Περίληψη:
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).
Συγγραφείς:
Tartaglione, I.
Origa, R.
Kattamis, A.
Pfeilstöcker, M.
Gunes, S.
Crowe, S.
Fagan, N.
Vincenzi, B.
Ruffo, G.B.
Λέξεις-κλειδιά:
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
