A real-world evidence analysis of the impact of switching from factor VIII to emicizumab prophylaxis in patients with hemophilia A without inhibitors

References

• Rind DM, Walton SM, Agboola F, et al. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value 2020. Institute for Clinical and Economic Review.Available from: https://icer.org/wp-content/uploads/2020/10/ICER_Hemophilia-A_Final-Report_112020.pdf
   Google Scholar
• Lyons J, Desai V, Xu Y, et al. Development and validation of an algorithm for identifying patients with hemophilia A in an administrative claims database. Value Health. 2018 Sep;21(9):1098–1103. PubMed PMID: 30224115. DOI:10.1016/j.jval.2018.03.008.
   PubMed Web of Science ®Google Scholar
• Prevention CfDCa. Data & Statistics on Hemophilia 2020 [cited. https://www.cdc.gov/ncbddd/hemophilia/data.html
   Google Scholar
• Webert KE. Acquired hemophilia A. Semin Thromb Hemost. 2012 Oct;38(7):735–741. doi:10.1055/s-0032-1326779. PubMed PMID: 22941793.
   PubMed Web of Science ®Google Scholar
• Knobe K, Berntorp E. Haemophilia and joint disease: pathophysiology, evaluation, and management. J Comorb. 2011;1:51–59. PubMed PMID: 29090136; PubMed Central PMCID: PMC5556421. DOI:10.15256/joc.2011.1.2.
   PubMedGoogle Scholar
• Makris M. Prophylaxis in haemophilia should be life-long. Blood Transfus. 2012 Apr;10(2):165–168. PubMed PMID: 22337280; PubMed Central PMCID: PMC3320774. DOI:10.2450/2012.0147-11.
   PubMed Web of Science ®Google Scholar
• Aledort L, Mannucci PM, Schramm W, et al. Factor VIII replacement is still the standard of care in haemophilia A. Blood Transfus. 2019 Nov;17(6):479–486. PubMed PMID: 31846611; PubMed Central PMCID: PMC6917528. DOI:10.2450/2019.0211-19.
   PubMed Web of Science ®Google Scholar
• Simpson ML, Valentino LA. Management of joint bleeding in hemophilia. Expert Rev Hematol. 2012 Aug;5(4):459–468. PubMed PMID: 22992238. DOI:10.1586/ehm.12.27.
   PubMed Web of Science ®Google Scholar
• Chhabra A, Spurden D, Fogarty PF, et al. Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B. Blood Coagul Fibrinolysis. 2020 Apr;31(3):186–192. PubMed PMID: 32271314; PubMed Central PMCID: PMC7195855. DOI:10.1097/MBC.0000000000000885.
   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 Aug;379(9):811–822. PubMed PMID: 30157389. DOI:10.1056/NEJMoa1803550.
   PubMed Web of Science ®Google Scholar
• Ebbert PT, Xavier F, Seaman CD, et al. Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors. Haemophilia. 2020 Jan;26(1):41–46. PubMed PMID: 31746522. DOI:10.1111/hae.13877.
   PubMed Web of Science ®Google Scholar
• Donners A, Rademaker CMA, Bevers LAH, et al. Pharmacokinetics and associated efficacy of emicizumab in humans: a systematic review. Clin Pharmacokinet. 2021 Nov;60(11):1395–1406. PubMed PMID: 34389928; PubMed Central PMCID: PMC8585815. DOI:10.1007/s40262-021-01042-w.
   PubMed Web of Science ®Google Scholar
• Genentech. Highlights of prescribing information: HEMLIBRA® (emicizumab). 2021. https://www.gene.com/download/pdf/hemlibra_prescribing.pdf
   Google Scholar
• Barg AA, Budnik I, Avishai E, et al. Emicizumab prophylaxis: prospective longitudinal real-world follow-up and monitoring. Haemophilia. 2021 May;27(3):383–391. PubMed PMID: 33892524. DOI:10.1111/hae.14318.
   PubMed Web of Science ®Google Scholar
• STATinMED. Real-world data for real-world evidence: STATinMED [cited 2022 Dec]. Available from: https://statinmed.com/data/
   Google Scholar
• Health insurance coverage of the total population: Kaiser Family Foundation; 2021 [cited 2023 Mar]. Available from: https://www.kff.org/other/state-indicator/total-population/?currentTimeframe=0&sortModel=%7B%22colId%22:%22Location%22,%22sort%22:%22asc%22%7D
   Google Scholar
• Morris SB, DeShon RP. Combining effect size estimates in meta-analysis with repeated measures and independent-groups designs. Psychol Methods. 2002 Mar;7(1):105–125. PubMed PMID: 11928886. DOI:10.1037/1082-989x.7.1.105.
