Association of patient, treatment and disease characteristics with patient-reported outcomes
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Association of patient, treatment and disease characteristics with patient-reported outcomes : Results of the ECHO Registry. / Hay, Charles R M; Makris, Michael; Shima, Midori; Nagao, Azusa; Jiménez-Yuste, Víctor; Skinner, Mark; Kessler, Craig M; von Mackensen, Sylvia.
In: HAEMOPHILIA, Vol. 30, No. 1, 01.2024, p. 106-115.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Association of patient, treatment and disease characteristics with patient-reported outcomes
T2 - Results of the ECHO Registry
AU - Hay, Charles R M
AU - Makris, Michael
AU - Shima, Midori
AU - Nagao, Azusa
AU - Jiménez-Yuste, Víctor
AU - Skinner, Mark
AU - Kessler, Craig M
AU - von Mackensen, Sylvia
N1 - © 2023 The Authors. Haemophilia published by John Wiley & Sons Ltd.
PY - 2024/1
Y1 - 2024/1
N2 - 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.
AB - 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.
KW - Humans
KW - United States
KW - Hemophilia A/drug therapy
KW - Treatment Outcome
KW - Quality of Life
KW - Prospective Studies
KW - Registries
KW - Pain
KW - Surveys and Questionnaires
KW - Patient Reported Outcome Measures
U2 - 10.1111/hae.14895
DO - 10.1111/hae.14895
M3 - SCORING: Journal article
C2 - 38030962
VL - 30
SP - 106
EP - 115
JO - HAEMOPHILIA
JF - HAEMOPHILIA
SN - 1351-8216
IS - 1
ER -