Burden of Treatment and Disease on People with Hemophilia: Global Real-World Survey.

INTRODUCTION: Although prophylactic treatments can reduce bleeding risk for people with hemophilia (PwH), burdens associated with treatment and physical and psychological symptoms may persist. AIM: To determine treatment and disease burden associated with hemophilia from a global perspective. METHODS: This cross-sectional patient survey captured patient-reported outcomes, experiences and clinical data from PwH in eight countries between December 2023 and March 2024. Treatment burden was measured using the child/adult Hemophilia Treatment Experience Measure (Hemo-TEM), validated questionnaires covering five domains. Disease burden was measured using the Patient-Reported Outcomes Measurement Information System 29+2 version 2.1 (PROMIS-29+2)/PROMIS Parent Proxy, validated questionnaires comprising eight domains. Here we report data on treatment, physical and psychological burdens for people with moderate and severe hemophilia without inhibitors by treatment class: standard half-life and extended half-life factor replacement for hemophilia A (HA) and hemophilia B (HB), and non-factor therapy for HA. RESULTS: Data from 495 PwH and parents/guardians of PwH were analyzed. Across the five Hemo-TEM domains, adults with HA experienced similar degrees of treatment burden regardless of treatment class, with comparable results observed for adults with HB. Around one-third of PwH experienced soreness (33% HA; 31% HB) and pain (31% HA; 26% HB) due to their current treatment at least sometimes, and one in five experienced some level of treatment bother. PROMIS-29+2 scores indicated that people with HA and HB experienced similar degrees of physical and psychological burden regardless of treatment class. Anxiety and worry were prevalent among PwH. CONCLUSION: PwH continue to experience treatment- and disease-related burdens. Managing hemophilia still requires a balance between bleed protection, quality of life and ease of treatment administration.