{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,14]],"date-time":"2025-05-14T01:11:44Z","timestamp":1747185104193,"version":"3.40.5"},"reference-count":28,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2024,2,5]],"date-time":"2024-02-05T00:00:00Z","timestamp":1707091200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Recombinant porcine factor VIII (rpFVIII; susoctocog alfa) is predicted to provide functional FVIII activity in patients with congenital haemophilia A with inhibitors (CHAWI).<\/jats:p><\/jats:sec><jats:sec><jats:title>Aims<\/jats:title><jats:p>To evaluate the efficacy and safety of rpFVIII in patients with CHAWI undergoing invasive procedures.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This phase 3, multicentre, single\u2010arm, open\u2010label study (NCT02895945) enrolled males aged 12\u201375 years with severe\/moderately severe CHAWI who required surgical\/invasive procedures. Patients received a loading dose of rpFVIII 1\u20132\u00a0h before surgery. The primary outcome was the proportion of all procedures with a \u2018good\u2019 or \u2018excellent\u2019 response (treatment success) on the global haemostatic efficacy assessment score.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Of the eight dosed patients, five completed the study. Six of seven surgeries (85.7%; 95% confidence interval, 42.1\u201399.6) achieved treatment success; five were rated \u2018excellent\u2019, one was rated \u2018good\u2019. Seven surgery\u2010related bleeding episodes occurred in three patients during the study, with none requiring additional surgical intervention. Overall, six of eight patients experienced 17 treatment\u2010emergent adverse events. Three patients developed de novo inhibitors to rpFVIII. Five patients reported anamnestic reactions, three to both human (h) FVIII (i.e., alloantibodies to exogenous FVIII detected with anti\u2010hFVIII assays) and rpFVIII, and two to hFVIII only. Four serious adverse events were considered related to rpFVIII (three anti\u2010rpFVIII antibody positive; one anamnestic reaction to hFVIII and rpFVIII).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Good haemostasis was achieved with rpFVIII during the immediate perioperative period. The study was terminated early because the study sponsor and health authorities determined that the risk of anamnestic reactions outweighs the benefits in this study population.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14932","type":"journal-article","created":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T07:01:15Z","timestamp":1707289275000},"page":"395-403","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Recombinant porcine factor VIII in patients with congenital haemophilia A with inhibitors undergoing surgery: Phase 3, single\u2010arm, open\u2010label study"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0485-7402","authenticated-orcid":false,"given":"Christian","family":"Pfrepper","sequence":"first","affiliation":[{"name":"Division of Hemostaseology Department of Hematology Cellular Therapy, Hemostaseology and Infectiology University Hospital Leipzig  Leipzig Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paolo","family":"Radossi","sequence":"additional","affiliation":[{"name":"Oncohematology Department Istituto Oncologico Veneto Castelfranco Veneto Hospital  Castelfranco Veneto Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7877-4784","authenticated-orcid":false,"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"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4910-2142","authenticated-orcid":false,"given":"Kaan","family":"Kavakli","sequence":"additional","affiliation":[{"name":"Ege University Faculty of Medicine  Izmir Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2762-6033","authenticated-orcid":false,"given":"Roger","family":"Schutgens","sequence":"additional","affiliation":[{"name":"Center for Benign Haematology, Thrombosis and Haemostasis Van Creveldkliniek University Medical Centre Utrecht University Utrecht  Utrecht Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1599-774X","authenticated-orcid":false,"given":"Nazan","family":"Sarper","sequence":"additional","affiliation":[{"name":"Kocaeli University Medical Faculty  Kocaeli Turkey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joan","family":"Gu","sequence":"additional","affiliation":[{"name":"Takeda Development Center Americas, Inc.  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