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Cost-effectiveness analysis of haploidentical vs matched unrelated allogeneic hematopoietic stem cells transplantation in patients older than 55 years

Abstract

Due to limited donor availability, high comorbidities, and cost issues, allogeneic hematopoietic stem cell transplant is not universally accessible. The aim of this study was to conduct a cost-effectiveness analysis of haploidentical vs matched unrelated transplant. This retrospective study included patients with hematological malignancies older than 55 years who underwent haploidentical or matched unrelated transplant between 2011 and 2013 in Marseille. The incremental cost-effectiveness ratio has been calculated using the mean overall survival and the mean transplant costs. Costs were calculated using a micro-costing strategy from the hospital perspective and a time horizon at 2 years. Haploidentical transplant was considered an innovative procedure and matched unrelated transplant as the reference. Probabilistic and sensitivity analyses were performed on the incremental cost-effectiveness ratio. During inclusion, 29 patients underwent haploidentical transplant and 63 matched unrelated transplant. In haploidentical and matched unrelated transplant, the mean overall survival was 19.4 (1.6) months and 15.1 (1.2) months (p = 0.06), respectively, and the mean cost was 98,304 (40,872) € and 151,373 (65,742) € (p < 0.01), respectively. The incremental cost-effectiveness ratio was assessed to −148,485 (−1,265,550; −64,368) € per life year gained. Among older patients suffering from hematological malignancies, haploidentical transplant seemed in our analysis to be cost-effective compared with matched unrelated transplant.

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References

  1. Copelan EA. Hematopoietic stem-cell transplantation. N Engl J Med. 2006;354:1813–26.

    Article  CAS  Google Scholar 

  2. Gratwohl A, Baldomero H, Aljurf M, Pasquini MC, Bouzas LF, Yoshimi A, et al. Hematopoietic stem cell transplantation: a global perspective. JAMA. 2010;303:1617–24.

    Article  CAS  Google Scholar 

  3. Khera N, Zeliadt SB, Lee SJ. Economics of hematopoietic cell transplantation. Blood. 2012;120:1545–51.

    Article  CAS  Google Scholar 

  4. Gratwohl A, Pasquini MC, Aljurf M, Atsuta Y, Baldomero H, Foeken L, et al. One million haemopoietic stem-cell transplants: a retrospective observational study. Lancet Haematol. 2015;2:e91–100.

    Article  Google Scholar 

  5. Passweg JR, Baldomero H, Bader P, Bonini C, Cesaro S, Dreger P, et al. Hematopoietic SCT in Europe 2013: recent trends in the use of alternative donors showing more haploidentical donors but fewer cord blood transplants. Bone Marrow Transplant. 2015;50:476–82.

    Article  CAS  Google Scholar 

  6. Blaise D, Devillier R, Lecoroller-Sorriano A-G, Boher J-M, Boyer-Chammard A, Tabrizi R, et al. Low non-relapse mortality and long-term preserved quality of life in older patients undergoing matched related donor allogeneic stem cell transplantation: a prospective multicenter phase II trial. Haematologica . 2015;100:269–74.

    Article  Google Scholar 

  7. Gragert L, Eapen M, Williams E, Freeman J, Spellman S, Baitty R, et al. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014;371:339–48.

    Article  CAS  Google Scholar 

  8. Saber W, Opie S, Rizzo JD, Zhang M-J, Horowitz MM, Schriber J. Outcomes after matched unrelated donor versus identical sibling hematopoietic cell transplantation in adults with acute myelogenous leukemia. Blood. 2012;119:3908–16.

    Article  CAS  Google Scholar 

  9. Appelbaum FR. Allogeneic hematopoietic cell transplantation for acute myeloid leukemia when a matched related donor is not available. Hematology. 2008;2008:412–7.

    Article  Google Scholar 

  10. Joshua TV, Rizzo JD, Zhang M-J, Hari PN, Kurian S, Pasquini M, et al. Access to hematopoietic stem cell transplantation: effect of race and sex. Cancer. 2010;116:3469–76.

    Article  Google Scholar 

  11. Petersdorf EW. Limits of HLA mismatching in unrelated hematopoietic cell transplantation. Blood. 2004;104:2976–80.

    Article  CAS  Google Scholar 

  12. Kekre N, Antin JH. Hematopoietic stem cell transplantation donor sources in the 21st century: choosing the ideal donor when a perfect match does not exist. Blood. 2014;124:334–43.

    Article  CAS  Google Scholar 

  13. Appelbaum FR. Alternative donor transplantation for adults with acute leukemia. Best Pract Res Clin Haematol. 2014;27:272–7.

    Article  Google Scholar 

  14. Raiola AM, Dominietto A, di Grazia C, Lamparelli T, Gualandi F, Ibatici A, et al. Unmanipulated haploidentical transplants compared with other alternative donors and matched sibling grafts. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2014;20:1573–9.

