ISSN 2997-1036
International Journal of Hematology | Vol. 5, No. 6, June 2014 | pp. 41–48
DOI: 10.46882/2014/IJH/000054
Original Article
Title: Efficacy and compliance tracking of low-dose deferasirox therapy in transfusion-dependent beta-thalassemia major patients
Names of Authors: S. T. Adeyemi¹, U. V. Okoye², W. X. Salami³
Authors’ Affiliations: ¹Department of Haematology and Blood Transfusion, Obafemi Awolowo University, Ile-Ife, Nigeria; ²Department of Medicine, University of Nigeria Teaching Hospital, Enugu, Nigeria; ³Department of Paediatrics, Ahmadu Bello University, Zaria, Nigeria
Abstract: Chronic red blood cell transfusions are mandatory for survival in beta-thalassemia major but lead to systemic iron overload, requiring effective chelation therapy. This 12-month prospective study evaluated the efficacy, safety, and compliance tracking of a low-dose oral deferasirox regimen (15 to 20 mg/kg/day) in 35 transfusion-dependent pediatric and young adult patients. Iron overload was monitored via serial serum ferritin levels at 3-month intervals, while monthly creatinine and liver enzyme checks tracked drug safety. Baseline mean serum ferritin was 3,450 ± 620 ng/ml. After 12 months of deferasirox therapy, mean serum ferritin fell significantly to 2,120 ± 450 ng/ml (P < 0.01) in compliant individuals. Drug adherence, assessed via pill counts and structured interviews, was high at 82.8%. Mild, transient increases in serum creatinine (> 33% above baseline) occurred in 11.4% of patients but resolved without dose interruption. Gastrointestinal side effects, mostly mild diarrhea or nausea, occurred in 22.8% of cases. Low-dose oral deferasirox demonstrates satisfactory efficacy in reducing systemic iron burden with a manageable safety profile, making it a viable options for improving compliance in low-resource environments.
Keywords: Beta-thalassemia major, iron overload, deferasirox, iron chelation therapy, ferritin
Manuscript Timeline: Received: March 15, 2014; Revised: April 24, 2014; Accepted: May 12, 2014; Published: June 18, 2014