{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,26]],"date-time":"2026-04-26T00:01:29Z","timestamp":1777161689401,"version":"3.51.4"},"reference-count":23,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2019,2,6]],"date-time":"2019-02-06T00:00:00Z","timestamp":1549411200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004337","name":"Roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004337","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2019,3]]},"abstract":"<jats:sec><jats:title>Introduction<\/jats:title><jats:p>Prospectively collected real\u2010world data on bleeds, haemophilia treatment and safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited. A global, non\u2010interventional study (NIS; NCT02476942) prospectively collected real\u2010world data in PwHA who were treated per local routine clinical practice.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>Assess annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs) in adult\/adolescent PwHA without inhibitors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Eligible participants aged \u226512\u00a0years with severe HA without history of inhibitors prospectively collected bleeding and treatment information.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Ninety\u2010four participants were enrolled (median [range] age, 34 [12\u201076]\u00a0years) and monitored for a median (range) of 29.8 (12.4\u201047.7)\u00a0weeks. In the episodic (n\u00a0=\u00a045) and prophylactic (n\u00a0=\u00a049) treatment groups, respectively, 872\/1066 (81.8%) and 151\/189 (79.9%), bleeds were treated; ABRs (95% confidence interval) were 36.1 (30.8\u201042.3) and 5.0 (3.3\u20107.5), respectively, for treated bleeds and 43.1 (36.5\u201050.9) and 6.2 (4.2\u20109.2), respectively, for all bleeds, and median (interquartile range) ABRs were 31.1 (19.8\u201051.6) and 1.9 (0.0\u20108.2), respectively, for treated bleeds and 35.3 (21.7\u201062.9) and 2.7 (0.0\u20109.4), respectively, for all bleeds. Half of the participants on FVIII prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median doses\/wk of standard and extended half\u2010life FVIII, respectively. Serious AEs included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE was viral upper respiratory tract infection.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>PwHA without inhibitors continue to bleed on prophylaxis, consistent with the literature, and require treatment for breakthrough bleeds. This prospective NIS demonstrates the need for more efficacious haemostatic approaches.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.13655","type":"journal-article","created":{"date-parts":[[2019,2,6]],"date-time":"2019-02-06T12:45:37Z","timestamp":1549457137000},"page":"213-220","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non\u2010interventional study in a real\u2010world setting"],"prefix":"10.1111","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6547-5240","authenticated-orcid":false,"given":"Rebecca","family":"Kruse\u2010Jarres","sequence":"first","affiliation":[{"name":"Washington Center for Bleeding Disorders at Bloodworks Northwest Seattle Washington"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3038-2145","authenticated-orcid":false,"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"Universit\u00e4tsklinikum Bonn Bonn Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elena","family":"Santagostino","sequence":"additional","affiliation":[{"name":"Centro Emofilia e Trombosi A. Bianchi Bonomi IRCCS Fondazione Ca' Granda Milano Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Midori","family":"Shima","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Nara Medical University Kashihara Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2391-2016","authenticated-orcid":false,"given":"Christine L.","family":"Kempton","sequence":"additional","affiliation":[{"name":"Department of Hematology and Medical Oncology Emory School of Medicine Atlanta Georgia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Craig M.","family":"Kessler","sequence":"additional","affiliation":[{"name":"Lombardi Comprehensive Cancer Center Georgetown University Medical Center Washington District of Columbia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michaela","family":"Lehle","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd Basel Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sammy","family":"Chebon","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd Basel Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nives","family":"Selak Bienz","sequence":"additional","affiliation":[{"name":"F. Hoffmann\u2010La Roche Ltd Basel Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elina","family":"Asikanius","sequence":"additional","affiliation":[{"name":"F. 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