Efficacy and adverse effects of oral chelating therapy (deferasirox) in multi-transfused Pakistani children with β-thalassemia major

OBJECTIVE: To determine the efficacy and adverse effects of oral chelation therapy (deferasirox) in multi-transfused β-thalassemia major patients visiting pediatric thalassemia clinic in Civil Hospital Karachi. METHODS: This prospective study was conducted at pediatric thalassemia clinic of Civil Hospital Karachi. Hundred multi-transfused β-thalassemia patients registered in the clinic for oral iron chelation therapy were included in the study. Information regarding clinical and laboratory parameters including abdominal pain, jaundice, serum ferritin, creatinine and serum transaminase levels were recorded on a Performa and data was analyzed through SPSS 16. RESULTS: Hundred patients were stratified into two age groups, 54% were below and 46% were above nine year. Majority were males, 62% and 38% were females. Abdominal pain 41%, nausea 31%, vomiting 15%, jaundice 15% and elevated serum creatinine 11.5% were frequently observed clinical adverse effects in this study. Serum glutamic pyruvic transaminase (SGPT) level was statistically significant compared with initial visit and six months after optimal chelation therapy (p=0.030). Although Serum ferritin was decreased but it was not statistically significant (p=0.929). CONCLUSION: Deferasirox is an effective oral chelation agent for β-thalassemia major patients. Most common adverse effects of the drug are abdominal pain, nausea, vomiting, and elevation of liver enzymes.