Key clinical point: The addition of low-dose pegylated interferon-a2b (PegIFN-a2b) to dasatinib 3 months after initiation of tyrosine kinase inhibitor was effective and well tolerated without an increased risk for late toxicity in patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 5 years, rates of major molecular response (MR), MR4, and MR4.5 were 84.6%, 64.1%, and 51.3%, respectively. No disease progression or CML-related deaths were observed. Grade 2 or higher hematological adverse events were not observed after 12 months. After 24 months, pleural effusion was reported in 10% of patients.

Study details: Findings are from a 5-year follow-up of phase 2 NordCML007 trial including 40 adult patients with newly diagnosed CML-CP who were given upfront dasatinib. Low-dose PegIFN-a2b was added from months 4 to 15 after dasatinib initiation.

Disclosures: This study was sponsored by the Norwegian University of Science and Technology. No disclosures were reported.

Source: Flygt H et al. Eur J Haematol. 2021 Aug 21. doi: 10.1111/ejh.13699.

Key clinical point: The addition of low-dose pegylated interferon-a2b (PegIFN-a2b) to dasatinib 3 months after initiation of tyrosine kinase inhibitor was effective and well tolerated without an increased risk for late toxicity in patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 5 years, rates of major molecular response (MR), MR4, and MR4.5 were 84.6%, 64.1%, and 51.3%, respectively. No disease progression or CML-related deaths were observed. Grade 2 or higher hematological adverse events were not observed after 12 months. After 24 months, pleural effusion was reported in 10% of patients.

Study details: Findings are from a 5-year follow-up of phase 2 NordCML007 trial including 40 adult patients with newly diagnosed CML-CP who were given upfront dasatinib. Low-dose PegIFN-a2b was added from months 4 to 15 after dasatinib initiation.

Disclosures: This study was sponsored by the Norwegian University of Science and Technology. No disclosures were reported.

Source: Flygt H et al. Eur J Haematol. 2021 Aug 21. doi: 10.1111/ejh.13699.

Key clinical point: The addition of low-dose pegylated interferon-a2b (PegIFN-a2b) to dasatinib 3 months after initiation of tyrosine kinase inhibitor was effective and well tolerated without an increased risk for late toxicity in patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML-CP).

Major finding: At 5 years, rates of major molecular response (MR), MR4, and MR4.5 were 84.6%, 64.1%, and 51.3%, respectively. No disease progression or CML-related deaths were observed. Grade 2 or higher hematological adverse events were not observed after 12 months. After 24 months, pleural effusion was reported in 10% of patients.

Study details: Findings are from a 5-year follow-up of phase 2 NordCML007 trial including 40 adult patients with newly diagnosed CML-CP who were given upfront dasatinib. Low-dose PegIFN-a2b was added from months 4 to 15 after dasatinib initiation.

Disclosures: This study was sponsored by the Norwegian University of Science and Technology. No disclosures were reported.

Source: Flygt H et al. Eur J Haematol. 2021 Aug 21. doi: 10.1111/ejh.13699.

Key clinical point: Although all starting ponatinib doses showed clinical benefit among patients with resistant chronic-phase chronic myeloid leukemia (CML-CP), the optimal benefit-risk ratio was observed with 45 mg ponatinib reduced to 15 mg on achievement of a response.

Major finding: The response rates at 12 months in patients receiving 45 mg, 30 mg, or 15 mg ponatinib were 44.1% (98.3% CI 31.7%-57.0%), 29.0% (98.3% CI 18.4%-41.6%), and 23.1% (98.3% CI 13.4%-35.3%), respectively. Grade 3-5 treatment-emergent arterial occlusive events were observed in 5, 5, and 3 patients in the 45, 30, and 15 mg ponatinib cohorts, respectively.

Study details: The phase 2 OPTIC trial included 283 adult patients with CML-CP resistant/intolerant to at least 2 prior tyrosine kinase inhibitors or with T315l mutation, randomly assigned to receive a once-daily starting dose of 45 mg, 30 mg, or 15 mg ponatinib. Patients receiving 45 mg or 30 mg doses were reduced to 15 mg upon achievement of a response.

