{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T04:13:53Z","timestamp":1773029633308,"version":"3.50.1"},"reference-count":13,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2019,6,6]],"date-time":"2019-06-06T00:00:00Z","timestamp":1559779200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100010793","name":"Pfizer Japan","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100010793","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2019,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>In 2010, nonacog alfa became the first recombinant factor IX (rFIX) available in Japan for patients with haemophilia B.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To determine real\u2010world safety (adverse events, incidence of inhibitors) and effectiveness of nonacog alfa in Japan.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This multicentre, prospective, observational, postmarketing surveillance study enrolled previously treated and untreated patients (PTPs and PUPs, respectively) who were observed for 1 and 2\u00a0years, respectively, after initiating nonacog alfa therapy. Safety and effectiveness were assessed for each treatment type. Annualized bleeding rate (ABR) and incremental recovery of rFIX were also evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, 312 of 314 patients enrolled from 173 sites were eligible for the safety analysis set (PTPs, 281; PUPs, 28; other, 3). Mean age was 25.4 (PTPs) and 14.8 (PUPs) years. Haemophilic severity ranged from mild to severe, and 133 (42.6%) patients had haemophilic arthropathy. Of 285 patients (PTPs, 257; PUPs, 28) in the effectiveness set, 112 received on\u2010demand treatment for 1161 bleeding episodes (effectiveness rate, 93.7%) and 185 received routine prophylaxis (effectiveness rate, 95.5%). No spontaneous bleeding was observed in 52.4% of patients during prophylactic treatment. Median ABR was lower during routine prophylaxis (2.0) vs the rest of the observation period (8.3). A weak negative correlation was found between body weight and the reciprocal of rFIX recovery. Eleven adverse drug reactions occurred in 7 PTPs (2.2% [7\/312]); recurrence of inhibitor was observed in 1 patient, but no new inhibitor developed in PTPs or PUPs.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Nonacog alfa therapy is safe and effective in the real\u2010world scenario in Japan.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.13783","type":"journal-article","created":{"date-parts":[[2019,6,6]],"date-time":"2019-06-06T07:41:40Z","timestamp":1559806900000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Postmarketing safety and effectiveness of recombinant factor IX (nonacog alfa) in Japanese patients with haemophilia B"],"prefix":"10.1111","volume":"25","author":[{"given":"Katsuyuki","family":"Fukutake","sequence":"first","affiliation":[{"name":"Department of Laboratory Medicine Tokyo Medical University Tokyo Japan"},{"name":"Department of Molecular Genetics of Coagulation Disorders Tokyo Medical University Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masashi","family":"Taki","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, St. Marianna University School of Medicine Yokohama City Seibu Hospital Yokohama Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tadashi","family":"Matsushita","sequence":"additional","affiliation":[{"name":"Department of Transfusion Medicine Nagoya University Hospital Nagoya Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michio","family":"Sakai","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Occupational and Environmental Health Kitakyushu Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ami","family":"Takata","sequence":"additional","affiliation":[{"name":"Pfizer Japan Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiromi","family":"Yamaguchi","sequence":"additional","affiliation":[{"name":"Pfizer Japan Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6230-6679","authenticated-orcid":false,"given":"Toshiyuki","family":"Karumori","sequence":"additional","affiliation":[{"name":"Pfizer Japan Tokyo Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2019,6,6]]},"reference":[{"key":"e_1_2_10_2_1","unstructured":"World Federation of Hemophilia. Report on the annual global survey 2016.http:\/\/www1.wfh.org\/publications\/files\/pdf-1690.pdf. Accessed July 3 2018."},{"key":"e_1_2_10_3_1","unstructured":"World Federation of Hemophilia. Guidelines for the Management of Hemophilia 2nd edition.http:\/\/www1.wfh.org\/publications\/files\/pdf-1472.pdf. Accessed July 3 2018."},{"key":"e_1_2_10_4_1","unstructured":"Medical and Scientific Advisory Council (MASAC). MASAC Recommendation Concerning Prophylaxis.https:\/\/www.hemophilia.org\/sites\/default\/files\/document\/files\/241Prophylaxis.pdf. Accessed July 3 2018."},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa067659"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0037-1963(03)80732-1"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02162.x"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1159\/000488060"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13409"},{"key":"e_1_2_10_10_1","unstructured":"Project entrusted by Ministry of Health Labour and Welfare. Nationwide survey on coagulation disorders 2014. Published by Japan Foundation for AIDS Prevention.http:\/\/api-net.jfap.or.jp\/library\/alliedEnt\/02\/backNumber.html. Accessed July 3 2018."},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2005.1.429"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12229"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.1996.tb00006.x"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2796.1992.tb00546.x"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.13783","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/hae.13783","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.13783","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,10]],"date-time":"2023-09-10T04:38:58Z","timestamp":1694320738000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.13783"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,6,6]]},"references-count":13,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2019,7]]}},"alternative-id":["10.1111\/hae.13783"],"URL":"https:\/\/doi.org\/10.1111\/hae.13783","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,6,6]]},"assertion":[{"value":"2019-01-21","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-05-13","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-06-06","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}