1002/hon.2851.

Key clinical point: Even after effective treatment with tyrosine kinase inhibitors, patients with chronic myeloid leukemia (CML) had notably altered global quality of life (QoL) and health utility scores.

Major finding: Compared with the general population, patients with CML had notably affected QoL in terms of social, role, and cognitive functioning with a mean difference of −16.0, −13.1, and −11.7, respectively. The utility score had a deviation from the reference norm of −0.15 on an average (standard deviation, 0.25) compared with the general population.

Study details: Findings are from a prospective web-based survey that assessed QoL and health utility scores in 383 patients with CML (92% in chronic-phase) in France.

Disclosures: This study was funded by the French National Agency for Research and the French Institute for Public Health Research. The authors declared no conflicts of interest.

Source: Foulon S et al. Qual Life Res. 2021 Mar 2. doi: 10.1007/s11136-021-02794-5.

Key clinical point: Even after effective treatment with tyrosine kinase inhibitors, patients with chronic myeloid leukemia (CML) had notably altered global quality of life (QoL) and health utility scores.

Major finding: Compared with the general population, patients with CML had notably affected QoL in terms of social, role, and cognitive functioning with a mean difference of −16.0, −13.1, and −11.7, respectively. The utility score had a deviation from the reference norm of −0.15 on an average (standard deviation, 0.25) compared with the general population.

Study details: Findings are from a prospective web-based survey that assessed QoL and health utility scores in 383 patients with CML (92% in chronic-phase) in France.

Disclosures: This study was funded by the French National Agency for Research and the French Institute for Public Health Research. The authors declared no conflicts of interest.

Source: Foulon S et al. Qual Life Res. 2021 Mar 2. doi: 10.1007/s11136-021-02794-5.

Key clinical point: Even after effective treatment with tyrosine kinase inhibitors, patients with chronic myeloid leukemia (CML) had notably altered global quality of life (QoL) and health utility scores.

Major finding: Compared with the general population, patients with CML had notably affected QoL in terms of social, role, and cognitive functioning with a mean difference of −16.0, −13.1, and −11.7, respectively. The utility score had a deviation from the reference norm of −0.15 on an average (standard deviation, 0.25) compared with the general population.

Study details: Findings are from a prospective web-based survey that assessed QoL and health utility scores in 383 patients with CML (92% in chronic-phase) in France.

Disclosures: This study was funded by the French National Agency for Research and the French Institute for Public Health Research. The authors declared no conflicts of interest.

Source: Foulon S et al. Qual Life Res. 2021 Mar 2. doi: 10.1007/s11136-021-02794-5.

Key clinical point: Combination of dasatinib and nivolumab was safe but did not show any clinical activity in patients with difficult-to-treat chronic myeloid leukemia (CML) in chronic phase (CP) or accelerated phase (AP) who received at least 2 prior tyrosine kinase inhibitors (TKIs).

Major finding: No drug limiting toxicities were observed even with the maximum dose of both drugs. The most common grade 3 or higher adverse event (AE) of any cause was diarrhea (13%), of which only 3% were considered drug related. Serious AEs occurred in 48% of patients; however, only 2 were considered drug related. The overall response rate was low with none being durable.

Study details: Findings are from a phase 1b dose-escalation study involving 31 patients with CML in CP (n=24) or AP (n=7) who received 2 or more prior TKIs and showed progression, resistance, suboptimal response, or intolerance to the most recent therapy.

Disclosures: This trial was funded by Bristol Myers Squibb, Princeton, NJ, USA. The lead author along with other authors declared receiving grants, advisory boards, honoraria, and/or research funding from various pharmaceutical companies including Bristol Myers Squibb. R Swanink and PM Regueira were employees of Bristol Myers Squibb.

Source: Martínez-López J et al. Leuk Lymphoma. 2021 Mar 2. doi: 10.1080/10428194.2021.1889536.

Key clinical point: Combination of dasatinib and nivolumab was safe but did not show any clinical activity in patients with difficult-to-treat chronic myeloid leukemia (CML) in chronic phase (CP) or accelerated phase (AP) who received at least 2 prior tyrosine kinase inhibitors (TKIs).

Major finding: No drug limiting toxicities were observed even with the maximum dose of both drugs. The most common grade 3 or higher adverse event (AE) of any cause was diarrhea (13%), of which only 3% were considered drug related. Serious AEs occurred in 48% of patients; however, only 2 were considered drug related. The overall response rate was low with none being durable.

Study details: Findings are from a phase 1b dose-escalation study involving 31 patients with CML in CP (n=24) or AP (n=7) who received 2 or more prior TKIs and showed progression, resistance, suboptimal response, or intolerance to the most recent therapy.

Disclosures: This trial was funded by Bristol Myers Squibb, Princeton, NJ, USA. The lead author along with other authors declared receiving grants, advisory boards, honoraria, and/or research funding from various pharmaceutical companies including Bristol Myers Squibb. R Swanink and PM Regueira were employees of Bristol Myers Squibb.

Source: Martínez-López J et al. Leuk Lymphoma. 2021 Mar 2. doi: 10.1080/10428194.2021.1889536.

Key clinical point: Combination of dasatinib and nivolumab was safe but did not show any clinical activity in patients with difficult-to-treat chronic myeloid leukemia (CML) in chronic phase (CP) or accelerated phase (AP) who received at least 2 prior tyrosine kinase inhibitors (TKIs).