    Article  Google Scholar 

  15. Di Stasi A, Milton DR, Poon LM, Hamdi A, Rondon G, Chen J, et al. Similar transplantation outcomes for acute myeloid leukemia and myelodysplastic syndrome patients with haploidentical versus 10/10 human leukocyte antigen-matched unrelated and related donors. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2014;20:1975–81.

    Article  Google Scholar 

  16. Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, et al. Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. 2015;126:1033–40.

    Article  CAS  Google Scholar 

  17. Shabbir-Moosajee M, Lombardi L, Ciurea SO. An overview of conditioning regimens for haploidentical stem cell transplantation with post-transplantation cyclophosphamide. Am J Hematol. 2015;90:541–8.

    Article  CAS  Google Scholar 

  18. Goldman JM, Szydlo R, Horowitz MM, Gale RP, Ash RC, Atkinson K, et al. Choice of pretransplant treatment and timing of transplants for chronic myelogenous leukemia in chronic phase. Blood. 1993;82:2235–8.

    CAS  PubMed  Google Scholar 

  19. Jourdan E, Maraninchi D, Reiffers J, Archimbaud E, Michallet M, Harousseau JL, et al. Allogeneic bone marrow transplantation remains an efficient consolidation for adults with acute myeloid leukemia even when performed very soon after diagnosis (<100 days). The SFGM (Société Française de Greffe de Moelle. Leukemia. 1995;9:1068–71.

    CAS  PubMed  Google Scholar 

  20. Gratwohl A. The EBMT risk score. Bone Marrow Transplant. 2012;47:749–56.

    Article  CAS  Google Scholar 

  21. Blaise D, Fürst S, Crocchiolo R, El-Cheikh J, Granata A, Harbi S, et al. Haploidentical T cell-replete transplantation with post-transplantation cyclophosphamide for patients in or above the sixth decade of age compared with allogeneic hematopoietic stem cell transplantation from an human leukocyte antigen-matched related or unrelated donor. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2015;22:119–24.

    Article  Google Scholar 

  22. Burns R, Leal J, Sullivan R, Luengo-Fernandez R. Economic burden of malignant blood disorders across Europe: a population-based cost analysis. Lancet Haematol. 2016;3:e362–70.

    Article  Google Scholar 

  23. Saito AM, Cutler C, Zahrieh D, Soiffer RJ, Ho VT, Alyea EP, et al. Costs of allogeneic hematopoietic cell transplantation with high-dose regimens. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2008;14:197–207.

    Article  Google Scholar 

  24. Khera N, Emmert A, Storer BE, Sandmaier BM, Alyea EP, Lee SJ. Costs of allogeneic hematopoietic cell transplantation using reduced intensity conditioning regimens. Oncologist. 2014;19:639–44.

    Article  Google Scholar 

  25. Svahn B-M, Remberger M, Alvin O, Karlsson H, Ringdén O. Increased costs after allogeneic haematopoietic SCT are associated with major complications and re-transplantation. Bone Marrow Transplant. 2012;47:706–15.

    Article  Google Scholar 

  26. Suh KJ, Kim I, Lim J, Ha H, Park S, Koh Y, et al. Total costs and clinical outcome of hematopoietic stem cell transplantation in adults with leukemia: comparison between reduced-intensity and myeloablative conditioning. Clin Transplant. 2015;29:124–33.

    Article  Google Scholar 

  27. Matthes-Martin S, Pötschger U, Barr R, Martin M, Boztug H, Klingebiel T, et al. Costs and cost-effectiveness of allogeneic stem cell transplantation in children are predictable. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2012;18:1533–9.

    Article  Google Scholar 

  28. Fuchs EJ. Related haploidentical donors are a better choice than matched unrelated donors: point. Blood Adv. 2017;1:397–400.

    Article  Google Scholar 

  29. Shawn BE. Related haploidentical donors are a better choice than matched unrelated donors: counterpoint. Blood Adv. 2017;1:401–6.

    Article  Google Scholar 

  30. Agence Technique de l’Information sur l’Hospitalisation. Référentiel de couts MCO 2014 [Internet]. Agence Technique de l’Information sur l’Hospitalisation; 2016. Available from: http://www.scansante.fr/r%C3%A9f%C3%A9rentiel-de-co%C3%BBts-mco-2014

  31. Agence Technique de l’Information sur l’Hospitalisation. Référentiel de couts SSR 2014 [Internet]. Agence Technique de l’Information sur l’Hospitalisation; 2016. Available from: http://www.scansante.fr/r%C3%A9f%C3%A9rentiel-de-co%C3%BBts-ssr-2014

  32. Caisse Nationale de l’Assurance Maladie des Travailleurs Salariés. Nomenclature des Actes de Biologie Médicale v 42. Caisse Nationale de l’Assurance Maladie des Travailleurs Salariés; 2014.

  33. Centre Hospitalier Universitaire de Montpellier. Référentiel des Actes Hors Nomenclatures v 5.3 [Internet]. Centre Hospitalier Universitaire de Montpellier; 2014. Available from: http://www.chu-montpellier.fr/fileadmin/user_upload/Pole_BiologiePathologie/BHN/RHNBio.V5.3.pdf

  34. Caisse Nationale de l’Assurance Maladie des Travailleurs Salariés. Classification Commune des Actes Médicaux v 37. Caisse Nationale de l’Assurance Maladie des Travailleurs Salariés; 2014.

