A 3-year study of deferasirox therapy in sickle cell disease patients in Basra, Southern Iraq.

Background: Patients with sickle cell disease (SCD) may require repeated transfusions, which inevitably lead to iron overload (IOL).

Aims: : This study aims to assess the effectiveness and safety of oral deferasirox (DFX) in patients with SCD and transfusional IOL.

Patients and Methods: A descriptive study has been performed on patients with SCD who have completed at least 3 years on DFX. Height and weight were checked every 3-6 months. The efficacy was assessed based on serum ferritin (SF) levels. The safety was assessed based on adverse events (AEs), alanine aminotransferase (ALT), and serum creatinine (S. Cr) levels.

Results: : A total of 102 patients (61 males and 41 females) were recruited. Their mean daily iron intake was 0.13 ± 0.06 mg/kg. SF levels declined significantly from 2434.1 ± 132.9 ng/ml at the start of the study to 1655.8 ± 154.2 ng/ml at the end of the study (P < 0.05), with significant decreases observed after increasing the DFX dose to ≥ 30 mg/kg/day. ALT (12.8 ± 9.9 vs. 12.1 ± 7.1 U/L) and S. Cr (72.4 ± 9.2 vs. 74.1 ± 7.9 mmol/L) levels did not show significant differences from the start to the end of the study (P > 0.05). Thirty-eight patients (37%) developed AEs. The most common were abdominal pain (24.5%), diarrhea (8.0%), and nausea (7.8%). AEs were predominantly transient and mild to moderate in nature.

Conclusions: This study has revealed that DFX is a safe, tolerable, and effective drug for reducing IOL in SCD patients, though it is associated with mild and transient adverse events.