Economic impact model of delayed inhibitor development in patients with hemophilia a receiving emicizumab for the prevention of bleeding events

References

• Centers for Disease Control and Prevention. Data & statistics: hemophilia. 2016 [cited 2018 Aug 28]. Available from: https://www.cdc.gov/ncbddd/hemophilia/data.html.
   Google Scholar
• Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on‐demand and prophylaxis treatments in hemophilia A management. J Thrombosis Haemostasis. 2012;10(3):359–367.
   PubMed Web of Science ®Google Scholar
• Zhou ZY, Koerper MA, Johnson KA, et al. Burden of illness: direct and indirect costs among persons with hemophilia A in the United States. J Med Econ. 2015;18(6):457–465.
   PubMed Web of Science ®Google Scholar
• Fischer K, Ljung R. Primary prophylaxis in haemophilia care: guideline update 2016. Blood Cells Mol Dis. 2017;67:81–85.
   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.
   PubMed Web of Science ®Google Scholar
• O'Hara J, Sima CS, Frimpter J, et al. Long-term outcomes from prophylactic or episodic treatment of haemophilia A: a systematic review. Haemophilia. 2018;0(0):1–11.
   Google Scholar
• Rota M, Cortesi PA, Steinitz-Trost KN, et al. Meta-analysis on incidence of inhibitors in patients with haemophilia A treated with recombinant factor VIII products. Blood Coag Fibr. 2017;28(8):627–637.
   PubMed Web of Science ®Google Scholar
• Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–e47.
   PubMed Web of Science ®Google Scholar
• Gouw SC, van der Bom JG, Marijke van den Berg H. Marijke van den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood. 2007;109(11):4648–4654.
   PubMed Web of Science ®Google Scholar
• Hay CRM, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012;119(6):1335–1344.
   PubMed Web of Science ®Google Scholar
• Colowick AB, Bohn RL, Avorn J, et al. Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood. 2000;96(5):1698–1702.
   PubMed Web of Science ®Google Scholar
• Chen SL. Economic costs of hemophilia and the impact of prophylactic treatment on patient management. Am J Manag Care. 2016;22(5 Suppl):S126–S133.
   PubMed Web of Science ®Google Scholar
• Guh S, Grosse SD, McALISTER S, et al. Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008. Haemophilia. 2012;18(2):268–275.
   PubMed Web of Science ®Google Scholar
• Guh S, Grosse SD, McAlister S, et al. Health care expenditures for Medicaid-covered males with haemophilia in the United States, 2008. Haemophilia. 2012;18(2):276–283.
   PubMed Web of Science ®Google Scholar
• Armstrong EP, Malone DC, Krishnan S, et al. Costs and utilization of hemophilia A and B patients with and without inhibitors. J Med Econ. 2014;17(11):798–802.
   PubMedGoogle Scholar
• Morfini M, Haya S, Tagariello G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia. 2007;13(5):606–612.
   PubMed Web of Science ®Google Scholar
• McLaughlin JM, Munn JE, Anderson TL, et al. Predictors of quality of life among adolescents and young adults with a bleeding disorder. Health Qual Life Outcomes. 2017;15(1):67.
   PubMedGoogle Scholar
• HEMLIBRA [prescribing information]. Genentech Inc. South San Francisco, CA. November. 2018.
   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.
   PubMed Web of Science ®Google Scholar
• Young G, Sidonio RF, Liesner R, et al. HAVEN 2 updated analysis: multicenter, open-label, phase 3 study to evaluate efficacy, safety and pharmacokinetics of subcutaneous administration of emicizumab prophylaxis in pediatric patients with hemophilia a with inhibitors. Blood J. 2017;130:85.
   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.
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054–2064.
   PubMed Web of Science ®Google Scholar
• Antunes SV, Tangada S, Stasyshyn O, et al. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors. Haemophilia. 2014;20(1):65–72.
   PubMed Web of Science ®Google Scholar
• IBM Micromedex. Red book online [cited 7 Jan 2019]. Available from: http://www.micromedexsolutions.com/micromedex2/librarian/ (subscription required).
   Google Scholar
• ADVATE (antihemophilic factor [recombinant] package insert). Westlake Village, CA: Baxalta US Inc. 2016.
   Google Scholar
• FEIBA (anti-inhibitor coagulant complex) package insert. 2018. Available from: https://www.shirecontent.com/PI/PDFs/FEIBA_USA_ENG.pdf.
