Health-Related Quality of Life, Treatment Satisfaction, Adherence and Persistence in β-Thalassemia and Myelodysplastic Syndrome Patients with Iron Overload Receiving Deferasirox: Results from the EPIC Clinical Trial
John Porter,
Donald K. Bowden,
Marina Economou,
Jacques Troncy,
Arnold Ganser,
Dany Habr,
Nicolas Martin,
Adam Gater,
Diana Rofail,
Linda Abetz-Webb,
Helen Lau,
Maria Domenica Cappellini
Affiliations
John Porter
Department of Haematology, UCL Cancer Institute, University College London, Paul O'Gorman Building, 72 Huntley Street, London WC1E 6BT, UK
Donald K. Bowden
Monash Medical Centre, Melbourne, VIC 3168, Australia
Marina Economou
Thalassemia Clinical Care Services Unit, Hippokration General Hospital Thessaloniki, Egnatia Street 106, 54622 Thessaloniki, Greece
Jacques Troncy
Hematology, Hopital Edouard Herriot, 6 Rue Antoine Lumiere, 69008 Lyon, France
Arnold Ganser
Medizinische Hochschule Hannover (MHH), Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Carl-Neuberg Strasse 1, 30625 Hannover, Germany
Dany Habr
Novartis Pharmaceutical Corporation, 180 Park Avenue, 105-3E065, Florham Park, NJ 07932-1080, USA
Nicolas Martin
Novartis Pharma AG Postfach, 4002 Basel, Switzerland
Treatment of iron overload using deferoxamine (DFO) is associated with significant deficits in patients' health-related quality of life (HRQOL) and low treatment satisfaction. The current article presents patient-reported HRQOL, satisfaction, adherence, and persistence data from β-thalassemia (n=274) and myelodysplastic syndrome (MDS) patients (n=168) patients participating in the Evaluation of Patients' Iron Chelation with Exjade (EPIC) study (NCT00171821); a large-scale 1-year, phase IIIb study investigating the efficacy and safety of the once-daily oral iron chelator, deferasirox. HRQOL and satisfaction, adherence, and persistence to iron chelation therapy (ICT) data were collected at baseline and end of study using the Medical Outcomes Short-Form 36-item Health Survey (SF-36v2) and the Satisfaction with ICT Questionnaire (SICT). Compared to age-matched norms, β-thalassemia and MDS patients reported lower SF-36 domain scores at baseline. Low levels of treatment satisfaction, adherence, and persistence were also observed. HRQOL improved following treatment with deferasirox, particularly among β-thalassemia patients. Furthermore, patients reported high levels of satisfaction with deferasirox at end of study and greater ICT adherence, and persistence. Findings suggest deferasirox improves HRQOL, treatment satisfaction, adherence, and persistence with ICT in β-thalassemia and MDS patients. Improving such outcomes is an important long-term goal for patients with iron overload.
