Improved patient satisfaction, adherence and health-related quality of life with deferasirox (Exjade) in beta-thalassemia patients previously receiving other iron chelation therapies.

Introduction: Iron chelation therapy (ICT) is essential in removing excess iron deposited in body organs, ultimately preventing organ failure and extending the lives of patients (pts) with transfusion-dependent hematological disorders such as beta-thalassemia. Conventional ICT (deferoxamine, Desferal, DFO) requires a burdensome regimen (subcutaneous delivery 5-7 times a week) that has been shown to negatively impact pts' health-related quality of life (HRQL) and adherence to therapy. Adherence to ICT affects survival and therefore a well-tolerated and effective ICT regimen is required. The once-daily oral chelator, deferasirox (Exjade) offers 24-hour ICT, 7 days a week and is potentially less burdensome to pts. Method(s): As part of a larger single-arm, multicenter, open-label trial investigating the efficacy and safety of deferasirox (EPIC study), 217 beta-thalassemia pts >16 years of age were included in this subanalysis. Findings reported here investigate differences in satisfaction, adherence and HRQoL following treatment with deferasirox, in beta-thalassemia patients with prior experience of DFO monotherapy, or DFO in combination with deferiprone (Ferriprox, L1). All pts were asked at baseline and week 52 (end of study [EOS]) to complete the 19-item Satisfaction with ICT questionnaire (SICT) and the 36-item Short Form health survey (SF-36). Mean change scores between baseline and EOS on the four domains of the SICT (Perceived Effectiveness of ICT, Side Effects of ICT, Acceptance of ICT, and Burden of ICT) and the eight domains (Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health) and two summary scores (Physical and Mental Component Scores) of the SF-36 were calculated for patients who completed the respective instruments at both timepoints. Higher scores on the SICT and SF-36 represent greater HRQL, satisfaction with, and adherence to treatment. Adherence to deferasirox was assessed