Disclosures: No source of funding was identified other than Millennium Pharmaceuticals, Inc. for medical writing assistance. Some investigators, including the lead author, reported ties with various pharmaceutical companies.

Source: Cortes J et al. Blood. 2021 Aug 18. doi: 10.1182/blood.2021012082.

Key clinical point: Although all starting ponatinib doses showed clinical benefit among patients with resistant chronic-phase chronic myeloid leukemia (CML-CP), the optimal benefit-risk ratio was observed with 45 mg ponatinib reduced to 15 mg on achievement of a response.

Major finding: The response rates at 12 months in patients receiving 45 mg, 30 mg, or 15 mg ponatinib were 44.1% (98.3% CI 31.7%-57.0%), 29.0% (98.3% CI 18.4%-41.6%), and 23.1% (98.3% CI 13.4%-35.3%), respectively. Grade 3-5 treatment-emergent arterial occlusive events were observed in 5, 5, and 3 patients in the 45, 30, and 15 mg ponatinib cohorts, respectively.

Study details: The phase 2 OPTIC trial included 283 adult patients with CML-CP resistant/intolerant to at least 2 prior tyrosine kinase inhibitors or with T315l mutation, randomly assigned to receive a once-daily starting dose of 45 mg, 30 mg, or 15 mg ponatinib. Patients receiving 45 mg or 30 mg doses were reduced to 15 mg upon achievement of a response.

Disclosures: No source of funding was identified other than Millennium Pharmaceuticals, Inc. for medical writing assistance. Some investigators, including the lead author, reported ties with various pharmaceutical companies.

Source: Cortes J et al. Blood. 2021 Aug 18. doi: 10.1182/blood.2021012082.

Key clinical point: Although all starting ponatinib doses showed clinical benefit among patients with resistant chronic-phase chronic myeloid leukemia (CML-CP), the optimal benefit-risk ratio was observed with 45 mg ponatinib reduced to 15 mg on achievement of a response.

Major finding: The response rates at 12 months in patients receiving 45 mg, 30 mg, or 15 mg ponatinib were 44.1% (98.3% CI 31.7%-57.0%), 29.0% (98.3% CI 18.4%-41.6%), and 23.1% (98.3% CI 13.4%-35.3%), respectively. Grade 3-5 treatment-emergent arterial occlusive events were observed in 5, 5, and 3 patients in the 45, 30, and 15 mg ponatinib cohorts, respectively.

Study details: The phase 2 OPTIC trial included 283 adult patients with CML-CP resistant/intolerant to at least 2 prior tyrosine kinase inhibitors or with T315l mutation, randomly assigned to receive a once-daily starting dose of 45 mg, 30 mg, or 15 mg ponatinib. Patients receiving 45 mg or 30 mg doses were reduced to 15 mg upon achievement of a response.

Disclosures: No source of funding was identified other than Millennium Pharmaceuticals, Inc. for medical writing assistance. Some investigators, including the lead author, reported ties with various pharmaceutical companies.

Source: Cortes J et al. Blood. 2021 Aug 18. doi: 10.1182/blood.2021012082.

Key clinical point: Patients with chronic myeloid leukemia (CML) treated with first-line nilotinib vs. imatinib may have a higher risk for vascular adverse events (VAE), suggesting that older patients and those with a history of cerebrovascular diseases using nilotinib should be closely monitored.

Major finding: The risk for VAEs was significantly higher in patients treated with nilotinib vs. imatinib (hazard ratio 3.13; P < .05). Nilotinib use (odds ratio [OR] 3.43; P = .03), older age (OR, 1.04; P < .01), and history of cerebrovascular diseases (OR, 3.49; P = .02) were significant risk factors for VAEs.

Study details: Findings are from a retrospective analysis of 1,111 adult patients with CML treated with either frontline imatinib (n=565), nilotinib (n=306), or dasatinib (n=240).

Disclosures: No funding source or disclosures were reported.

Source: Chen MT et al. Oncologist. 2021 Aug 21. doi: 10.1002/onco.13944.

Key clinical point: Patients with chronic myeloid leukemia (CML) treated with first-line nilotinib vs. imatinib may have a higher risk for vascular adverse events (VAE), suggesting that older patients and those with a history of cerebrovascular diseases using nilotinib should be closely monitored.

