Pilot Study on the Efficacy and Safety of Thalidomide in Transfusion-Dependent and Non-transfusion-Dependent...

Thalassemia is a heterogeneous group of inherited hemoglobin disorders broadly classified into transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT). Chronic transfusion therapy in TDT often leads to complications, including iron overload and alloimmunization. Thalidomide, known for its immunomodulatory and fetal hemoglobin (HbF)-inducing properties, has shown promise in improving hematologic parameters in thalassemia. This study aimed to evaluate the efficacy and safety of thalidomide in patients with TDT and NTDT. A prospective, non-randomized study was conducted over two years at a tertiary care center in Eastern India. A total of 61 patients (25 TDT and 36 NTDT), aged 2-50 years, received thalidomide at 2-3 mg/kg/day for 24 weeks. Clinical and laboratory parameters, including hemoglobin levels, serum ferritin, HbF, organomegaly, and transfusion requirements, were assessed at baseline and post-treatment. Thalidomide therapy resulted in a significant rise in hemoglobin and HbF levels in both TDT and NTDT groups (p < 0.05). Serum ferritin levels, spleen and liver sizes, and transfusion needs were significantly reduced. By six months, 40% of TDT patients became transfusion-independent, while 80.5% of NTDT patients achieved a major hematologic response. Adverse effects were mild and included constipation, rash, and neutropenia. Thalidomide appears to be an effective and well-tolerated treatment in TDT and NTDT, offering benefits such as improved hemoglobin levels and reduced transfusion burden. Larger, randomized trials are warranted to confirm long-term safety and efficacy.