Skip to main content

Advertisement

Log in

Costs and management of patients with hemophilia A in France: the Hemraude study

  • Original Paper
  • Published:
The European Journal of Health Economics Aims and scope Submit manuscript

Abstract

Objective

The Hemraude study was conducted to describe the profile of patients with HA, disease management, and economic burden in a collective perspective.

Methods

This retrospective study was conducted using the French administrative healthcare claims database SNIIRAM/SNDS. Male patients treated for hemophilia A with a long-term illness (ALD) status or invalidity were included in the study between January 1, 2016 and December 31, 2017. Patients were classified in six treatment groups: no treatment, on-demand FVIII, prophylactic FVIII, FVIII in immune tolerance induction (ITI) protocol, on-demand bypassing agents, and prophylactic bypassing agents. Patients treated with FVIII in ITI protocol and those treated with bypassing agents are deemed to have developed inhibitors. HA patients were compared to a control population without coagulation disorder and matched (ratio 1:3) on age and sex.

Results

A total of 4172 patients were included in the analysis, aged on average 35.2 years, 5.3% had HIV infection, and 8.8% had hepatitis B or C. In 2017, half of the patients received no treatment for HA, 46.7% were treated with FVIII (25% on demand, 20.6% with prophylaxis, and 1.1% ITI), 1.5% with bypassing agents. Patients treated with prophylactic treatments, either inhibitor or non-inhibitor, were less likely to be hospitalized for severe bleeding compared to patients receiving on-demand treatments. The average annual costs for HA management per patient were 72,209.60 €. The highest costs were observed in patients treated with FVIII in ITI protocol and those receiving prophylactic bypassing agents.

Conclusion

Direct costs of HA treatments for HA may be very high especially in the small percentage of patients developing inhibitors or treated with ITI protocol.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Similar content being viewed by others

References

  1. Kloosterman, F., Zwagemaker, A.F., Abdi, A., Gouw, S., Castaman, G., Fijnvandraat, K.: Hemophilia management: huge impact of a tiny difference. Res Pract Thromb Haemost 4(3), 377–385 (2020). https://doi.org/10.1002/rth2.12314.PMID:32211572;PMCID:PMC7086468

    Article  PubMed  PubMed Central  Google Scholar 

  2. Wong, T., Recht, M.: Current options and new developments in the treatment of haemophilia. Drugs 71(3), 305–320 (2011)

    Article  Google Scholar 

  3. Mannucci, P.M., Tuddenham, E.G.: The hemophilias–from royal genes to gene therapy. N Engl J Med 344, 1773–1779 (2001)

    Article  CAS  Google Scholar 

  4. World Federation of Hemophilia: Report on the Annual Global Survey 2019. October 2020. https://elearning.wfh.org/resource/report-on-the-annual-global-survey-2019/ (2020). Accessed 03 Nov 2020

  5. Doncarli, A., Demiguel, V., Guseva Canu, I., Goulet, V., Bayart, S., Calvez, T., Castet, S., Dalibard, V., Demay, Y., Frotscher, B., Goudemand, J., Lambert, T., Milien, V., Oudot, C., Sannié, T., Chambost, H.: FranceCoag network. FranceCoag: a 22-year prospective follow-up of the national French cohort of patients with inherited bleeding disorders. Eur J Epidemiol 34(5), 521–532 (2019)

    Article  Google Scholar 

  6. Soucie, J.M., Monahan, P.E., Kulkarni, R., Konkle, B.A., Mazepa, M.A.: US hemophilia treatment center network. The frequency of joint hemorrhages and procedures in non-severe hemophilia A vs B. Blood Adv 2(16), 2136–2144 (2018)

    Article  Google Scholar 

  7. Srivastava, A., Brewer, A.K., Mauser-Bunschoten, E.P., et al.: Guidelines for the management of hemophilia. Haemophilia 19(1), e1–e47 (2013)

    Article  CAS  Google Scholar 

  8. Gringeri, A., Ewenstein, B., Reininger, A.: The burden of bleeding in haemophilia: is one bleed too many? Haemophilia 20(4), 459–463 (2014)

    Article  CAS  Google Scholar 

  9. Valentino, L.A.: Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost 8(9), 1895–1902 (2010)

    Article  CAS  Google Scholar 

  10. Manco-Johnson, M.J., Abshire, T.C., Shapiro, A.D., et al.: Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 357(6), 535–544 (2007)

    Article  CAS  Google Scholar 

  11. Garagiola, I., Palla, R., Peyvandi, F.: Risk factor for inhibitor development in severe hemophilia A. Thromb Res 168, 20–27 (2018)

