2021 update on clinical trials in β-thalassemia

被引：51

作者：

Musallam, Khaled M. ^{[1
,2
]}

Bou-Fakhredin, Rayan ^{[3
]}

Cappellini, Maria Domenica ^{[4
]}

Taher, Ali T. ^{[3
]}

机构：

[1] Burjeel Med City, Thalassemia Ctr, Abu Dhabi, U Arab Emirates

[2] Int Network Hematol, London, England

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

[4] Univ Milan, Dept Clin Sci & Community, Ca Granda Fdn IRCCS Maggiore Policlin Hosp, Milan, Italy

来源：

AMERICAN JOURNAL OF HEMATOLOGY | 2021年 / 96卷 / 11期

关键词：

REDUCES IRON OVERLOAD; FETAL-HEMOGLOBIN; GENE-THERAPY; INEFFECTIVE ERYTHROPOIESIS; MOUSE MODEL; ERYTHROID-CELLS; ANEMIA; INTERMEDIA; EFFICACY; HEPCIDIN;

D O I：

10.1002/ajh.26316

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The treatment landscape for patients with beta-thalassemia is witnessing a swift evolution, yet several unmet needs continue to persist. Patients with transfusion-dependent beta-thalassemia (TDT) primarily rely on regular transfusion and iron chelation therapy, which can be associated with considerable treatment burden and cost. Patients with non-transfusion-dependent beta-thalassemia (NTDT) are also at risk of significant morbidity due to the underlying anemia and iron overload, but treatment options in this patient subgroup are limited. In this review, we provide updates on clinical trials of novel therapies targeting the underlying pathology in beta-thalassemia, including the alpha/non-alpha-globin chain imbalance, ineffective erythropoiesis, and iron dysregulation.

引用

页码：1518 / 1531

页数：14

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