Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
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.ClinicalTrials.gov identifier: NCT02976753.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1080/16078454.2025.2513186 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Switching hemophilia A patients to an extended half-life agent on a prophylactic basis: an economic appraisal.
Expert Rev Pharmacoecon Outcomes Res
July 2025
Haemostasis Unit, Haemophilia Centre, 'Aghia Sophia' Children's Hospital, Athens, Greece.
Background: Gaining an understanding of transitioning hemophilia patients to an extended half-life (EHL) agent requires real-world data, encompassing various outcomes, which would help assessing the impact of the switch, for patients and the healthcare system. We investigate the economic implications of switching from standard half-life (SHL) recombinant factor VIII (rFVIII) from either prophylaxis or on-demand, to EHL rFVIII efmoroctocog alfa (FVIIIFc) prophylaxis.
Research Design And Methods: The study involved 48 patients with hemophilia A from the 5 specialized hemophilia centers in Greece.
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.
Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half-Life FVIII in Hemophilia A: A-SURE Study.
Eur J Haematol
February 2025
Global Medical Affairs and Clinical Development, Sobi, Stockholm, Sweden.
Objectives: The 24-month, prospective, non-interventional, European multicenter A-SURE study evaluated the real-world effectiveness of prophylaxis using an extended half-life recombinant factor VIII (FVIII) Fc fusion protein, efmoroctocog alfa (hereinafter rFVIIIFc), compared with prophylaxis using standard half-life (SHL) FVIII products in patients with hemophilia A.
Methods: Primary endpoints were annualized bleeding rate (ABR), annualized injection frequency, and annualized factor consumption. A comparative study design unique for an observational hemophilia study was implemented to reduce potential confounding in effectiveness estimates, wherein each patient prescribed rFVIIIFc was matched with one receiving SHL FVIII.
Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review.
Drugs R D
September 2024
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.
Damoctocog alfa pegol (BAY 94-9027, Jivi), is a site-specifically PEGylated, extended half-life recombinant factor VIII (FVIII) that is approved in several European and non-European countries for on-demand treatment and prophylaxis of bleeding in previously treated patients aged ≥ 12 years with hemophilia A. Reliable measurements can be obtained using most one-stage and chromogenic FVIII assays over a wide concentration range. The efficacy, safety and pharmacokinetics (PK) of damoctocog alfa pegol have been studied extensively in the PROTECT VIII clinical trials, and its long-term safety and effectiveness profile is continuing to build through observational and interventional real-world studies.
View Article and Find Full Text PDFReal-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany.
Res Pract Thromb Haemost
July 2024
Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
Background: Real-world experience with efmoroctocog alfa (a recombinant factor [F]VIII Fc fusion protein [rFVIIIFc]) and eftrenonacog alfa (a recombinant factor IX Fc fusion protein [rFIXFc]) is needed to bridge evidence gaps.
Objectives: To describe rFVIIIFc/rFIXFc usage and effectiveness over a 24-month prospective period.
Methods: PREVENT (NCT03055611), a noninterventional study across 25 German hemophilia treatment centers, enrolled previously treated persons with hemophilia A and B (all ages/severities) on individualized rFVIIIFc/rFIXFc prophylaxis before/at enrollment.