Hematology

Three GvHD prophylaxis strategies yield similar survival after haploidentical PBSC transplant for AML/MDS

In this phase III trial, 407 AML/MDS haplo-PBSCT recipients were randomized to low-dose ATG/PTCy, standard-dose ATG, or PTCy-based prophylaxis. Rates of grade II-IV aGvHD by day 100 were similar across all three arms, as were overall and GvHD/relapse-free survival. Moderate-to-severe cGvHD was numerically lower with ATG/PTCy and ATG than with PTCy alone, but differences were not statistically significant. Neutrophil and platelet recovery were significantly higher with the low-dose ATG/PTCy regimen, indicating faster hematologic recovery without compromising GvHD control. 1

PTCy-based prophylaxis substantially lowers bronchiolitis obliterans syndrome risk after allogeneic HCT

This multicenter cohort of 900 allogeneic HCT recipients compared BOS incidence between PTCy-based and non-PTCy GvHD prophylaxis. PTCy was associated with a 75% reduction in BOS risk (adjusted HR 0.25, 95% CI 0.09–0.74) after adjustment for key covariates. Adding cGvHD to the model attenuated the protective effect, indicating BOS reduction is largely mediated through cGvHD prevention. Results remained robust when haploidentical transplants were excluded, supporting use of PTCy-based regimens in patients at high BOS risk. 2

Fostamatinib achieves durable, steroid-sparing responses in refractory warm AIHA and Evans syndrome

This multicentre retrospective series evaluated fostamatinib in refractory warm autoimmune hemolytic anemia and Evans syndrome. Durable responses occurred in 46% of patients, with a 73% reduction in steroid use among responders. Toxicities were mainly hypertension, gastrointestinal symptoms, and neutropenia in 23%, with only one discontinuation and one dose reduction. In an eight-patient subset, migration inhibitory factor levels and specific naïve T-cell and HLA-DRhi monocyte subsets correlated with treatment response. 3

Quantitative MYD88 L265P ratio plus marrow flow cytometry refine risk in IgM gammopathies

The SAL-TO study analyzed 743 IgM gammopathy patients from Spain and Italy, including symptomatic and asymptomatic WM and IgM-MGUS. Adverse overall survival was associated with age over 65, male sex, symptomatic WM, and beta-2-microglobulin greater than 3 mg/L. Older age, bone marrow infiltration, anemia, and thrombocytopenia predicted shorter time to first treatment. A bone marrow MYD88 L265P/MYD88 wild-type ratio above 0.162 plus flow cytometry infiltration above 4.39% defined a high-risk group with worse survival and faster treatment need. 4