A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A
Background Despite recent advances, factor replacement therapy remains a cornerstone in hemophilia A treatment. Efmoroctocog alfa, a recombinant FVIII Fc fusion protein (rFVIIIFc), has an extended half-life allowing higher FVIII levels and less frequent dosing than standard half-life (SHL) products,...
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| Format: | Article |
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Taylor & Francis Group
2025-12-01
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| Series: | Hematology |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/16078454.2025.2513186 |
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| author | Johannes Oldenburg Charles Hay Flora Peyvandi Anna-Elina Lehtinen Ingrid Pabinger Eveline Nüesch Håkan Malmström Eva Bednar Stefan Lethagen |
| author_facet | Johannes Oldenburg Charles Hay Flora Peyvandi Anna-Elina Lehtinen Ingrid Pabinger Eveline Nüesch Håkan Malmström Eva Bednar Stefan Lethagen |
| author_sort | Johannes Oldenburg |
| collection | DOAJ |
| description | Background Despite recent advances, factor replacement therapy remains a cornerstone in hemophilia A treatment. Efmoroctocog alfa, a recombinant FVIII Fc fusion protein (rFVIIIFc), has an extended half-life allowing higher FVIII levels and less frequent dosing than standard half-life (SHL) products, without increasing factor consumption.Methods A-SURE was a 24-month prospective, non-interventional study assessing real-world effectiveness of rFVIIIFc prophylaxis. This post-hoc, intra-patient analysis included patients with hemophilia A (PwHA) who switched from SHL FVIII to rFVIIIFc prophylaxis. Effectiveness endpoints included annualised bleeding rate (ABR), annualised joint bleeding rate (AjBR), weekly injection frequency and weekly factor consumption.Results Of 131 PwHA eligible for analysis, mean ABR and AjBR decreased from 3.7 and 2.4 to 1.8 and 1.1, respectively, after switching (mean [95% confidence interval (CI)] change of −1.9 [−3.0, −0.8] and −1.2 [−2.0, −0.5]). Mean weekly injection frequency decreased from 3.1 to 2.3 (mean [95% CI] change of −0.8 [−1.0, −0.7]); weekly factor consumption reduced from 89.7 to 84.1 international units (IU)/kg, respectively (mean [95% CI] change of −5.7 [−10.7, −0.6]). These trends were consistent across age groups.Conclusion This intra-patient comparison demonstrates switching from SHL FVIII to rFVIIIFc prophylaxis reduces frequency of bleeds, injection frequency, and factor consumption, complementing previously reported data from A-SURE.Trial registration:ClinicalTrials.gov identifier: NCT02976753. |
| format | Article |
| id | doaj-art-d9563e8dfb22472e9c99a4e7ef3af0ba |
| institution | Kabale University |
| issn | 1607-8454 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
| record_format | Article |
| series | Hematology |
| spelling | doaj-art-d9563e8dfb22472e9c99a4e7ef3af0ba2025-08-20T03:30:14ZengTaylor & Francis GroupHematology1607-84542025-12-0130110.1080/16078454.2025.2513186A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia AJohannes Oldenburg0Charles Hay1Flora Peyvandi2Anna-Elina Lehtinen3Ingrid Pabinger4Eveline Nüesch5Håkan Malmström6Eva Bednar7Stefan Lethagen8Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, GermanyUniversity Department of Hematology, Manchester Royal Infirmary, Manchester, UKFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, Milan, ItalyCoagulation Disorders Unit, Department of Hematology, Helsinki University Hospital Comprehensive Cancer Centre and University of Helsinki, Helsinki, FinlandClinical Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, AustriaStatistical, Epidemiology and Data Sciences, Sobi, Basel, SwitzerlandStatistical, Epidemiology and Data Sciences, Sobi, Stockholm, SwedenGlobal Medical Affairs and Clinical Development, Sobi, Stockholm, SwedenGlobal Medical Affairs and Clinical Development, Sobi, Stockholm, SwedenBackground Despite recent advances, factor replacement therapy remains a cornerstone in hemophilia A treatment. Efmoroctocog alfa, a recombinant FVIII Fc fusion protein (rFVIIIFc), has an extended half-life allowing higher FVIII levels and less frequent dosing than standard half-life (SHL) products, without increasing factor consumption.Methods A-SURE was a 24-month prospective, non-interventional study assessing real-world effectiveness of rFVIIIFc prophylaxis. This post-hoc, intra-patient analysis included patients with hemophilia A (PwHA) who switched from SHL FVIII to rFVIIIFc prophylaxis. Effectiveness endpoints included annualised bleeding rate (ABR), annualised joint bleeding rate (AjBR), weekly injection frequency and weekly factor consumption.Results Of 131 PwHA eligible for analysis, mean ABR and AjBR decreased from 3.7 and 2.4 to 1.8 and 1.1, respectively, after switching (mean [95% confidence interval (CI)] change of −1.9 [−3.0, −0.8] and −1.2 [−2.0, −0.5]). Mean weekly injection frequency decreased from 3.1 to 2.3 (mean [95% CI] change of −0.8 [−1.0, −0.7]); weekly factor consumption reduced from 89.7 to 84.1 international units (IU)/kg, respectively (mean [95% CI] change of −5.7 [−10.7, −0.6]). These trends were consistent across age groups.Conclusion This intra-patient comparison demonstrates switching from SHL FVIII to rFVIIIFc prophylaxis reduces frequency of bleeds, injection frequency, and factor consumption, complementing previously reported data from A-SURE.Trial registration:ClinicalTrials.gov identifier: NCT02976753.https://www.tandfonline.com/doi/10.1080/16078454.2025.2513186Hemophilia Astandard half-lifeextended half-lifefactor consumptioninjection frequencyreal-world |
| spellingShingle | Johannes Oldenburg Charles Hay Flora Peyvandi Anna-Elina Lehtinen Ingrid Pabinger Eveline Nüesch Håkan Malmström Eva Bednar Stefan Lethagen A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A Hematology Hemophilia A standard half-life extended half-life factor consumption injection frequency real-world |
| title | A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A |
| title_full | A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A |
| title_fullStr | A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A |
| title_full_unstemmed | A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A |
| title_short | A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A |
| title_sort | a sure intra patient comparison of prophylactic effectiveness of a recombinant factor viii fc fusion protein versus standard half life factor viii in hemophilia a |
| topic | Hemophilia A standard half-life extended half-life factor consumption injection frequency real-world |
| url | https://www.tandfonline.com/doi/10.1080/16078454.2025.2513186 |
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