{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T07:04:56Z","timestamp":1777878296002,"version":"3.51.4"},"reference-count":20,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Baxalta Innovations GmbH, a Takeda company"},{"name":"Baxalta US Inc, a Takeda company"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Hematology"],"published-print":{"date-parts":[[2023,1]]},"abstract":"\n Background:<\/jats:title>\n The phase 3, prospective PROPEL study demonstrated that pharmacokinetic (PK)-guided prophylaxis targeting elevated factor VIII (FVIII) troughs in patients with hemophilia A resulted in lower annualized bleeding rates (ABRs) and a higher proportion of patients experiencing zero bleeds in the second 6 months of treatment when targeting a FVIII trough of 8\u201312% versus 1\u20133%.<\/jats:p>\n <\/jats:sec>\n \n Objective:<\/jats:title>\n To investigate the benefit of PK-guided prophylaxis with rurioctocog alfa pegol targeting two FVIII trough levels in specific patient subgroups in a post hoc analysis using data from PROPEL.<\/jats:p>\n <\/jats:sec>\n \n Design:<\/jats:title>\n This is a post hoc analysis of data from the PROPEL study. The design and primary outcomes of the prospective, randomized PROPEL study (NCT02585960) have been reported previously.<\/jats:p>\n <\/jats:sec>\n \n Methods:<\/jats:title>\n \n This post hoc analysis reports data stratified by FVIII half-life ( t\n 1\/2<\/jats:sub>\n ), hemophilic arthropathy status, number of target joints at screening, previous treatment regimen, and ABR range in the 12 months before study entry.\n <\/jats:p>\n <\/jats:sec>\n \n Results:<\/jats:title>\n \n Targeting an elevated FVIII trough of 8\u201312% was associated with higher average FVIII levels over time, regardless of FVIII t\n 1\/2<\/jats:sub>\n at baseline. The decrease in total ABR between the 8\u201312% and 1\u20133% arms was greatest in patients with a FVIII t\n 1\/2<\/jats:sub>\n of 6 to <12 h (0.7 versus 3.5); a higher number of target joints, that is, at least four target joints, at baseline (0.2 versus 1.6); the presence of arthropathy (0.1 versus 1.7); and those previously treated on-demand (0.3 versus 1.8).\n <\/jats:p>\n <\/jats:sec>\n \n Conclusion:<\/jats:title>\n \n These results support the feasibility of targeting elevated FVIII levels using personalized rurioctocog alfa pegol prophylaxis. These benefits may be especially important in patients with a short FVIII t\n 1\/2<\/jats:sub>\n and those receiving standard prophylaxis with frequent breakthrough bleeds, arthropathy, and target joints.\n <\/jats:p>\n <\/jats:sec>\n \n Registration:<\/jats:title>\n ClinicalTrials.gov Identifier: NCT02585960; https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02585960<\/jats:p>\n <\/jats:sec>","DOI":"10.1177\/20406207231178596","type":"journal-article","created":{"date-parts":[[2023,7,15]],"date-time":"2023-07-15T08:06:06Z","timestamp":1689408366000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":1,"title":["Targeting an elevated FVIII level using personalized rurioctocog alfa pegol prophylaxis in specific patient populations with hemophilia A:\n post hoc<\/i>\n subanalysis of the randomized, phase 3 PROPEL study"],"prefix":"10.1177","volume":"14","author":[{"given":"Carmen","family":"Escuriola-Ettingshausen","sequence":"first","affiliation":[{"name":"HZRM H\u00e4mophilie-Zentrum Rhein Main, M\u00f6rfelden-Walldorf, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Robert","family":"Klamroth","sequence":"additional","affiliation":[{"name":"Vivantes Klinikum im Friedrichshain, Berlin, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel","family":"Escobar","sequence":"additional","affiliation":[{"name":"The University of Texas Health Science Center at Houston, Houston, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Oleksandra","family":"Stasyshyn","sequence":"additional","affiliation":[{"name":"National Academy of Medical Sciences of Ukraine, Lviv, Ukraine"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Srilatha","family":"Tangada","sequence":"additional","affiliation":[{"name":"Takeda Development Center Americas, Inc., 650 East Kendall Street, Cambridge, MA 02142, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Werner","family":"Engl","sequence":"additional","affiliation":[{"name":"Baxalta Innovations GmbH, a Takeda Company, Vienna, Austria"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ivan","family":"Honauer","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG, Zurich, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hye-Youn","family":"Lee","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG, Zurich, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pratima","family":"Chowdary","sequence":"additional","affiliation":[{"name":"Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jerzy","family":"Windyga","sequence":"additional","affiliation":[{"name":"Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2023,7,15]]},"reference":[{"key":"bibr1-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"bibr2-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200106073442307"},{"key":"bibr3-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2006.01268.x"},{"key":"bibr4-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)61139-2"},{"key":"bibr5-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1177\/2040620718796429"},{"key":"bibr6-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1007\/s00228-009-0676-x"},{"key":"bibr7-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12270"},{"key":"bibr8-20406207231178596","unstructured":"Baxalta US Inc. ADYNOVATE [antihemophilic factor (recombinant), PEGylated] prescribing information, https:\/\/www.shirecontent.com\/PI\/PDFs\/ADYNOVATE_USA_ENG.pdf (2021, accessed 5 July 2022)."},{"key":"bibr9-20406207231178596","unstructured":"European Medicines Agency. ADYNOVI: rurioctocog alfa pegol. Adynovi: EPAR \u2013 product Information. Summary of product characteristics, https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/adynovi-epar-product-information_en.pdf (2022, accessed 5 July 2022)."},{"key":"bibr10-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2015-03-630897"},{"key":"bibr11-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13119"},{"key":"bibr12-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14052"},{"key":"bibr13-20406207231178596","first-page":"141","volume":"36","author":"Ar MC","year":"2019","journal-title":"Turk J Hematol"},{"key":"bibr14-20406207231178596","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.242735"},{"key":"bibr15-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020005673"},{"key":"bibr16-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"bibr17-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13807"},{"key":"bibr18-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1055\/s-0040-1709519"},{"key":"bibr19-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2020009603"},{"key":"bibr20-20406207231178596","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13361"}],"container-title":["Therapeutic Advances in Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406207231178596","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/20406207231178596","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/20406207231178596","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T23:16:41Z","timestamp":1777591001000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/20406207231178596"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1]]},"references-count":20,"alternative-id":["10.1177\/20406207231178596"],"URL":"https:\/\/doi.org\/10.1177\/20406207231178596","relation":{},"ISSN":["2040-6207","2040-6215"],"issn-type":[{"value":"2040-6207","type":"print"},{"value":"2040-6215","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,1]]},"article-number":"20406207231178596"}}