Low and fixed dose of hydroxyurea is effective and safe in patients with HbSβ+ thalassemia with IVS1-5(G→C) mutation

Background Despite compelling evidence that hydroxyurea is safe and effective in sickle cell disease, it is prescribed sparingly due to several barriers like knowledge gaps in certain genotypes, apprehension about its safety and toxicity, and limited resources. We undertook this study to find out the efficacy and safety of HU in patients with HbSβ+‐thalassemia with IVS1–5(G→C) mutation. Procedure We registered 318 patients with HbSβ+‐thalassemia with IVS1–5(G→C) mutation. Of these, 203 were enrolled for hydroxyurea treatment at a low and fixed dose of 10 mg/kg/day. One hundred four patients (Group‐I: 37 children and Group‐II: 67 adults) with ≥2 years of hydroxyurea treatment were studied. Results The rate of vaso‐occlusive crises, requirement of blood transfusion and rate of hospitalization reduced from 3 to 0.5, 1 to 0 and 1 to 0 in Group‐I and 3 to 0, 1 to 0 and 0.5 to 0 in Group‐II respectively after HU therapy (P