Major finding: The risk for VAEs was significantly higher in patients treated with nilotinib vs. imatinib (hazard ratio 3.13; P < .05). Nilotinib use (odds ratio [OR] 3.43; P = .03), older age (OR, 1.04; P < .01), and history of cerebrovascular diseases (OR, 3.49; P = .02) were significant risk factors for VAEs.

Study details: Findings are from a retrospective analysis of 1,111 adult patients with CML treated with either frontline imatinib (n=565), nilotinib (n=306), or dasatinib (n=240).

Disclosures: No funding source or disclosures were reported.

Source: Chen MT et al. Oncologist. 2021 Aug 21. doi: 10.1002/onco.13944.

Key clinical point: Patients with chronic myeloid leukemia (CML) treated with first-line nilotinib vs. imatinib may have a higher risk for vascular adverse events (VAE), suggesting that older patients and those with a history of cerebrovascular diseases using nilotinib should be closely monitored.

Major finding: The risk for VAEs was significantly higher in patients treated with nilotinib vs. imatinib (hazard ratio 3.13; P < .05). Nilotinib use (odds ratio [OR] 3.43; P = .03), older age (OR, 1.04; P < .01), and history of cerebrovascular diseases (OR, 3.49; P = .02) were significant risk factors for VAEs.

Study details: Findings are from a retrospective analysis of 1,111 adult patients with CML treated with either frontline imatinib (n=565), nilotinib (n=306), or dasatinib (n=240).

Disclosures: No funding source or disclosures were reported.

Source: Chen MT et al. Oncologist. 2021 Aug 21. doi: 10.1002/onco.13944.

Key clinical point: Among patients with acute myeloid leukemia (AML) who underwent first allogeneic hematopoietic stem cell transplantation (allo-HSCT), children showed superior clinical outcomes compared to adolescents and postadolescents (APA) and young adults.

Major finding: At 2 years, the cumulative incidence of chronic graft-versus-host disease (P < .0001), relapse (P = .0254), and nonrelapse mortality (P < .0001) were significantly lower, and overall survival (P = .0009) and event-free survival (P = .0186) were higher in children compared to APA and young adult patients with AML.

Study details: Findings are from a retrospective analysis of children (age 0-14 years; n = 564), APA patients (age 15-25 years; n = 647), and young adults (age 26-40 years; n = 1,434) with AML who received a first allo-HSCT in the first or further remission or in refractory state.

Disclosures: This study did not receive any funding. The authors declared no conflicts of interest.

Source: Pochon C et al. J Cancer Res Clin Oncol. 2021 Sep 4. doi: 10.1007/s00432-021-03761-w.

Key clinical point: Among patients with acute myeloid leukemia (AML) who underwent first allogeneic hematopoietic stem cell transplantation (allo-HSCT), children showed superior clinical outcomes compared to adolescents and postadolescents (APA) and young adults.

Major finding: At 2 years, the cumulative incidence of chronic graft-versus-host disease (P < .0001), relapse (P = .0254), and nonrelapse mortality (P < .0001) were significantly lower, and overall survival (P = .0009) and event-free survival (P = .0186) were higher in children compared to APA and young adult patients with AML.

Study details: Findings are from a retrospective analysis of children (age 0-14 years; n = 564), APA patients (age 15-25 years; n = 647), and young adults (age 26-40 years; n = 1,434) with AML who received a first allo-HSCT in the first or further remission or in refractory state.

Disclosures: This study did not receive any funding. The authors declared no conflicts of interest.

Source: Pochon C et al. J Cancer Res Clin Oncol. 2021 Sep 4. doi: 10.1007/s00432-021-03761-w.

Key clinical point: Among patients with acute myeloid leukemia (AML) who underwent first allogeneic hematopoietic stem cell transplantation (allo-HSCT), children showed superior clinical outcomes compared to adolescents and postadolescents (APA) and young adults.

Major finding: At 2 years, the cumulative incidence of chronic graft-versus-host disease (P < .0001), relapse (P = .0254), and nonrelapse mortality (P < .0001) were significantly lower, and overall survival (P = .0009) and event-free survival (P = .0186) were higher in children compared to APA and young adult patients with AML.

