Efficacy and safety of luspatercept in the treatment of anemia in allo-HSCT for patients with hematological diseases

作者：Shiwei Jin, Mengmeng Pan, Weiping Zhang, Jiong Hu, Jian‐Qing Mi, Ling Wang · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-2438 · 被引用次数：1 · 研究领域：Hematopoietic Stem Cell Transplantation、Hemoglobinopathies and Related Disorders、Acute Myeloid Leukemia Research

Abstract Background: Persistent anemia affects up to 30% of long-term survivors after allogeneic hematopoietic stem-cell transplantation (allo-HSCT). Current treatments have limited efficacy and substantial side effects. Luspatercept is a first-in-class erythroid maturation agent, with several encouraging results reported in small retrospective studies. We therefore conducted a relative larger, rigorously defined retrospective analysis to clarify the efficacy and safety of luspatercept in adults with anaemia persisting after allo-HSCT. Methods: This retrospective analysis included 96 consecutive patients treated at our center between November 2022 and December 2024. The eligibility criteria were post-transplant anemia (hemoglobin <65g/L despite ≥4 weeks of standard care), complete donor chimerism (≥95%), underlying disease in complete remission, and no evidence of haemolysis, bleeding or nutritional deficiency. Diagnosis included AML (47), ALL (30), aplastic anemia (15), MDS (3), and myelofibrosis (1). All patients with haematological malignancies received myeloablative conditioning regimens based on fludarabine/busulfan/melphalan or fludarabine/busulfan/TMI. The patient with aplastic anaemia received an immuno-myeloablative regimen. The primary end-point was erythroid response defined as a sustained (≥14 consecutive days) rise in haemoglobin ≥15 g/L from baseline without red-cell transfusion. Secondary endpoints included hemoglobin ≥80 g/L, time to response, and safet...