Hematology

Low-dose induction matches standard efficacy in pediatric AML with faster recovery

This multicenter randomized noninferiority trial compared low-dose versus standard-dose induction in children with AML receiving identical post-remission therapy. CR/CRi rates were nearly identical, 95.1% with low-dose and 95.3% with standard-dose induction. MRD <0.1% after second induction occurred in 87.4% versus 87.1% of patients, confirming comparable depth of response. Four-year OS and EFS were similar between arms, with no significant differences reported. Neutrophil and platelet recovery were significantly faster with low-dose induction, indicating potential reductions in cytopenia-related morbidity and resource use. 1

Imatinib delivers durable 25-year control in chronic-phase CML

This retrospective monocentric cohort followed 210 adults with chronic-phase CML treated with imatinib between 2000 and 2025. At 25 years, overall survival was 71% and progression-free survival 88%, demonstrating lasting disease control in real-world practice. The ELTS score independently predicted OS, EFS, and PFS, and lower-risk patients achieved MMR faster and more often reached deep molecular responses. Dose reductions, frequently applied in older and comorbid patients, did not compromise molecular or survival outcomes. Higher Charlson comorbidity index was associated with inferior OS, underscoring the impact of competing mortality in survivorship discussions. 2

Permissive HLA-DPB1 mismatch with PTCy reduces relapse after unrelated allo-HCT

Investigators analyzed 541 adults undergoing 8/8 unrelated donor SCT with post-transplant cyclophosphamide for myeloid neoplasms. Patients were classified as DPB1-matched, permissive mismatched, or non-permissive in graft-versus-host or host-versus-graft directions. Two-year relapse incidence was lower with permissive mismatching versus matching (18% vs 28%; HR 0.6, 95% CI 0.4–0.9). The relapse reduction was most pronounced in high-risk AML/MDS, where outcomes approached those of lower-risk disease. GVHD incidence was not increased, and OS and PFS were similar, making DPB1-permissive mismatching an actionable donor-selection criterion. 3

Trilaciclib limits chemotherapy-driven expansion of TP53-mutant clonal hematopoiesis

Across four randomized trials, trilaciclib with diverse chemotherapy regimens mitigated expansion of clonal hematopoiesis bearing DNA damage response mutations, including TP53. A syngeneic TP53-mutant mouse model showed trilaciclib blocked platinum-induced TP53-mutant competitive repopulation. Mechanistically, trilaciclib promoted hematopoietic stem and progenitor quiescence and reduced the stemness advantage of TP53-mutant clones. These data provide proof of concept that CDK4/6 inhibition may pharmacologically limit chemotherapy-induced expansion of high-risk preleukemic clones. 4