Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias
AT Taher, R Origa, S Perrotta, A Kouraklis… - Health and quality of life …, 2018 - Springer
AT Taher, R Origa, S Perrotta, A Kouraklis, GB Ruffo, A Kattamis, AS Goh, V Huang, A Zia…
Health and quality of life outcomes, 2018•SpringerBackground Adherence to long-term chelation therapy in transfusion-dependent patients is
critical to prevent iron overload-related complications. Once-daily deferasirox dispersible
tablets (DT) have proven long-term efficacy and safety in patients≥ 2 years old with chronic
transfusional iron overload. However, barriers to optimal adherence remain, including
palatability, preparation time, and requirements for fasting state. A new film-coated tablet
(FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light …
critical to prevent iron overload-related complications. Once-daily deferasirox dispersible
tablets (DT) have proven long-term efficacy and safety in patients≥ 2 years old with chronic
transfusional iron overload. However, barriers to optimal adherence remain, including
palatability, preparation time, and requirements for fasting state. A new film-coated tablet
(FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light …
Background
Adherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications. Once-daily deferasirox dispersible tablets (DT) have proven long-term efficacy and safety in patients ≥2 years old with chronic transfusional iron overload. However, barriers to optimal adherence remain, including palatability, preparation time, and requirements for fasting state. A new film-coated tablet (FCT) formulation was developed, swallowed once daily (whole/crushed) with/without a light meal.
Methods
The open-label, Phase II ECLIPSE study evaluated patient-reported outcomes (PROs) in transfusion-dependent thalassemia or lower-risk myelodysplastic syndromes patients randomized 1:1 to receive deferasirox DT or FCT over 24 weeks as a secondary outcome of the study. Three PRO questionnaires were developed to evaluate both deferasirox formulations: 1) Modified Satisfaction with Iron Chelation Therapy Questionnaire; 2) Palatability Questionnaire; 3) Gastrointestinal (GI) Symptom Diary.
Results
One hundred seventy three patients were enrolled; 87 received the FCT and 86 the DT formulation. FCT recipients consistently reported better adherence (easier to take medication, less bothered by time to prepare medication and waiting time before eating), greater satisfaction/preference (general satisfaction and with administration of medicine), and fewer concerns (less worry about not swallowing enough medication, fewer limitations in daily activities, less concern about side effects). FCT recipients reported no taste or aftertaste and could swallow all their medicine with an acceptable amount of liquid. GI summary scores were low for both formulations.
Conclusions
These findings suggest a preference in favor of the deferasirox FCT formulation regardless of underlying disease or age group. Better patient satisfaction and adherence to chelation therapy may reduce iron overload-related complications.
Trial registration
ClinicalTrials.gov identifier: NCT02125877; registered April 26, 2014.
Springer
以上显示的是最相近的搜索结果。 查看全部搜索结果