{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T00:54:16Z","timestamp":1790729656257,"version":"4.1.0"},"reference-count":15,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2016,6,28]],"date-time":"2016-06-28T00:00:00Z","timestamp":1467072000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100004191","name":"Novo Nordisk","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004191","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008322","name":"CSL Behring","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008322","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2016,7]]},"abstract":"<jats:sec><jats:title>Introduction<\/jats:title><jats:p>Health\u2010related quality of life (<jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL) of individuals with haemophilia has greatly improved with the use of factor replacement and routine prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To explore the <jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL of individuals with haemophilia B treated with nonacog beta pegol, an extended half\u2010life recombinant factor <jats:styled-content style=\"fixed-case\">IX<\/jats:styled-content>, in a single\u2010blind, randomized multinational phase <jats:styled-content style=\"fixed-case\">III<\/jats:styled-content> pivotal trial (paradigm<jats:sup>\u2122<\/jats:sup>2) and its open\u2010label extension (paradigm<jats:sup>\u2122<\/jats:sup>4).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In the pivotal trial, adolescents and adults with haemophilia B were allocated to 28\u2010week on\u2010demand treatment or randomized to 52 weeks of prophylaxis with 10 or 40 <jats:styled-content style=\"fixed-case\">IU<\/jats:styled-content> kg<jats:sup>\u22121<\/jats:sup> nonacog beta pegol administered every seven days. In the extension trial, patients could continue on the same treatment or switch to the alternate dosing regimen at any time. <jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL was assessed with the <jats:styled-content style=\"fixed-case\">HAEMO<\/jats:styled-content>\u2010<jats:styled-content style=\"fixed-case\">QOL<\/jats:styled-content>\/<jats:styled-content style=\"fixed-case\">HAEM<\/jats:styled-content>\u2010A\u2010<jats:styled-content style=\"fixed-case\">QOL<\/jats:styled-content> age\u2010specific questionnaires and the <jats:styled-content style=\"fixed-case\">EQ<\/jats:styled-content>\u20105D.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In the pivotal trial, adults receiving 40 <jats:styled-content style=\"fixed-case\">IU<\/jats:styled-content> kg<jats:sup>\u22121<\/jats:sup> prophylaxis reported significant improvements in the \u2018<jats:styled-content style=\"fixed-case\">HAEM<\/jats:styled-content>\u2010A\u2010<jats:styled-content style=\"fixed-case\">QOL<\/jats:styled-content> Total\u2019 score (\u22126.4 \u00b1 8.5, <jats:italic>P<\/jats:italic> = 0.017) and in \u2018Sport\u2019 (\u221215.3 \u00b1 8.5, <jats:italic>P<\/jats:italic> = 0.020), \u2018Feeling\u2019 (\u221215.2 \u00b1 18.3, <jats:italic>P<\/jats:italic> = 0.010) and \u2018Partnership\u2019 (\u22129.6 \u00b1 15.5, <jats:italic>P<\/jats:italic> = 0.046) domain scores; no significant improvements were seen in the other arms. At the pivotal trial end, fewer patients reported problems in the <jats:styled-content style=\"fixed-case\">EQ<\/jats:styled-content>\u20105D \u2018Mobility\u2019 and \u2018Pain\/Discomfort\u2019 dimensions, in particular those receiving prophylaxis. In the extension trial, adult patients switching from 10 to 40 <jats:styled-content style=\"fixed-case\">IU<\/jats:styled-content> kg<jats:sup>\u22121<\/jats:sup> prophylaxis showed significant improvements in \u2018<jats:styled-content style=\"fixed-case\">HAEM<\/jats:styled-content>\u2010A\u2010<jats:styled-content style=\"fixed-case\">QOL<\/jats:styled-content> Total\u2019 score (\u221212.5 \u00b1 8.7, <jats:italic>P<\/jats:italic> = 0.016) and \u2018Physical health\u2019 domain (\u221223.1 \u00b1 14.4, <jats:italic>P<\/jats:italic> = 0.016).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Prophylactic treatment with nonacog beta pegol 40 <jats:styled-content style=\"fixed-case\">IU<\/jats:styled-content> kg<jats:sup>\u22121<\/jats:sup> once weekly leads to <jats:styled-content style=\"fixed-case\">HRQ<\/jats:styled-content>oL benefits in individuals with haemophilia B; this might be related to fewer bleeding episodes and higher <jats:styled-content style=\"fixed-case\">FIX<\/jats:styled-content> activity levels.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.12995","type":"journal-article","created":{"date-parts":[[2016,6,29]],"date-time":"2016-06-29T04:13:19Z","timestamp":1467173599000},"source":"Crossref","is-referenced-by-count":26,"title":["Improvement in health\u2010related quality of life in patients with haemophilia B treated with nonacog beta pegol, a new extended half\u2010life recombinant <scp>FIX<\/scp> product"],"prefix":"10.1111","volume":"22","author":[{"given":"P.","family":"Chowdary","sequence":"first","affiliation":[{"name":"Katharine Dormandy Haemophilia Centre and Thrombosis Unit Royal Free London NHS Foundation Trust London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S.","family":"Kearney","sequence":"additional","affiliation":[{"name":"CHCMN Hemophilia and Thrombosis Center Children's Hospital and Clinics of Minnesota Minneapolis MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Regnault","sequence":"additional","affiliation":[{"name":"Patient\u2010Centered Outcomes Mapi Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. S.","family":"Hoxer","sequence":"additional","affiliation":[{"name":"Novo Nordisk A\/S S\u00f8borg Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D. L.","family":"Yee","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Hematology\u2010Oncology Section Baylor College of Medicine Texas Children's Hemophilia &amp; Thrombosis Center Houston TX USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,6,28]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02909.x"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH11-03-0193"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2516.1999.00347.x"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-7-24"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12315"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12000"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-02-335596"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-05-573055"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2016.02.030"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1355-0691.2004.00875.x"},{"issue":"2","key":"e_1_2_8_12_1","first-page":"115","article-title":"Validation of the haemophilia\u2010specific quality of life questionnaire for adult patients with haemophilia (Haem\u2010A\u2010QoL)","volume":"90","author":"Mackensen S","year":"2005","journal-title":"Haematologica"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/0168-8510(90)90421-9"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2006.01257.x"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1155\/2014\/968081"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12375"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1111%2Fhae.12995","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.12995","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,23]],"date-time":"2023-09-23T23:40:02Z","timestamp":1695512402000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.12995"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,6,28]]},"references-count":15,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2016,7]]}},"alternative-id":["10.1111\/hae.12995"],"URL":"https:\/\/doi.org\/10.1111\/hae.12995","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,6,28]]}}}