Current Hematologic Malignancy Reports

, Volume 13, Issue 2, pp 125–134 | Cite as

Value-Based Care in Hematopoietic Cell Transplantation and Cellular Therapy: Challenges and Opportunities

  • Gunjan L. ShahEmail author
  • Navneet Majhail
  • Nandita Khera
  • Sergio Giralt
Health Economics (N Khera, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Health Economics


Purpose of Review

Improved tolerability and outcomes after hematopoietic cell transplantation (HCT), along with the availability of alternative donors, have expanded its use. With this growth, and the development of additional cellular therapies, we also aim to increase effectiveness, efficiency, and the quality of the care provided. Fundamentally, the goal of value-based care is to have better health outcomes with streamlined processes, improved patient experience, and lower costs for both the patients and the health care system. HCT and cellular therapy treatments are multiphase treatments which allow for interventions at each juncture.

Recent Findings

We present a summary of the current literature with focus on program structure and overall system capacity, coordination of therapy across providers, standardization across institutions, diversity and disparities in care, patient quality of life, and cost implications.


Each of these topics provides challenges and opportunities to improve value-based care for HCT and cellular therapy patients.


Value-base care Hematopoietic cell transplantation CAR-T cell Cellular therapy 


Compliance with Ethical Standards

Conflict of Interest

Gunjan L. Shah, Nandita Khera, and Sergio Giralt declare no conflict of interest. Navneet Majhail is a consultant for Anthem, Inc.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

