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

Muhammad Mohsin Shoukat; Hafiza Khadija Tariq; Fareed Ullah; Muhammad Arslan Liaqat; Areeba Sajjad; Nauman Akhtar

doi:10.61919/e41dwh72

Authors

Muhammad Mohsin Shoukat Consultant Pediatrician, THQ Hospital, Hasilpur, District Bahawalpur, Pakistan Author
Hafiza Khadija Tariq Final Year Pharm-D Student, NIST Multan, affiliated with Bahauddin Zakariya University, Multan, Pakistan Author
Fareed Ullah Medical Officer, Mufti Mehmood Memorial Hospital, Quetta, Pakistan Author
Muhammad Arslan Liaqat Doctor of Medicine, Asian Medical Institute, Kant, Kyrgyzstan Author
Areeba Sajjad Department of Pharmacy, The University of Faisalabad, Faisalabad, Pakistan, Faculty of Pharmaceutical Sciences, Lahore University of Medical and Applied Sciences, Lahore, Pakistan Author
Nauman Akhtar PAF Hospital, Faisal, Karachi, Pakistan Author

DOI:

https://doi.org/10.61919/e41dwh72

Keywords:

Adherence; Deferasirox; Deferoxamine; Iron Overload; Randomized Controlled Trial; Thalassemia; Transfusion-Dependent

Abstract

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.

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