Study details: Findings are from a retrospective analysis of children (age 0-14 years; n = 564), APA patients (age 15-25 years; n = 647), and young adults (age 26-40 years; n = 1,434) with AML who received a first allo-HSCT in the first or further remission or in refractory state.

Disclosures: This study did not receive any funding. The authors declared no conflicts of interest.

Source: Pochon C et al. J Cancer Res Clin Oncol. 2021 Sep 4. doi: 10.1007/s00432-021-03761-w.

Key clinical point: The maximum tolerated dose (MTD) of combined romidepsin and azacitidine (ROM/AZA) therapy was well tolerated and clinically active in patients with newly diagnosed, relapsed or refractory (R/R) acute myeloid leukemia (AML) and unfit for intensive chemotherapy.

Major finding: Overall, 23.7% of patients treated with the MTD had a complete remission (CR)/incomplete CR or partial response by cycle 6. The combination therapy was well tolerated with 5 grade 3 or higher nonhematologic treatment-related adverse events affecting at least 10% of patients. Only 4 patients discontinued treatment because of toxicity.

Study details: Findings are from the phase 1/2 ROMAZA trial including 48 patients with newly diagnosed, R/R AML ineligible for intensive chemotherapy. The MTD of ROM/AZA therapy was established as 12 mg/m² romidepcin on days 8 and 15 with 75 mg/m² azacitidine on days 1-9.

Disclosures: This study was funded by Bloodwise and Celgene. The lead author reported ties with Novartis, Daichi-Sankyo, Pfizer, Janssen, and Amgen. Some investigators reported ties with various pharmaceutical companies, including Celgene.

Source: Loke J et al. Br J Haematol. 2021 Sep 6. doi: 10.1111/bjh.17823.

Key clinical point: The maximum tolerated dose (MTD) of combined romidepsin and azacitidine (ROM/AZA) therapy was well tolerated and clinically active in patients with newly diagnosed, relapsed or refractory (R/R) acute myeloid leukemia (AML) and unfit for intensive chemotherapy.

Major finding: Overall, 23.7% of patients treated with the MTD had a complete remission (CR)/incomplete CR or partial response by cycle 6. The combination therapy was well tolerated with 5 grade 3 or higher nonhematologic treatment-related adverse events affecting at least 10% of patients. Only 4 patients discontinued treatment because of toxicity.

Study details: Findings are from the phase 1/2 ROMAZA trial including 48 patients with newly diagnosed, R/R AML ineligible for intensive chemotherapy. The MTD of ROM/AZA therapy was established as 12 mg/m² romidepcin on days 8 and 15 with 75 mg/m² azacitidine on days 1-9.

Disclosures: This study was funded by Bloodwise and Celgene. The lead author reported ties with Novartis, Daichi-Sankyo, Pfizer, Janssen, and Amgen. Some investigators reported ties with various pharmaceutical companies, including Celgene.

Source: Loke J et al. Br J Haematol. 2021 Sep 6. doi: 10.1111/bjh.17823.

Key clinical point: The maximum tolerated dose (MTD) of combined romidepsin and azacitidine (ROM/AZA) therapy was well tolerated and clinically active in patients with newly diagnosed, relapsed or refractory (R/R) acute myeloid leukemia (AML) and unfit for intensive chemotherapy.

Major finding: Overall, 23.7% of patients treated with the MTD had a complete remission (CR)/incomplete CR or partial response by cycle 6. The combination therapy was well tolerated with 5 grade 3 or higher nonhematologic treatment-related adverse events affecting at least 10% of patients. Only 4 patients discontinued treatment because of toxicity.

Study details: Findings are from the phase 1/2 ROMAZA trial including 48 patients with newly diagnosed, R/R AML ineligible for intensive chemotherapy. The MTD of ROM/AZA therapy was established as 12 mg/m² romidepcin on days 8 and 15 with 75 mg/m² azacitidine on days 1-9.

Disclosures: This study was funded by Bloodwise and Celgene. The lead author reported ties with Novartis, Daichi-Sankyo, Pfizer, Janssen, and Amgen. Some investigators reported ties with various pharmaceutical companies, including Celgene.

