| 論文種別 | 原著(症例報告除く) |
| 言語種別 | 英語 |
| 査読の有無 | 査読あり |
| 表題 | Association of patient, treatment and disease characteristics with patient-reported outcomes: Results of the ECHO Registry. |
| 掲載誌名 | 正式名：Haemophilia : the official journal of the World Federation of Hemophilia 略 称：Haemophilia ISSNコード：13652516/13518216 |
| 掲載区分 | 国外 |
| 巻・号・頁 | 30(1),pp.106-115 |
| 著者・共著者 | Charles R M Hay, Michael Makris, Midori Shima, Azusa Nagao, Víctor Jiménez-Yuste, Mark Skinner, Craig M Kessler, Sylvia von Mackensen |
| 発行年月 | 2024/01 |
| 概要 | 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. |
| DOI | 10.1111/hae.14895 |
| PMID | 38030962 |