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

Authors: Hay, CRM.; Makris, M; Shima, M; Nagao, A; Jiménez-Yuste, V; Skinner, M; Kessler, CM.; von Mackensen, S.

Affiliations: Manchester University Department of Haematology, Manchester, UK. Department of Infection, Immunity and Cardiovascular Disease, University of Sheffield, Sheffield, UK. Nara Medical University, Kashihara, Japan. Department of Blood Coagulation, Ogikubo Hospital, Tokyo, Japan. Autónoma University Madrid and La Paz University Hospital, Madrid, Spain. Institute for Policy Advancement Ltd., Washington, DC, USA. Department of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, Ontario, Canada. Georgetown University Medical Center, Washington, DC, USA. Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.

Publication: Haemophilia. 2023

ABSTRACT: 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.