{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T04:54:58Z","timestamp":1790571298887,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,11,25]],"date-time":"2025-11-25T00:00:00Z","timestamp":1764028800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,11,25]],"date-time":"2025-11-25T00:00:00Z","timestamp":1764028800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100003246","name":"Nederlandse Organisatie voor Wetenschappelijk Onderzoek","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003246","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008322","name":"CSL Behring","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008322","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004326","name":"Bayer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004326","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012112","name":"Swedish Orphan Biovitrum","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012112","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Efanesoctocog alfa is a novel factor VIII (FVIII) concentrate with a unique molecular design that enables Von Willebrand Factor\u2010independent clearance in patients with haemophilia A. Limited sampling strategies (LSSs) are necessary to implement accurate pharmacokinetic (PK)\u2010guided dosing for efanesoctocog alfa in clinical practice.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>This in silico study aims to evaluate the predictive performance of 10 LSSs with one to three samples for estimating individual PK profiles of efanesoctocog alfa.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      Monte Carlo simulations based on a published population PK model generated individual FVIII activity\u2010time profiles for a virtual population. LSSs were applied to sample from these profiles, and PK parameters, FVIII activity peak level at 0.5\u00a0h (C\n                      <jats:sub>0.5<\/jats:sub>\n                      ), FVIII activity trough level at 168\u00a0h (C\n                      <jats:sub>168<\/jats:sub>\n                      ) and time above FVIII activity thresholds (5, 10 and 40\u00a0IU\/dL) were estimated using Bayesian forecasting.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      All LSSs complied with our requirements of a relative mean prediction error of &lt;\u00b15% and a relative root mean square error of &lt;25%. For prophylactic dosing advice, a LSS was considered clinically suitable if at least 80% of predicted C\n                      <jats:sub>168<\/jats:sub>\n                      had a prediction error within \u22123 to 3\u00a0IU\/dL. This criterion was met by one two\u2010sample LSS and three three\u2010sample LSSs. For pre\u2010operative dosing advice, suitability required at least 80% of predicted C\n                      <jats:sub>0.5<\/jats:sub>\n                      within \u201320 to 20\u00a0IU\/dL, fulfilled by one two\u2010sample and six three\u2010sample LSSs.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Several LSSs demonstrated adequate predictive performance for PK\u2010guided dosing of efanesoctocog alfa.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70169","type":"journal-article","created":{"date-parts":[[2025,11,25]],"date-time":"2025-11-25T14:25:56Z","timestamp":1764080756000},"page":"87-97","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["In Silico Assessment of Limited Blood Sampling Strategies for Individualised Pharmacokinetic\u2010guided Dosing of Efanesoctocog Alfa in Haemophilia A Patients"],"prefix":"10.1111","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-5883-0475","authenticated-orcid":false,"given":"Jelien","family":"den Hollander","sequence":"first","affiliation":[{"name":"Hospital Pharmacy\u2010Clinical Pharmacology, Academic Medical Centre Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1557-2995","authenticated-orcid":false,"given":"Marjon H.","family":"Cnossen","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children's Hospital University Medical Centre Rotterdam  Rotterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8401-0879","authenticated-orcid":false,"given":"Ron A. A.","family":"Math\u00f4t","sequence":"additional","affiliation":[{"name":"Hospital Pharmacy\u2010Clinical Pharmacology, Academic Medical Centre Amsterdam  Amsterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,11,25]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265\u2010023\u201001866\u20109"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2209226"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood\u20102023\u2010188739"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.100176"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14826"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325\u2010022\u201002220\u20103"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13540"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2023.101098"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/jcph.70008"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1177\/204062072096688"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10928\u2010024\u201009929\u20104"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1097\/MBC.0000000000001204"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00228\u2010021\u201003173\u20102"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.13771"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2015.136275"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12464"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.15185"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/psp4.12938"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15778"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70169","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/hae.70169","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70169","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T03:55:26Z","timestamp":1790567726000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.70169"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,25]]},"references-count":20,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["10.1111\/hae.70169"],"URL":"https:\/\/doi.org\/10.1111\/hae.70169","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,11,25]]},"assertion":[{"value":"2025-06-24","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-11-03","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-11-25","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}