  1. 1.
    Majhail NS. Optimizing quality and efficiency of healthcare delivery in hematopoietic cell transplantation. Curr Hematol Malig Rep. 2015;10:199–204.CrossRefPubMedGoogle Scholar
  2. 2.
    Brown M, Brown G, Sharma S. Evidence-based to value-based medicine. Chicago: AMA Press; 2005.Google Scholar
  3. 3.
    Conrad DA. The theory of value-based payment incentives and their application to health care. Health Serv Res. 2015;50:2057–89.CrossRefPubMedPubMedCentralGoogle Scholar
  4. 4.
    Porter ME. What is value in health care? NEJM. 2010;363:2477–81.CrossRefPubMedGoogle Scholar
  5. 5.
    Hashmi SK, Bredeson C, Duarte RF, et al. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report. BBMT. 2017;23:717–25.Google Scholar
  6. 6.
    Denzen EM, Majhail NS, Stickney Ferguson S, et al. Hematopoietic cell transplantation in 2020: summary of year 2 recommendations of the National Marrow Donor Program’s System Capacity Initiative. BBMT. 2013;19:4–11.Google Scholar
  7. 7.
    Majhail NS, Murphy EA, Denzen EM, et al. The National Marrow Donor Program’s Symposium on Hematopoietic Cell Transplantation in 2020: a health care resource and infrastructure assessment. BBMT. 2012;18:172–82.Google Scholar
  8. 8.
    Neumann JL, Mau L-W, Virani S, Denzen EM, Boyle DA, Boyle NJ, et al. Burnout, moral distress, work–life balance, and career satisfaction among hematopoietic cell transplantation professionals. BBMT. 2017;
  9. 9.
    Majhail NS, Murphy EA, Omondi NA, Robinett P, Gajewski JL, LeMaistre CF, et al. Allogeneic transplant physician and center capacity in the United States. BBMT. 2011;17:956–61.Google Scholar
  10. 10.
    Majhail NS, Mau LW, Chitphakdithai P, et al. National survey of hematopoietic cell transplantation center personnel, infrastructure, and models of care delivery. BBMT. 2015;21:1308–14.Google Scholar
  11. 11.
    Nivison-Smith I, Milliken S, Dodds AJ, Gottlieb D, Kwan J, Ma DDF, et al. Activity and capacity profile of transplant physicians and centers in Australia and New Zealand. BBMT. 2018;24:169–74.Google Scholar
  12. 12.
    Merten JA, Shapiro JF, Gulbis AM, et al. Utilization of collaborative practice agreements between physicians and pharmacists as a mechanism to increase capacity to care for hematopoietic stem cell transplant recipients. BBMT. 2013;19:509–18.Google Scholar
  13. 13.
    Komanduri KV. Pharmacists and physicians in hematopoietic stem cell transplantation: advances and opportunities to use collaborative practice agreements to improve care. BBMT. 2013;19:505–8.Google Scholar
  14. 14.
    Medicare and Medicaid Programs. Reform of hospital and critical access hospital conditions of participation. Fed Regist. 2012;77:29034–76. To be codified at 42 CFR x482 and x48Google Scholar
  15. 15.
    Vulaj V, Hough S, Bedard L, Farris K, Mackler E (2018) Oncology pharmacist opportunities: closing the gap in quality care. JOP JOP2017026666.Google Scholar
  16. 16.
    Stickney Ferguson S, Randall J, Dabney J, Kalbacker ME, Boyle N, Thao V, et al. Perceived workforce challenges among clinical social workers in hematopoietic cell transplantation programs. BBMT. 2017;
  17. 17.
    LeMaistre CF, Farnia S, Crawford S, McGuirk J, Maziarz RT, Coates J, et al. Standardization of terminology for episodes of hematopoietic stem cell patient transplant care. BBMT. 2013;19:851–7.Google Scholar
  18. 18.
    Majhail NS, Giralt S, Bonagura A, Crawford S, Farnia S, Omel JL, et al. Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials. BBMT. 2015;21:583–8.Google Scholar
  19. 19.
    Khera N, Martin P, Edsall K, Bonagura A, Burns LJ, Juckett M, et al. Patient-centered care coordination in hematopoietic cell transplantation. Blood Adv. 2017;1:1617–27.CrossRefPubMedPubMedCentralGoogle Scholar
  20. 20.
    Khera N, Gooley T, Flowers MED, Sandmaier BM, Loberiza F, Lee SJ, et al. Association of distance from transplantation center and place of residence on outcomes after allogeneic hematopoietic cell transplantation. BBMT. 2016;22:1319–23.Google Scholar
  21. 21.
    Morrison CF, Martsolf DM, Wehrkamp N, Tehan R, Pai ALH. Medication adherence in hematopoietic stem cell transplantation: a review of the literature. BBMT. 2017;23:562–8.Google Scholar
  22. 22.
