Abstract
BackgroundAdherence 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.MethodsThe 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.ResultsOne 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.ConclusionsThese 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 registrationClinicalTrials.gov identifier: NCT02125877; registered April 26, 2014.
Highlights
Adherence to long-term chelation therapy in transfusion-dependent patients is critical to prevent iron overload-related complications
Barriers still exist to optimal adherence with deferasirox dispersible tablets (DT), including the need to take the drug in a fasting state, requirements for careful dispersion prior to ingestion, the chalky consistency, and suboptimal gastrointestinal (GI) tolerability [17]
The treatment groups were balanced with respect to type of anemia and prior chelation therapy, with 70 transfusion-dependent thalassemia (TDT) patients in each arm and 77 and 79 previously chelated patients in the DT and film-coated tablet (FCT) arm, respectively
Summary
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. In recognition of the importance of patient adherence to chelation therapy, a film-coated tablet (FCT) formulation for oral administration (either whole or crushed and mixed with soft foods) was developed as an alternative treatment option intended to improve patient acceptability and compliance. Both FCT and DT contain the same active substance (deferasirox) and are administered once daily. In contrast to the DT, the FCT does not contain the excipients lactose and sodium lauryl sulphate and can be taken with or without a light meal [19, 20] offering a more simple and convenient mode of administration with potential improvements in GI tolerability
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