@article {150453, title = {Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST).}, journal = {Pediatric blood \& cancer}, volume = {64}, year = {2017}, month = {2017 Sep}, abstract = {

BACKGROUND: Children with red blood cell disorders may receive regular transfusions from an early age and consequently accumulate iron. Adequate iron chelation therapy can prevent organ damage and delayed growth/development. Deferasirox is indicated for treatment of pediatric patients with chronic iron overload due to transfusional hemosiderosis; however, fewer than 10\% of patients in the registration studies were aged 2 to less than 6 years.

PROCEDURE: Deferasirox, a once-daily oral iron chelator, was evaluated in young pediatric patients with transfusional hemosiderosis during the observational 5-year ENTRUST study. Patients aged 2 to less than 6 years at enrollment received deferasirox according to local prescribing information, with the primary objective of evaluating safety, specifically renal and hepatic function. Serum ferritin was observed as a surrogate efficacy parameter.

RESULTS: In total, 267 patients (mean age 3.2 years) predominantly with β-thalassemia (n = 176, 65.9\%) were enrolled. Mean {\textpm} standard deviation deferasirox dose was 25.8 {\textpm} 6.5 mg/kg per day over a median of 59.9 months. A total of 145 patients (54.3\%) completed 5 years{\textquoteright} treatment. The proportion of patients with two or more consecutive postbaseline measurements (>=7 days apart) of serum creatinine higher than age-adjusted upper limit of normal (ULN) and alanine aminotransferase more than five times the ULN was 4.4\% (95\% confidence interval [CI]: 2.1-7.9) and 4.0\% (95\% CI: 1.8-7.4), respectively. Median serum ferritin decreased from 1,702 ng/ml at baseline to 1,127 ng/ml at 5~years. There were no new safety signals.

CONCLUSIONS: Safety and efficacy of deferasirox in young pediatric patients in this long-term, observational study in everyday clinical practice were consistent with the known deferasirox profile.

}, keywords = {Benzoates, Chelation Therapy, Child, Preschool, Female, Hematologic Diseases, Hemosiderosis, Humans, Iron Chelating Agents, Male, Transfusion Reaction, Triazoles}, issn = {1545-5017}, doi = {10.1002/pbc.26507}, author = {Vichinsky, Elliott and Amal El-Beshlawy and Al Zoebie, Azzam and Kamdem, Annie and Koussa, Suzanne and Chotsampancharoen, Thirachit and Bruederle, Andreas and Gilotti, Geralyn and Han, Jackie and Mohsen Elalfy} }