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
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...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-07-01
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| Series: | Research and Practice in Thrombosis and Haemostasis |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S247503792500295X |
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| author | Teruhisa Fujii Keiji Nogami Akihiro Sawada Azusa Nagao Chiai Nagae Masanori Nojima Nobuaki Suzuki Mika Kawano Tomomi Shimura Yoshimasa Sugao Kagehiro Amano |
| author_facet | Teruhisa Fujii Keiji Nogami Akihiro Sawada Azusa Nagao Chiai Nagae Masanori Nojima Nobuaki Suzuki Mika Kawano Tomomi Shimura Yoshimasa Sugao Kagehiro Amano |
| author_sort | Teruhisa Fujii |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-08ebd8be368445ea967320f9bce6d326 |
| institution | Kabale University |
| issn | 2475-0379 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Research and Practice in Thrombosis and Haemostasis |
| spelling | doaj-art-08ebd8be368445ea967320f9bce6d3262025-08-20T04:02:23ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792025-07-019510297110.1016/j.rpth.2025.102971TSUBASA study: evaluation of the quality and content of daily life of people with hemophilia A without factor VIII inhibitors on prophylactic treatment with emicizumabTeruhisa Fujii0Keiji Nogami1Akihiro Sawada2Azusa Nagao3Chiai Nagae4Masanori Nojima5Nobuaki Suzuki6Mika Kawano7Tomomi Shimura8Yoshimasa Sugao9Kagehiro Amano10Division of Transfusion Medicine, Hemophilia Treatment Center, Hiroshima University Hospital, Hiroshima, Japan; Correspondence Teruhisa Fujii, Division of Transfusion Medicine, AIDS Care Team, Hemophilia Treatment Center, Hiroshima University Hospital, 1-2-3, Kasumi, Minami-ku, Hiroshima City, Hiroshima, 734-8551, Japan.Department of Pediatrics, Nara Medical University, Nara, JapanDepartment of Respiratory Medicine and Hematology, Hyogo Medical University, Hyogo, JapanDepartment of Hematology and Oncology, Kansai Medical University Hospital, Osaka, JapanDepartment of Pediatrics, St. Marianna University School of Medicine, Kanagawa, JapanDivision of Advanced Medicine Promotion/Center for Translational Research, Institute of Medical Science, University of Tokyo, Tokyo, JapanDepartment of Transfusion Medicine, Nagoya University Hospital, Aichi, JapanChugai Pharmaceutical Co, Ltd, Tokyo, JapanChugai Pharmaceutical Co, Ltd, Tokyo, JapanChugai Pharmaceutical Co, Ltd, Tokyo, JapanDepartment of Laboratory Medicine, Tokyo Medical University, Tokyo, JapanBackground: 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.http://www.sciencedirect.com/science/article/pii/S247503792500295XhemophiliaemicizumabJapanprospective studyquality of life |
| spellingShingle | Teruhisa Fujii Keiji Nogami Akihiro Sawada Azusa Nagao Chiai Nagae Masanori Nojima Nobuaki Suzuki Mika Kawano Tomomi Shimura Yoshimasa Sugao Kagehiro Amano 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 Research and Practice in Thrombosis and Haemostasis hemophilia emicizumab Japan prospective study quality of life |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | hemophilia emicizumab Japan prospective study quality of life |
| url | http://www.sciencedirect.com/science/article/pii/S247503792500295X |
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