  35. MINISTÈRE DES AFFAIRES SOCIALES ET DE LA SANTÉ. Arrêté du 7 avril 2014 modifiant l’arrêté du 9 mars 2010 modifié relatif au tarif de cession des produits sanguins labiles [Internet]. avril, 2014. Available from: https://www.legifrance.gouv.fr/affichTexte.do?cidTexte = JORFTEXT000028867487

  36. Haute Autorité de Santé. Choix méthodologiques pour l’évaluation économique à la HAS. Saint-Denis La Plaine: HAS; 2011 Oct.

  37. Drummond MF, Sculpher MJ, Claxton K, Stoddart GL, Torrance GW. Methods for the economic evaluation of health care programmes.. 4 ed. Oxford: Oxford Univ. Press; 2015. p. 445.

    Google Scholar 

  38. De Peretti C, Siani C. Decision-making with the incremental cost-effectiveness ratio under uncertainty. Health Syst Sci. 2006;9:111–45.

    Google Scholar 

  39. Fieller EC. Some problems in interval estimation. J R Stat Soc Ser B Methodol. 1954;16:175–85.

    Google Scholar 

  40. Siani C, De Peretti C. The performance of Fieller’s method in problematic cases often occuring in practice. Health Syst Sci. 2006;9:205–26.

    Google Scholar 

  41. Claxton K, Sculpher M, McCabe C, Briggs A, Akehurst R, Buxton M, et al. Probabilistic sensitivity analysis for NICE technology assessment: not an optional extra. Health Econ. 2005;14:339–47.

    Article  Google Scholar 

  42. Majhail NS, Mothukuri JM, Macmillan ML, Verneris MR, Orchard PJ, Wagner JE, et al. Costs of pediatric allogeneic hematopoietic-cell transplantation. Pediatr Blood Cancer. 2010;54:138–43.

    Article  Google Scholar 

  43. Preussler JM, Denzen EM, Majhail NS. Costs and cost-effectiveness of hematopoietic cell transplantation. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2012;18:1620–8.

    Article  Google Scholar 

  44. Busse R, Schreyögg J, Smith PC. Variability in healthcare treatment costs amongst nine EU countries—results from the HealthBASKET project. Health Econ. 2008;17(1 Suppl):S1–8.

    Article  Google Scholar 

  45. Majhail NS, Mau LW, Denzen EM, Arneson TJ. Costs of autologous and allogeneic hematopoietic cell transplantation in the United States: a study using a large national private claims database. Bone Marrow Transplant. 2013;48:294–300.

    Article  CAS  Google Scholar 

  46. van Agthoven M, Groot MT, Verdonck LF, Löwenberg B, Schattenberg AVMB, Oudshoorn M. et al. Cost analysis of HLA-identical sibling and voluntary unrelated allogeneic bone marrow and peripheral blood stem cell transplantation in adults with acute myelocytic leukaemia or acute lymphoblastic leukaemia. Bone Marrow Transplant. 2002;30:243–51.

    Article  Google Scholar 

  47. European Central Bank. Inflation dashboard [Internet]. European Central Bank; [cited 13 August 2016]. Available from: https://www.ecb.europa.eu/stats/prices/hicp/html/inflation.en.html

  48. Passweg JR, Baldomero H, Bregni M, Cesaro S, Dreger P, Duarte RF, et al. Hematopoietic SCT in Europe: data and trends in 2011. Bone Marrow Transplant. 2013;48:1161–7.

    Article  CAS  Google Scholar 

  49. Sorror ML. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood . 2005;106:2912–9.

    Article  CAS  Google Scholar 

  50. Armand P, Gibson CJ, Cutler C, Ho VT, Koreth J, Alyea EP, et al. A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood . 2012;120:905–13.

    Article  CAS  Google Scholar 

  51. Blaise D, Tabrizi R, Boher J-M, Le Corroller-Soriano A-G, Bay J-O, Fegueux N, et al. Randomized study of 2 reduced-intensity conditioning strategies for human leukocyte antigen-matched, related allogeneic peripheral blood stem cell transplantation: prospective clinical and socioeconomic evaluation. Cancer . 2013;119:602–11.

    Article  CAS  Google Scholar 

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Acknowledgements

We thank Christian Naït-Akli, Najoua Guelmani, Karolina Griffiths, and Jaïs Troian.

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Correspondence to Anne-Gaëlle Le Corroller Soriano.

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These authors contributed equally: Didier Blaise, Anne-Gaëlle Le Corroller Soriano.

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Debals-Gonthier, M., Siani, C., Faucher, C. et al. Cost-effectiveness analysis of haploidentical vs matched unrelated allogeneic hematopoietic stem cells transplantation in patients older than 55 years. Bone Marrow Transplant 53, 1096–1104 (2018). https://doi.org/10.1038/s41409-018-0133-5

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