Major finding: No drug limiting toxicities were observed even with the maximum dose of both drugs. The most common grade 3 or higher adverse event (AE) of any cause was diarrhea (13%), of which only 3% were considered drug related. Serious AEs occurred in 48% of patients; however, only 2 were considered drug related. The overall response rate was low with none being durable.

Study details: Findings are from a phase 1b dose-escalation study involving 31 patients with CML in CP (n=24) or AP (n=7) who received 2 or more prior TKIs and showed progression, resistance, suboptimal response, or intolerance to the most recent therapy.

Disclosures: This trial was funded by Bristol Myers Squibb, Princeton, NJ, USA. The lead author along with other authors declared receiving grants, advisory boards, honoraria, and/or research funding from various pharmaceutical companies including Bristol Myers Squibb. R Swanink and PM Regueira were employees of Bristol Myers Squibb.

Source: Martínez-López J et al. Leuk Lymphoma. 2021 Mar 2. doi: 10.1080/10428194.2021.1889536.

Key clinical point: Imatinib treatment following interferon-α failure showed long-term efficacy in a real-life setting of patients with late chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: After a median of 4.5 months of therapy initiation, 84% of patients achieved complete cytogenic response as their best cytogenetic response. The estimated 18-year overall survival, event-free survival, and progression-free survival were 64.8%, 69%, and 64.4%, respectively. Imatinib discontinuation and CML-related deaths occurred in 36% and 34% of patients, respectively, and 86% of patients were in treatment-free remission at the last follow-up.

Study details: Findings are from a retrospective analysis of 139 patients with late CML-CP treated with imatinib following interferon-α therapy failure followed up for a median of 16.6 years.

Disclosures: No specific funding source was identified. The authors declared no conflicts of interest.

Source: Pepe S et al. Leuk Lymphoma. 2021 Mar 16. doi: 10.1080/10428194.2021.1901094.

Key clinical point: Imatinib treatment following interferon-α failure showed long-term efficacy in a real-life setting of patients with late chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: After a median of 4.5 months of therapy initiation, 84% of patients achieved complete cytogenic response as their best cytogenetic response. The estimated 18-year overall survival, event-free survival, and progression-free survival were 64.8%, 69%, and 64.4%, respectively. Imatinib discontinuation and CML-related deaths occurred in 36% and 34% of patients, respectively, and 86% of patients were in treatment-free remission at the last follow-up.

Study details: Findings are from a retrospective analysis of 139 patients with late CML-CP treated with imatinib following interferon-α therapy failure followed up for a median of 16.6 years.

Disclosures: No specific funding source was identified. The authors declared no conflicts of interest.

Source: Pepe S et al. Leuk Lymphoma. 2021 Mar 16. doi: 10.1080/10428194.2021.1901094.

Key clinical point: Imatinib treatment following interferon-α failure showed long-term efficacy in a real-life setting of patients with late chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: After a median of 4.5 months of therapy initiation, 84% of patients achieved complete cytogenic response as their best cytogenetic response. The estimated 18-year overall survival, event-free survival, and progression-free survival were 64.8%, 69%, and 64.4%, respectively. Imatinib discontinuation and CML-related deaths occurred in 36% and 34% of patients, respectively, and 86% of patients were in treatment-free remission at the last follow-up.

Study details: Findings are from a retrospective analysis of 139 patients with late CML-CP treated with imatinib following interferon-α therapy failure followed up for a median of 16.6 years.

Disclosures: No specific funding source was identified. The authors declared no conflicts of interest.

Source: Pepe S et al. Leuk Lymphoma. 2021 Mar 16. doi: 10.1080/10428194.2021.1901094.

Key clinical point: Dasatinib and nilotinib as second-line treatments were equally effective, with a high molecular response (MR) and a tolerable safety profile in real-life patients with chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 12 months, 47% and 38% of patients had a major MR and 18.2% and 16.6% had a deep MR in the dasatinib and nilotinib groups, respectively (P = .481). Grade 3-4 adverse events were more frequent in dasatinib vs. nilotinib groups (P = .003) with no effect on MR.

Study details: Findings are from a retrospective analysis of 131 patients with CML-CP who switched to second-line treatment with either dasatinib (n=72) or nilotinib (n=59) after frontline imatinib intolerance/resistance.

Disclosures: The authors did not declare any source of funding. M Breccia, M Martelli, and F Efficace reported receiving honoraria, personal fees, and grants from and being on the advisory board for various pharmaceutical companies. Other authors had no disclosures.

Source: Scalzulli E et al. Ann Hematol. 2021 Mar 7. doi: 10.1007/s00277-021-04477-0.

Key clinical point: Dasatinib and nilotinib as second-line treatments were equally effective, with a high molecular response (MR) and a tolerable safety profile in real-life patients with chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 12 months, 47% and 38% of patients had a major MR and 18.2% and 16.6% had a deep MR in the dasatinib and nilotinib groups, respectively (P = .481). Grade 3-4 adverse events were more frequent in dasatinib vs. nilotinib groups (P = .003) with no effect on MR.

Study details: Findings are from a retrospective analysis of 131 patients with CML-CP who switched to second-line treatment with either dasatinib (n=72) or nilotinib (n=59) after frontline imatinib intolerance/resistance.

Disclosures: The authors did not declare any source of funding. M Breccia, M Martelli, and F Efficace reported receiving honoraria, personal fees, and grants from and being on the advisory board for various pharmaceutical companies. Other authors had no disclosures.

