| 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 | Five-Year Longitudinal Changes in Treatment Strategies and Clinical Outcomes in Adults With Haemophilia: The ADVANCE Japan Cohort Study. |
| 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 | Online ahead of print |
| 著者・共著者 | Azusa Nagao, Akihiro Sawada, Takeshi Kanematsu, Naoya Yamasaki, Hideyuki Takedani, Masanori Nojima, Yukio Hiroi, Teruhisa Fujii, Nobuaki Suzuki, Tadashi Matsushita, Satoshi Higasa, Kagehiro Amano, Yushi Chikasawa |
| 発行年月 | 2026/08 |
| 概要 | BACKGROUND:Prophylactic treatment for haemophilia has evolved with the introduction of extended half-life (EHL) and nonfactor products. However, long-term real-world data describing how these advances have affected adults with haemophilia remain limited.OBJECTIVES:To evaluate 5-year changes in treatment strategies and clinical outcomes in adults aged ≥40 years with haemophilia.METHODS:We analyzed 5-year longitudinal data from ADVANCE Japan, a nationwide prospective registry of adults aged ≥40 years with haemophilia.RESULTS:Of approximately 600 registered participants, 568 inhibitor-negative individuals were included in this analysis. The median follow-up duration was 4.99 years (Q1-Q3, 4.89-5.92). In haemophilia A, treatment strategies shifted towards EHL and nonfactor therapies over time. Among participants with severe haemophilia A receiving prophylaxis, median injection frequency decreased from 2.0 to 1.0 injection per week over 5 years (p < 0.001). In contrast, among participants with severe haemophilia B, injection frequency remained unchanged (p = 0.563). Despite these therapeutic transitions, annualized bleeding rate (ABR) and annualized joint bleeding rate (AjBR) showed no significant longitudinal changes across age groups in either haemophilia A or B. The proportion of participants with at least one target joint decreased from 88/568 (15.5%) at baseline to 72/548 (13.1%) in 2024.CONCLUSIONS:Over 5 years, treatment strategies in adults with haemophilia A shifted towards EHL and nonfactor therapies, with reduced injection frequency among severe haemophilia A receiving prophylaxis. Bleeding rates remained stable and target joints modestly decreased, suggesting that bleeding outcomes alone may not fully reflect the evolving clinical burden in aging adults with haemophilia.TRIAL REGISTRATION:This study is registered in the Japan Registry of Clinical Trials (JRCT; https://jrct.mhlw.go.jp/latest-detail/jRCT1030190046). |
| DOI | 10.1111/hae.70370 |
| PMID | 42649344 |