   PubMed Web of Science ®Google Scholar
• Levy-Mendelovich S, Brutman-Barazani T, Budnik I, et al. Real-world data on bleeding patterns of hemophilia A patients treated with emicizumab. J Clin Med. 2021 Sep:10(19): DOI:10.3390/jcm10194303. PubMed PMID: 34640320; PubMed Central PMCID: PMC8509656.
   PubMed Web of Science ®Google Scholar
• Barg AA, Livnat T, Budnik I, et al. Emicizumab treatment and monitoring in a paediatric cohort: real-world data. Br J Haematol. 2020 Oct;191(2):282–290. PubMed PMID: 32656767. DOI:10.1111/bjh.16964.
   PubMed Web of Science ®Google Scholar
• Misgav M, Brutman-Barazani T, Budnik I, et al. Emicizumab prophylaxis in haemophilia patients older than 50 years with cardiovascular risk factors: real-world data. Haemophilia. 2021 Mar;27(2):253–260. PubMed PMID: 33595174. DOI:10.1111/hae.14261.
   PubMed Web of Science ®Google Scholar
• Lewandowska M, Randall N, Bakeer N, et al. Management of people with haemophilia A undergoing surgery while receiving emicizumab prophylaxis: real-world experience from a large comprehensive treatment centre in the US. Haemophilia. 2021 Jan;27(1):90–99. PubMed PMID: 33245841; PubMed Central PMCID: PMC7898816. DOI:10.1111/hae.14212.
   PubMed Web of Science ®Google Scholar
• Wall C, Xiang H, Palmer B, et al. Emicizumab prophylaxis in people with severe haemophilia A without inhibitors: outcomes from the UK Haemophilia Centre Doctors‘ Organisation. International Society on Thrombosis and Haemostasis. 2022; [cited 2022 Jul]. https://abstracts.isth.org/abstract/emicizumab-prophylaxis-in-people-with-severe-haemophilia-a-without-inhibitors-outcomes-from-the-uk-haemophilia-centre-doctors-organisation/
   Google Scholar
• Hsiang WR, Lukasiewicz A, Gentry M, et al. Medicaid patients have greater difficulty scheduling health care appointments compared with private insurance patients: a meta-analysis. Inquiry. Jan 2019;56:46958019838118. PubMed PMID: 30947608; PubMed Central PMCID: PMC6452575. DOI:10.1177/0046958019838118.
   PubMed Web of Science ®Google Scholar
• Ayanian JZ. Medicaid expansion and access to care for low-income adults in 4 southern states during COVID-19. JAMA Health Forum. 2021 Aug;2(8):e212000. PubMed PMID: 36218714. DOI:10.1001/jamahealthforum.2021.2000.
   PubMedGoogle Scholar
• Chu WM, Ho HE, Wang JD, et al. Risk of major comorbidities among workers with hemophilia: a 14-year population-based study. Medicine (Baltimore). 2018 Feb;97(6):e9803. PubMed PMID: 29419677; PubMed Central PMCID: PMC5944666. DOI:10.1097/MD.0000000000009803.
   PubMed Web of Science ®Google Scholar
• Gualtierotti R, Solimeno LP, Peyvandi F. Hemophilic arthropathy: current knowledge and future perspectives. J Thromb Haemost. 2021 Sep;19(9):2112–2121. PubMed PMID: 34197690; PubMed Central PMCID: PMC8456897. DOI:10.1111/jth.15444.
   PubMed Web of Science ®Google Scholar
• Avina-Zubieta JA, Galindo-Rodriguez G, Lavalle C. Rheumatic manifestations of hematologic disorders. Curr Opin Rheumatol. 1998 Jan;10(1):86–90. PubMed PMID: 9448995. DOI:10.1097/00002281-199801000-00013.
   PubMedGoogle Scholar
• Hassan E, Motwani J. Breakthrough bleeding episodes in pediatric severe hemophilia A patients with and without inhibitors receiving emicizumab prophylaxis: a single-center retrospective review. Pediatr Hematol Oncol. 2022 Aug;39(5):418–426. PubMed PMID: 35170384. DOI:10.1080/08880018.2021.2004269.
   PubMed Web of Science ®Google Scholar
• Batt K, Schultz BG, Caicedo J, et al. A real-world study comparing pre-post billed annualized bleed rates and total cost of care among non-inhibitor patients with hemophilia A switching from FVIII prophylaxis to emicizumab. Curr Med Res Opin. 2022 Oct;38(10):1685–1693. PubMed PMID: 35880468. DOI:10.1080/03007995.2022.2105072.
   PubMed Web of Science ®Google Scholar