Session: 322. Disorders of Coagulation or Fibrinolysis: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Bleeding and Clotting, Adults, hemophilia, Workforce, Diseases, Real World Evidence, Study Population
Methods: The German AHEAD study is a non-interventional, prospective, multicenter study. This analysis focused on patients switched from rAHF to rurioctocog alfa pegol or enrolled on rurioctocog alfa pegol during 7 years of follow-up (data cutoff: Jun 30, 2020), using a time-free method of analysis to provide average values for each outcome per patient per treatment year, irrespective of the duration of study follow-up. Key outcomes included annualized bleeding rates (ABRs [all bleeds]), annualized joint bleeding rates (AJBRs), factor consumption, and factor VIII trough levels expressed in patient-years. Adverse events were also reported. In the analyses presented for bleeding rates and consumption, the switch year was excluded. These results focus on rurioctocog alfa pegol; however, outcomes for rAHF are included in the table for comparison. This study was reviewed/approved by the relevant institutional review boards/ethics committees of all participating centers.
Results: A total of 382 patients were receiving rAHF at baseline; 60 of these switched from rAHF to rurioctocog alfa pegol during the study. Two additional patients were enrolled on rurioctocog alfa pegol, giving a total of 62 patients in the rurioctocog alfa pegol group (severe HA, n=55; moderate HA, n=7; PKP, n=27; SP, n=35). Median age (range) for the 322 patients who received rAHF throughout the study (severe HA, n=258; moderate HA, n=64) was 24 (1-80) years. In the time-free analysis of bleeding rates, the combined rurioctocog alfa pegol data for enrolled patients and switched patients with severe HA resulted in a total of 23 patient-years of data for rurioctocog alfa pegol (SP, n=8 patient-years; PKP, n=15 patient-years). Median (Q1-Q3) ABRs were lower in patients receiving PKP with rurioctocog alfa pegol vs SP (0 [0-2.9] vs 0.5 [0-1.6]). Similar median AJBRs were observed between the PKP and SP groups (Table). A higher proportion of patients receiving PKP vs SP had an ABR or AJBR of zero (ABRs, 60% vs 50%; AJBRs, 80% vs 75%, respectively). Mean annualized total dose for rurioctocog alfa pegol was 4154 IU/kg for SP and 4662 IU/kg for PKP. Mean (median [Q1-Q3]) target FVIII trough levels for patients with severe HA receiving rurioctocog alfa pegol PKP were 6.0% (5.0% [3.2%-7.9%]). For patients receiving rAHF PKP, the mean (median [Q1-Q3]) target trough levels were 3.5% (3.0% [2.0%-5.0%]). Adverse events occurred in 9/62 (14.5%) patients treated with rurioctocog alfa pegol in the time-free analysis; 4 (6.5%) were serious adverse events and none were treatment related. De novo inhibitors were reported for 3 patients in the rAHF group and for none in the rurioctocog alfa pegol group.
Conclusion: These real-world data showed lower bleeding rates with rurioctocog alfa pegol PKP versus SP in the time-free analysis.
Disclosures: Klamroth: Daiichi Sankyo: Other, Speakers Bureau; LEO: Other, Research Funding, Speakers Bureau; Uniqure: Consultancy, Other; Sobi: Consultancy, Other, Speakers Bureau; Sanofi: Consultancy, Other: Clinical trial investigator, Travel support, Speakers Bureau; Octapharma: Consultancy, Other: Clinical trial investigator, Travel support, Speakers Bureau; Roche/Cugai: Consultancy, Other: Clinical trial investigator, Travel support, Speakers Bureau; Biotest: Consultancy, Other: Travel support, Speakers Bureau; BioMarin: Consultancy, Other: Clinical trial investigator, Travel support, Speakers Bureau; Shire (a Takeda company): Consultancy, Other: Clinical trial investigator, Travel support, Research Funding, Speakers Bureau; Pfizer: Consultancy, Other: Clinical trial investigator, Travel support, Research Funding, Speakers Bureau; Novo Nordisk: Consultancy, Other: Clinical trial investigator, Travel support, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Other: Clinical trial investigator, Travel support, Research Funding, Speakers Bureau; Bayer: Consultancy, Other: Clinical trial investigator, Travel support, Research Funding, Speakers Bureau; Grifols: Speakers Bureau. Kurnik: Bayer: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Novo Nordisk: Consultancy, Research Funding, Speakers Bureau; Pfizer: Research Funding; Roche: Consultancy, Research Funding, Speakers Bureau; Shire (a Takeda company): Consultancy, Research Funding, Speakers Bureau; Sobi: Consultancy, Research Funding; Biotest: Consultancy, Speakers Bureau. Wenning: Bayer: Research Funding; Biotest: Consultancy, Research Funding; CSL Behring: Research Funding; Octapharma: Research Funding; Shire (a Takeda company): Consultancy, Research Funding; Sobi: Consultancy, Research Funding; Roche: Consultancy, Research Funding, Speakers Bureau. Escuriola-Ettingshausen: Biotest: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Octapharma: Consultancy, Research Funding, Speakers Bureau; Shire (a Takeda company): Consultancy, Research Funding, Speakers Bureau; Sobi: Consultancy, Research Funding, Speakers Bureau; BioMarin: Consultancy; Grifols: Consultancy, Speakers Bureau; Novo Nordisk: Consultancy, Speakers Bureau; Roche/Chugai: Consultancy, Speakers Bureau; Bayer: Speakers Bureau; Kedrion: Speakers Bureau; Pfizer: Speakers Bureau. Regensburger: Takeda Pharma Vertrieb GmbH & Co. KG: Current Employment. Tang: Takeda Pharmaceuticals International AG: Current Employment; Takeda: Current equity holder in publicly-traded company. Gu: Takeda Development Center Americas, Inc.: Current Employment; Takeda: Current equity holder in publicly-traded company. Guerra: Takeda Pharmaceuticals: Current Employment; Takeda: Current equity holder in publicly-traded company. Oldenburg: Novo Nordisk: Consultancy, Honoraria, Research Funding, Speakers Bureau; Freeline: Consultancy, Honoraria, Speakers Bureau; Grifols: Consultancy, Honoraria, Speakers Bureau; Chugai: Consultancy, Honoraria, Research Funding, Speakers Bureau; CSL-Behring: Consultancy, Honoraria, Research Funding, Speakers Bureau; Biomarin: Consultancy, Honoraria, Speakers Bureau; Biotest: Consultancy, Honoraria, Research Funding, Speakers Bureau; Bayer: Consultancy, Honoraria, Research Funding, Speakers Bureau; Biogen Idec: Consultancy, Honoraria, Speakers Bureau; University Clinic Bonn AöR: Current Employment; Octapharma: Consultancy, Honoraria, Research Funding, Speakers Bureau; Pfizer: Consultancy, Honoraria, Research Funding, Speakers Bureau; Shire (a Takeda company): Consultancy, Research Funding, Speakers Bureau; Roche: Consultancy, Speakers Bureau; Sobi: Consultancy, Speakers Bureau.