ASH 2016 | Film-coated tablet deferasirox formulation for thalassemia and myelodysplastic syndromes (MDS)

Ali T. Taher, MD, PhD, from the American University of Beirut Medical Center, Beirut, Lebanon, discusses the safety and efficacy of a new deferasirox formulation for thalassemia and myelodysplastic syndromes (MDS) at the American Society of Hematology (ASH) Congress 2016 in San Diego, CA. He explains that thalassemia patients require continuous blood transfusion, and as a result of this they also need a chelator to remove excess iron from their body. Dr Taher describes how both iron chelators currently available, deferasirox and deferoxamine, have problems which resulted in decreased patient adherence to treatment. He introduces the Phase II randomized multicenter E.C.L.I.P.S.E. trial (NCT02125877), which included 173 patients with thalassemia and myelodysplasia (MDS) who were separated into two arms, receiving either the standard of care dispersible tablet deferasirox treatment or a film-coated tablet formulation of deferasirox. Dr Taher explains why standard of care deferasirox treatment had problems with adherence, as it needed to be dissolved for over an hour, and taken on an empty stomach early in the morning. The trial aimed to assess the safety of the new film-coated formulation, as well as determining patient-reported outcomes. He summarizes data showing the drug is very well tolerated, with less gastrointestinal side-effects than the standard of care formulation. Patients taking the new formulation also showed a trend of reduction in their serum ferritin. Dr Taher also discusses patient reported outcomes of this trial, which showed that patients felt the drug was well tolerated and reported adherence and compliance, with no palatability problems, and no time missed from work and school.