Long-Term Therapy with Deferasirox in Young Pediatric Patients with Transfusional Hemosiderosis Completing up to 5 Years of Treatment in the Observational E.N.T.R.U.S.T. Study

• Published in: Blood 2016-12, Vol.128 (22), p.2470-2470
• Main Authors: Vichinsky, Elliott, El-Beshlawy, Amal, Alzoebie, Azzam, Kamdem, Annie, Koussa, Suzanne, Chotsampancharoen, Thirachit, Bruederle, Andreas, Han, Jackie, Elalfy, Mohsen
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V128.22.2470.2470

▪ Introduction:Regular supportive transfusion therapy from an early age is common practice in young children with β thalassemia major (βTM), sickle-cell disease (SCD) and Diamond-Blackfan anemia (DBA). Patients consequently accumulate iron that can affect organ function and delay growth/development. As such, iron chelation therapy is often necessary from an early age, with lifelong requirements for patients who cannot be cured with hematopoietic stem cell transplantation. Deferasirox, a once-daily oral iron chelator, has demonstrated efficacy and safety in adult and pediatric patients with chronic iron overload. Long-term evaluation of deferasirox in young pediatric patients with transfusional hemosiderosis is valuable, as conducted in the 5-yr multinational, observational ENTRUST study. Here, we report safety and efficacy outcomes in pediatric patients with transfusional hemosiderosis receiving up to 5 yrs of continuous deferasirox treatment in clinical practice. Methods:Patients aged 2-