Source: Scalzulli E et al. Ann Hematol. 2021 Mar 7. doi: 10.1007/s00277-021-04477-0.

Key clinical point: Dasatinib and nilotinib as second-line treatments were equally effective, with a high molecular response (MR) and a tolerable safety profile in real-life patients with chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 12 months, 47% and 38% of patients had a major MR and 18.2% and 16.6% had a deep MR in the dasatinib and nilotinib groups, respectively (P = .481). Grade 3-4 adverse events were more frequent in dasatinib vs. nilotinib groups (P = .003) with no effect on MR.

Study details: Findings are from a retrospective analysis of 131 patients with CML-CP who switched to second-line treatment with either dasatinib (n=72) or nilotinib (n=59) after frontline imatinib intolerance/resistance.

Disclosures: The authors did not declare any source of funding. M Breccia, M Martelli, and F Efficace reported receiving honoraria, personal fees, and grants from and being on the advisory board for various pharmaceutical companies. Other authors had no disclosures.

Source: Scalzulli E et al. Ann Hematol. 2021 Mar 7. doi: 10.1007/s00277-021-04477-0.

Key clinical point: More than 3 years of frontline nilotinib treatment was effective with sustained long-term high treatment-free remission (TFR) rates and manageable safety in patients with Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: Among patients who entered TFR, 41.6% remained in major molecular response (MMR), with 40.0% in MR^4.5, whereas 98.9% of patients who lost MMR regained after treatment reinitiation. No disease progression or CML-related deaths were observed, and the adverse event profile was consistent with that reported previously.

Study details: Findings are from a 5-year follow-up of phase 2 ENESTfreedom trial including 190 adult patients with Ph+ CML-CP who received at least 2 years of frontline nilotinib treatment, achieved MR^4.5, and attempted TFR after undergoing a 1-year nilotinib treatment consolidation phase.

Disclosures: This study was funded by Novartis Pharmaceuticals. The lead author along with other authors reported ties with various pharmaceutical companies including Novartis. P Aimone, S Li, and K Titorenko reported being employees of Novartis.

Source: Radich JP et al. Leukemia. 2021 Mar 11. doi: 10.1038/s41375-021-01205-5.

Key clinical point: More than 3 years of frontline nilotinib treatment was effective with sustained long-term high treatment-free remission (TFR) rates and manageable safety in patients with Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: Among patients who entered TFR, 41.6% remained in major molecular response (MMR), with 40.0% in MR^4.5, whereas 98.9% of patients who lost MMR regained after treatment reinitiation. No disease progression or CML-related deaths were observed, and the adverse event profile was consistent with that reported previously.

Study details: Findings are from a 5-year follow-up of phase 2 ENESTfreedom trial including 190 adult patients with Ph+ CML-CP who received at least 2 years of frontline nilotinib treatment, achieved MR^4.5, and attempted TFR after undergoing a 1-year nilotinib treatment consolidation phase.

Disclosures: This study was funded by Novartis Pharmaceuticals. The lead author along with other authors reported ties with various pharmaceutical companies including Novartis. P Aimone, S Li, and K Titorenko reported being employees of Novartis.

Source: Radich JP et al. Leukemia. 2021 Mar 11. doi: 10.1038/s41375-021-01205-5.

Key clinical point: More than 3 years of frontline nilotinib treatment was effective with sustained long-term high treatment-free remission (TFR) rates and manageable safety in patients with Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: Among patients who entered TFR, 41.6% remained in major molecular response (MMR), with 40.0% in MR^4.5, whereas 98.9% of patients who lost MMR regained after treatment reinitiation. No disease progression or CML-related deaths were observed, and the adverse event profile was consistent with that reported previously.

Study details: Findings are from a 5-year follow-up of phase 2 ENESTfreedom trial including 190 adult patients with Ph+ CML-CP who received at least 2 years of frontline nilotinib treatment, achieved MR^4.5, and attempted TFR after undergoing a 1-year nilotinib treatment consolidation phase.

Disclosures: This study was funded by Novartis Pharmaceuticals. The lead author along with other authors reported ties with various pharmaceutical companies including Novartis. P Aimone, S Li, and K Titorenko reported being employees of Novartis.

Source: Radich JP et al. Leukemia. 2021 Mar 11. doi: 10.1038/s41375-021-01205-5.

Key clinical point: Initial rate of BCR-ABL1 decline, measured as halving time, was a strong predictor of sustained treatment-free remission (TFR) post-tyrosine kinase inhibitor (TKI) cessation in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with firstline TKI therapies.

Major finding: Patients with a sustained TFR had a shorter BCR-ABL1 halving time vs. those with molecular relapse (10.1 vs. 21.7 days; P less than .001). The probability of sustained TFR was 80% vs. 4% among patients with halving time less than 9.35 days vs. more than 21.85 days (P less than .001).

Study details: Findings are from a retrospective analysis of 115 adult patients with CML-CP who attempted TFR and were followed up for at least 12 months post-TKI discontinuation.

Disclosures: The authors did not declare any source of funding. Some of the investigators including the lead author reported receiving honoraria and travel and accommodation expenses; being on the advisory board; and receiving research funding from various pharmaceutical companies.

Source: Shanmuganathan N et al. Blood. 2021 Mar 4. doi: 10.1182/blood.2020005514.

Key clinical point: Initial rate of BCR-ABL1 decline, measured as halving time, was a strong predictor of sustained treatment-free remission (TFR) post-tyrosine kinase inhibitor (TKI) cessation in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with firstline TKI therapies.