    • Battiwalla M, Hashmi S, Majhail N, Pavletic S, Savani BN, Shelburne N. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: developing recommendations to improve survivorship and long-term outcomes. BBMT. 2017;23:6–9. First in series of papers describing the National Institutes of Health HCT Late Effects Initiative process and recommendations. Google Scholar
  23. 23.
    Gea-Banacloche J, Komanduri KV, Carpenter P, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Immune Dysregulation and Pathobiology Working Group Report. BBMT. 2017;23:870–81.Google Scholar
  24. 24.
    Armenian SH, Chemaitilly W, Chen M, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Cardiovascular Disease and Associated Risk Factors Working Group Report. BBMT. 2017;23:201–10.Google Scholar
  25. 25.
    Morton LM, Saber W, Baker KS, Barrett AJ, Bhatia S, Engels EA, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Subsequent Neoplasms Working Group Report. BBMT. 2017;23:367–78.Google Scholar
  26. 26.
    Bevans M, El-Jawahri A, Tierney DK, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Patient-Centered Outcomes Working Group Report. BBMT. 2017;23:538–51.Google Scholar
  27. 27.
    Hashmi SK, Bredeson C, Duarte RF, et al. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: the Healthcare Delivery Working Group Report. BBMT. 2017;23:717–25.Google Scholar
  28. 28.
    Shaw BE, Hahn T, Martin PJ, Mitchell SA, Petersdorf EW, Armstrong GT, et al. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: the Research Methodology and Study Design Working Group Report. BBMT. 2017;23:10–23.Google Scholar
  29. 29.
    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.CrossRefPubMedPubMedCentralGoogle Scholar
  30. 30.
    Baker KS, Davies SM, Majhail NS, et al. Race and socioeconomic status influence outcomes of unrelated donor hematopoietic cell transplantation. BBMT. 2009;15:1543–54.Google Scholar
  31. 31.
    Rao K, Darrington DL, Schumacher JJ, Devetten M, Vose JM, Loberiza FR. Disparity in survival outcome after hematopoietic stem cell transplantation for hematologic malignancies according to area of primary residence. BBMT. 2007;13:1508–14.Google Scholar
  32. 32.
    Majhail NS, Nayyar S, Santibañez MEB, Murphy EA, Denzen EM. Racial disparities in hematopoietic cell transplantation in the United States. BMT. 2012;47:1385–90.Google Scholar
  33. 33.
    Hamilton BK, Rybicki L, Arai S, et al. Association of Socioeconomic Status with chronic graft-versus-host disease outcomes. BBMT. 2018;24:393–9.Google Scholar
  34. 34.
    Ailawadhi S, Frank RD, Advani P, Swaika A, Temkit M', Menghani R, et al. Racial disparity in utilization of therapeutic modalities among multiple myeloma patients: a SEER-medicare analysis. Cancer Med. 2017;6:2876–85.CrossRefPubMedPubMedCentralGoogle Scholar
  35. 35.
    Majhail NS, Jagasia M. Referral to transplant center for hematopoietic cell transplantation. Hematol Oncol Clin North Am. 2014;28:1201–13.CrossRefPubMedGoogle Scholar
  36. 36.
    Pidala J, Craig BM, Lee SJ, Majhail N, Quinn G, Anasetti C. Practice variation in physician referral for allogeneic hematopoietic cell transplantation. BMT. 2013;48:63–7.Google Scholar
  37. 37.
    Preston E, Chinapen S, Borrill T, et al. Disease progression is main barrier to allogeneic hematopoietic stem cell transplantation (HCT) in patients with newly diagnosed and relapsed acute leukemia. BBMT Abstract. 2018; 465.Google Scholar
  38. 38.
    Cleeland CS. Symptom burden: multiple symptoms and their impact as patient-reported outcomes. J Natl Cancer Inst Monogr. 2007;2007:16–21.CrossRefGoogle Scholar
  39. 39.
    Pidala J, Anasetti C, Jim H. Quality of life after allogeneic hematopoietic cell transplantation. Blood. 2009;114:7–19.CrossRefPubMedPubMedCentralGoogle Scholar
  40. 40.
    Rodgers C, Wills-Bagnato P, Sloane R, Hockenberry M. Health-related quality of life among children and adolescents during hematopoietic stem cell transplant recovery. J Pediatr Oncol Nurs. 2015;32:329–36.CrossRefPubMedPubMedCentralGoogle Scholar
  41. 41.
    Lawitschka A, Güclü ED, Varni JW, Putz M, Wolff D, Pavletic S, et al. Health-related quality of life in pediatric patients after allogeneic SCT: development of the PedsQL Stem Cell Transplant module and results of a pilot study. BMT. 2014;49:1093–7.Google Scholar
  42. 42.
