PRO136 PATIENT-REPORTED BURDEN OF TRANSFUSION-DEPENDENT BETA-THALASSEMIA IN THE USA AND UK MEASURED USING A DIGITAL APP

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Evaluate real-world management and symptom burden of transfusion-dependent β-thalassemia (TDT) in the United States and United Kingdom. Individuals with TDT were recruited to a 3-month study to investigate the time and symptom burden of TDT and its impact on health-related quality of life (HRQoL). Adult participants or caregivers for individuals with TDT aged 12–17 years, provided data via MyThalLog – a bespoke smartphone app. Daily burden assessments were made via the following: • Time burden questionnaire • Brief Fatigue Inventory (BFI) ‘worst fatigue’ question. • Brief Pain Inventory - Short Form ‘worst pain’ question Participants also completed the Transfusion-dependent quality of life (TranQoL) questionnaire and full BFI monthly. The time burden of TDT was substantial on transfusion days (mean: 8.8 hours, median: 8.2, Interquartile Range [IQR]: 3.4). The average weekly time burden in non-transfusion weeks was 41.4 hours (median: 10.2, IQR: 87.5 - variation driven by time for chelation preparation) for participants on non-oral chelators and 2.87 hours (median: 1.2, IQR: 1.2) for those on oral chelation only. Worst fatigue and pain scores observed in the five-day, pre-transfusion period were high (mean: 5.6 and 4.8, respectively; 10-point scale; 10 = worst symptoms). There was a clinically relevant reduction in fatigue post-transfusion (mean reductions versus transfusion day, over days one to three: 1.2, 1.4, 1.8 points, respectively; minimal clinically important difference [MCID]: 1 point). Pain remained persistent post-transfusion (mean reductions over days one to three: 0.9, 1.1, 1.4 points, respectively; MCID: 2 points). TranQoL (mean: 62.9, median: 63.7, IQR: 23.9; 100-point scale; 100 = best HRQoL) and BFI (mean: 5.5, median: 6.2, IQR: 4.0; 10-point scale; 10 = worst symptoms) scores were lower (i.e., worse) than reported previously in TDT and oncology populations from TranQoL and BFI validation studies, respectively. Time managing disease, fatigue, pain, and HRQoL impairment contributed to participant reported TDT burden.