3,509
Views
5
CrossRef citations to date
0
Altmetric
Hematology

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

ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon & ORCID Icon show all
Pages 1685-1693 | Received 24 Jun 2022, Accepted 19 Jul 2022, Published online: 03 Aug 2022

References

  • Soucie JM, Evatt B, Jackson D. Occurrence of hemophilia in the United States. Am J Hematol. 1998;59(4):288–294.
  • Soucie JM, Miller CH, Dupervil B, et al. Occurrence rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia. 2020;26(3):487–493.
  • Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1-47–47.
  • Simpson ML, Valentino LA. Management of joint bleeding in hemophilia. Expert Rev Hematol. 2012;5(4):459–468.
  • 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;31(3):186–192.
  • Santagostino E, Young G, Carcao M, et al. A contemporary look at FVIII inhibitor development: still a great influence on the evolution of hemophilia therapies. Expert Rev Hematol. 2018;11(2):87–97.
  • Walsh CE, Soucie JM, Miller CH. Impact of inhibitors on hemophilia a mortality in the United States: Impact of inhibitors on hemophilia. Am. J. Hematol. 2015;90(5):400–405.
  • Brown TM, Lee WC, Joshi AV, et al. Health-related quality of life and productivity impact in haemophilia patients with inhibitors. Haemophilia. 2009;15(4):911–917.
  • Osooli M, Donfield SM, Carlsson KS, et al. Joint comorbidities among Swedish carriers of haemophilia: a register‐based cohort study over 22 years. Haemophilia. 2019;25(5):845–850.
  • Ragni MV, Berntorp E, Carcao M, et al. Chapter 8 inhibitors to clotting factor. WFH guidelines for the management of hemophilia 3rd edition. Haemophilia. 2020:26;107–120.
  • Gelbenegger G, Schoergenhofer C, Knoebl P, et al. Bridging the missing link with emicizumab: a bispecific antibody for treatment of hemophilia A. Thromb Haemost. 2020;120(10):1357–1370.
  • 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.
  • 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):e295-305–e305.
  • Reyes A, Révil 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.
  • Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia a with inhibitors. N Engl J Med. 2017;377(9):809–818.
  • Shrestha A, Eldar-Lissai A, Hou N, et al. Real-world resource use and costs of haemophilia A-related bleeding. Haemophilia. 2017;23(4):e267-75–e275.
  • Fryar CD, Kruszon-Moran D, Gu Q, et al. Mean body weight, height, waist circumference, and body mass index among adults: United States, 1999–2000 through 2015–2016. Natl Health Stat Rep. 2018;(122):1–16.
  • Kuczmarski RJ, Ogden CL, Guo SS, et al. CDC growth charts for the United States: methods and development. Vital Health Stat 11. 2000;2002(246):1–190.
  • Su J, Zhou J, Buckley B, et al. The immune tolerance induction factor utilizations and costs for the management of male hemophilia-a patients who developed inhibitors. Blood. 2016;128(22):4758–4758.
  • Quan H, Sundararajan V, Halfon P, et al. Coding algorithms for defining comorbidities in ICD-9-CM and ICD-10 administrative data. Med Care. 2005;43:1130–1139.
  • Seaman SR, Richardson S. Equivalence of prospective and retrospective models in the bayesian analysis of case-control studies. Biometrika. 2005;92(2):505–505.
  • 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;10(19):4303.
  • Barg AA, Livnat T, Budnik I, et al. Emicizumab treatment and monitoring in a paediatric cohort: real‐world data. Br J Haematol. 2020;191(2):282–290.
  • Barg AA, Budnik I, Avishai E, et al. Emicizumab prophylaxis: prospective longitudinal real‐world follow‐up and monitoring. Haemophilia. 2021;27(3):383–391.
  • Barg AA, Avishai E, Budnik I, et al. Emicizumab prophylaxis among infants and toddlers with severe hemophilia a and inhibitors—a single‐center cohort. Pediatr Blood Cancer. 2019;66(11):e27886.
  • 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;27(2):253–260.
  • Ebbert PT, Xavier F, Seaman CD, et al. Emicizumab prophylaxis in patients with haemophilia a with and without inhibitors. Haemophilia. 2020;26(1):41–46.
  • 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;27(1):90–99.
  • Caplan EO, Patel AM, DeClue RW, et al. Real-world treatment, clinical outcomes and healthcare resource utilization among persons with hemophilia a by age. J Comp Eff Res. 2021;10(15):1121–1131.
  • Samelson‐Jones BJ, Guelcher C, Kuhn J, et al. Real‐world cost estimates of initiating emicizumab in US patients with haemophilia A. Haemophilia. 2021;27(4):591–598.
  • McCary I, Guelcher C, Kuhn J, et al. Real-World use of emicizumab in patients with hemophilia with and without inhibitors. Blood. 2019;134(1):1137–1137.
  • Kakani P, Chernew M, Chandra A. Rebates in the pharmaceutical industry: evidence from medicines sold in retail pharmacies in the US. No. w26846. National Bureau of Economic Research. 2020.
  • Klamroth R, Wojciechowski P, Aballéa S, et al. 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. J Blood Med. 2021;12:115–122.
  • Cortesi PA, Castaman G, Trifirò G, et al. Cost-effectiveness and budget impact of emicizumab prophylaxis in haemophilia A patients with inhibitors. Thromb Haemost. 2020;120(2):216–228.
  • Lee H, Cho H, Han JW, et al. Cost‐utility analysis of emicizumab prophylaxis in haemophilia A patients with factor VIII inhibitors in Korea. Haemophilia. 2021;27(1):e12–e21.
  • Sun SX, Wu Y, McDermott M, et al. Cost-Effectiveness model of recombinant FVIII versus emicizumab treatment of patients with severe hemophilia A without inhibitors. Blood. 2019;134(1):2102–2102.
  • Stonebraker JS, Ducore JM. Modelling future usage and cost of factor and emicizumab to treat haemophilia a for the US Western states region IX haemophilia treatment centres. Haemophilia. 2021;27(1):e22–e29.
  • Zhou ZY, Raimundo K, Patel AM, et al. Model of short- and Long-Term outcomes of emicizumab prophylaxis treatment for persons with hemophilia A. J Manag Care Spec Pharm. 2020;26(9):1109–1120.