Alternative therapies of manifest forms of β-thalassemia: stimulation of fetal hemoglobin synthesis

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Abstract

An imbalance in the synthesis and ratio of α and non-α globin chains is the major pathophysiological mechanism leading to ineffective erythropoiesis and hemolysis in severe form of the β-thalassemia. Phenotype in patients who retain the capacity to synthesize fetal hemoglobin (HbF) is milder due to decreasing α- /non-α globin chain imbalance. Induction of HbF might be an effective therapeutic strategy for severe form of β-thalassemia. This literature review presents data about efficiency safeness of different medication and their combination, which stimulate synthesis of HbF. This review can provide a new insight into the current status and future perspectives in HbF reactivation as treatment option for severe form of β-thalassemia.

About the authors

M. V. Krasilnikova

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology

Email: marina.krasilnikova@fcchomoscow.ru
ORCID iD: 0000-0002-0929-4792

MD, hematologist, Department of Hematology, Institute of Hematology, Immunology and Cell Technology

Russia 117997, Moscow, Samory Mashela st., 1

 

Russian Federation

N. A. Karamjan

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology

Author for correspondence.
Email: marina.krasilnikova@fcchomoscow.ru
ORCID iD: 0000-0002-9865-527X
Russian Federation

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Copyright (c) 2017 Krasilnikova M.V., Karamjan N.A.

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