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2021 update on clinical trials in β‐thalassemia
Author(s) -
Musallam Khaled M.,
BouFakhredin Rayan,
Cappellini Maria Domenica,
Taher Ali T.
Publication year - 2021
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.26316
Subject(s) - thalassemia , medicine , ineffective erythropoiesis , anemia , beta thalassemia , clinical trial , erythropoiesis , chelation therapy , intensive care medicine , blood transfusion , transfusion therapy , pediatrics
The treatment landscape for patients with β‐thalassemia is witnessing a swift evolution, yet several unmet needs continue to persist. Patients with transfusion‐dependent β‐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 β‐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 β‐thalassemia, including the α/non‐α‐globin chain imbalance, ineffective erythropoiesis, and iron dysregulation.