Research Article: 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

Date Published: August 12, 2012

Publisher: Hindawi Publishing Corporation

Author(s): 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.


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.

Partial Text

Regular blood transfusions are essential for the management of haematological conditions such as β-thalassemia major and myelodysplastic syndromes (MDS). As a result, however, patients with these conditions are susceptible to the development of transfusion-dependent iron overload (hemosiderosis or secondary iron overload). In the absence of a naturally occurring physiological mechanism for the removal of excess iron in the body, life-long treatment and adherence to iron chelation therapy (ICT) are necessary to prevent the morbidity and mortality that may result if excess iron is allowed to accumulate [1, 2].

The EPIC study is the largest prospective evaluation of any iron chelation therapy conducted to date. Findings from this study have demonstrated that deferasirox is an efficacious and generally well-tolerated treatment for the treatment of iron overload in patients with transfusion-dependent disorders such as β-thalassemia and MDS [12, 13]. In addition, this study provided a unique opportunity to collect data concerning the role that deferasirox can play in addressing HRQOL concerns associated with iron chelation therapies and how deferasirox may help address issues related to treatment satisfaction, adherence, and persistence among patients with iron overload.

This substudy represents the largest prospective evaluation of patient-reported outcomes with deferasirox to date. Findings indicate improvements in patient-reported HRQOL, ICT satisfaction, adherence, and persistence following treatment with deferasirox particularly among β-thalassemia patients, the majority of whom had been using infused ICT prior to enrolment in the study. Patient satisfaction with deferasirox is high and patients receiving deferasirox report being more likely to adhere and persist with ICT. Such evaluations are vital for improving both the long-term health outcomes and survival of patients with transfusion-dependent iron overload and minimising future health resource use.