Deferasirox use after hematopoietic stem cell transplantation in pediatric patients with beta-thalassemia major: preliminary results.

摘要

There are limited data on the posttransplantation pharmacological treatment of iron overload in ex-thalassemic patients and the current approach is phlebotomy. The authors chelated 2 ex-thalassemic patients after hematopoietic stem cell transplantation with deferasirox for 6 and 24 months. Although serum ferritin levels decreased, cardiac and hepatic iron load, measured by T2* magnetic resonance imaging (MRI), showed decrease in iron overload in these organs. The drug was tolerated well by both patients and no adverse effect on donor hematopoiesis was observed. This preliminary study demonstrates that deferasirox is well tolerated in these patients and will be a good potential therapy when more data have been obtained from larger studies.