Regular
blood transfusion is the mainstay of treatment in transfusion-dependent β-
thalassemia (TDT); however, transfusions culminate in an array of serious complications. Therefore, a single-arm, non-randomized clinical trial was conducted in
hydroxyurea refractory TDT patients to explore the long-term safety and efficacy of
thalidomide. The primary outcomes for efficacy were rise in
hemoglobin (Hb) level and changes in transfusion frequency. Whereas, several clinical and laboratory parameters were assessed for safety of
thalidomide. Secondary outcomes included changes in serum
ferritin, serum
lactate dehydrogenase (LDH), serum
uric acid, red blood cell indices, and size of liver and spleen. A total of 532 patients were followed for a period of 30 months. Significant increase in mean Hb level was identified at 6 months (1.4 g/dL, p ≤ 0.001) and 30 months (2 g/dL, p ≤ 0.001) in comparison with baseline. A total of 408 (76.7%) patients responded to
thalidomide therapy (excellent responders 25.8%, good responders 31%, and partial responders 19.9%) and attained transfusion independence within 6 months of
therapy. A significant decline in mean
ferritin, LDH level, liver size, and spleen size was observed. No unfavorable effects were observed on kidney and liver functions. Mild adverse events were reported in 48 (9%) patients and serious adverse events, including cerebral vascular accident and portal vein
thrombosis were reported in two patients each. This study concludes that
thalidomide is an effective and well-tolerated
drug that can improve Hb levels and reduce transfusion burden in
hydroxyurea refractory TDT patients.Trial registration: This trial is registered at http://www.clinicaltrial.gov as # NCT03651102.