   Google Scholar
• Neufeld EJ, Sidonio RF, O’Day K, et al. Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States. J Med Econ. 2018;21(8):762–769.
   PubMed Web of Science ®Google Scholar
• NOVOSEVEN (Coagulation - Factor VIIa [Recombinant]) package insert. Accessed 7 January 2018.
   Google Scholar
• Centers for Disease Control and Prevention. Data table for boys length-for-age and weight-for-age charts [cited 23 Jul 2018]. Available from: https://www.cdc.gov/growthcharts/who/boys_length_weight.htm.
   Google Scholar
• World Health Organization. Data table for boys length-for-age and weight-for-age charts [cited 23 Jul 2018]. Available from: https://www.cdc.gov/growthcharts/data/zscore/wtage.xls.
   Google Scholar
• Batsuli G, Zimowski KL, Tickle K, et al. Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis. Haemophilia. 2019. doi: 10.1111/hae.13819.
   PubMedGoogle Scholar
• Centers for Disease Control and Prevention. Data & statistics: hemophilia [cited 23 Jul 2018]. Available from: https://www.cdc.gov/ncbddd/hemophilia/data.html.
   Google Scholar
• Centers for Disease Control and Prevention. Report on the Universal Data Collection Program, 2005–2009 [cited 6 Aug 2018]. Available from: https://www.cdc.gov/ncbddd/blooddisorders/udc/documents/report-udcprogram_january2005-december-2009_jan-2014.pdf.
   Google Scholar
• American Thrombosis & Hemostasis Network. ATHN Research Report Brief - September 30, 2017 [cited 28 Aug 2018]. Available from: https://athn.org/documents/document_file/302.
   Google Scholar
• Nagao A, Yeung CHT, Germini F, et al. Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing. Thromb Res. 2019;173:79–84.
   PubMed Web of Science ®Google Scholar
• Megias-Vericat JE, Bonanad S, Haya S, et al. Bayesian pharmacokinetic-guided prophylaxis with recombinant factor VIII in severe or moderate haemophilia A. Thromb Res. 2019;174:151–162.
   PubMed Web of Science ®Google Scholar
• Mingot-Castellano ME, Parra R, Nunez R, et al. Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic-guided prophylaxis based on Bayesian models with myPKFiT((R)). Haemophilia. 2018;24(5):e338–e343.
   PubMed Web of Science ®Google Scholar
• Iorio A. Using pharmacokinetics to individualize hemophilia therapy. Hematology Am Soc Hematol Educ Program. 2017;2017(1):595–604.
   PubMedGoogle Scholar
• Iorio A, Edginton AN, Blanchette V, et al. Performing and interpreting individual pharmacokinetic profiles in patients with Hemophilia A or B: Rationale and general considerations. Res Pract Thromb Haemost. 2018;2(3):535–548.
   PubMed Web of Science ®Google Scholar
• McMullen S, Buckley B, Hall E, et al. Budget impact analysis of prolonged half-life recombinant FVIII therapy for hemophilia in the United States. Value Health. 2017;20(1):93–99.
   PubMed Web of Science ®Google Scholar
• 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:e27886.
   PubMed Web of Science ®Google Scholar
• Pierce GF, Hart DP, Kaczmarek R. Safety and efficacy of emicizumab and other novel agents in newborns and infants. Haemophilia. 2019. doi: 10.1111/hae.13822.
   Web of Science ®Google Scholar
• Komvilaisak P, Connolly B, Naqvi A, et al. Overview of the use of implantable venous access devices in the management of children with inherited bleeding disorders. Haemophilia. 2006;12(s6):87–93.
   PubMedGoogle Scholar
• Santagostino E, Mancuso ME. Venous access in haemophilic children: choice and management. Haemophilia. 2010;16(s1):20–24.
   PubMedGoogle Scholar
• Walsh CE, Valentino LA. Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices. Haemophilia. 2009;15(5):1014–1021.
   PubMed Web of Science ®Google Scholar
• Ljung R, Gretenkort Andersson N. The current status of prophylactic replacement therapy in children and adults with haemophilia. Br J Haematol. 2015;169(6):777–786.
   PubMed Web of Science ®Google Scholar
• Scott MJ, Xiang H, Hart DP, et al. Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study. Haemophilia. 2018;25(2):205–212.
   PubMed Web of Science ®Google Scholar