, Volume 37, Issue 12, pp 1421–1449 | Cite as

Cost Effectiveness of Transplant, Conventional Chemotherapy, and Novel Agents in Multiple Myeloma: A Systematic Review

  • Shuangshuang Fu
  • Chi-Fang Wu
  • Michael Wang
  • David R. LairsonEmail author
Review Article



Treatments for multiple myeloma (MM) have been rapidly evolving. Newly developed treatment regimens are likely to be more effective but also cost more than conventional therapies.


We conducted a systematic review to compare the cost effectiveness of different classes of MM treatment.


We searched the PubMed, MEDLINE, Web of Science, and EMBASE databases for studies published during 1990–2018 comparing the cost effectiveness of transplant, chemotherapeutic and novel MM treatments. Titles and abstracts were independently reviewed for eligibility by two investigators. The quality of the included studies was evaluated using the 16-item, validated Quality of Health Economics Studies instrument.


Twenty-four publications were included in the systematic review and summarized according to treatment regimen and line. For first-line treatment, transplant was the most cost-effective option for transplant-eligible MM patients [the incremental cost-effectiveness ratio (ICER) was $4053–€45,460 per quality-adjusted life-year (QALY) gained, and $3848–$72,852 per life-year gained (LYG)], and the ICER for novel agents compared with conventional chemotherapy was $59,076 per QALY and $220,681 per LYG. For second-line treatment, in comparisons of novel agent-based regimens, ICERs were inconsistent. However, bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone (ICERs showed cost saving, £30,153 per QALY gained, and €39,911 per LYG, respectively).


For transplant-eligible MM patients, transplant is a cost-effective first-line treatment. More cost-effectiveness analyses comparing novel agents in the first-line treatment regimen are warranted to determine which agent or regimen is the most cost effective. In the second-line setting, it is unclear which novel agent-based regimen is most cost effective, but bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone.



The authors acknowledge the assistance with the literature search provided by Greg Pratt in the Research Medical Library, and the editorial services provided by Sarah Bronson in Scientific Publications at The University of Texas MD Anderson Cancer Center.

Author Contributions

SF: study concept and design, data collection and interpretation, and manuscript drafting and revision. CFW: data collection and interpretation, and manuscript drafting and revision. MW: data interpretation, and manuscript drafting and revision. DRL: study concept and design, data interpretation, and manuscript drafting and revision.

Compliance with Ethical Standards

Conflict of interest

Shuangshuang Fu, Chi-Fang Wu, Michael Wang and David R. Lairson have no conflicts of interest to declare.

