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Open Access 01-12-2018 | Research

Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias

Authors: Ali T. Taher, Raffaella Origa, Silverio Perrotta, Alexandra Kouraklis, Giovan Battista Ruffo, Antonis Kattamis, Ai-Sim Goh, Vicky Huang, Aiesha Zia, Raquel Merino Herranz, John B. Porter

Published in: Health and Quality of Life Outcomes | Issue 1/2018

Abstract

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.

Available only for authorised users

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Title: Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias
Authors: Ali T. Taher
Raffaella Origa
Silverio Perrotta
Alexandra Kouraklis
Giovan Battista Ruffo
Antonis Kattamis
Ai-Sim Goh
Vicky Huang
Aiesha Zia
Raquel Merino Herranz
John B. Porter
Publication date: 01-12-2018
Publisher: BioMed Central
Published in: Health and Quality of Life Outcomes / Issue 1/2018
Electronic ISSN: 1477-7525
DOI: https://doi.org/10.1186/s12955-018-1041-5

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias

Abstract

Background

Methods

Results

Conclusions

Trial registration

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

Trial registration

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