Luspatercept for the treatment of anemia in myelodysplastic syndromes and primary myelofibrosis

被引：82

作者：

Fenaux, Pierre ^{[1
,2
]}

Kiladjian, Jean Jacques ^{[2
,3
]}

Platzbecker, Uwe ^{[4
,5
]}

机构：

[1] Hop St Louis, AP HP, Serv Hematol Seniors, F-75475 Paris, France

[2] Univ Paris Diderot, Dept Hematol, Paris, France

[3] Ctr Invest Cliniques, AP HP, Paris, France

[4] Leipzig Univ Hosp, Med Clin, Leipzig, Germany

[5] Leipzig Univ Hosp, Hematol & Cellular Therapy, Policlin 1, Leipzig, Germany

来源：

BLOOD | 2019年 / 133卷 / 08期

关键词：

TRANSFUSION-DEPENDENT PATIENTS; FC FUSION PROTEIN; LOWER-RISK MDS; SCORING SYSTEM; ERYTHROPOIESIS; RECEPTOR; BETA; SOTATERCEPT; PLACEBO; AZACITIDINE;

D O I：

10.1182/blood-2018-11-876888

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Anemia of lower-risk myelodysplastic syndromes (MDSs) and primary myelofibrosis (PMF) generally becomes resistant to available treatments, leading to red blood cell (RBC) transfusions, iron overload, shortened survival, and poor quality of life. The transforming growth factor-b superfamily, including activins and growth differentiation factors (GDFs), is aberrantly expressed in lower-risk MDSs and PMF. Luspatercept (and sotatercept), ligand traps that particularly inhibit GDF11, lead to RBC transfusion independence in 10% to 50% of lower-risk MDSs resistant to available treatments, and have started to be used in PMF.

引用

页码：790 / 794

页数：5

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