Sources of funding



  1. 1.
    Surveillance, epidemiology and end results program. Cancer Stat Facts: Myeloma. SEER; 2017.Google Scholar
  2. 2.
    American Society of Hematology. Myeloma; 2018. Accessed 8 Apr 2018.
  3. 3.
    Kumar SK, et al. Multiple myeloma, Version 3.2017, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Cancer Netw. 2017;15:230–69.CrossRefGoogle Scholar
  4. 4.
    Naymagon L, Abdul-Hay M. Novel agents in the treatment of multiple myeloma: a review about the future. J Hematol Oncol. 2016;9:52.CrossRefGoogle Scholar
  5. 5.
    Jemal A, et al. Annual Report to the Nation on the Status of Cancer, 1975–2014, featuring survival. J Natl Cancer Inst. 2017;109:djx030.CrossRefGoogle Scholar
  6. 6.
    Kumar A, et al. Management of multiple myeloma: a systematic review and critical appraisal of published studies. Lancet Oncol. 2018;4:293–304.CrossRefGoogle Scholar
  7. 7.
    Gaultney JG, et al. Critical review of economic evaluations in multiple myeloma: An overview of the economic evidence and quality of the methodology. Eur J Cancer. 2011;47(10):1458–67.CrossRefGoogle Scholar
  8. 8.
    Zhang W, et al. Systematic review of cost-effectiveness analyses of treatments for psoriasis. PharmacoEconomics. 2015;33:327–40.CrossRefGoogle Scholar
  9. 9.
    Chiou CF, et al. Development and validation of a grading system for the quality of cost-effectiveness studies. Med Care. 2003;41(1):32–44.CrossRefGoogle Scholar
  10. 10.
    Henon PD, Donatini B, Eisenmann JC, Becker M, Beck-Wirth G. Comparative survival, quality of life and cost-effectiveness of intensive therapy with autologous blood cell transplantation or conventional chemotherapy in multiple myeloma. Bone Marrow Transplant. 1995;16(1):19–25.PubMedGoogle Scholar
  11. 11.
    Sampson FC, Beard SM, Scott F, Vandenberghe E. Cost-effectiveness of high-dose chemotherapy in first-line treatment of advanced multiple myeloma. Br J Haematol. 2001;113(4):1015–9.CrossRefGoogle Scholar
  12. 12.
    Kouroukis CT, O’Brien BJ, Benger A, Marcellus D, Foley R, Garner J, et al. Cost-effectiveness of a transplantation strategy compared to melphalan and prednisone in younger patients with multiple myeloma. Leuk Lymphoma. 2003;44(1):29–37.CrossRefGoogle Scholar
  13. 13.
    Prinja SK, Kaur G, Malhotra P, Jyani G, Ramachandran R, Bahuguna P, et al. Cost-effectiveness of autologous stem cell treatment as compared to conventional chemotherapy for treatment of multiple myeloma in India. Indian J Hematol Blood Transfus. 2017;33(1):31–40.CrossRefGoogle Scholar
  14. 14.
    Garrison LP Jr, Wang ST, Huang H, Ba-Mancini A, Shi H, Chen K, et al. The cost-effectiveness of initial treatment of multiple myeloma in the U.S. with bortezomib plus melphalan and prednisone versus thalidomide plus melphalan and prednisone or lenalidomide plus melphalan and prednisone with continuous lenalidomide maintenance treatment. Oncologist. 2013;18(1):27–36.CrossRefGoogle Scholar
  15. 15.
    Kim MY, Sposto R, Swaika A, Asano H, Alamgir A, Chanan-Khan A, et al. Pharmacoeconomic implications of lenalidomide maintenance therapy in multiple myeloma. Oncology. 2014;87(4):224–31.CrossRefGoogle Scholar
  16. 16.
    Usmani SZ, Cavenagh JD, Belch AR, Hulin C, Basu S, White D, et al. Cost-effectiveness of lenalidomide plus dexamethasone vs. bortezomib plus melphalan and prednisone in transplant-ineligible U.S. patients with newly-diagnosed multiple myeloma. J Med Econ. 2016;19(3):243–58.CrossRefGoogle Scholar
  17. 17.
    Chen Y, Lairson DR, Chan W, Huo J, Du XL. Cost-effectiveness of novel agents in medicare patients with multiple myeloma: findings from a U.S. payer’s perspective. J Manag Care Spec Pharm. 2017;23(8):831–43.PubMedGoogle Scholar
  18. 18.
    Trippoli S, Messori A, Becagli P, Alterini R, Tendi E. Treatments for newly diagnosed multiple myeloma: analysis of survival data and cost-effectiveness evaluation. Oncol Rep. 1998;5(6):1475–82.PubMedGoogle Scholar
  19. 19.
    Corso A, Mangiacavalli S, Cocito F, Pascutto C, Ferretti VV, Pompa A, et al. Long term evaluation of the impact of autologous peripheral blood stem cell transplantation in multiple myeloma: a cost-effectiveness analysis [published erratum appears in PLoS One. 2014;9(1). doi:10.1371/annotation/949c27ad-65b0-4926-8c7b-5a45d67b1be9]. PLoS One. 2013;8(9):e75047.CrossRefGoogle Scholar
  20. 20.
    Shah GL, Winn AN, Lin PJ, Klein A, Sprague KA, Smith HP, et al. Cost-effectiveness of autologous hematopoietic stem cell transplantation for elderly patients with multiple myeloma using the surveillance, epidemiology, and end results-medicare database. Biol Blood Marrow Transplant. 2015;21(10):1823–9.CrossRefGoogle Scholar
  21. 21.
