Efficacy of rFVIIIFc versus Emicizumab for the Treatment of Patients with Hemophilia A without Inhibitors: Matching-Adjusted Indirect Comparison of A-LONG and HAVEN Trials

References

• Aledort L, Mannucci PM, Schramm W, Tarantino M. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus. 2019;17(6):479–486. doi:10.2450/2019.0211-19
   PubMed Web of Science ®Google Scholar
• National Organization for Rare Disorders (NORD®). Haemophilia A. Available from: https://rarediseases.org/rare-diseases/hemophilia-a/. Accessed October 2020.
   Google Scholar
• Valentino LA. Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost. 2010;8(9):1895–1902. doi:10.1111/j.1538-7836.2010.03962.x
   PubMed Web of Science ®Google Scholar
• Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535–544. doi:10.1056/NEJMoa067659
   PubMed Web of Science ®Google Scholar
• Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM, Group ES. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9(4):700–710. doi:10.1111/j.1538-7836.2011.04214.x
   PubMed Web of Science ®Google Scholar
• Morfini M, Benson G, Jimenez-Yuste V, et al. Tailoring care to haemophilia patients’ needs: which specialty and when? Blood Transfus. 2015;13(4):644–650. doi:10.2450/2015.0302-14
   Web of Science ®Google Scholar
• Bioverativ Therapeutics Inc. ELOCTATE® Prescribing Information; 2019. Available from: https://www.fda.gov/media/88746/download. Accessed October 2020.
   Google Scholar
• Swedish Orphan Biovitrum AB (publ). ELOCTA® summary of product characteristics; 2019. Available from: https://www.ema.europa.eu/en/documents/product-information/elocta-epar-product-information_en.pdf. Accessed October 2020.
   Google Scholar
• Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–325. doi:10.1182/blood-2013-10-529974
   PubMed Web of Science ®Google Scholar
• Young G, Mahlangu J, Kulkarni R, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015;13(6):967–977. doi:10.1111/jth.12911
   PubMed Web of Science ®Google Scholar
• Nolan B, Mahlangu J, Pabinger I, et al. Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: final results from the ASPIRE extension study. Haemophilia. 2020;26(3):494–502. doi:10.1111/hae.13953
   PubMed Web of Science ®Google Scholar
• Franchini M, Marano G, Pati I, et al. Emicizumab for the treatment of haemophilia A: a narrative review. Blood Transfus. 2019;17(3):223–228. doi:10.2450/2019.0026-19
   PubMed Web of Science ®Google Scholar
• Genetech Inc. Hemlibra® prescribing information; 2018. Available from: https://www.gene.com/download/pdf/hemlibra_prescribing.pdf. Accessed October 2020.
   Google Scholar
• Roche Pharma AG Hemlibra® summary of product characteristics; 2019. Available from: https://www.ema.europa.eu/en/documents/product-information/hemlibra-epar-product-information_en.pdf. Accessed October 2020.
   Google Scholar
• Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9):809–818. doi:10.1056/NEJMoa1703068
   PubMed Web of Science ®Google Scholar
• Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia a without inhibitors. N Engl J Med. 2018;379(9):811–822. doi:10.1056/NEJMoa1803550
   PubMed Web of Science ®Google Scholar
• Pipe SW, Shima M, Lehle M, et al. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. Lancet Haematol. 2019;6(6):e295–e305. doi:10.1016/S2352-3026(19)30054-7
   PubMed Web of Science ®Google Scholar
• Young G, Liesner R, Chang T, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019;134(24):2127–2138. doi:10.1182/blood.2019001869
   PubMed Web of Science ®Google Scholar
• Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, Welton NJ. NICE DSU Technical support document 18: methods for population-adjusted indirect comparisons in submission to NICE; 2016. Available from: http://nicedsu.org.uk/wp-content/uploads/2017/05/Population-adjustment-TSD-FINAL.pdf. Accessed October 2020.
   Google Scholar
• Signorovitch JE, Sikirica V, Erder MH, et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940–947. doi:10.1016/j.jval.2012.05.004
   PubMed Web of Science ®Google Scholar
• Higgins JPT, Green S. Cochrane handbook for systematic reviews of interventions version 5.1.0 (updated March 2011). Chochrane; 2011. Available from: www.training.cochrane.org/handbook. Accessed October 2020.
   Google Scholar
• Reyes A, Revil C, Niggli M, et al. Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial. Curr Med Res Opin. 2019;35(12):2079–2087. doi:10.1080/03007995.2019.1649378
   PubMed Web of Science ®Google Scholar
• Jain N, Lethagen S, Reyes A RE, et al. Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial. Curr Med Res Opin. 2020;36(7):1125–1127. doi:10.1080/03007995.2020.1744549
   PubMed Web of Science ®Google Scholar
• Batt K, Gao W, Ayyagari R, et al. Matching-adjusted indirect comparisons of annualized bleeding rate and utilization of BAY 94-9027 versus three recombinant factor VIII agents for prophylaxis in patients with severe hemophilia A. J Blood Med. 2019;10:147–159. doi:10.2147/JBM.S206806
   PubMed Web of Science ®Google Scholar
• Jimenez-Yuste V, Auerswald G, Benson G, et al. Achieving and maintaining an optimal trough level for prophylaxis in haemophilia: the past, the present and the future. Blood Transfus. 2014;12(3):314–319. doi:10.2450/2014.0298-13
   PubMed Web of Science ®Google Scholar