Analysis of Pooled Real-World Utilization and Outcomes Data of rVIII-Single Chain Compared With Standard and Extended Half-Life FVIII Products for Prophylaxis of Hemophilia A in France, Germany, and Italy
Author(s)
Maria Rosaria Villa, MD1, Abel Hassoun, MD, PhD2, Korinna Karampampa, MSc, PhD3, Ying Yang, PhD4, Marco Panebianco, MSc5, Mariano Ratto, MSc4, Xiang Zhang, PhD6, Florian Langer, MD7.
1Haemophilia and Thrombosis Centre, Hematology Unit, Ospedale del Mare, Naples, Italy, 2Haemophilia Treatment Center, Simone Veil Hospital, GH Eaubonne-Montmorency, Eaubonne, France, 3Director Global Market Access, CSL Behring, Stockholm, Sweden, 4Herspiegel, San Francisco, CA, USA, 5CSL Behring, Milan, Italy, 6CSL Behring, King of Prussia, PA, USA, 7Haemophilia Centre, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
1Haemophilia and Thrombosis Centre, Hematology Unit, Ospedale del Mare, Naples, Italy, 2Haemophilia Treatment Center, Simone Veil Hospital, GH Eaubonne-Montmorency, Eaubonne, France, 3Director Global Market Access, CSL Behring, Stockholm, Sweden, 4Herspiegel, San Francisco, CA, USA, 5CSL Behring, Milan, Italy, 6CSL Behring, King of Prussia, PA, USA, 7Haemophilia Centre, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
OBJECTIVES: To evaluate real-world utilisation and outcomes of rVIII-SingleChain prophylaxis in patients with moderate or severe haemophilia A versus extended half-life (EHL) and standard half-life (SHL) factor VIII (FVIII) products in France, Germany and Italy.
METHODS: De-identified patient chart data on prophylactic use (≥6 months) of rVIII-SingleChain, rFVIIIFc (EHL), octocog alfa and BAY 81-8973 (SHL) were collected from treatment centres in France, Germany and Italy. Descriptive statistics were summarised for FVIII consumption, annualised bleeding rate (ABR) and patients with zero bleeds. Generalised linear model regression was performed for FVIII consumption and ABR, adjusting for potential covariates using weights from propensity score matching performed at inclusion. Data were pooled and sub-analysed by disease severity and country.
RESULTS: Overall, 549 patients aged ≥12 years were included (rVIII-SingleChain, n=118; rFVIIIFc, n=175; octocog alfa, n=117; BAY 81-8973, n=139). After covariate adjustment, mean FVIII consumption with rVIII-SingleChain (84.2 IU/kg/week) was lower versus octocog alfa (100.4 IU/kg/week, p=0.011) and comparable with BAY 81-8973 and rFVIIIFc (87.2 IU/kg/week, p=0.637; 85.4 IU/kg/week, p=0.815); in moderate patients, mean FVIII consumption was lower with rVIII-SingleChain versus rFVIIIFc in France and Germany (46.4 vs 77.3 IU/kg/week, p=0.020; 51.1 vs 77.2 IU/kg/week, p=0.029, respectively), and comparable in Italy (89.5 vs 90.0 IU/kg/week, p=0.933). After covariate adjustment, ABR was lower with rVIII-SingleChain versus BAY 81-8973 (1.06 vs 1.41, p=0.024) and comparable with octocog alfa (1.25, p=0.306) and rFVIIIFc (1.10, p=0.614); in Italy, ABR was lower with rVIII-SingleChain versus BAY 81-8973 (0.61 vs 1.12, p=0.002) and octocog alfa (1.14, p=0.013). Zero bleeds occurred in 35.6%, 30.3%, 20.5% and 25.9% of rVIII-SingleChain-, rFVIIIFc-, octocog alfa- and BAY 81-8973-treated patients, respectively. FVIII consumption and bleeding data in patients aged ≥18 years (n=415) supported these findings.
CONCLUSIONS: Prophylaxis with rVIII-SingleChain may reduce consumption and improve bleed protection compared with SHL FVIII products, and may provide comparable protection to EHL FVIII products.
METHODS: De-identified patient chart data on prophylactic use (≥6 months) of rVIII-SingleChain, rFVIIIFc (EHL), octocog alfa and BAY 81-8973 (SHL) were collected from treatment centres in France, Germany and Italy. Descriptive statistics were summarised for FVIII consumption, annualised bleeding rate (ABR) and patients with zero bleeds. Generalised linear model regression was performed for FVIII consumption and ABR, adjusting for potential covariates using weights from propensity score matching performed at inclusion. Data were pooled and sub-analysed by disease severity and country.
RESULTS: Overall, 549 patients aged ≥12 years were included (rVIII-SingleChain, n=118; rFVIIIFc, n=175; octocog alfa, n=117; BAY 81-8973, n=139). After covariate adjustment, mean FVIII consumption with rVIII-SingleChain (84.2 IU/kg/week) was lower versus octocog alfa (100.4 IU/kg/week, p=0.011) and comparable with BAY 81-8973 and rFVIIIFc (87.2 IU/kg/week, p=0.637; 85.4 IU/kg/week, p=0.815); in moderate patients, mean FVIII consumption was lower with rVIII-SingleChain versus rFVIIIFc in France and Germany (46.4 vs 77.3 IU/kg/week, p=0.020; 51.1 vs 77.2 IU/kg/week, p=0.029, respectively), and comparable in Italy (89.5 vs 90.0 IU/kg/week, p=0.933). After covariate adjustment, ABR was lower with rVIII-SingleChain versus BAY 81-8973 (1.06 vs 1.41, p=0.024) and comparable with octocog alfa (1.25, p=0.306) and rFVIIIFc (1.10, p=0.614); in Italy, ABR was lower with rVIII-SingleChain versus BAY 81-8973 (0.61 vs 1.12, p=0.002) and octocog alfa (1.14, p=0.013). Zero bleeds occurred in 35.6%, 30.3%, 20.5% and 25.9% of rVIII-SingleChain-, rFVIIIFc-, octocog alfa- and BAY 81-8973-treated patients, respectively. FVIII consumption and bleeding data in patients aged ≥18 years (n=415) supported these findings.
CONCLUSIONS: Prophylaxis with rVIII-SingleChain may reduce consumption and improve bleed protection compared with SHL FVIII products, and may provide comparable protection to EHL FVIII products.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO10
Topic
Clinical Outcomes, Medical Technologies, Real World Data & Information Systems
Topic Subcategory
Clinical Outcomes Assessment
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)