Evaluating Effectiveness of Deferasirox Versus Deferoxamine in Improving Clinical Outcomes among Patients with Transfusion-Dependent Thalassemia: A Randomized Controlled Trial

Background: Transfusion-dependent β-thalassemia necessitates lifelong blood transfusions that progressively accumulate systemic iron, causing cardiomyopathy, hepatic cirrhosis, and endocrine failure without effective chelation. Deferasirox, a once-daily oral iron chelator, offers a pharmacological and administration advantage over subcutaneous deferoxamine, yet comparative randomized evidence in South Asian clinical populations remains limited. Objective: To compare the efficacy, safety, and treatment adherence profiles of deferasirox versus deferoxamine among patients with transfusion-dependent β-thalassemia in the Islamabad–Rawalpindi region of Pakistan. Methods: A parallel-group, open-label randomized controlled trial was conducted over five months. Seventy-two patients (aged 8–35 years) were randomized 1:1 to deferasirox (20–30 mg/kg/day orally) or deferoxamine (40 mg/kg/day subcutaneously, five days per week). The primary outcome was change in serum ferritin; secondary outcomes included ALT, renal function, adverse events (CTCAE v5.0), and adherence (MMAS-8). Results: Deferasirox produced a significantly greater mean ferritin reduction (715 ± 150 ng/mL; 95% CI [666, 764]) than deferoxamine (335 ± 120 ng/mL; 95% CI [296, 374]; between-group difference 380 ng/mL [317, 443]; p = 0.003; Cohen's d = 2.80). ALT normalized in the deferasirox group (38.2 IU/L) but not in the deferoxamine group (42.4 IU/L; p = 0.01). Composite adverse event rates were 27.8% versus 50.0% (p = 0.04). High adherence was recorded in 83.3% versus 66.7% of patients. Conclusion: Deferasirox demonstrated superior iron chelation efficacy, hepatic enzyme normalization, tolerability, and adherence compared with deferoxamine and should be considered a preferred chelation strategy in routine transfusion-dependent thalassemia management.