Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia
Issued Date
2023-01-01
Resource Type
ISSN
09395555
eISSN
14320584
Scopus ID
2-s2.0-85160286785
Pubmed ID
37227493
Journal Title
Annals of Hematology
Rights Holder(s)
SCOPUS
Bibliographic Citation
Annals of Hematology (2023)
Suggested Citation
Viprakasit V., Hamdy M.M., Hassab H.M.A., Sherief L.M., Al-Bagshi M., Khattab M., Chuncharunee S., Dung P.C., Küpesiz A., Shekhawat A., Sonawane Y., Perez L.T., Slader C., Taher A.T. Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia. Annals of Hematology (2023). doi:10.1007/s00277-023-05240-3 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/82956
Title
Patient preference for deferasirox film-coated versus dispersible tablet formulation: a sequential-design phase 2 study in patients with thalassemia
Author's Affiliation
Ramathibodi Hospital
Siriraj Hospital
American University of Beirut Medical Center
Faculty of Medicine
Zagazig University, Faculty of Medicine
Cairo University
Akdeniz Üniversitesi
Novartis International AG
Ibn Sina Hospital, Agdal Rabat
Hereditary Blood Diseases Centre
Novartis Healthcare Private Limited
Blood Transfusion and Hematology Hospital
Siriraj Hospital
American University of Beirut Medical Center
Faculty of Medicine
Zagazig University, Faculty of Medicine
Cairo University
Akdeniz Üniversitesi
Novartis International AG
Ibn Sina Hospital, Agdal Rabat
Hereditary Blood Diseases Centre
Novartis Healthcare Private Limited
Blood Transfusion and Hematology Hospital
Other Contributor(s)
Abstract
Iron chelation therapy (ICT) is the mainstay of treatment in patients with thalassemia requiring blood transfusions. This phase 2 JUPITER study evaluated patient preference between film-coated tablet (FCT) and dispersible tablet (DT) in transfusion-dependent thalassemia (TDT) or non-TDT (NTDT) patients treated with both formulations in a sequential manner. The primary endpoint was patient-reported preference for FCT over DT, while secondary outcomes included patient reported outcomes (PROs) evaluated by overall preference, and by age, thalassemia transfusion status, and previous ICT status. Out of 183 patients screened, 140 and 136 patients completed the treatment periods 1 and 2 of the core study, respectively. At week 48, the majority of patients preferred FCT over DT (90.3 vs. 7.5%; difference of percentage: 0.83 [95% confidence interval (CI), 0.75–0.89; P < 0.0001]). FCT scored better on secondary PROs and showed less severe gastrointestinal symptoms than DT, except in the change of modified Satisfaction with Iron Chelation Therapy (mSICT) preference scores, which were similar for both the formulations. Patients with TDT had stable ferritin levels, while it showed a downward trend up to week 48 in patients with NTDT on deferasirox treatment. Overall, 89.9% of patients reported ≥ 1 adverse event (AE), of which 20.3% experienced ≥ 1 serious AE. The most common treatment-emergent AEs were proteinuria, pyrexia, urine protein/creatinine ratio increase, diarrhea, upper respiratory tract infections, transaminase increase, and pharyngitis. Overall, this study reinforced the observations from the previous study by showing a distinct patient preference for FCT over DT formulation and further supported the potential benefits of life-long compliance with ICT.