論文種別	原著(症例報告除く)
言語種別	英語
査読の有無	その他(不明)
表題	TSUBASA study: evaluation of the quality and content of daily life of people with hemophilia A without factor VIII inhibitors on prophylactic treatment with emicizumab.
掲載誌名	正式名：Research and practice in thrombosis and haemostasis 略　 称：Res Pract Thromb Haemost ISSNコード：24750379/24750379
掲載区分	国外
巻・号・頁	9(5),pp.102971
著者・共著者	Teruhisa Fujii, Keiji Nogami, Akihiro Sawada, Azusa Nagao, Chiai Nagae, Masanori Nojima, Nobuaki Suzuki, Mika Kawano, Tomomi Shimura, Yoshimasa Sugao, Kagehiro Amano
発行年月	2025/07
概要	BACKGROUND:Hemophilia A (HA) negatively impacts quality of life (QoL). Treatment with the bispecific antibody emicizumab has shown efficacy and safety in people with HA in clinical trials, but long-term QoL data are limited.OBJECTIVES:To investigate the QoL of people with HA receiving emicizumab over 97 weeks in the prospective, observational TSUBASA study in Japan.METHODS:Data were collected from participants aged ≥6 years with HA without factor VIII inhibitors and caregivers of participants of any age from November 2019 to October 2023. Quality of daily life was measured via the 36-Item Short Form Health Survey, the International Physical Activity Questionnaire, the Work Productivity and Activity Impairment Questionnaire and Classroom Impairment Questionnaire: Hemophilia Specific, and a survey-based questionnaire evaluating daily life, completed by the participant and their caregiver.RESULTS:Overall, 104 participants aged ≥6 years were enrolled. The median (range) age was 39.0 (6-73) years. Eighty-five (81.7%) participants had severe HA; 21 (20.2%) had target joints. The 36-Item Short Form Health Survey scores were mostly unchanged across the study period and comparable with Japanese national standard values. The proportion of participants engaging in high physical activity increased from 20.2% to 27.3% between baseline and week 97. The Work Productivity and Activity Impairment Questionnaire and Classroom Impairment Questionnaire: Hemophilia Specific scores were generally stable. The questionnaires showed improvements in activity, motivation for work/school, and bleed anxiety, as judged by participants and caregivers.CONCLUSION:QoL outcomes remained largely unchanged across the study period. Notable improvements were observed in physical activity levels, motivation for work/school, and anxiety related to bleeding, as reported by participants and caregivers.
DOI	10.1016/j.rpth.2025.102971
PMID	40821744