Zaidel Bianca, Garland Stephanie, Merkeley Hayley
Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Red Cell Disorders Program of British Columbia, Vancouver, British Columbia, Canada.
Br J Haematol. 2025 Jun;206(6):1806-1810. doi: 10.1111/bjh.20131. Epub 2025 Apr 30.
Patients with transfusion-dependent thalassaemia (TDT) require chronic, life-sustaining red blood cell transfusions, which contribute to iron overload and associated morbidity and mortality. Luspatercept, an inhibitor of the transforming growth factor-beta pathway, decreases transfusion requirements in a subset of transfusion-dependent β-thalassaemia patients and is increasingly prescribed in clinical practice. Subsequent to regulatory approvals, several reports have identified the risk of extra-medullary haematopoiesis, which resulted in updated safety labelling. In this report, we present a case of a patient with TDT who developed severe splenomegaly, haemolysis and thrombocytopenia necessitating treatment discontinuation.
依赖输血的地中海贫血（TDT）患者需要长期进行维持生命的红细胞输血，这会导致铁过载以及相关的发病和死亡风险。罗特西普是一种转化生长因子-β通路抑制剂，可减少部分依赖输血的β地中海贫血患者的输血需求，且在临床实践中的处方量日益增加。在监管部门批准后，多项报告发现了髓外造血的风险，这导致了安全标签的更新。在本报告中，我们介绍了一例TDT患者的病例，该患者出现了严重脾肿大、溶血和血小板减少，需要停药。
