Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report
Pedro Henrique Prata, Dirk-Jan Eikema, Brian Piepenbroek et al. · 2025 · Haematologica
Aplastic anemia (AA) transformation into myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) is associated with a dismal prognosis. Hematopoietic stem cell transplant offers the sole possibility of cure, but data on long-term survival are scarce. We retrospectively analyzed 270 patients transplanted for MDS, AML, or an isolated cytogenetic abnormality after a diagnosis of AA or paroxysmal nocturnal hemoglobinuria reported to the EBMT. The median age at transplantation was 39 years. The 5-year overall survival (OS) rate was 64%, unaffected by chromosome 7 abnormalities, age at trans
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