Source: Loke J et al. Br J Haematol. 2021 Sep 6. doi: 10.1111/bjh.17823.

Key clinical point: Patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) during their first complete remission (CR) from a mismatched unrelated donor (MMUD), umbilical cord blood (UCB), or a haploidentical-related donor (Haplo) had similar outcomes, making all of them a viable option in the absence of a matched related or unrelated donor.

Major finding: For patients who had transplants between 2015 and 2018, the 3-year overall survival of MMUD, UCB, and Haplo groups were 60%, 66%, and 63%, respectively (P = .693). The rates of nonrelapse mortality (P = .147), relapse (P = .075), and graft-versus-host disease-free/relapse-free survival (P = .365) were also similar between the 3 groups.

Study details: Findings are from a retrospective analysis of 1,932 patients with AML who underwent allo-HSCT during their first CR from MMUD, UCB or Haplo.

Disclosures: This study was supported by grants from the Practical Research Project for Allergic Diseases and Immunology from the Japan Agency for Medical Research and Development and the Aichi Cancer Research Foundation. The authors declared no conflicts of interest.

Source: Yanada M et al. Transplant Cell Ther. 2021 Sep 6. doi: 10.1016/j.jtct.2021.08.027.

Key clinical point: Patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) during their first complete remission (CR) from a mismatched unrelated donor (MMUD), umbilical cord blood (UCB), or a haploidentical-related donor (Haplo) had similar outcomes, making all of them a viable option in the absence of a matched related or unrelated donor.

Major finding: For patients who had transplants between 2015 and 2018, the 3-year overall survival of MMUD, UCB, and Haplo groups were 60%, 66%, and 63%, respectively (P = .693). The rates of nonrelapse mortality (P = .147), relapse (P = .075), and graft-versus-host disease-free/relapse-free survival (P = .365) were also similar between the 3 groups.

Study details: Findings are from a retrospective analysis of 1,932 patients with AML who underwent allo-HSCT during their first CR from MMUD, UCB or Haplo.

Disclosures: This study was supported by grants from the Practical Research Project for Allergic Diseases and Immunology from the Japan Agency for Medical Research and Development and the Aichi Cancer Research Foundation. The authors declared no conflicts of interest.

Source: Yanada M et al. Transplant Cell Ther. 2021 Sep 6. doi: 10.1016/j.jtct.2021.08.027.

Key clinical point: Patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) during their first complete remission (CR) from a mismatched unrelated donor (MMUD), umbilical cord blood (UCB), or a haploidentical-related donor (Haplo) had similar outcomes, making all of them a viable option in the absence of a matched related or unrelated donor.

Major finding: For patients who had transplants between 2015 and 2018, the 3-year overall survival of MMUD, UCB, and Haplo groups were 60%, 66%, and 63%, respectively (P = .693). The rates of nonrelapse mortality (P = .147), relapse (P = .075), and graft-versus-host disease-free/relapse-free survival (P = .365) were also similar between the 3 groups.

Study details: Findings are from a retrospective analysis of 1,932 patients with AML who underwent allo-HSCT during their first CR from MMUD, UCB or Haplo.

Disclosures: This study was supported by grants from the Practical Research Project for Allergic Diseases and Immunology from the Japan Agency for Medical Research and Development and the Aichi Cancer Research Foundation. The authors declared no conflicts of interest.

Source: Yanada M et al. Transplant Cell Ther. 2021 Sep 6. doi: 10.1016/j.jtct.2021.08.027.

Key clinical point: De-escalation of cytarabine exposure by elimination of the fifth chemotherapy course should only be considered in pediatric patients with low-risk acute myeloid leukemia (LR-AML) with favorable cytogenetic/molecular features and negative minimal residual disease (MRD) at the end of first induction (EOI1).

Major finding: Overall, patients who received 4 vs. 5 chemotherapy courses had worse disease-free survival (DFS; hazard ratio [HR] 1.45; P = .009) and relapse risk (HR 1.40; P = .019). However, patients with favorable cytogenetic/molecular features and negative MRD at EOI1 had no difference in DFS (P = .934) and overall survival (P = .928) with 4 vs. 5 chemotherapy courses.

