{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T03:57:08Z","timestamp":1791863828525,"version":"4.3.4"},"reference-count":49,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,10,30]],"date-time":"2023-10-30T00:00:00Z","timestamp":1698624000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Hereditary factor X (FX) deficiency (HFXD) is an autosomal recessive rare bleeding disorder that leads to defects in the FX protein. Depending on the degree of deficiency, patients may be at risk of life\u2010threatening bleeding episodes. Historical treatments for FX deficiency include prothrombin complex concentrates, which can increase the risk of thrombosis, and fresh frozen plasma, which can cause volume overload and transfusion reactions. Plasma\u2010derived FX (pdFX), a single\u2010factor, high\u2010purity, high\u2010potency human FX treatment, was approved in 2015 in the United States and in 2016 in Europe for on\u2010demand treatment and prophylaxis of bleeding episodes and perioperative management of patients with HFXD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Five studies that examined the use of pdFX in patients with mild (plasma FX activity [FX:C] \u22655\u00a0IU\/dL), moderate (FX:C \u22651 and\u00a0&lt;5\u00a0IU\/dL), or severe (FX:C\u00a0&lt;\u00a01\u00a0IU\/dL) HFXD were reviewed: TEN01, TEN02 and TEN03 were prospective, open\u2010label, multicentre, nonrandomised studies, and TEN05 and TEN06 were multicentre retrospective studies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>When used as an on\u2010demand treatment, pdFX was judged by investigators to be successful in treating 41\/42 (97.6%), 2\/3 (66.6%) and 79\/79 (100%) bleeds in TEN01, TEN02 and TEN05, respectively. When used prophylactically, pdFX was judged \u2018excellent\u2019 for the prevention of bleeds in nine (100%) and eight (100%) patients in TEN02 and TEN05, respectively. Perioperative treatment and pharmacokinetics were also assessed. pdFX was safe and well tolerated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Together, these studies support the use of pdFX for on\u2010demand treatment of bleeding, routine prophylaxis, and perioperative management of bleeding in patients with HFXD.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14894","type":"journal-article","created":{"date-parts":[[2023,10,30]],"date-time":"2023-10-30T13:06:41Z","timestamp":1698671201000},"page":"59-67","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Plasma\u2010derived human factor X concentrate for the treatment of patients with hereditary factor X deficiency"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2944-0240","authenticated-orcid":false,"given":"Miguel A.","family":"Escobar","sequence":"first","affiliation":[{"name":"University of Texas Health Science Center at Houston\u2010McGovern Medical School and the Gulf States Hemophilia and Thrombophilia Center  Houston Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1174-1958","authenticated-orcid":false,"given":"Kaan","family":"Kavakli","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Division of Hemato\u2010Oncology Ege University Faculty of Medicine  \u0130zmir Turkey"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,10,30]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13058"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-08-532820"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2141.1998.00806.x"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1080\/17425255.2016.1237504"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V70.1.165.165"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2012.04653.x"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2008.01856.x"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1097\/MBC.0000000000000563"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1097\/MBC.0000000000000787"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1097\/MBC.0000000000000435"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2004-02-0595"},{"key":"e_1_2_9_13_1","volume-title":"The Human Gene Mutation Database at the Institute of Medical Genetics in Cardiff","author":"Cooper DN","year":"2017"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-017-1779-6"},{"key":"e_1_2_9_15_1","volume-title":"Williams Hematology","author":"Zivelin A","year":"2010"},{"key":"e_1_2_9_16_1","unstructured":"National Center for Biotechnology Information.NCBI Gene. 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