Major finding: Patients with a sustained TFR had a shorter BCR-ABL1 halving time vs. those with molecular relapse (10.1 vs. 21.7 days; P less than .001). The probability of sustained TFR was 80% vs. 4% among patients with halving time less than 9.35 days vs. more than 21.85 days (P less than .001).

Study details: Findings are from a retrospective analysis of 115 adult patients with CML-CP who attempted TFR and were followed up for at least 12 months post-TKI discontinuation.

Disclosures: The authors did not declare any source of funding. Some of the investigators including the lead author reported receiving honoraria and travel and accommodation expenses; being on the advisory board; and receiving research funding from various pharmaceutical companies.

Source: Shanmuganathan N et al. Blood. 2021 Mar 4. doi: 10.1182/blood.2020005514.

Key clinical point: Initial rate of BCR-ABL1 decline, measured as halving time, was a strong predictor of sustained treatment-free remission (TFR) post-tyrosine kinase inhibitor (TKI) cessation in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with firstline TKI therapies.

Major finding: Patients with a sustained TFR had a shorter BCR-ABL1 halving time vs. those with molecular relapse (10.1 vs. 21.7 days; P less than .001). The probability of sustained TFR was 80% vs. 4% among patients with halving time less than 9.35 days vs. more than 21.85 days (P less than .001).

Study details: Findings are from a retrospective analysis of 115 adult patients with CML-CP who attempted TFR and were followed up for at least 12 months post-TKI discontinuation.

Disclosures: The authors did not declare any source of funding. Some of the investigators including the lead author reported receiving honoraria and travel and accommodation expenses; being on the advisory board; and receiving research funding from various pharmaceutical companies.

Source: Shanmuganathan N et al. Blood. 2021 Mar 4. doi: 10.1182/blood.2020005514.

Key clinical point: High red blood cell distribution width (RDW) at diagnosis was associated with poor prognosis and treatment response in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with tyrosine kinase inhibitors (TKIs).

Major finding: High RDW was a significant predictor of poor overall survival (hazard ratio, [HR], 9.741; P = .005) and progression-free survival (HR, 16.74; P = .009). Patients with high RDW had worse treatment responses at 3 months (P = .03) and 6 months (P = .02).

Study details: Findings are from a retrospective analysis of 93 patients with newly diagnosed CML-CP and treated with TKIs. Patients were categorized into low (18.65% or lesser; n=58) and high (more than 18.65%; n=35) RDW groups.

Disclosures: No funding source was reported. The authors declared no conflicts of interest.

Source: Mao XL et al. Medicine. 2021 Mar 12. doi: 10.1097/MD.0000000000024003.

Key clinical point: High red blood cell distribution width (RDW) at diagnosis was associated with poor prognosis and treatment response in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with tyrosine kinase inhibitors (TKIs).

Major finding: High RDW was a significant predictor of poor overall survival (hazard ratio, [HR], 9.741; P = .005) and progression-free survival (HR, 16.74; P = .009). Patients with high RDW had worse treatment responses at 3 months (P = .03) and 6 months (P = .02).

Study details: Findings are from a retrospective analysis of 93 patients with newly diagnosed CML-CP and treated with TKIs. Patients were categorized into low (18.65% or lesser; n=58) and high (more than 18.65%; n=35) RDW groups.

Disclosures: No funding source was reported. The authors declared no conflicts of interest.

Source: Mao XL et al. Medicine. 2021 Mar 12. doi: 10.1097/MD.0000000000024003.

Key clinical point: High red blood cell distribution width (RDW) at diagnosis was associated with poor prognosis and treatment response in patients with chronic-phase chronic myeloid leukemia (CML-CP) treated with tyrosine kinase inhibitors (TKIs).

Major finding: High RDW was a significant predictor of poor overall survival (hazard ratio, [HR], 9.741; P = .005) and progression-free survival (HR, 16.74; P = .009). Patients with high RDW had worse treatment responses at 3 months (P = .03) and 6 months (P = .02).

Study details: Findings are from a retrospective analysis of 93 patients with newly diagnosed CML-CP and treated with TKIs. Patients were categorized into low (18.65% or lesser; n=58) and high (more than 18.65%; n=35) RDW groups.

Disclosures: No funding source was reported. The authors declared no conflicts of interest.

Source: Mao XL et al. Medicine. 2021 Mar 12. doi: 10.1097/MD.0000000000024003.

Among individuals who have received a hematopoietic stem cell transplant (HSCT), often used in the treatment of blood cancers, rates of survival are poor for those who develop COVID-19.

The probability of survival 30 days after being diagnosed with COVID-19 is only 68% for persons who have received an allogeneic HSCT and 67% for autologous HSCT recipients, according to new data from the Center for International Blood and Marrow Transplant Research.

These findings underscore the need for “stringent surveillance and aggressive treatment measures” in this population, Akshay Sharma, MBBS, of St. Jude Children’s Research Hospital, Memphis, and colleagues wrote.

The findings were published online March 1, 2021, in The Lancet Haematology.

The study is “of importance for physicians caring for HSCT recipients worldwide,” Mathieu Leclerc, MD, and Sébastien Maury, MD, Hôpital Henri Mondor, Créteil, France, commented in an accompanying editorial.
 

Study details

For their study, Dr. Sharma and colleagues analyzed outcomes for all HSCT recipients who developed COVID-19 and whose cases were reported to the CIBMTR. Of 318 such patients, 184 had undergone allogeneic HSCT, and 134 had undergone autologous HSCT.