Study details: This prospective study assessed 923 pediatric patients with LR-AML treated in either AAML0531 (n = 225; excluding those treated with gemtuzumab ozogamicin) or AAML1031 (n = 698) trials with 4 (79%) or 5 (21%) courses of frontline chemotherapy.

Disclosures: This study was supported by National Clinical Trials Network (NCTN) Operations Center, NCTN Statistics & Data Center, St. Baldrick’s Foundation, Alex’s Lemonade Stand Foundation, and National Heart Lung and Blood Institute. The authors declared no conflicts of interest.

Source: Getz KD et al. Pediatr Blood Cancer. 2021 Sep 2. doi: 10.1002/pbc.29313.

Key clinical point: De-escalation of cytarabine exposure by elimination of the fifth chemotherapy course should only be considered in pediatric patients with low-risk acute myeloid leukemia (LR-AML) with favorable cytogenetic/molecular features and negative minimal residual disease (MRD) at the end of first induction (EOI1).

Major finding: Overall, patients who received 4 vs. 5 chemotherapy courses had worse disease-free survival (DFS; hazard ratio [HR] 1.45; P = .009) and relapse risk (HR 1.40; P = .019). However, patients with favorable cytogenetic/molecular features and negative MRD at EOI1 had no difference in DFS (P = .934) and overall survival (P = .928) with 4 vs. 5 chemotherapy courses.

Study details: This prospective study assessed 923 pediatric patients with LR-AML treated in either AAML0531 (n = 225; excluding those treated with gemtuzumab ozogamicin) or AAML1031 (n = 698) trials with 4 (79%) or 5 (21%) courses of frontline chemotherapy.

Disclosures: This study was supported by National Clinical Trials Network (NCTN) Operations Center, NCTN Statistics & Data Center, St. Baldrick’s Foundation, Alex’s Lemonade Stand Foundation, and National Heart Lung and Blood Institute. The authors declared no conflicts of interest.

Source: Getz KD et al. Pediatr Blood Cancer. 2021 Sep 2. doi: 10.1002/pbc.29313.

Key clinical point: De-escalation of cytarabine exposure by elimination of the fifth chemotherapy course should only be considered in pediatric patients with low-risk acute myeloid leukemia (LR-AML) with favorable cytogenetic/molecular features and negative minimal residual disease (MRD) at the end of first induction (EOI1).

Major finding: Overall, patients who received 4 vs. 5 chemotherapy courses had worse disease-free survival (DFS; hazard ratio [HR] 1.45; P = .009) and relapse risk (HR 1.40; P = .019). However, patients with favorable cytogenetic/molecular features and negative MRD at EOI1 had no difference in DFS (P = .934) and overall survival (P = .928) with 4 vs. 5 chemotherapy courses.

Study details: This prospective study assessed 923 pediatric patients with LR-AML treated in either AAML0531 (n = 225; excluding those treated with gemtuzumab ozogamicin) or AAML1031 (n = 698) trials with 4 (79%) or 5 (21%) courses of frontline chemotherapy.

Disclosures: This study was supported by National Clinical Trials Network (NCTN) Operations Center, NCTN Statistics & Data Center, St. Baldrick’s Foundation, Alex’s Lemonade Stand Foundation, and National Heart Lung and Blood Institute. The authors declared no conflicts of interest.

Source: Getz KD et al. Pediatr Blood Cancer. 2021 Sep 2. doi: 10.1002/pbc.29313.

Key clinical point: Mutations of the CCAAT/enhancer-binding protein alpha gene (CEBPA) in the basic leucine zipper domain (bZIP), even when detected as single-mutated CEBPA, were associated with favorable prognosis in patients with de novo acute myeloid leukemia (AML).

Major finding: Patients with compared to without CEBPA mutation in bZIP had a higher rate of complete remission, longer overall survival (OS), and lower cumulative incidence of relapse (all P < .001). Among patients with single-mutated CEBPA, those with CEBPA mutation in compared to out-of bZIP had a significantly longer OS (P = .008).

Study details: Findings are from a retrospective analysis of 1,028 patients with de novo AML.

Disclosures: No source of funding was identified. The authors declared no conflict of interests.

