{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:32:37Z","timestamp":1790814757387,"version":"4.1.0"},"reference-count":27,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2020,12,16]],"date-time":"2020-12-16T00:00:00Z","timestamp":1608076800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2021,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Congenital fibrinogen deficiency (CFD) is a rare, inherited disorder affecting normal blood clotting function, where patients can experience severe and\/or frequent bleeding episodes (BEs). Treatment with human fibrinogen concentrate (HFC) can prevent\/arrest bleeding. There is a need for more data on the efficacy, pharmacokinetics (PK) and safety of HFC treatment in paediatric patients with CFD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Haemostatic efficacy of HFC (<jats:italic>Fibryga<jats:sup>\u00ae<\/jats:sup><\/jats:italic>, Octapharma AG) for on\u2010demand treatment of bleeding and surgical prophylaxis in patients &lt;12\u00a0years old was assessed by investigators and an Independent Data Monitoring and Endpoint Adjudication Committee (IDMEAC) based on an objective 4\u2010point efficacy scale. Maximum clot firmness (MCF; surrogate marker of haemostatic efficacy), single\u2010dose PK and safety were also assessed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Of 14 patients receiving HFC (median [range] age 6.0\u00a0years [1.0\u201310.0]), eight received HFC for 10 BEs, three for surgical prophylaxis and 13 for PK. The IDMEAC rated haemostatic efficacy as 100% successful for on\u2010demand BE treatment (95% CI 69.15\u2013100.00) and surgical prophylaxis (95% CI 29.24\u2013100.00). After a mean first dose of 70.78\u00a0mg\/kg for BEs, mean (\u00b1SD) MCF significantly increased from pre\u2010treatment to 1\u2010hour post\u2010infusion (3.3\u00a0mm [\u00b11.77]; <jats:italic>P<\/jats:italic>\u00a0=\u00a00.0002), coinciding with haemostatic efficacy. PK parameters were favourable. Two possibly related adverse events occurred, including one serious (portal vein thrombosis). No allergic\/hypersensitivity reactions or deaths were observed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>HFC treatment for on\u2010demand treatment of BEs and surgical prophylaxis was efficacious for this ultra\u2010rare paediatric population with congenital afibrinogenaemia and showed a favourable PK and safety profile.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14230","type":"journal-article","created":{"date-parts":[[2020,12,18]],"date-time":"2020-12-18T03:04:33Z","timestamp":1608260673000},"page":"283-292","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Efficacy and safety of fibrinogen concentrate for on\u2010demand treatment of bleeding and surgical prophylaxis in paediatric patients with congenital fibrinogen deficiency"],"prefix":"10.1111","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5584-5049","authenticated-orcid":false,"given":"Claudia","family":"Djambas Khayat","sequence":"first","affiliation":[{"name":"Hotel Dieu de France Hospital, Saint Joseph University Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sunil","family":"Lohade","sequence":"additional","affiliation":[{"name":"Sahyadri Specialty Hospital Pune India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fulton","family":"D\u2019Souza","sequence":"additional","affiliation":[{"name":"St. John\u2019s Medical College Hospital Bangalore India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Latha Gowda","family":"Shamanur","sequence":"additional","affiliation":[{"name":"S.S Institute of Medical Science and Research Center Davangere India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omid Reza","family":"Zekavat","sequence":"additional","affiliation":[{"name":"Hematology Research Center Nemazee Hospital, Shiraz University of Medical Sciences Shiraz Iran"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Kruzhkova","sequence":"additional","affiliation":[{"name":"Research &amp; Development Department Octapharma Lachen Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruce","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Clinical Research &amp; Development Octapharma Paramus NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Solomon","sequence":"additional","affiliation":[{"name":"Research &amp; Development Department Octapharma Lachen Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sigurd","family":"Knaub","sequence":"additional","affiliation":[{"name":"Research &amp; Development Department Octapharma Lachen Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flora","family":"Peyvandi","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,12,16]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2004-02-0595"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.14216"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2006.02014.x"},{"key":"e_1_2_9_5_1","doi-asserted-by":"crossref","unstructured":"LaffanM.Fibrinogen. 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