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

Although prophylactic treatments can reduce bleeding risk for people with hemophilia (PwH), burdens associated with treatment and physical and psychological symptoms may persist.

To determine treatment and disease burden associated with hemophilia from a global perspective.

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.

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.

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.