Source: Wakita S et al. Blood Adv. 2021 Aug 27. doi: 10.1182/bloodadvances.2021004292.

Key clinical point: Mutations of the CCAAT/enhancer-binding protein alpha gene (CEBPA) in the basic leucine zipper domain (bZIP), even when detected as single-mutated CEBPA, were associated with favorable prognosis in patients with de novo acute myeloid leukemia (AML).

Major finding: Patients with compared to without CEBPA mutation in bZIP had a higher rate of complete remission, longer overall survival (OS), and lower cumulative incidence of relapse (all P < .001). Among patients with single-mutated CEBPA, those with CEBPA mutation in compared to out-of bZIP had a significantly longer OS (P = .008).

Study details: Findings are from a retrospective analysis of 1,028 patients with de novo AML.

Disclosures: No source of funding was identified. The authors declared no conflict of interests.

Source: Wakita S et al. Blood Adv. 2021 Aug 27. doi: 10.1182/bloodadvances.2021004292.

Key clinical point: Mutations of the CCAAT/enhancer-binding protein alpha gene (CEBPA) in the basic leucine zipper domain (bZIP), even when detected as single-mutated CEBPA, were associated with favorable prognosis in patients with de novo acute myeloid leukemia (AML).

Major finding: Patients with compared to without CEBPA mutation in bZIP had a higher rate of complete remission, longer overall survival (OS), and lower cumulative incidence of relapse (all P < .001). Among patients with single-mutated CEBPA, those with CEBPA mutation in compared to out-of bZIP had a significantly longer OS (P = .008).

Study details: Findings are from a retrospective analysis of 1,028 patients with de novo AML.

Disclosures: No source of funding was identified. The authors declared no conflict of interests.

Source: Wakita S et al. Blood Adv. 2021 Aug 27. doi: 10.1182/bloodadvances.2021004292.

Key clinical point: Oral azacitidine (OA) maintenance had a manageable safety profile in patients with newly diagnosed acute myeloid leukemia (AML) who attained the first complete remission (CR) or CR with incomplete blood count recovery (CRi) after induction chemotherapy (IC). However, blood count monitoring for at least the first 2 treatment cycles is recommended.

Major finding: Gastrointestinal (91% vs. 62%) and hematologic (66% vs. 47%) adverse events were common with OA vs. placebo. Adverse events were mostly manageable with dose interruptions (43%) or reductions (16%) and infrequently led to OA discontinuation (13%).

Study details: This safety analysis of the QUAZAR AML-001 trial included 469 patients with newly diagnosed AML with intermediate- or poor-risk cytogenetics who attained the first CR/CRi after IC, randomly assigned to receive either 300 mg OA (n = 236) or placebo (n = 233).

Disclosures: This study was funded by Celgene, a Bristol Myers Squibb Company. Some investigators, including the lead author, reported ties with various pharmaceutical companies including Celgene and Bristol Myers Squibb.

Source: Ravandi F et al. J Hematol Oncol. 2021 Aug 28. doi: 10.1186/s13045-021-01142-x.

Key clinical point: Oral azacitidine (OA) maintenance had a manageable safety profile in patients with newly diagnosed acute myeloid leukemia (AML) who attained the first complete remission (CR) or CR with incomplete blood count recovery (CRi) after induction chemotherapy (IC). However, blood count monitoring for at least the first 2 treatment cycles is recommended.

Major finding: Gastrointestinal (91% vs. 62%) and hematologic (66% vs. 47%) adverse events were common with OA vs. placebo. Adverse events were mostly manageable with dose interruptions (43%) or reductions (16%) and infrequently led to OA discontinuation (13%).

Study details: This safety analysis of the QUAZAR AML-001 trial included 469 patients with newly diagnosed AML with intermediate- or poor-risk cytogenetics who attained the first CR/CRi after IC, randomly assigned to receive either 300 mg OA (n = 236) or placebo (n = 233).

Disclosures: This study was funded by Celgene, a Bristol Myers Squibb Company. Some investigators, including the lead author, reported ties with various pharmaceutical companies including Celgene and Bristol Myers Squibb.

