{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,4,24]],"date-time":"2025-04-24T04:23:42Z","timestamp":1745468622585,"version":"3.40.4"},"reference-count":28,"publisher":"S. Karger AG","license":[{"start":{"date-parts":[[2024,4,23]],"date-time":"2024-04-23T00:00:00Z","timestamp":1713830400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2024,4,23]],"date-time":"2024-04-23T00:00:00Z","timestamp":1713830400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Acta Haematol"],"abstract":"<jats:p>&lt;b&gt;&lt;i&gt;Introduction:&lt;\/i&gt;&lt;\/b&gt; The phase 2\/3 PROTECT VIII study demonstrated long-term efficacy and safety of damoctocog alfa pegol (BAY 94-9027; Jivi&lt;sup&gt;\u00ae&lt;\/sup&gt;), a B-domain-deleted recombinant factor VIII (FVIII), site-specifically PEGylated to improve its pharmacokinetic profile. We report a post hoc assessment of bleeding and safety outcomes in the subgroup of patients, aged 12\u2013&amp;lt;18 years at enrolment. &lt;b&gt;&lt;i&gt;Method:&lt;\/i&gt;&lt;\/b&gt; PROTECT VIII was a multicentre, open-label study of previously treated males aged 12\u201365 years with severe haemophilia A (FVIII &amp;lt;1%). Twelve patients were included in this analysis. All received damoctocog alfa pegol prophylaxis for the total time in study (median [range] time in study 4.0 [1.3\u20136.2] years). &lt;b&gt;&lt;i&gt;Results:&lt;\/i&gt;&lt;\/b&gt; Overall median (Q1; Q3) total and joint annualised bleeding rates were 1.8 (0.4; 5.1) and 0.7 (0.2; 1.8), respectively, for the entire study. During the last 6 months of treatment, eight (66.7%) and ten (83.3%) out of 12 patients experienced zero total and joint bleeds, respectively. No patient developed FVIII inhibitors. No deaths or thrombotic events were reported. &lt;b&gt;&lt;i&gt;Conclusion:&lt;\/i&gt;&lt;\/b&gt; Efficacy and safety of damoctocog alfa pegol were confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood. <\/jats:p>","DOI":"10.1159\/000538702","type":"journal-article","created":{"date-parts":[[2024,4,23]],"date-time":"2024-04-23T21:00:36Z","timestamp":1713906036000},"page":"1-10","source":"Crossref","is-referenced-by-count":0,"title":["Long-Term Efficacy and Safety of Damoctocog Alfa Pegol Prophylaxis in Patients with Haemophilia A Aged 12\u2013&amp;lt;18 Years at Enrolment into PROTECT VIII"],"prefix":"10.1159","author":[{"given":"Mark T.","family":"Reding","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mindy","family":"Simpson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan","family":"Ducore","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P\u00e5l Andr\u00e8","family":"Holme","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monika","family":"Maas Enriquez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Elisa","family":"Mancuso","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"127","published-online":{"date-parts":[[2024,4,23]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","DOI":"10.1038\/nrd.2018.70"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1586\/17474086.2015.1002465"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02813.x"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12618"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12951"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S139851"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.2450\/2019.0040-19"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2008.01843.x"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.12582"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.25807"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12506"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1111\/jth.13597"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14297"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-019-03747-2"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-020-04280-3"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13853"},{"key":"ref17","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2019.08.023"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14319"},{"key":"ref20","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13875"},{"key":"ref21","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejps.2019.01.015"},{"key":"ref22","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12159"},{"key":"ref23","doi-asserted-by":"publisher","DOI":"10.1160\/TH14-05-0407"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14270"},{"key":"ref25","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2020-044997"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13712"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.1111\/jth.14959"},{"key":"ref28","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13953"}],"container-title":["Acta Haematologica"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/karger.com\/aha\/article-pdf\/doi\/10.1159\/000538702\/4231229\/000538702.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/karger.com\/aha\/article-pdf\/doi\/10.1159\/000538702\/4231229\/000538702.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,23]],"date-time":"2025-04-23T15:15:00Z","timestamp":1745421300000},"score":1,"resource":{"primary":{"URL":"https:\/\/karger.com\/article\/doi\/10.1159\/000538702"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,23]]},"references-count":28,"URL":"https:\/\/doi.org\/10.1159\/000538702","archive":["Portico"],"relation":{"is-supplemented-by":[{"id-type":"doi","id":"10.6084\/m9.figshare.25574097","asserted-by":"subject"}]},"ISSN":["0001-5792","1421-9662"],"issn-type":[{"type":"print","value":"0001-5792"},{"type":"electronic","value":"1421-9662"}],"subject":[],"published":{"date-parts":[[2024,4,23]]}}}