Objective: To evaluated serum ferritin levels in children having beta-thalassemia major with iron-overload treated with deferasirox.

Study Design: A prospective open-label observational study.

Place and Duration of the Study: The Outpatient Department of Hematological Disorder, Thalassemia and Bone Marrow Transplantation Centre, and Department of Biochemistry, “Bahawal Victoria Hospital, Quaid e Azam Medical College”, Bahawalpur, Pakistan from February 2020 to January 2021.

Material and Methods: Children aged 2 to 17 years with diagnosis of β-thalassemia major having chronic iron overload (serum ferritin > 1000 ng/ml) and 2 or more blood transfusions per month were included. Demographic information like gender, age, and residential status, contact number along with assessment of hepatitis serology, HIV status, electrocardiogram and ophthalmic findings were noted on a pre-designed proforma specially designed for this study. Deferasirox was initiated in a dose of 20 mg/kg/day and was adjusted at 3-months interval. Serum ferritin levels were measured at baseline, 3-months, 6-months, 9-months and 12-months.

Results: In a total of 40 children, there were 23 (57.5%) male. Overall, mean age was calculated to be 9.1+4.2 years. Residential status of 28 (70.0%) children was rural. Mothers of 27 (67.5%) children were illiterate. At baseline, mean serum ferritin level was noted to be 4137+2319 ng/ml. Significant reduction in mean serum ferritin levels during the course of this study was found with the use of deferasirox as mean serum ferritin levels reduced significantly from baseline to 3-months, 6-months, 9-months and at 12-months as 3637+2119 ng/ml, 4081+2050 ng/ml, 4346+2349 ng/ml, 3252+2464 ng/ml and 2188+1195 ng/ml respectively (p<0.001).

Conclusion: Deferasirox was found to be an effective iron chelation therapy among transfusion dependent thalassemia children with iron-overload.

Keywords: serum ferritin, Thalassemia, iron-overload, deferasirox.