{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T12:39:50Z","timestamp":1788871190059,"version":"build-2803163510"},"reference-count":27,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2014,11,7]],"date-time":"2014-11-07T00:00:00Z","timestamp":1415318400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/3.0\/"}],"funder":[{"name":"CSL Behring (King of Prussia, PA, USA)"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2015,1]]},"abstract":"Summary<\/jats:title>\n \n Congenital factor\n XIII<\/jats:styled-content>\n (\n FXIII<\/jats:styled-content>\n ) deficiency is an extremely rare, potentially life\u2010threatening bleeding disorder. Routine prophylactic management is recommended for individuals with clinically relevant\n FXIII<\/jats:styled-content>\n deficiency. This prospective, multicentre, open\u2010label study evaluated the long\u2010term efficacy and safety of prophylactic infusions of\n FXIII<\/jats:styled-content>\n concentrate (human) 40\u00a0IU\u00a0kg\n \u22121<\/jats:sup>\n in patients with congenital\n FXIII<\/jats:styled-content>\n deficiency.\n FXIII<\/jats:styled-content>\n concentrate (human) was administered every 4\u00a0weeks for 12\u00a0months. Dosing was adjusted to maintain trough\n FXIII<\/jats:styled-content>\n activity levels of 5\u201320%. Logistical and ethical constraints precluded use of a placebo control group. Annualized incidence of spontaneous bleeding was compared with historical rates; safety was assessed as a secondary objective. Forty\u2010one patients were enrolled and completed the study. The annualized rate for spontaneous bleeding episodes requiring\n FXIII<\/jats:styled-content>\n treatment was 0.000 episodes per patient\u2010year (95%\n CI<\/jats:styled-content>\n : 0.000; 0.097). The study met its primary endpoint: the upper limit of the 95%\n CI<\/jats:styled-content>\n was substantially below the historical rate of 2.5 bleeding episodes per patient\u2010year. Five spontaneous bleeding episodes (involving three patients; none requiring\n FXIII<\/jats:styled-content>\n treatment) and eight trauma\u2010related bleeding episodes (two requiring\n FXIII<\/jats:styled-content>\n treatment) occurred. Five patients had surgery during the study, only one of whom required\n FXIII<\/jats:styled-content>\n treatment for post\u2010surgical bleeding. Most patients (\u226585%) had trough\n FXIII<\/jats:styled-content>\n activity levels \u226510%. No patient discontinued treatment due to an adverse event. No adverse events related to thromboembolism or viral transmission were reported. Prophylactic treatment with\n FXIII<\/jats:styled-content>\n concentrate (human) was well tolerated and prevented spontaneous bleeding episodes that were serious enough to require treatment with\n FXIII<\/jats:styled-content>\n \u2010containing product. Clinical trial registration:\n www.clinicaltrials.gov\/ct2\/show\/NCT00885742<\/jats:ext-link>\n .\n <\/jats:p>","DOI":"10.1111\/hae.12524","type":"journal-article","created":{"date-parts":[[2014,11,7]],"date-time":"2014-11-07T04:50:47Z","timestamp":1415335847000},"page":"102-108","source":"Crossref","is-referenced-by-count":20,"title":["Efficacy and safety of prophylactic treatment with plasma\u2010derived factor\n XIII<\/scp>\n concentrate (human) in patients with congenital factor\n XIII<\/scp>\n deficiency"],"prefix":"10.1111","volume":"21","author":[{"given":"C.","family":"Ashley","sequence":"first","affiliation":[{"name":"Alabama Clinical Therapeutics LLC Birmingham AL USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E.","family":"Chang","sequence":"additional","affiliation":[{"name":"St. Luke's Mountain State Tumor Institute Boise ID USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J.","family":"Davis","sequence":"additional","affiliation":[{"name":"University of Miami\/Jackson Memorial Hospital Miami FL USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.","family":"Mangione","sequence":"additional","affiliation":[{"name":"CSL Behring King of Prussia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V.","family":"Frame","sequence":"additional","affiliation":[{"name":"CSL Behring King of Prussia PA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D. J.","family":"Nugent","sequence":"additional","affiliation":[{"name":"Children's Hospital of Orange County (CHOC) Orange CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2014,11,7]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1537-2995.2012.03865.x"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/0268-960X(93)90010-2"},{"key":"e_1_2_9_4_1","first-page":"245","article-title":"Human factor XIII: fibrin\u2010stabilizing factor","volume":"5","author":"Lorand L","year":"1980","journal-title":"Prog Hemost Thromb"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/MOH.0b013e3283497e3e"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2141.1999.01648.x"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.109.2.e32"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2008.01857.x"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02123.x"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2006.02.009"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH07-01-0034"},{"key":"e_1_2_9_12_1","volume-title":"MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Other Bleeding Disorders","author":"National Hemophilia Foundation"},{"key":"e_1_2_9_13_1","volume-title":"FDA News Release. Transfusion\u2010related acute lung injury (TRALI)","author":"Food and Drug Administration (FDA)","year":"2001"},{"key":"e_1_2_9_14_1","unstructured":"National Hemophilia Foundation.Factor XIII deficiency.http:\/\/www.hemophilia.org\/Bleeding-Disorders\/Types-of-Bleeding-Disorders\/Other-Factor-Deficiencies\/Factor-XIII. Accessed September 2014."},{"key":"e_1_2_9_15_1","unstructured":"CSL Behring.Corifact\u00ae. Highlights of Prescribing Information.http:\/\/www.corifact.com\/docs\/Corifact-Prescribing-Information.pdf. Accessed October 2014."},{"key":"e_1_2_9_16_1","unstructured":"Food and Drug Administration (FDA).FDA approves product to prevent bleeding in people with rare genetic defect.http:\/\/www.fda.gov\/NewsEvents\/Newsroom\/PressAnnouncements\/ucm243856.htm. Accessed October 2014."},{"key":"e_1_2_9_17_1","doi-asserted-by":"crossref","unstructured":"NugentDJ AshleyC Garc\u00eda\u2010TalaveraJ LoLC MehdiAS MangioneA.Pharmacokinetics and safety of plasma\u2010derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. DOI:10.1111\/hae.12505.","DOI":"10.1111\/hae.12505"},{"key":"e_1_2_9_18_1","unstructured":"PeyvandiF PallaR MenegattiM.Rare bleeding disorders (RBDs) \u2013 a European response.http:\/\/www.ehc.eu\/fileadmin\/about_bleeding_disorders\/ENGLISH.pdf. 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