Overall, about half of these patients (49%) had mild COVID-19.

Severe COVID-19 that required mechanical ventilation developed in 15% and 13% of the allogeneic and autologous HSCT recipients, respectively.

About one-fifth of patients died: 22% and 19% of allogeneic and autologous HSCT recipients, respectively.

Factors associated with greater mortality risk included age of 50 years or older (hazard ratio, 2.53), male sex (HR, 3.53), and development of COVID-19 within 12 months of undergoing HSCT (HR, 2.67).

Among autologous HSCT recipients, lymphoma was associated with higher mortality risk in comparison with a plasma cell disorder or myeloma (HR, 2.41), the authors noted.

“Two important messages can be drawn from the results reported by Sharma and colleagues,” Dr. Leclerc and Dr. Maury wrote in their editorial. “The first is the confirmation that the prognosis of COVID-19 is particularly poor in HSCT recipients, and that its prevention, in the absence of any specific curative treatment with sufficient efficacy, should be at the forefront of concerns.”

The second relates to the risk factors for death among HSCT recipients who develop COVID-19. In addition to previously known risk factors, such as age and gender, the investigators identified transplant-specific factors potentially associated with prognosis – namely, the nearly threefold increase in death among allogeneic HSCT recipients who develop COVID-19 within 12 months of transplant, they explained.

However, the findings are limited by a substantial amount of missing data, short follow-up, and the possibility of selection bias, they noted.

“Further large and well-designed studies with longer follow-up are needed to confirm and refine the results,” the editorialists wrote.

“[A] better understanding of the distinctive features of COVID-19 infection in HSCT recipients will be a necessary and essential step toward improvement of the remarkably poor prognosis observed in this setting,” they added.

The study was funded by the American Society of Hematology; the Leukemia and Lymphoma Society; the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institutes of Health; the Health Resources and Services Administration; and the Office of Naval Research. Dr. Sharma receives support for the conduct of industry-sponsored trials from Vertex Pharmaceuticals, CRISPR Therapeutics, and Novartis and consulting fees from Spotlight Therapeutics. Dr. Leclerc and Dr. Maury disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Among individuals who have received a hematopoietic stem cell transplant (HSCT), often used in the treatment of blood cancers, rates of survival are poor for those who develop COVID-19.

The probability of survival 30 days after being diagnosed with COVID-19 is only 68% for persons who have received an allogeneic HSCT and 67% for autologous HSCT recipients, according to new data from the Center for International Blood and Marrow Transplant Research.

These findings underscore the need for “stringent surveillance and aggressive treatment measures” in this population, Akshay Sharma, MBBS, of St. Jude Children’s Research Hospital, Memphis, and colleagues wrote.

The findings were published online March 1, 2021, in The Lancet Haematology.

The study is “of importance for physicians caring for HSCT recipients worldwide,” Mathieu Leclerc, MD, and Sébastien Maury, MD, Hôpital Henri Mondor, Créteil, France, commented in an accompanying editorial.
 

Study details

For their study, Dr. Sharma and colleagues analyzed outcomes for all HSCT recipients who developed COVID-19 and whose cases were reported to the CIBMTR. Of 318 such patients, 184 had undergone allogeneic HSCT, and 134 had undergone autologous HSCT.

Overall, about half of these patients (49%) had mild COVID-19.

Severe COVID-19 that required mechanical ventilation developed in 15% and 13% of the allogeneic and autologous HSCT recipients, respectively.

About one-fifth of patients died: 22% and 19% of allogeneic and autologous HSCT recipients, respectively.

Factors associated with greater mortality risk included age of 50 years or older (hazard ratio, 2.53), male sex (HR, 3.53), and development of COVID-19 within 12 months of undergoing HSCT (HR, 2.67).

Among autologous HSCT recipients, lymphoma was associated with higher mortality risk in comparison with a plasma cell disorder or myeloma (HR, 2.41), the authors noted.

“Two important messages can be drawn from the results reported by Sharma and colleagues,” Dr. Leclerc and Dr. Maury wrote in their editorial. “The first is the confirmation that the prognosis of COVID-19 is particularly poor in HSCT recipients, and that its prevention, in the absence of any specific curative treatment with sufficient efficacy, should be at the forefront of concerns.”

The second relates to the risk factors for death among HSCT recipients who develop COVID-19. In addition to previously known risk factors, such as age and gender, the investigators identified transplant-specific factors potentially associated with prognosis – namely, the nearly threefold increase in death among allogeneic HSCT recipients who develop COVID-19 within 12 months of transplant, they explained.

However, the findings are limited by a substantial amount of missing data, short follow-up, and the possibility of selection bias, they noted.

“Further large and well-designed studies with longer follow-up are needed to confirm and refine the results,” the editorialists wrote.

“[A] better understanding of the distinctive features of COVID-19 infection in HSCT recipients will be a necessary and essential step toward improvement of the remarkably poor prognosis observed in this setting,” they added.

The study was funded by the American Society of Hematology; the Leukemia and Lymphoma Society; the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institutes of Health; the Health Resources and Services Administration; and the Office of Naval Research. Dr. Sharma receives support for the conduct of industry-sponsored trials from Vertex Pharmaceuticals, CRISPR Therapeutics, and Novartis and consulting fees from Spotlight Therapeutics. Dr. Leclerc and Dr. Maury disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Among individuals who have received a hematopoietic stem cell transplant (HSCT), often used in the treatment of blood cancers, rates of survival are poor for those who develop COVID-19.

