title: 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.
abstract: 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.
authors.affiliation: Department of Haematology, UCL Cancer Institute, University College London, Paul O'Gorman Building, 72 Huntley Street, London WC1E 6BT, UK.
authors.forename: John
authors.initials: J
authors.lastname: Porter
authors.forename: Donald K
authors.initials: DK
authors.lastname: Bowden
authors.forename: Marina
authors.initials: M
authors.lastname: Economou
authors.forename: Jacques
authors.initials: J
authors.lastname: Troncy
authors.forename: Arnold
authors.initials: A
authors.lastname: Ganser
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Nicolas
authors.initials: N
authors.lastname: Martin
authors.forename: Adam
authors.initials: A
authors.lastname: Gater
authors.forename: Diana
authors.initials: D
authors.lastname: Rofail
authors.forename: Linda
authors.initials: L
authors.lastname: Abetz-Webb
authors.forename: Helen
authors.initials: H
authors.lastname: Lau
authors.forename: Maria Domenica
authors.initials: MD
authors.lastname: Cappellini