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

Abstract

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.

Keywords: Basra, deferasirox, sickle cell disease