The probability of survival 30 days after being diagnosed with COVID-19 is only 68% for persons who have received an allogeneic HSCT and 67% for autologous HSCT recipients, according to new data from the Center for International Blood and Marrow Transplant Research.

These findings underscore the need for “stringent surveillance and aggressive treatment measures” in this population, Akshay Sharma, MBBS, of St. Jude Children’s Research Hospital, Memphis, and colleagues wrote.

The findings were published online March 1, 2021, in The Lancet Haematology.

The study is “of importance for physicians caring for HSCT recipients worldwide,” Mathieu Leclerc, MD, and Sébastien Maury, MD, Hôpital Henri Mondor, Créteil, France, commented in an accompanying editorial.
 

Study details

For their study, Dr. Sharma and colleagues analyzed outcomes for all HSCT recipients who developed COVID-19 and whose cases were reported to the CIBMTR. Of 318 such patients, 184 had undergone allogeneic HSCT, and 134 had undergone autologous HSCT.

Overall, about half of these patients (49%) had mild COVID-19.

Severe COVID-19 that required mechanical ventilation developed in 15% and 13% of the allogeneic and autologous HSCT recipients, respectively.

About one-fifth of patients died: 22% and 19% of allogeneic and autologous HSCT recipients, respectively.

Factors associated with greater mortality risk included age of 50 years or older (hazard ratio, 2.53), male sex (HR, 3.53), and development of COVID-19 within 12 months of undergoing HSCT (HR, 2.67).

Among autologous HSCT recipients, lymphoma was associated with higher mortality risk in comparison with a plasma cell disorder or myeloma (HR, 2.41), the authors noted.

“Two important messages can be drawn from the results reported by Sharma and colleagues,” Dr. Leclerc and Dr. Maury wrote in their editorial. “The first is the confirmation that the prognosis of COVID-19 is particularly poor in HSCT recipients, and that its prevention, in the absence of any specific curative treatment with sufficient efficacy, should be at the forefront of concerns.”

The second relates to the risk factors for death among HSCT recipients who develop COVID-19. In addition to previously known risk factors, such as age and gender, the investigators identified transplant-specific factors potentially associated with prognosis – namely, the nearly threefold increase in death among allogeneic HSCT recipients who develop COVID-19 within 12 months of transplant, they explained.

However, the findings are limited by a substantial amount of missing data, short follow-up, and the possibility of selection bias, they noted.

“Further large and well-designed studies with longer follow-up are needed to confirm and refine the results,” the editorialists wrote.

“[A] better understanding of the distinctive features of COVID-19 infection in HSCT recipients will be a necessary and essential step toward improvement of the remarkably poor prognosis observed in this setting,” they added.

The study was funded by the American Society of Hematology; the Leukemia and Lymphoma Society; the National Cancer Institute; the National Heart, Lung and Blood Institute; the National Institute of Allergy and Infectious Diseases; the National Institutes of Health; the Health Resources and Services Administration; and the Office of Naval Research. Dr. Sharma receives support for the conduct of industry-sponsored trials from Vertex Pharmaceuticals, CRISPR Therapeutics, and Novartis and consulting fees from Spotlight Therapeutics. Dr. Leclerc and Dr. Maury disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Omidubicel, an investigational enriched umbilical cord blood product being developed by Gamida Cell for transplantation in patients with blood cancers, appears to have some advantages over standard umbilical cord blood.

The results come from a global phase 3 trial (NCT02730299) presented at the annual meeting of the European Society for Blood and Bone Marrow Transplantation.

“Transplantation with omidubicel, compared to standard cord blood transplantation, results in faster hematopoietic recovery, fewer infections, and fewer days in hospital,” said coinvestigator Guillermo F. Sanz, MD, PhD, from the Hospital Universitari i Politècnic la Fe in Valencia, Spain.

“Omidubicel should be considered as the new standard of care for patients eligible for umbilical cord blood transplantation,” Dr. Sanz concluded.

Zachariah DeFilipp, MD, from Mass General Cancer Center in Boston, a hematopoietic stem cell transplantation specialist who was not involved in the study, said in an interview that “omidubicel significantly improves the engraftment after transplant, as compared to standard cord blood transplant. For patients that lack an HLA-matched donor, this approach can help overcome the prolonged cytopenias that occur with standard cord blood transplants in adults.”

Gamida Cell plans to submit these data for approval of omidubicel by the Food and Drug Administration in the fourth quarter of 2021.

Omidubicel is also being evaluated in a phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937).
 

Expanding possibilities

Although umbilical cord blood stem cell grafts come from a readily available source and show greater tolerance across HLA barriers than other sources (such as bone marrow), the relatively low dose of stem cells in each unit results in delayed hematopoietic recovery, increased transplant-related morbidity and mortality, and longer hospitalizations, Dr. Sanz said.

Omidubicel consists of two cryopreserved fractions from a single cord blood unit. The product contains both noncultured CD133-negative cells, including T cells, and CD133-positive cells that are then expanded ex vivo for 21 days in the presence of nicotinamide.

“Nicotinamide increases stem and progenitor cells, inhibits differentiation and increases migration, bone marrow homing, and engraftment efficiency while preserving cellular functionality and phenotype,” Dr. Sanz explained during his presentation.

In an earlier phase 1/2 trial in 36 patients with high-risk hematologic malignancies, omidubicel was associated with hematopoietic engraftment lasting at least 10 years.
 

