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

Taher AT 1 , Origa R 2 , Perrotta S 3 , Kouraklis A 4 , Ruffo GB 5 , Kattamis A 6 Show all authors , Goh AS 7 , Huang V 8 , Zia A 9 , Herranz RM 9 , Porter JB 10

Affiliations 

  • 1 Department of Internal Medicine, Director - Fellowship and Residents Research Program, Faculty of Medicine American University of Beirut Medical Center, Beirut, Lebanon. [email protected]
  • 2 Ospedale Pediatrico Microcitemico 'A.Cao', A.O. 'G. Brotzu', Cagliari, Italy
  • 3 Università della Campania "L. Vanvitelli,", Caserta, Italy
  • 4 Hematology Division, Department of Internal Medicine, University of Patras Medical School, Patras, Greece
  • 5 U.O.C. Ematolog. Con Talassemia, A.O. Civico-Di Cristina-Benfratelli, Palermo, Italy
  • 6 First Department of Pediatrics, University of Athens, Athens, Greece
  • 7 Department of Medicine, Hospital Pulau Pinang, Georgetown, Penang, Malaysia
  • 8 Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA
  • 9 Novartis Pharma AG, Basel, Switzerland
  • 10 University College London, London, UK
Health Qual Life Outcomes, 2018 Nov 19;16(1):216.
PMID: 30453981 DOI: 10.1186/s12955-018-1041-5

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.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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