Curtis et al. (July 17 issue)1 report evidence of longer graft-versus-host disease (GVHD)–free, relapse-free survival with post-transplantation cyclophosphamide–based prophylaxis than with cyclosporin–methotrexate among patients receiving a stem-cell transplant from a matched related donor after myeloablative conditioning or reduced-intensity conditioning.

UBTF-TD has been reported in a significant percentage of childhood MDS-EB and has been associated with inferior survival compared to that of patients with the wild-type gene. We treated three consecutive pediatric patients affected by UBTF-TD MDS-EB with venetoclax and azacitidine (ven/aza) in combination as 28-day cycles on a compassionate use basis three consecutive pediatric patients affected by UBTF-TD MDS-EB as a bridge to allogeneic HSCT. Treatment with ven/aza was well-tolerated, and all patients responded to the ven/aza course, achieving CR with flow-cytometry negativity.

Luspatercept has emerged as a novel therapy for anemia in transfusion-dependent (TD) lower-risk

How should patients with VEXAS (an acronym for vacuoles [in myeloid and erythroid precursors], E1 enzyme, X-linked, autoinflammatory, somatic) syndrome be treated? Until now, we have only had case reports and small series to guide us.

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