Association of patient, treatment and disease characteristics with patient‐reported outcomes: Results of the ECHO Registry

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2024-01, Vol.30 (1), p.106-115
• Main Authors: Hay, Charles R. M., Makris, Michael, Shima, Midori, Nagao, Azusa, Jiménez‐Yuste, Víctor, Skinner, Mark, Kessler, Craig M., Mackensen, Sylvia
• Format: Article
• Language: English
• Subjects: body mass index; Body weight; Clinical outcomes; Data collection; factor VIII; haemophilia A; Hemophilia; Pain; patient reported outcome measures; Patients; Population studies; Quality of life; registries; Statistical analysis
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.14895

Introduction Patient‐reported outcomes (PROs) in people living with haemophilia A (PLWHA) are often under‐reported. Investigating PROs from a single study with a diverse population of PLWHA is valuable, irrespective of FVIII product or regimen. Aim To report available data from the Expanding Communications on Haemophilia A Outcomes (ECHO) registry investigating the associations of patient, treatment and disease characteristics with PROs and clinical outcomes in PLWHA. Methods ECHO (NCT02396862), a prospective, multinational, observational registry, enrolled participants aged ≥16 years with moderate or severe haemophilia A using any product or treatment regimen. Data collection, including a variety of PRO questionnaires, was planned at baseline and annually for ≥2 years. Associations between PRO scores and patient, treatment and disease characteristics were determined by statistical analyses. Results ECHO was terminated early owing to logistical constraints. Baseline data were available from 269 PLWHA from Europe, the United States and Japan. Most participants received prophylactic treatment (76.2%), with those using extended‐half‐life products (10.0%) reporting higher treatment satisfaction. Older age and body weight >30 kg/m2 (>BMI) were associated with poorer joint health. Older age was associated with poorer physical functioning and work productivity. Health‐related quality of life and pain interference also deteriorated with age and >BMI; >BMI also increased pain severity scores. Conclusion ECHO captured a variety of disease characteristics, treatment patterns, PROs and clinical outcomes obtained in real‐world practice with ≤1 year's follow‐up. Older age, poorer joint health and >BMI adversely affected multiple aspects of participant well‐being.