Aplastic Anemia

Abstract 6508 Background: Advances in hematopoietic stem cell transplantation (HSCT) have been driven by progress in supportive care, conditioning regimens, and graft-versus-host disease (GvHD) prophylaxis. Post-transplant morbidity and mortality are most pronounced in the first…

Haploidentical bone marrow transplantation (haplo-BMT) with post-transplant cyclophosphamide (PT-Cy) has become an established alternative for patients lacking HLA-matched donors, with outcomes comparable to those of matched-sibling and unrelated-donor transplants [1]. While CD34…

Abstract Background Paroxysmal nocturnal hemoglobinuria (PNH) clones are detected in up to 60% of patients with aplastic anemia (AA); however, their prognostic impact remains incompletely defined, particularly in the context of frontline immunosuppressive therapy (IST) or…

Key Points A large retrospective study evaluating association between pregnancy after HCT with both conditioning protocols and assisted reproduction. Findings include that some protocols appear more sterilizing than others and the impact of assisted reproduction is substantial…

Abstract Purpose of Review In inherited bone marrow failure syndromes (IBMFS), clonal hematopoiesis (CH) has been increasingly recognized as a molecular fingerprint of the underlying pathophysiology. A specific clonal profile has been reported in telomere biology disorders (TBDs)…

Abstract Background Clonal hematopoiesis (CH) is a feature of severe aplastic anemia (SAA), but its clinical significance is debated. Methods We integrated longitudinal clinical and CH data from patients with SAA treated with immunosuppression plus eltrombopag (IST-EPAG) in a…

Key Points Frontline eltrombopag plus IST is cost-effective compared with IST alone for adults with severe aplastic anemia. Incremental net monetary benefit per patient treated with frontline eltrombopag is ∼$64 000. Abstract The randomized phase 3 RACE trial demonstrated…

ABSTRACT Allogeneic hematopoietic cell transplantation (allo-HCT) is an effective treatment for patients with high-risk hematologic malignancies. Over the last decade, gut microbiota composition during allo-HCT has been associated with patients' outcomes. Treatment-related…