{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T12:43:02Z","timestamp":1790772182737,"version":"4.1.0"},"reference-count":23,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,8,8]],"date-time":"2023-08-08T00:00:00Z","timestamp":1691452800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007723","name":"Takeda Pharmaceuticals U.S.A.","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007723","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>In recent years, there has been increased focus on individualizing treatment for persons with hemophilia including pharmacokinetic\u2010guided (PK) dosing.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aims<\/jats:title><jats:p>In this retrospective study clinical outcomes before and after PK\u2010guided prophylaxis were examined.<\/jats:p><\/jats:sec><jats:sec><jats:title>Materials and methods<\/jats:title><jats:p>Eight Haemophilia Treatment Centres from the United States participated in the study and included 132 patients classified into two cohorts: those undergoing a PK\u2010assessment for product switch (switchers) or to optimize treatment (non\u2010switchers). Subset analyses for the two most common products and patients with dosing per prescription label were included for annual bleeding rates (ABR), mean weekly consumption outcomes, and annualized cost of prophylaxis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The most common products before and after index date were octocog alfa, rurioctocog alfa pegol, and efmoroctocog alfa. Seventy\u2010four (56%) patients were identified as switchers and 58 (44%) patients were classified as non\u2010switchers. The majority of patients (78.0%) experienced either a decrease in ABR post\u2010index or maintained 0 ABR during pre\u2010 and post\u2010index time periods, with similar proportions identified in both switchers (77.0%) and non\u2010switchers (79.3%) populations. Non\u2010switchers were identified as having no significant change in cost of therapy, while switchers experienced increased cost of therapy driven by higher price of extended half\u2010life products. Within subset analyses, patients receiving rurioctocog alfa pegol and efmoroctocog alfa had mean ABR under 1 after index date.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>PK\u2010guided prophylaxis has the potential to improve clinical outcomes without increase in cost of therapy for patients maintaining product and can aid in maintaining effective protection against bleeds in those switching product.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14826","type":"journal-article","created":{"date-parts":[[2023,8,9]],"date-time":"2023-08-09T06:58:45Z","timestamp":1691564325000},"page":"1234-1242","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Effects of PK\u2010guided prophylaxis on clinical outcomes and FVIII consumption for patients with moderate to severe Haemophilia A"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6013-1254","authenticated-orcid":false,"given":"Guy","family":"Young","sequence":"first","affiliation":[{"name":"Children's Hospital Los Angeles University of Southern California Keck School of Medicine  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael U.","family":"Callaghan","sequence":"additional","affiliation":[{"name":"Children's Hospital of Michigan  Detroit Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vinod","family":"Balasa","sequence":"additional","affiliation":[{"name":"Hemotology\/Oncology Valley Children's Healthcare  Madera California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amit","family":"Soni","sequence":"additional","affiliation":[{"name":"Center for Inherited Blood Disorders  Orange California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanjay","family":"Ahuja","sequence":"additional","affiliation":[{"name":"UH Rainbow Babies and Children's Hospital  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9415-9543","authenticated-orcid":false,"given":"Jonathan C.","family":"Roberts","sequence":"additional","affiliation":[{"name":"Bleeding &amp; Clotting Disorders Institute  Peoria Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mindy L.","family":"Simpson","sequence":"additional","affiliation":[{"name":"Rush University Medical Center  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0323-2571","authenticated-orcid":false,"given":"Hande","family":"Kizilocak","sequence":"additional","affiliation":[{"name":"Children's Hospital Los Angeles University of Southern California Keck School of Medicine  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2700-9382","authenticated-orcid":false,"given":"Andrew","family":"Frick","sequence":"additional","affiliation":[{"name":"Trio Health Inc.  Louisville Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ali G.","family":"Mokdad","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals U.S.A, 95 Hayden Ave  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shan","family":"Xing","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals U.S.A, 95 Hayden Ave  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jorge","family":"Caicedo","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals U.S.A, 95 Hayden Ave  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,8,8]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1080\/14728214.2021.1988073"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.13298"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13438"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-07-360594"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12985"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020005673"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02838.x"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02191.x"},{"key":"e_1_2_10_10_1","unstructured":"LeeM MorfiniM SchulmanS IngerslevJ.The Factor VIII\/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis: the design and analysis of pharmacokinetic studies of coagulation factors.2001;Accessed May 2022 at:https:\/\/cdn.ymaws.com\/www.isth.org\/resource\/"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-0038-1646423"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13540"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF01061728"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12204"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1345\/aph.1D374"},{"key":"e_1_2_10_16_1","unstructured":"ASP Drug Pricing. 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