    Pandya C, Hashmi S, Khera N, Gertz MA, Dispenzieri A, Hogan W, et al. Cost-effectiveness analysis of early vs. late autologous stem cell transplantation in multiple myeloma. Clin Transplant. 2014;28(10):1084–91.CrossRefGoogle Scholar
  22. 22.
    Mehta JD, Duff SB, Gupta S. Cost effectiveness of bortezomib in the treatment of advanced multiple myeloma. Manag Care Interface. 2004;17(9):52–61.PubMedGoogle Scholar
  23. 23.
    Hornberger J, Rickert J, Dhawan R, Liwing J, Aschan J, Lothgren M. The cost-effectiveness of bortezomib in relapsed/refractory multiple myeloma: Swedish perspective. Eur J Haematol. 2010;85(6):484–91.CrossRefGoogle Scholar
  24. 24.
    Moller J, Nicklasson L, Murthy A. Cost-effectiveness of novel relapsed-refractory multiple myeloma therapies in Norway: lenalidomide plus dexamethasone vs bortezomib. J Med Econ. 2011;14(6):690–7.CrossRefGoogle Scholar
  25. 25.
    Brown RE, Stern S, Dhanasiri S, Schey S. Lenalidomide for multiple myeloma: cost-effectiveness in patients with one prior therapy in England and Wales. Eur J Health Econ. 2013;14(3):507–14.CrossRefGoogle Scholar
  26. 26.
    Fragoulakis V, Kastritis E, Psaltopoulou T, Maniadakis N. Economic evaluation of therapies for patients suffering from relapsed-refractory multiple myeloma in Greece. Cancer Manag Res. 2013;5:37–48.CrossRefGoogle Scholar
  27. 27.
    Carlson JJ, Guzauskas GF, Chapman RH, Synnott PG, Liu S, Russo ET, et al. Cost-effectiveness of drugs to treat relapsed/refractory multiple myeloma in the United States. J Manag Care Spec Pharm. 2018;24(1):29–38.PubMedGoogle Scholar
  28. 28.
    Borg S, Nahi H, Hansson M, Lee D, Elvidge J, Persson U. Cost effectiveness of pomalidomide in patients with relapsed and refractory multiple myeloma in Sweden. Acta Oncol. 2016;55(5):554–60.CrossRefGoogle Scholar
  29. 29.
    Pelligra CG, Parikh K, Guo S, Chandler C, Mouro J, Abouzaid S, et al. Cost-effectiveness of pomalidomide, carfilzomib, and daratumumab for the treatment of patients with heavily pretreated relapsed-refractory multiple myeloma in the United States. Clin Ther. 2017;39(10):1986–2005.e5.CrossRefGoogle Scholar
  30. 30.
    Gueneau P, Chretien ML, Cransac-Miet A, Aho LS, Lafon I, Favennec C, et al. Efficacy, safety, and cost of pomalidomide in relapsed and refractory multiple myeloma. Eur J Haematol. 2018;100(5):518–25.CrossRefGoogle Scholar
  31. 31.
    Jakubowiak AJ, Campioni M, Benedict A, Houisse I, Tichy E, Giannopoulou A, et al. Cost-effectiveness of adding carfilzomib to lenalidomide and dexamethasone in relapsed multiple myeloma from a US perspective. J Med Econ. 2016;19(11):1061–74.CrossRefGoogle Scholar
  32. 32.
    Jakubowiak AJ, Houisse I, Majer I, Benedict A, Campioni M, Panjabi S, et al. Cost-effectiveness of carfilzomib plus dexamethasone compared with bortezomib plus dexamethasone for patients with relapsed or refractory multiple myeloma in the United States. Expert Rev Hematol. 2017;10(12):1107–19.CrossRefGoogle Scholar
  33. 33.
    Blommestein HM, Verelst SG, de Groot S, Huijgens PC, Sonneveld P, Uyl-de Groot CA. A cost-effectiveness analysis of real-world treatment for elderly patients with multiple myeloma using a full disease model. Eur J Haematol. 2016;96(2):198–208.CrossRefGoogle Scholar
  34. 34.
    Zhang W, et al. Systematic review of cost-effectiveness analyses of treatments for psoriasis. Pharmacoeconomics. 2015;33(4):327–40.CrossRefGoogle Scholar
  35. 35.
    Neumann PJ, Ganiats TG, Russell LB, Sanders GD, Siegel JE, Oxford University Press. Cost-effectiveness in health and medicine. New York: Oxford University Press; 2017.Google Scholar
  36. 36.
    Hay JW. Now is the time for transparency in value-based healthcare decision modeling. Value Health. 2019;22(5):564–9.CrossRefGoogle Scholar
  37. 37.
    Aguiar PM, Lima TM, Storpirtis S. Systematic review of the economic evaluations of novel therapeutic agents in multiple myeloma: what is the reporting quality? J Clin Pharm Ther. 2016;41(2):189–97.CrossRefGoogle Scholar
  38. 38.
    Chen W, Yang Y, Chen Y, Du F, Zhan H. Cost-effectiveness of bortezomib for multiple myeloma: a systematic review. Clinicoeconomics Outcomes Res. 2016;8:137–51.CrossRefGoogle Scholar

Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.Division of Cancer Prevention and Population Sciences, Department of Health Services ResearchThe University of Texas MD Anderson Cancer CenterHoustonUSA
  2. 2.Division of Management, Policy, and Community Health, School of Public HealthThe University of Texas Health Science Center at HoustonHoustonUSA
  3. 3.Department of Lymphoma and MyelomaThe University of Texas MD Anderson Cancer CenterHoustonUSA

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