New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, Phase II ECLIPSE study

American University of Beirut Medical Center, Beirut, Lebanon; Ospedale Pediatrico Microcitemico 'A.Cao', University of Cagliari, Italy; Second University of Naples, Naples, Italy; Hematology Division, Department of Internal Medicine, University of Patras Medical School, Patras, Greece; U.O.C. Ematolog. Con Talassemia, A.O. Civico-Di Cristina-Benfratelli, Palermo, Italy; First Department of Pediatrics, University of Athens, Athens, Greece; Department of Medicine, Hospital Pulau Pinang, Georgetown, Penang, Malaysia; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Novartis Pharma AG, Basel, Switzerland; University College London, London, UK.

American Journal of Hematology. 2017;92((5):):420-428
Once-daily deferasirox dispersible tablets (DT) have a well-defined safety and efficacy profile and, compared with parenteral deferoxamine, provide greater patient adherence, satisfaction, and quality of life. However, barriers still exist to optimal adherence, including gastrointestinal tolerability and palatability, leading to development of a new film-coated tablet (FCT) formulation that can be swallowed with a light meal, without the need to disperse into a suspension prior to consumption. The randomized, open-label, Phase II ECLIPSE study evaluated the safety of deferasirox DT and FCT formulations over 24 weeks in chelation-naive or pre-treated patients aged ≥10 years, with transfusion-dependent thalassemia or IPSS-R very-low-, low- or intermediate-risk myelodysplastic syndromes. 173 patients were randomized 1:1 to DT (n=86) or FCT (n=87). Adverse events (overall), consistent with the known deferasirox safety profile, were reported in similar proportions of patients for each formulation (DT 89.5%; FCT 89.7%), with a lower frequency of severe events observed in patients receiving FCT (19.5% vs. 25.6% DT). Laboratory parameters (serum creatinine, creatinine clearance, alanine aminotransferase, aspartate aminotransferase and urine/protein creatinine ratio) generally remained stable throughout the study. Patient-reported outcomes showed greater adherence and satisfaction, better palatability and fewer concerns with FCT than DT. Treatment compliance by pill count was higher with FCT (92.9%) than with DT (85.3%). This analysis suggests deferasirox FCT offers an improved formulation with enhanced patient satisfaction, which may improve adherence, thereby reducing frequency and severity of iron overload-related complications. This article is protected by copyright. All rights reserved.
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