{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T01:11:00Z","timestamp":1747185060252,"version":"3.40.5"},"reference-count":22,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,5,2]],"date-time":"2022-05-02T00:00:00Z","timestamp":1651449600000},"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":[[2022,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Hereditary factor X (FX) deficiency (FXD) is a rare autosomal recessive bleeding disorder. Plasma\u2010derived FX (pdFX) is a high\u2010purity FX concentrate approved in the United States and Europe for the treatment and prophylaxis of bleeding episodes and for peri\u2010operative management in patients with hereditary FXD (HFXD).<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To review pharmacokinetic dosing, efficacy, and safety data for pdFX as routine prophylaxis for HFXD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Summary of the published pharmacokinetic and safety data from TEN01, TEN02, TEN05, and real\u2010world publications of pdFX for prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Pharmacokinetic modelling data from the phase 3 TEN01 study supported administration of pdFX 25\u00a0IU\/kg twice weekly for routine prophylaxis in adolescents\/adults (aged \u226512\u00a0years). Results from nine paediatric patients in the phase 3 TEN02 study and eight adolescents\/adults (aged \u226512\u00a0years) in the retrospective data\u2010collection TEN05 study, along with real\u2010world evidence, showed that routine prophylaxis with pdFX \u224840\u00a0IU\/kg twice weekly in patients aged\u00a0&lt;12\u00a0years and pdFX \u224825\u00a0IU\/kg twice weekly in patients aged \u226512\u00a0years was effective in bleeding prevention.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>pdFX was well tolerated in clinical studies, with no new safety signals identified during routine prophylactic use. Based on current evidence, it is recommended that routine prophylaxis with pdFX be initiated at 25\u00a0IU\/kg twice weekly in adults\/adolescents \u226512\u00a0years of age, and at a dosage of 40\u00a0IU\/kg twice weekly in children\u00a0&lt;12\u00a0years of age. Thereafter, FX levels should be closely monitored, and dosages should be adjusted according to clinical response and to maintain trough levels \u22655\u00a0IU\/dl.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14570","type":"journal-article","created":{"date-parts":[[2022,5,2]],"date-time":"2022-05-02T13:43:50Z","timestamp":1651499030000},"page":"523-531","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["A review of the pharmacokinetics, efficacy and safety of high\u2010purity factor X for the prophylactic treatment of hereditary factor X deficiency"],"prefix":"10.1111","volume":"28","author":[{"given":"Jeanette","family":"Payne","sequence":"first","affiliation":[{"name":"Department of Paediatric Haematology Sheffield Children's NHS Foundation Trust  Sheffield UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0122-9320","authenticated-orcid":false,"given":"Glaivy","family":"Batsuli","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Division of Pediatric Hematology and Oncology Emory University Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta  Atlanta Georgia USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andrew D.","family":"Leavitt","sequence":"additional","affiliation":[{"name":"Departments of Laboratory Medicine &amp; Medicine (Hematology) University of California San Francisco  San Francisco California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mary","family":"Mathias","sequence":"additional","affiliation":[{"name":"Great Ormond Street Hospital for Children NHS Foundation Trust Haemophilia Comprehensive Care Centre  London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3421-0925","authenticated-orcid":false,"given":"Catherine E.","family":"McGuinn","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Division of Pediatric Hematology Oncology Weill Cornell Medicine  New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2022,5,2]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13058"},{"key":"e_1_2_9_3_1","unstructured":"National Haemophilia Foundation.Factor X deficiency.2021.https:\/\/www.hemophilia.org\/bleeding\u2010disorders\u2010a\u2010z\/types\/other\u2010factor\u2010deficiencies\/factor\u2010xAccessed."},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2012.04844.x"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2004.00944.x"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1080\/17425255.2016.1237504"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.thromres.2019.07.014"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-06-820738"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12440"},{"key":"e_1_2_9_10_1","unstructured":"Bio Products Laboratory Ltd.COAGADEX\u00ae (human coagulation Factor X).2018.https:\/\/www.fda.gov\/media\/94797\/downloadAccessed."},{"key":"e_1_2_9_11_1","unstructured":"European Medicines Agency.Coagadex (human coagulation factor X): summary of product characteristics.2021.https:\/\/www.ema.europa.eu\/en\/documents\/product\u2010information\/coagadex\u2010epar\u2010product\u2010information_en.pdfAccessed 12 April 2022."},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12894"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13500"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12550"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.14985"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/trf.15308"},{"key":"e_1_2_9_17_1","first-page":"389","article-title":"Treatment of a subdural hematoma and long\u2010term secondary prophylaxis in a patient with severe factor X (FX) deficiency treated with a high\u2010purity plasma\u2010derived factor X (pdFX) concentrate","volume":"1","author":"Kavakli K","year":"2017","journal-title":"Res Pract Thromb 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