    Article  CAS  Google Scholar 

  12. Dekoven, M., Wisniewski, T., Petrilla, A., et al.: Health-related quality of life in haemophilia patients with inhibitors and their caregivers. Haemophilia 19(2), 287–293 (2013)

    Article  CAS  Google Scholar 

  13. Kroner, B.L.: Comparison of the international immune tolerance registry and the North American immune tolerance registry. Vox Sang. 77(Suppl 1), 33–37 (1999)

    Article  Google Scholar 

  14. Yoo, K.Y., Joo, S.C., Choi, Y.M.: Long-term course of anti-factor VIII antibody in patients with hemophilia A at a single center. Blood Res. 51(1), 37–43 (2016)

    Article  CAS  Google Scholar 

  15. Ljung, R.C.R.: How I manage patients with inherited haemophilia A and B and factor inhibitors. Br J Haematol 180(4), 501–510 (2018)

    Article  Google Scholar 

  16. Kitazawa, T., Igawa, T., Sampei, Z., et al.: A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 18(10), 1570–1574 (2012)

    Article  CAS  Google Scholar 

  17. Harkins Druzgal, C., Kizilocak, H., Brown, J., Sennett, M., Young, G.: Neutralizing antidrug antibody to emicizumab in a patient with severe hemophilia A with inhibitors: new case with detailed laboratory evaluation. J Thromb Haemost (2020). https://doi.org/10.1111/jth.14957

    Article  PubMed  Google Scholar 

  18. Colowick, A.B., Bohn, R.L., Avorn, J., Ewenstein, B.M.: Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood 96(5), 1698–1702 (2000)

    Article  CAS  Google Scholar 

  19. Grimes, D.A., Schulz, K.F.: Compared to what? Finding controls for case-control studies. Lancet 365(9468), 1429–1433 (2005)

    Article  Google Scholar 

  20. Bouillon, K., Bertrand, M., Maura, G., Blotière, P.O., Ricordeau, P., Zureik, M.: Risk of bleeding and arterial thromboembolism in patients with non-valvular atrial fibrillation either maintained on a vitamin K antagonist or switched to a non-vitamin K-antagonist oral anticoagulant: a retrospective, matched-cohort study. Lancet Haematol 2(4), e150–e159 (2015)

    Article  Google Scholar 

  21. O’Hara, J., Hughes, D., Camp, C., Burke, T., Carroll, L., Diego, D.G.: The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis 12(1), 106 (2017)

    Article  Google Scholar 

  22. Valentino, L.A., Pipe, S.W., Tarantino, M.D., Ye, X., Xiong, Y., Luo, M.P.: Healthcare resource utilization among haemophilia A patients in the United States. Haemophilia 18, 332–338 (2012)

    Article  CAS  Google Scholar 

  23. Guh, S., Grosse, S.D., McAlister, S., Kessler, C.M., Soucie, J.M.: Health care expenditures for Medicaid-covered males with haemophilia in the United States, 2008. Haemophilia 18, 276–283 (2012)

    Article  CAS  Google Scholar 

  24. Dalton, D.R.: Hemophilia in the managed care. Am J Manag Care 21(suppl 6), S123–S130 (2015)

    PubMed  Google Scholar 

  25. Zhou, Z.Y., Koerper, M.A., Johnson, K.A., Riske, B., Baker, J.R., Ullman, M., Curtis, R.G., Poon, J.L., Lou, M., Nichol, M.B.: Burden of illness: direct and indirect costs among persons with hemophilia A in the United States. J Med Econ 18(6), 457–465 (2015). https://doi.org/10.3111/13696998.2015.1016228

    Article  PubMed  Google Scholar 

  26. Tencer, T., Friedman, H.S., Li-McLeod, J., Johnson, K.: Medical costs and resource utilization for hemophilia patients with and without HIV or HCV infection. J Manag Care Pharm 13(9), 790–798 (2007)

    Article  Google Scholar 

Download references

Funding

This study was supported by an unrestricted grant from Roche.

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to B. Detournay.

Additional information

Publisher's Note

Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Supplementary Information

Below is the link to the electronic supplementary material.

Supplementary file1 (DOCX 144 KB)

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Laurendeau, C., Goudemand, J., Trossaert, M. et al. Costs and management of patients with hemophilia A in France: the Hemraude study. Eur J Health Econ 23, 23–32 (2022). https://doi.org/10.1007/s10198-021-01339-4

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s10198-021-01339-4

Keywords

JEL Classification

Navigation