Efficacy of decitabine as hemoglobin F inducer in HbE/β-thalassemia.

To study safety, efficacy (hemoglobin and hemoglobin F percentage increment in non-transfusion-dependent patients and decrease in transfusion frequency in transfusion-dependent patients), and determinants of response of decitabine in patients with HbE/β-thalassemia. Thirty patients of HbE/β-thalassemia (age > 18 years) were enrolled. Both transfusion-dependent (TDT) and non-transfusion-dependent (NTDT) patients were included after obtaining informed consent. Participants received 0.2 mg/kg of 5-aza-2'-deoxycytidine (decitabine) subcutaneously on 2 consecutive days a week for at least 12 weeks. Complete hemogram was done every 2 weeks and HPLC at every 4-week interval, until 2 months after last dose of drug for response assessment. Various factors like XMN1 polymorphism, IVS 1-5, alpha deletion, alpha triplication, baseline hemoglobin F, and baseline total hemoglobin were evaluated as determinants of response. Mean therapy period was 20.32 weeks. For NTDT group, peak mean increment in hemoglobin was 0.938 g/dl (p value < .001) and hemoglobin F percentage was 9.62% (p value < .001). Transfusion requirement decreased to 0.25 units compared to 0.96 units per patient per month for TDT patients over a period of last 1 year. Common side effects were respiratory tract infection (grade I/II) in three patients, chest tightness in one patient (grade I), and gastric erosion (grade III) in one patient. Decitabine is safe and efficacious in HbE/β-thalassemia.