Abstract
This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden. Randomization will be stratified by age groups (2 to <10 years, 10 to><18 years) and prior iron chelation therapy (yes no). there will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either deferasirox dt or granules, and an optional extension phase where all patients will receive the granules up to 5 years. patients who demonstrated benefit to granules or dt in the core phase, and or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or fct) or up to 5 years, whichever occurs first.>
