Management of luspatercept therapy in patients with transfusion-dependent β-thalassaemia

被引：9

作者：

Sheth, Sujit ^{[1
]}

Taher, Ali T. ^{[2
]}

Coates, Thomas D. ^{[3
,4
]}

Kattamis, Antonis ^{[5
]}

Cappellini, Maria Domenica ^{[6
,7
]}

机构：

[1] Weill Cornell Med, Div Pediat Hematol Oncol, New York, NY USA

[2] Amer Univ Beirut, Dept Internal Med, Med Ctr, Beirut, Lebanon

[3] Childrens Hosp Angeles, Canc & Blood Dis Inst, Los Angeles, CA USA

[4] USC Keck Sch Med, Los Angeles, CA USA

[5] Natl & Kapodistrian Univ Athens, Dept Pediat 1, Athens, Greece

[6] Univ Milan, Fdn IRCCS Ca Granda Policlin Hosp, Milan, Italy

[7] Univ Milan, Fdn IRCCS Ca Granda Policlin Hosp, Pad Granelli, Via Francesco Sforza 35, I-20122 Milan, Italy

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2023年 / 201卷 / 05期

关键词：

adverse event management; dose management; haemoglobin; luspatercept; beta-thalassaemia; ERYTHROPOIESIS; DISEASE;

D O I：

10.1111/bjh.18801

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Patients with transfusion-dependent beta-thalassaemia require lifelong, regular red blood cell transfusions for survival; however, frequent blood transfusions are associated with an increased risk of iron overload, transfusion-transmitted disease and alloimmunization, as well as reduced quality of life. Luspatercept, an erythroid maturation agent that promotes late-stage erythroid maturation independently of erythropoietin, has demonstrated efficacy in reducing transfusion burden in patients with transfusion-dependent beta-thalassaemia. In this review, we discuss treatment initiation, ongoing evaluation, dose adjustment and management of adverse events in transfusion-dependent patients with beta-thalassaemia receiving luspatercept, and we provide guidance on how to determine whether patients are deriving clinical benefit.

引用

页码：824 / 831

页数：8

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