title: 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.
abstract: 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-naïve or pre-treated patients aged ≥10 years, with transfusion-dependent thalassemia or IPSS-R very-low-, low-, or intermediate-risk myelodysplastic syndromes. One hundred seventy-three 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.
authors.affiliation: American University of Beirut Medical Center, Beirut, Lebanon.
authors.forename: Ali T
authors.identifier: http://orcid.org/0000-0001-8515-2238
authors.initials: AT
authors.lastname: Taher
authors.affiliation: Ospedale Pediatrico Microcitemico "A. Cao,", University of Cagliari, Cagliari, Italy.
authors.forename: Raffaella
authors.initials: R
authors.lastname: Origa
authors.affiliation: Department of Pediatrics, Second University of Naples, Naples, Italy.
authors.forename: Silverio
authors.initials: S
authors.lastname: Perrotta
authors.affiliation: Hematology Division, Department of Internal Medicine, University of Patras Medical School, Patras, Greece.
authors.forename: Alexandra
authors.initials: A
authors.lastname: Kourakli
authors.affiliation: U.O.C. Ematolog. Con Talassemia, A.O. Civico-Di Cristina-Benfratelli, Palermo, Italy.
authors.forename: Giovan Battista
authors.initials: GB
authors.lastname: Ruffo
authors.affiliation: First Department of Pediatrics, University of Athens, Athens, Greece.
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.affiliation: Department of Medicine, Hospital Pulau Pinang, Georgetown, Penang, Malaysia.
authors.forename: Ai-Sim
authors.initials: AS
authors.lastname: Goh
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
authors.forename: Annelore
authors.initials: A
authors.lastname: Cortoos
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
authors.forename: Vicky
authors.initials: V
authors.lastname: Huang
authors.affiliation: Novartis Pharma AG, Basel, Switzerland.
authors.forename: Marine
authors.initials: M
authors.lastname: Weill
authors.affiliation: Novartis Pharma AG, Basel, Switzerland.
authors.forename: Raquel
authors.initials: R
authors.lastname: Merino Herranz
authors.affiliation: Department of Haematology, University College London, London, UK.
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000008:administration & dosage*; D002626:Chemistry, Pharmaceutical / Q000379:methods; D002648:Child; D000077588:Deferasirox; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D010349:Patient Compliance; D011788:Quality of Life; D013608:Tablets, Enteric-Coated / Q000008:administration & dosage / Q000009:adverse effects / Q000737:chemistry; D013789:Thalassemia / Q000188:drug therapy*; D014230:Triazoles / Q000008:administration & dosage*; D055815:Young Adult
chemical_list: D001565:Benzoates; D013608:Tablets, Enteric-Coated; D014230:Triazoles; D000077588:Deferasirox