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

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/m(2) (>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.

Automated summary

This study investigates the associations between patient-reported outcomes (PROs) and patient, treatment, and disease characteristics in people living with haemophilia A (PLWHA). The results show that older age, poorer joint health, and higher body mass index (BMI) adversely affect multiple aspects of participant well-being.