    Rodgers C, Wills-Bagnato P, Sloane R, Hockenberry M. Health-related quality of life among children and adolescents during hematopoietic stem cell transplant recovery. J Pediatr Oncol Nurs. 2015;32:329–36.CrossRefPubMedPubMedCentralGoogle Scholar
  43. 43.
    Parsons SK, Tighiouart H, Terrin N. Assessment of health-related quality of life in pediatric hematopoietic stem cell transplant recipients: progress, challenges and future directions. Expert Rev Pharmacoecon Outcomes Res. 2013;13:217–25.CrossRefPubMedPubMedCentralGoogle Scholar
  44. 44.
    Liu Y-M, Jaing T-H, Chen Y-C, Tang S-T, Li C-Y, Wen Y-C, et al. Quality of life after hematopoietic stem cell transplantation in pediatric survivors. Cancer Nurs. 2016;39:502–9.CrossRefPubMedGoogle Scholar
  45. 45.
    Mosher CE, DuHamel KN, Rini C, Corner G, Lam J, Redd WH. Quality of life concerns and depression among hematopoietic stem cell transplant survivors. Support Care Cancer. 2011;19:1357–65.CrossRefPubMedGoogle Scholar
  46. 46.
    Bevans MF, Mitchell SA, Barrett AJ, Bishop M, Childs R, Fowler D, et al. Function, adjustment, quality of life and symptoms (FAQS) in allogeneic hematopoietic stem cell transplantation (HSCT) survivors: a study protocol. Heal Qual Life Outcomes. 2011;9:24.CrossRefGoogle Scholar
  47. 47.
    Hamilton JG, Wu LM, Austin JE, Valdimarsdottir H, Basmajian K, Vu A, et al. Economic survivorship stress is associated with poor health-related quality of life among distressed survivors of hematopoietic stem cell transplantation. Psychooncology. 2013;22:911–21.CrossRefPubMedGoogle Scholar
  48. 48.
    de Wreede LC, Watson M, van Os M, Milligan D, van Gelder M, Michallet M, et al. Improved relapse-free survival after autologous stem cell transplantation does not translate into better quality of life in chronic lymphocytic leukemia: lessons from the randomized European Society for Blood and Marrow Transplantation-Intergroup study. Am J Hematol. 2014;89:174–80.CrossRefPubMedGoogle Scholar
  49. 49.
    Kenzik K, Huang I-C, Rizzo JD, Shenkman E, Wingard J. Relationships among symptoms, psychosocial factors, and health-related quality of life in hematopoietic stem cell transplant survivors. Support Care Cancer. 2015;23:797–807.CrossRefPubMedGoogle Scholar
  50. 50.
    Pillay B, Lee SJ, Katona L, De Bono S, Burney S, Avery S. A prospective study of the relationship between sense of coherence, depression, anxiety, and quality of life of haematopoietic stem cell transplant patients over time. Psychooncology. 2015;24:220–7.CrossRefPubMedGoogle Scholar
  51. 51.
    Landau H, Wood K, Chung DJ, Koehne G, Lendvai N, Hassoun H, et al. Fractionated stem cell infusions for patients with plasma cell myeloma undergoing autologous hematopoietic cell transplantation. Leuk Lymphoma. 2016;57:1781–5.CrossRefPubMedGoogle Scholar
  52. 52.
    Deng G, Giralt S, Chung DJ, Landau H, Siman J, Search B, et al. Acupuncture for reduction of symptom burden in multiple myeloma patients undergoing autologous hematopoietic stem cell transplantation: a randomized sham-controlled trial. Support Care Cancer. 2018;26:657–65.CrossRefPubMedGoogle Scholar
  53. 53.
    Anderson KO, Giralt SA, Mendoza TR, Brown JO, Neumann JL, Mobley GM, et al. Symptom burden in patients undergoing autologous stem-cell transplantation. BMT. 2007;39:759–66.Google Scholar
  54. 54.
    Bevans MF, Mitchell SA, Barrett JA, et al. Symptom distress predicts long-term health and well-being in allogeneic stem cell transplantation survivors. BBMT. 2014;20:387–95.Google Scholar
  55. 55.
    Dueck AC, Mendoza TR, Mitchell SA, Reeve BB, Castro KM, Rogak LJ, et al. Validity and reliability of the US National Cancer Institute’s Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). JAMA Oncol. 2015;1:1051–9.Google Scholar
  56. 56.
    Shah N, Shi Q, Williams LA, et al. Higher stem cell dose infusion after intensive chemotherapy does not improve symptom burden in older patients with multiple myeloma and amyloidosis. BBMT. 2016;22:226–31.Google Scholar
  57. 57.
    Khera N, Chang Y, Hashmi S, et al. Financial burden in recipients of allogeneic hematopoietic cell transplantation. BBMT. 2014;20:1375–81.Google Scholar
  58. 58.
