{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T22:50:04Z","timestamp":1780527004517,"version":"3.54.1"},"reference-count":29,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,12,17]],"date-time":"2025-12-17T00:00:00Z","timestamp":1765929600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,12,17]],"date-time":"2025-12-17T00:00:00Z","timestamp":1765929600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Efanesoctocog alfa is a novel, \u201cultra\u2010extended half\u2010life\u201d FVIII concentrate for bleed treatment and prevention in haemophilia A. Clinical trials excluded individuals with active or prior FVIII inhibitors, those on emicizumab, and previously untreated patients (PUPs).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>This retrospective, single\u2010centre study evaluated the management and outcomes of pediatric clinic patients who initiated efanesoctocog alfa for haemophilia A bleed treatment or prevention.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Clinical characteristics, pharmacokinetic data, hemostatic outcomes, and non\u2010standardised patient and caregiver feedback were collected by medical record review.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Overall, 24 patients initiated efanesoctocog alfa, including seven who switched from emicizumab, five who previously achieved immune tolerance induction for FVIII inhibitors, and one PUP, who was successfully treated with a single infusion for his initial joint bleed. Mean FVIII activity on day 6 was 13.4\u00a0\u00b1\u00a06.1\u00a0IU\/dL by one\u2010stage assay for patients who switched from FVIII concentrate prophylaxis. Five switched from emicizumab without a washout, preventing the use of a one\u2010stage assay, and had a mean chromogenic FVIII activity of 23.8\u00a0\u00b1\u00a05.2\u00a0IU\/dL on day 6. Compared with prior prophylaxis, efanesoctocog alfa was associated with a lower mean treated annualised bleed rate (1.3\u00a0\u00b1\u00a01.5\u00a0vs 0.1\u00a0\u00b1\u00a00.3,\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.001) and annualised emergency department bleed evaluations (0.8\u00a0\u00b1\u00a01.4\u00a0vs 0.1\u00a0\u00b1\u00a00.4,\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.04). No recurrence of FVIII inhibitors occurred over a median follow\u2010up of 22.6 months (range: 6.4\u201325.4). Patients\/caregivers reported improvements in musculoskeletal discomfort, perception of hemostatic coverage, and confidence to pursue physical activity.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Individuals with clinically severe haemophilia A, including subgroups previously excluded from clinical trials, successfully transitioned to efanesoctocog alfa with excellent hemostatic outcomes and no inhibitor recurrence.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.70175","type":"journal-article","created":{"date-parts":[[2025,12,17]],"date-time":"2025-12-17T08:55:35Z","timestamp":1765961735000},"page":"402-409","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Integration of Efanesoctocog Alfa in Clinical Practice for Children, Adolescents, and Young Adults With Severe Haemophilia A"],"prefix":"10.1111","volume":"32","author":[{"given":"Debbie","family":"Jiang","sequence":"first","affiliation":[{"name":"Hematology Division Massachusetts General Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie","family":"Johnston","sequence":"additional","affiliation":[{"name":"Boston Children's Hospital, Boston Bleeding Disorders Center  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Echelman","sequence":"additional","affiliation":[{"name":"Dana Farber and Boston Children's Blood and Cancer Center  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stacy E.","family":"Croteau","sequence":"additional","affiliation":[{"name":"Boston Children's Hospital, Boston Bleeding Disorders Center  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,12,17]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmoa067659"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538\u20107836.2011.04214.x"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019001311"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2011.02682.x"},{"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\u20102516.2010.02383.x"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/AJH.27602"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.27039"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1080\/17474086.2023.2198207"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.102238"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2023012285"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2018020552"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325\u2010024\u201003031\u20104"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.3390\/pharmacy13010016"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1182\/BLOOD.2019001292"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmoa2209226"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMOA2312611"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/HAE.14831"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12672"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2024.12.034"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.2196\/RESPROT.6558"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2007.01497.x"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1182\/BLOOD\u20102011\u201009\u2010379453"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/JTH.14153"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1111\/HAE.13882"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1111\/HAE.70017"},{"key":"e_1_2_10_29_1","doi-asserted-by":"publisher","DOI":"10.1182\/BLOOD\u20102023\u2010181973"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1111\/CTS.12794"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70175","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/hae.70175","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.70175","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T13:35:20Z","timestamp":1773408920000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.70175"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,12,17]]},"references-count":29,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2026,3]]}},"alternative-id":["10.1111\/hae.70175"],"URL":"https:\/\/doi.org\/10.1111\/hae.70175","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,12,17]]},"assertion":[{"value":"2025-09-11","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication 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