Details of phase 3 trial results

The global phase 3 trial was conducted in 125 patients (aged 13-65 years) with high-risk malignancies, including acute myeloid and lymphoblastic leukemias, myelodysplastic syndrome, chronic myeloid leukemia, lymphomas, and rare leukemias. These patients were all eligible for allogeneic stem cell transplantation but did not have matched donors.

Patients were randomly assigned to receive hematopoietic reconstitution with either omidubicel (n = 52) or standard cord blood (n = 58).

At 42 days of follow-up, the median time to neutrophil engraftment in the intention-to-treat (ITT) population, the primary endpoint, was 12 days with omidubicel versus 22 days with standard cord blood (P < .001).

In the as-treated population – the 108 patients who actually received omidubicel or standard cord blood – median time to engraftment was 10.0 versus 20.5 days, respectively (P < .001).

Rates of neutrophil engraftment at 42 days were 96% with omidubicel versus 89% with standard cord blood.

The secondary endpoint of time-to-platelet engraftment in the ITT population also favored omidubicel, with a cumulative day 42 incidence rate of 55%, compared with 35% with standard cord blood (P = .028).

In the as-treated population, median times to platelet engraftment were 37 days and 50 days, respectively (P = .023). The cumulative rates of platelet engraftment at 100 days of follow-up were 83% and 73%, respectively.

The incidence of grade 2 or 3 bacterial or invasive fungal infections by day 100 in the ITT population was 37% among patients who received omidubicel, compared with 57% for patients who received standard cord blood (P = .027). Viral infections occurred in 10% versus 26% of patients, respectively.

The incidence of acute graft versus host disease at day 100 was similar between treatment groups, and there was no significant difference at 1 year.

Relapse and nonrelapse mortality rates, as well as disease-free and overall survival rates also did not differ between groups.

In the first 100 days post transplant, patients who received omidubicel were alive and out of the hospital for a median of 60.5 days, compared with 48 days for patients who received standard cord blood (P = .005).

The study was funded by Gamida Cell. Dr. Sanz reported receiving research funding from the company and several others, and consulting fees, honoraria, speakers bureau activity, and travel expenses from other companies. Dr. DeFilipp reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Omidubicel, an investigational enriched umbilical cord blood product being developed by Gamida Cell for transplantation in patients with blood cancers, appears to have some advantages over standard umbilical cord blood.

The results come from a global phase 3 trial (NCT02730299) presented at the annual meeting of the European Society for Blood and Bone Marrow Transplantation.

“Transplantation with omidubicel, compared to standard cord blood transplantation, results in faster hematopoietic recovery, fewer infections, and fewer days in hospital,” said coinvestigator Guillermo F. Sanz, MD, PhD, from the Hospital Universitari i Politècnic la Fe in Valencia, Spain.

“Omidubicel should be considered as the new standard of care for patients eligible for umbilical cord blood transplantation,” Dr. Sanz concluded.

Zachariah DeFilipp, MD, from Mass General Cancer Center in Boston, a hematopoietic stem cell transplantation specialist who was not involved in the study, said in an interview that “omidubicel significantly improves the engraftment after transplant, as compared to standard cord blood transplant. For patients that lack an HLA-matched donor, this approach can help overcome the prolonged cytopenias that occur with standard cord blood transplants in adults.”

Gamida Cell plans to submit these data for approval of omidubicel by the Food and Drug Administration in the fourth quarter of 2021.

Omidubicel is also being evaluated in a phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937).
 

Expanding possibilities

Although umbilical cord blood stem cell grafts come from a readily available source and show greater tolerance across HLA barriers than other sources (such as bone marrow), the relatively low dose of stem cells in each unit results in delayed hematopoietic recovery, increased transplant-related morbidity and mortality, and longer hospitalizations, Dr. Sanz said.

Omidubicel consists of two cryopreserved fractions from a single cord blood unit. The product contains both noncultured CD133-negative cells, including T cells, and CD133-positive cells that are then expanded ex vivo for 21 days in the presence of nicotinamide.

“Nicotinamide increases stem and progenitor cells, inhibits differentiation and increases migration, bone marrow homing, and engraftment efficiency while preserving cellular functionality and phenotype,” Dr. Sanz explained during his presentation.

In an earlier phase 1/2 trial in 36 patients with high-risk hematologic malignancies, omidubicel was associated with hematopoietic engraftment lasting at least 10 years.
 

Details of phase 3 trial results

The global phase 3 trial was conducted in 125 patients (aged 13-65 years) with high-risk malignancies, including acute myeloid and lymphoblastic leukemias, myelodysplastic syndrome, chronic myeloid leukemia, lymphomas, and rare leukemias. These patients were all eligible for allogeneic stem cell transplantation but did not have matched donors.

Patients were randomly assigned to receive hematopoietic reconstitution with either omidubicel (n = 52) or standard cord blood (n = 58).

At 42 days of follow-up, the median time to neutrophil engraftment in the intention-to-treat (ITT) population, the primary endpoint, was 12 days with omidubicel versus 22 days with standard cord blood (P < .001).

In the as-treated population – the 108 patients who actually received omidubicel or standard cord blood – median time to engraftment was 10.0 versus 20.5 days, respectively (P < .001).

Rates of neutrophil engraftment at 42 days were 96% with omidubicel versus 89% with standard cord blood.

The secondary endpoint of time-to-platelet engraftment in the ITT population also favored omidubicel, with a cumulative day 42 incidence rate of 55%, compared with 35% with standard cord blood (P = .028).

