A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with Β-Thalassemia Major

• Deferasirox up to 20 mg/kg/day significantly reduced serum ferritin in children with β-thalassemia major post-HSCT.• Deferasirox also decreased LIC and increased cardiac T2* levels.• Deferasirox was well tolerated with only 6 AEs requiring dose adjustment or interruption.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research