Τίτλος:
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
Γλώσσες Τεκμηρίου:
Αγγλικά
Περίληψη:
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. © 2017 Wiley Periodicals, Inc.
Συγγραφείς:
Taher, A.T.
Origa, R.
Perrotta, S.
Kourakli, A.
Ruffo, G.B.
Kattamis, A.
Goh, A.-S.
Cortoos, A.
Huang, V.
Weill, M.
Merino Herranz, R.
Porter, J.B.
Περιοδικό:
American Journal of Hematology
Εκδότης:
Wiley-Liss, Inc.
Λέξεις-κλειδιά:
alanine aminotransferase; aspartate aminotransferase; creatinine; deferasirox; deferiprone; deferoxamine; ferritin; benzoic acid derivative; deferasirox; enteric coated tablet; triazole derivative, abdominal pain; alanine aminotransferase blood level; Article; aspartate aminotransferase blood level; constipation; controlled study; creatinine blood level; creatinine clearance; dehydration; diarrhea; drug combination; drug overdose; drug safety; drug tolerability; drug withdrawal; dysphagia; ferritin blood level; gastrointestinal symptom; headache; human; iron chelation; iron overload; kidney failure; major clinical study; medication compliance; nausea; palatability; patient compliance; patient satisfaction; patient-reported outcome; phase 2 clinical trial; priority journal; protein urine level; proteinuria; randomized controlled trial; tablet formulation; thalassemia; treatment response; virus infection; vomiting; administration and dosage; adolescent; adult; adverse effects; aged; chemistry; child; clinical trial; enteric coated tablet; female; male; medicinal chemistry; middle aged; Myelodysplastic Syndromes; procedures; quality of life; thalassemia; very elderly; young adult, Adolescent; Adult; Aged; Aged, 80 and over; Benzoates; Chemistry, Pharmaceutical; Child; Female; Humans; Male; Middle Aged; Myelodysplastic Syndromes; Patient Compliance; Quality of Life; Tablets, Enteric-Coated; Thalassemia; Triazoles; Young Adult