    Farnia S, Ganetsky A, Silver A, Hwee T, Preussler J, Griffin J, et al. Challenges around access to and cost of life-saving medications after allogeneic hematopoietic cell transplantation for Medicare patients. BBMT. 2017;23:1387–92.Google Scholar
  59. 59.
    Bestvina CM, Zullig LL, Rushing C, Chino F, Samsa GP, Altomare I, et al. Patient-oncologist cost communication, financial distress, and medication adherence. J Oncol Pr. 2014;10:162–7.CrossRefGoogle Scholar
  60. 60.
    Preussler JM, Farnia SH, Denzen EM, Majhail NS. Variation in medicaid coverage for hematopoietic cell transplantation. J Oncol Pr. 2014;10:e196–200.CrossRefGoogle Scholar
  61. 61.
    Maziarz RT, Driscoll D. Hematopoietic stem cell transplantation and implications for cell therapy reimbursement. Cell Stem Cell. 2011;8:609–12.CrossRefPubMedGoogle Scholar
  62. 62.
    • Maziarz RT, Farnia S, Martin P, Komanduri KV. Optimal benefits for hematopoietic stem cell transplantation: a consensus opinion. BBMT. 2014;20:1671–6. Recommendations by the Financial Working Group of the National Marrow Donor Program-sponsored System Capacity Initiative on what to include in a bundled insurance payment. Google Scholar
  63. 63.
    Khera N, Zeliadt SB, Lee SJ. Economics of hematopoietic cell transplantation. Blood. 2012;120:1545–51.CrossRefPubMedGoogle Scholar
  64. 64.
    Preussler JM, Mau L-W, Majhail NS, et al. Administrative claims data for economic analyses in hematopoietic cell transplantation: challenges and opportunities. BBMT. 2016;22:1738–46.Google Scholar
  65. 65.
    Preussler JM, Denzen EM, Majhail NS. Costs and cost-effectiveness of hematopoietic cell transplantation. BBMT. 2012;18:1620–8.Google Scholar
  66. 66.
    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. BMT. 2013;48:294–300.Google Scholar
  67. 67.
    Lee SJ, Anasetti C, Kuntz KM, Patten J, Antin JH, Weeks JC. The costs and cost-effectiveness of unrelated donor bone marrow transplantation for chronic phase chronic myelogenous leukemia. Blood. 1998;92:4047–52.PubMedGoogle Scholar
  68. 68.
    Shah GL, Winn AN, Lin P-J, 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. BBMT. 2015;21:1823–9.Google Scholar
  69. 69.
    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 Transpl. 2014;28:1084–91.CrossRefGoogle Scholar
  70. 70.
    Worcester S (2017) CAR T-cell therapy: moving from cost to value. In: Hematol. News. Accessed 15 Jan 2018.
  71. 71.
    Bach PB, Giralt SA, Saltz LB. FDA approval of Tisagenlecleucel. JAMA. 2017;318:1861–2.CrossRefPubMedGoogle Scholar
  72. 72.
    LeMaistre CF, Farnia SH. Goals for pay for performance in hematopoietic cell transplantation: a primer. BBMT. 2015;21:1367–72.Google Scholar
  73. 73.
    Farnia S, Shah J, Rinkle V, Gajewski J. ASBMT autologous CAR-T coding guidance. 2017Google Scholar
  74. 74.
    •• Tice JA, Walsh JM, Otuonye I, Chapman R, Kumar V, Seidner M, Ollendorf DA, Pearson SD (2017) Chimeric antigen receptor T-cell therapy for B-cell cancers: effectiveness and value. Inst Clin Econ Rev 1–120. Draft evidence report by the Institute for Clinical and Economic Review summarizing currently known clinical data on CAR T cells and presenting economic models. Google Scholar
  75. 75.
    Stranges E, Russo C, Friedman B. Procedures with the most rapidly increasing hospital costs, 2004–2007 [HCUP statistical brief 82]. Rockville, MD. 2009Google Scholar
  76. 76.
    Gajewski JL, McClellan MB, Majhail NS, et al. Payment and care for hematopoietic cell transplantation patients: toward a specialized medical home for complex care patients. BBMT. 2018;24:4–12.Google Scholar
  77. 77.
    Transplantation C for B and M (2017) CIBMTR report of survival statistics for blood and marrow transplantation. Accessed 15 Jan 2018.
  78. 78.
    Hippen BE, Maddux FW. Integrating kidney transplantation into value-based care for people with renal failure. Am J Transpl. 2018;18:43–52.CrossRefGoogle Scholar

Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  • Gunjan L. Shah
    • 1
    Email author
  • Navneet Majhail
    • 2
  • Nandita Khera
    • 3
  • Sergio Giralt
    • 1
  1. 1.Adult Bone Marrow Transplant Service, Department of MedicineMemorial Sloan Kettering Cancer CenterNew YorkUSA
  2. 2.Blood and Marrow Transplant ProgramCleveland ClinicClevelandUSA
  3. 3.Division of Hematology/OncologyMayo ClinicPhoenixUSA

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