OBJECTIVE: To determine the efficacy and adverse effects of deferiprone in patients with Thalassemia Major.

METHODS: A prospective case series study was conducted at the Fatimid Foundation Blood Bank and Haematological Diseases center Lahore. A total of 87 patients entered into the study between September 2005 and November 2006. Deferiprone was given at subsidized rates at a dose of 75/mg/day for seven days. Physical examination and initial Laboratory investigations were done in all patients at the start of the study. Physical and laboratory data were filled on a questionnaire and analyzed using SPSS version 10.0.

RESULTS: Eighty seven patients with mean age of 10.0 +/- 4.33 years (range 4-27 years) were included in the study. Mean follow up was 8 +/- 3.94 months (range 2-12 months). The mean Ferritin at the start of study was 4656 +/- 2052.5 ug/L (range 1200-14630 ug/L) and at the end of study period was 4139 +/- 1710.4 ug/L (range 749-8961) (p < 0.001). Adverse events were joint pains in 10% patients, gastrointestinal symptoms in 11% and no adverse events in 79% patients. There was no evidence of agranulocytosis in any patient.

CONCLUSION: Deferiprone was well tolerated, had few adverse effects and was effective in lowering the patient's serum ferritin level.