Transfusing children with hemoglobinopathies

Abstract

Thalassemia and sickle cell disease (SCD) are among the most common inherited diseases worldwide. Red blood cell transfusion is a cornerstone of their treatment, but its indications have significantly changed over the past years. New therapies are emerging in both syndromes: among them, hematopoietic stem cell transplantation is now routinely proposed, and gene therapy has shown promising preliminary results.

Keywords: Drépanocytose; Gene therapy; Greffe de cellules souches hématopoïétiques; Hematopoietic stem cell transplantation; Hydroxyurea; Hydroxyurée; Red blood cell transfusion; Sickle cell disease; Thalassemia; Thalassémie; Thérapie génique; Transfusion érythrocytaire.