Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study
Norbert Gattermann, Carlo Finelli, Matteo Della Porta, Pierre Fenaux, Arnold Ganser, Agnes Guerci-Bresler, Mathias Schmid, Kerry Taylor, Dominique Vassilieff, Dany Habr, Gabor Domokos, Bernard Roubert, Christian Rose
Leukemia Research | PERGAMON-ELSEVIER SCIENCE LTD | Published : 2010
The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500ng/mL, approximately 50% were chelation-naïve. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naïve or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reducti..View full abstract
The study was sponsored by Novartis Pharma AG (Basel, Switzerland) and designed by the sponsor in consultation with a board of expert hematologists. The sponsor and research organization working under contract for the sponsor monitored the investigational sites, collected and managed the data, and conducted the statistical analysis. An independent Study Monitoring Committee (SMC) over saw the execution of the study and the lead investigators and SMC had access to the final study data and provided significant contributions to the data interpretation. Dr Gattermann prepared the manuscript with the assistance of a medical writer. Financial support for medical editorial assistance was provided by Novartis. All other named authors contributed to the interpretation of the results, critically reviewed and commented on the manuscript, and approved the final version before submission. Dr Gattermann made all final decisions about all aspects of publication. We thank Andrew Jones, PhD for medical editorial assistance with this manuscript.