Study on Safety and Efficacy of Deferasirox in the treatment of Thalassemia in a South Indian Tertiary Care Hospital

Background: There were very less number of research studies been done in Indian settings or around the world about the efficacy and safety of Deferasirox in thalassemia patients. The objective of this study was to assess the efficacy and safety of Deferasirox in thalassemia patients. Methodology: It is a prospective, observational study conducted for a period of eight months that assessed the laboratory data of thalassemia patients aged ≤ 15 years who were on Deferasirox therapy. The study was conducted in a tertiary care hospital situated in Nellore Andhra Pradesh, India. The study involved the day-to-day review of medication orders and laboratory data in the pediatrics wards. Evaluation included the collection of laboratory data such as serum ferritin levels for the efficacy assessment and alanine transaminase (ALT), serum creatinine, White Blood Cells (WBC) counts and platelet counts for safety assessment. Results: Total numbers of patients enrolled in the study were twenty-four out of 63 thalassemia patients treated in the hospital. Efficacy outcomes resulted that 18 (75%) out of 24 patients responded positively to the Deferasirox, whereas remaining 6 (25%) patients were observed with no changes in their serum ferritin levels. The safety outcomes stated that 11 (45.83%) out of 24 patients were observed with increased ALT levels whereas, the other safety parameters such as serum creatinine, WBCs and platelet counts were observed to be with mild deviations causing no clinical harm to the patients. Conclusion: This study has shown a consistent efficacy in 75% of patients and a manageable safety profile. Thus, it demonstrates that the oral chelation therapy of Deferasirox was efficient and well tolerated, especially in moderately iron loaded transfusion-dependent thalassemia patients.