@article{3078247,
    title = "Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: Phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study",
    author = "Tartaglione, I. and Origa, R. and Kattamis, A. and Pfeilstöcker, M. and Gunes, S. and Crowe, S. and Fagan, N. and Vincenzi, B. and Ruffo, G.B.",
    journal = "Experimental Hematology and Oncology",
    year = "2020",
    volume = "9",
    number = "1",
    publisher = "BioMed Central Ltd.",
    doi = "10.1186/s40164-020-00174-2",
    keywords = "deferasirox;  ferritin, abdominal pain;  adult;  Article;  asthenia;  controlled study;  coughing;  diarrhea;  disease severity;  drug efficacy;  drug safety;  drug tolerability;  drug withdrawal;  female;  ferritin blood level;  fever;  film coating;  gastroenteritis;  headache;  human;  hypertransaminasemia;  influenza;  major clinical study;  male;  monotherapy;  multicenter study;  musculoskeletal pain;  myelodysplastic syndrome;  nausea;  oropharynx pain;  outcome assessment;  pharyngitis;  phase 3 clinical trial;  priority journal;  proteinuria;  randomized controlled trial;  rhinitis;  tablet;  thalassemia;  upper abdominal pain;  urinary tract infection;  vomiting",
    abstract = "Background: Despite the proven benefits of iron chelation therapy (ICT) in the management of chronic iron overload and related complications, compliance to long-term ICT is challenging. Results from the ECLIPSE study, an open-label, randomized, multicenter, 2-arm, phase 2 study evaluated the safety of deferasirox dispersible tablet and film-coated tablet (FCT) formulations in patients with transfusion-dependent thalassemia (TDT) or very low, low, or intermediate risk myelodysplastic syndrome (MDS) treated over 24 weeks. Methods: The aim of the current study (a 2-year, open-label, multicenter, single-arm, phase 3 study) is to evaluate the long-term safety and efficacy of deferasirox FCT in a subset of patients with TDT or lower/intermediate-risk MDS treated for 2 years after the completion of 24 weeks of treatment with deferasirox in the ECLIPSE phase 2 study. Results: Of 53 patients enrolled, 34 (64.2%) completed treatment and study. Adverse events (AEs) reported in most patients (~ 70%) were of mild to moderate severity. Headache and diarrhea were the most frequently (> 25%) reported AEs. None of the serious AEs (including 1 death) were considered treatment related. No new safety signal was identified, and long-term safety of deferasirox FCT was consistent with the known safety profile of deferasirox. No major concerns associated with gastrointestinal tolerability, renal safety, or hematological abnormalities (thrombocytopenia/neutropenia) were reported during the 2 years. Patients receiving deferasirox FCT had a treatment compliance (by pill count) of ~ 90% and persistence (continuous use for ≥ 30 days) of > 95%. Reduction in serum ferritin level was almost consistent starting from week 2 across all post-baseline time points (relative reduction: month 6, 19%; month 12, 29%). Conclusions: The results from this 2-year interventional study suggest that the recommended dosing of deferasirox FCT, with better tolerability, palatability, and compliance, offers a favorable option of ICT for long-term management of iron overload and associated complications in TDT. Trial registration ClinicalTrials.gov, NCT02720536. Registered 28 March 2016, https://www.clinicaltrials.gov/ct2/show/NCT02720536 © 2020 The Author(s)."
}