Source: Ravandi F et al. J Hematol Oncol. 2021 Aug 28. doi: 10.1186/s13045-021-01142-x.

Key clinical point: Oral azacitidine (OA) maintenance had a manageable safety profile in patients with newly diagnosed acute myeloid leukemia (AML) who attained the first complete remission (CR) or CR with incomplete blood count recovery (CRi) after induction chemotherapy (IC). However, blood count monitoring for at least the first 2 treatment cycles is recommended.

Major finding: Gastrointestinal (91% vs. 62%) and hematologic (66% vs. 47%) adverse events were common with OA vs. placebo. Adverse events were mostly manageable with dose interruptions (43%) or reductions (16%) and infrequently led to OA discontinuation (13%).

Study details: This safety analysis of the QUAZAR AML-001 trial included 469 patients with newly diagnosed AML with intermediate- or poor-risk cytogenetics who attained the first CR/CRi after IC, randomly assigned to receive either 300 mg OA (n = 236) or placebo (n = 233).

Disclosures: This study was funded by Celgene, a Bristol Myers Squibb Company. Some investigators, including the lead author, reported ties with various pharmaceutical companies including Celgene and Bristol Myers Squibb.

Source: Ravandi F et al. J Hematol Oncol. 2021 Aug 28. doi: 10.1186/s13045-021-01142-x.

Key clinical point: Low-intensity treatment with low-dose cytarabine (LD-AC) combined with cladribine offered therapeutic benefit with an acceptable safety profile in elderly patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy.

Major finding: Rates of overall response and 56-day mortality were 54.0% and 20.5%, respectively. The median overall survival was 6.9 months (95% CI 4.9-9.6) and superior in patients who achieved an objective response (hazard ratio [HR] 0.04; P < .0001) or partial remission (HR 0.31; P = .0002) compared to nonresponders.

Study details: Findings are from a prospective analysis of 117 elderly patients (age, 60 years or older) with AML with poor performance status or a high comorbidity index treated with frontline LD-AC and cladribine.

Disclosures: This study was supported by the Institute of Hematology and Transfusion Medicine in Warsaw and Polish Adult Leukemia Group. The authors declared no conflicts of interest.

Source: Budziszewska BK et al. Cancers. 2021 Aug 20. doi: 10.3390/cancers13164189.

Key clinical point: Low-intensity treatment with low-dose cytarabine (LD-AC) combined with cladribine offered therapeutic benefit with an acceptable safety profile in elderly patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy.

Major finding: Rates of overall response and 56-day mortality were 54.0% and 20.5%, respectively. The median overall survival was 6.9 months (95% CI 4.9-9.6) and superior in patients who achieved an objective response (hazard ratio [HR] 0.04; P < .0001) or partial remission (HR 0.31; P = .0002) compared to nonresponders.

Study details: Findings are from a prospective analysis of 117 elderly patients (age, 60 years or older) with AML with poor performance status or a high comorbidity index treated with frontline LD-AC and cladribine.

Disclosures: This study was supported by the Institute of Hematology and Transfusion Medicine in Warsaw and Polish Adult Leukemia Group. The authors declared no conflicts of interest.

Source: Budziszewska BK et al. Cancers. 2021 Aug 20. doi: 10.3390/cancers13164189.

Key clinical point: Low-intensity treatment with low-dose cytarabine (LD-AC) combined with cladribine offered therapeutic benefit with an acceptable safety profile in elderly patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy.

Major finding: Rates of overall response and 56-day mortality were 54.0% and 20.5%, respectively. The median overall survival was 6.9 months (95% CI 4.9-9.6) and superior in patients who achieved an objective response (hazard ratio [HR] 0.04; P < .0001) or partial remission (HR 0.31; P = .0002) compared to nonresponders.

Study details: Findings are from a prospective analysis of 117 elderly patients (age, 60 years or older) with AML with poor performance status or a high comorbidity index treated with frontline LD-AC and cladribine.

Disclosures: This study was supported by the Institute of Hematology and Transfusion Medicine in Warsaw and Polish Adult Leukemia Group. The authors declared no conflicts of interest.

Source: Budziszewska BK et al. Cancers. 2021 Aug 20. doi: 10.3390/cancers13164189.