{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T06:16:59Z","timestamp":1790835419143,"version":"4.1.0"},"reference-count":44,"publisher":"Georg Thieme Verlag KG","issue":"05","license":[{"start":{"date-parts":[[2023,2,9]],"date-time":"2023-02-09T00:00:00Z","timestamp":1675900800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"CSL Behring"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2023,5]]},"abstract":"<jats:p>Background\u2003rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A.<\/jats:p><jats:p>Objectives\u2003To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis.<\/jats:p><jats:p>Methods\u2003This interim analysis includes data, collected between January 2018 \u2014 September 2021, from patients treated with rVIII-SingleChain prophylaxis at French Hemophilia Treatment centers. Data on annualized bleeding rates, dosing frequency, and consumption before and after switching to rVIII-SingleChain were recorded. A population PK analysis was also conducted to estimate PK parameters.<\/jats:p><jats:p>Results\u2003Overall, 43 patients switched to prophylaxis with rVIII-SingleChain either from a previous prophylaxis regimen or from on-demand treatment. Following the switch to rVIII-SingleChain, patients maintained excellent bleed control. After switching to rVIII-SingleChain, most patients maintained or reduced their regimen. Interestingly, a majority of patients treated &gt;2\u2009\u00d7\/weekly with a standard half-life FVIII reduced both injection frequency and FVIII consumption with rVIII-SingleChain. A PK analysis revealed a lower clearance of rVIII-SingleChain (1.9 vs. 2.1 dL\/h) and a longer half-life both in adolescents\/adults (n\u2009=\u200928) and pediatric (n\u2009=\u20096) patients (15.5 and 11.9\u2009hours, respectively vs. 14.5 and 10.3\u2009hours) than previously reported.<\/jats:p><jats:p>Conclusions\u2003Patients who switched to rVIII-SingleChain prophylaxis demonstrated excellent bleed control and a reduction in infusion frequency. A population PK analysis revealed improved PK parameters compared with those reported in the clinical trial.<\/jats:p>","DOI":"10.1055\/s-0043-1761449","type":"journal-article","created":{"date-parts":[[2023,2,10]],"date-time":"2023-02-10T00:04:51Z","timestamp":1675987491000},"page":"490-500","source":"Crossref","is-referenced-by-count":5,"title":["A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis"],"prefix":"10.1055","volume":"123","author":[{"given":"Benoit","family":"Guillet","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, University Hospital, Rennes, France"},{"name":"Univ Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en sant\u00e9, environnement et travail) - UMR_S 1085, Rennes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abel","family":"Hassoun","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, Simone Veil Hospital, GH Eaubonne-Montmorency, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e9n\u00e9dicte","family":"Wibaut","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Centre, National Reference Willebrand Centre, University Hospital, Lille, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annie","family":"Harroche","sequence":"additional","affiliation":[{"name":"Department of Hematology, Haemophilia Treatment Centre, University Hospital Necker Enfants Malades, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Biron-Andr\u00e9ani","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Centre, University Hospital, Montpellier, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yohan","family":"Repesse","sequence":"additional","affiliation":[{"name":"Haematology Laboratory and Haemophilia Reference Centre, Centre Hospitalier Universitaire de Caen, Caen, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roseline","family":"d'Oiron","sequence":"additional","affiliation":[{"name":"CRH, CRC-MHC (Centre de R\u00e9f\u00e9rence de l'H\u00e9mophilie, Centre de Ressource et de Comp\u00e9tence des Maladies H\u00e9morragiques Constitutionnelles), H\u00f4pital Bic\u00eatre, AP-HP, Universit\u00e9 Paris-Saclay, Paris, France"},{"name":"HITh, UMR_S1176, INSERM, Universit\u00e9 Paris-Saclay, Le Kremlin Bic\u00eatre, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brigitte","family":"Tardy","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, University Hospital, Saint-Etienne, France"},{"name":"Inserm CIC 1408, Saint-Etienne University Hospital Center, Saint-Etienne, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brigitte","family":"Pan Petesch","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, Morvan University Hospital, Saint-Etienne Brest, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Chamouni","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, University Hospital, Rouen, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Val\u00e9rie","family":"Gay","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, Hospital, Chambery, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Fouassier","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, H\u00f4tel-Dieu University Hospital, Nantes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire","family":"Pouplard","sequence":"additional","affiliation":[{"name":"Haemophilia Treatment Center, Hospital, Tours, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C\u00e9dric","family":"Martin","sequence":"additional","affiliation":[{"name":"CSL Behring, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hasan","family":"Catovic","sequence":"additional","affiliation":[{"name":"CSL Behring, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7134-0713","authenticated-orcid":false,"given":"Xavier","family":"Delavenne","sequence":"additional","affiliation":[{"name":"INSERM, UMR 1059, Dysfonction Vasculaire et de l'H\u00e9mostase, Universit\u00e9 de Lyon, Saint Etienne, France"},{"name":"Laboratoire de Pharmacologie \u2013 Toxicologie, CHU de Saint-Etienne, Saint-Etienne, 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