Improved Patient Satisfaction, Adherence and Health-Related Quality of Life with Deferasirox (Exjade®) in β-Thalassemia Patients Previously Receiving Other Iron Chelation Therapies.
Authors: Porter John B. , Athanasiou-Metaxa Miranda , Bowden Donald K. , Troncy Jacques , Habr Dany , Domokos Gabor , Roubert Bernard , Rofail Diana , Gater Adam , Baladi Jean-François , Cappellini M. Domenica 
Published: October 2019

Abstract
Abstract 2486
Poster Board II-463

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 β-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.

Methods:
As part of a larger single-arm, multicenter, open-label trial investigating the efficacy and safety of deferasirox (EPIC study), 217 β-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 β-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 by two SICT items asking pts how often they thought about stopping their ICT, and how often they took their ICT exactly as directed by their doctor.

Results:
217 β-thalassemia pts were recruited (mean age 30.6 ± 7.7); 42.9% (n = 93) male, 57.1% (n = 124) female. Of 216 pts with prior history of ICT, 62.0% (n=134) had prior DFO monotherapy and 37.0% (n=80) had prior DFO in combination with L1 (2 patients had prior LI monotherapy; not included in this analysis due to the small patient number). SICT and SF-36 mean-change domain scores were based on patients who completed questionnaires at baseline and EOS (SICT: 105 DFO and 46 DFO+L1 pts; SF-36: 90 DFO and 42 DFO+L1 pts). Significant improvements (p<0.0001) in mean-change SICT domain scores were reported at EOS for both pts having previously received DFO and DFO+L1: Side Effects of ICT (DFO: 1.50 ± 1.28; DFO+L1: 1.19 ± 1.32); Acceptance of ICT (DFO: 1.73 ± 1.18; DFO+L1: 1.49 ± 1.26); and Burden of ICT (DFO: 1.49 ± 1.00; DFO+L1: 1.04 ± 1.06). No statistically significant differences in mean-change scores for the Perceived Effectiveness of ICT domain were found for either group. At EOS, a greater proportion of pts in both groups reported always following their ICT regimen, compared with baseline (DFO: 69.2% vs 36.0%; DFO+L1: 69.5% vs 32.8%) and never thinking about stopping ICT (DFO: 80.0% vs 44.0%; DFO+L1: 74.6% vs 38.8%). Mean change SF-36 domain scores in pts with prior DFO monotherapy were positive for all domains at EOS (indicating improvement from baseline). Mean change scores for Physical Functioning (5.60 ± 20.08, p <0.005), Bodily Pain (10.13 ± 28.81, p <0.001), General Health Perceptions (3.47 ± 17.62, p <0.05), Social Functioning (6.53 ± 26.65, p <0.05) and the Physical Component score (2.71 ± 8.80, p<0.005) all reached statistical significance. Mean change SF-36 domain scores in pts with prior DFO+L1 therapy were also positive for domains of Role-Physical, Bodily Pain, Vitality, Mental Health and Physical and Mental Component Scores. However, none of these scores reached statistical significance at the p<0.05 level.

Conclusions:
β-thalassemia pts with prior history of ICT (DFO monotherapy or DFO+L1) demonstrated improved satisfaction and adherence to ICT following treatment with deferasirox. Improvements in HRQL were also observed and were generally greater in pts previously treated with DFO monotherapy compared with pts who previously received DFO+L1 combination therapy.

Disclosures:
Porter: Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Vifor International: Membership on an entity's Board of Directors or advisory committees. Habr:Novartis Pharmaceuticals: Employment. Domokos:Novartis Pharma AG: Employment. Roubert:Novartis Pharma AG: Employment. Rofail:Employed by Mapi Values, a health outcomes research agency that consults to the pharmaceutical industry. We are paid consultants for Novartis Pharmaceutical Corporation for patient-reported outcomes in iron overload: Consultancy. Gater:Employed by Mapi Values, a health outcomes research agency that consults to the pharmaceutical industry. We are paid consultants for Novartis Pharmaceutical Corporation for patient-reported outcomes in iron overload: Consultancy. Baladi:Novartis Pharmaceuticals: Employment. Cappellini:Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Genzyme: Membership on an entity's Board of Directors or advisory committees.