In the as-treated population, median times to platelet engraftment were 37 days and 50 days, respectively (P = .023). The cumulative rates of platelet engraftment at 100 days of follow-up were 83% and 73%, respectively.

The incidence of grade 2 or 3 bacterial or invasive fungal infections by day 100 in the ITT population was 37% among patients who received omidubicel, compared with 57% for patients who received standard cord blood (P = .027). Viral infections occurred in 10% versus 26% of patients, respectively.

The incidence of acute graft versus host disease at day 100 was similar between treatment groups, and there was no significant difference at 1 year.

Relapse and nonrelapse mortality rates, as well as disease-free and overall survival rates also did not differ between groups.

In the first 100 days post transplant, patients who received omidubicel were alive and out of the hospital for a median of 60.5 days, compared with 48 days for patients who received standard cord blood (P = .005).

The study was funded by Gamida Cell. Dr. Sanz reported receiving research funding from the company and several others, and consulting fees, honoraria, speakers bureau activity, and travel expenses from other companies. Dr. DeFilipp reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Omidubicel, an investigational enriched umbilical cord blood product being developed by Gamida Cell for transplantation in patients with blood cancers, appears to have some advantages over standard umbilical cord blood.

The results come from a global phase 3 trial (NCT02730299) presented at the annual meeting of the European Society for Blood and Bone Marrow Transplantation.

“Transplantation with omidubicel, compared to standard cord blood transplantation, results in faster hematopoietic recovery, fewer infections, and fewer days in hospital,” said coinvestigator Guillermo F. Sanz, MD, PhD, from the Hospital Universitari i Politècnic la Fe in Valencia, Spain.

“Omidubicel should be considered as the new standard of care for patients eligible for umbilical cord blood transplantation,” Dr. Sanz concluded.

Zachariah DeFilipp, MD, from Mass General Cancer Center in Boston, a hematopoietic stem cell transplantation specialist who was not involved in the study, said in an interview that “omidubicel significantly improves the engraftment after transplant, as compared to standard cord blood transplant. For patients that lack an HLA-matched donor, this approach can help overcome the prolonged cytopenias that occur with standard cord blood transplants in adults.”

Gamida Cell plans to submit these data for approval of omidubicel by the Food and Drug Administration in the fourth quarter of 2021.

Omidubicel is also being evaluated in a phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937).
 

Expanding possibilities

Although umbilical cord blood stem cell grafts come from a readily available source and show greater tolerance across HLA barriers than other sources (such as bone marrow), the relatively low dose of stem cells in each unit results in delayed hematopoietic recovery, increased transplant-related morbidity and mortality, and longer hospitalizations, Dr. Sanz said.

Omidubicel consists of two cryopreserved fractions from a single cord blood unit. The product contains both noncultured CD133-negative cells, including T cells, and CD133-positive cells that are then expanded ex vivo for 21 days in the presence of nicotinamide.

“Nicotinamide increases stem and progenitor cells, inhibits differentiation and increases migration, bone marrow homing, and engraftment efficiency while preserving cellular functionality and phenotype,” Dr. Sanz explained during his presentation.

In an earlier phase 1/2 trial in 36 patients with high-risk hematologic malignancies, omidubicel was associated with hematopoietic engraftment lasting at least 10 years.
 

Details of phase 3 trial results

The global phase 3 trial was conducted in 125 patients (aged 13-65 years) with high-risk malignancies, including acute myeloid and lymphoblastic leukemias, myelodysplastic syndrome, chronic myeloid leukemia, lymphomas, and rare leukemias. These patients were all eligible for allogeneic stem cell transplantation but did not have matched donors.

Patients were randomly assigned to receive hematopoietic reconstitution with either omidubicel (n = 52) or standard cord blood (n = 58).

At 42 days of follow-up, the median time to neutrophil engraftment in the intention-to-treat (ITT) population, the primary endpoint, was 12 days with omidubicel versus 22 days with standard cord blood (P < .001).

In the as-treated population – the 108 patients who actually received omidubicel or standard cord blood – median time to engraftment was 10.0 versus 20.5 days, respectively (P < .001).

Rates of neutrophil engraftment at 42 days were 96% with omidubicel versus 89% with standard cord blood.

The secondary endpoint of time-to-platelet engraftment in the ITT population also favored omidubicel, with a cumulative day 42 incidence rate of 55%, compared with 35% with standard cord blood (P = .028).

In the as-treated population, median times to platelet engraftment were 37 days and 50 days, respectively (P = .023). The cumulative rates of platelet engraftment at 100 days of follow-up were 83% and 73%, respectively.

The incidence of grade 2 or 3 bacterial or invasive fungal infections by day 100 in the ITT population was 37% among patients who received omidubicel, compared with 57% for patients who received standard cord blood (P = .027). Viral infections occurred in 10% versus 26% of patients, respectively.

The incidence of acute graft versus host disease at day 100 was similar between treatment groups, and there was no significant difference at 1 year.

Relapse and nonrelapse mortality rates, as well as disease-free and overall survival rates also did not differ between groups.

In the first 100 days post transplant, patients who received omidubicel were alive and out of the hospital for a median of 60.5 days, compared with 48 days for patients who received standard cord blood (P = .005).

The study was funded by Gamida Cell. Dr. Sanz reported receiving research funding from the company and several others, and consulting fees, honoraria, speakers bureau activity, and travel expenses from other companies. Dr. DeFilipp reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.