Official reprint from UpToDate®
www.uptodate.com ©2017 UpToDate, Inc. and/or its affiliates. All Rights Reserved.

Determining eligibility for allogeneic hematopoietic cell transplantation

H Joachim Deeg, MD
Brenda M Sandmaier, MD
Section Editor
Nelson J Chao, MD
Deputy Editor
Alan G Rosmarin, MD


Hematopoietic cell transplantation (HCT) is a general term that encompasses a series of procedures in which the patient is treated with chemotherapy, radiation therapy, or both (referred to as the "preparative/conditioning regimen") followed by the infusion of hematopoietic stem/progenitor cells. Various strategies for HCT have been developed and are applied, dependent upon the patient’s disease and disease stage, the hematopoietic cell donor, and the source of hematopoietic progenitor cells. The assessment of those parameters, in turn, will affect the selection of the preparative regimen. These factors influence the efficacy of HCT and the short and long-term toxicities associated with the procedure. (See "The approach to hematopoietic cell transplantation survivorship", section on 'Terminology'.)

Allogeneic HCT (allo-HCT) uses hematopoietic progenitor cells collected from a healthy person (not from the patients themselves). Allo-HCT is increasingly used to treat a variety of hematologic neoplasms and nonmalignant marrow disorders (acquired and inherited), including inborn errors of metabolism. Eligibility for allo-HCT varies across countries and institutions. Ultimately, decisions regarding transplant eligibility should be made on a case-by-case basis dependent upon a risk-benefit assessment, and the needs and wishes of the patient. Here we discuss eligibility for allo-HCT. Eligibility for autologous HCT, the use of HCT in specific disease settings, and the short- and long-term complications of HCT are discussed separately. (See "Determining eligibility for autologous hematopoietic cell transplantation" and "Hematopoietic support after hematopoietic cell transplantation" and "Quality of life following hematopoietic cell transplantation" and "Management of the hematopoietic cell transplant recipient in the immediate post-transplant period" and "The approach to hematopoietic cell transplantation survivorship".)


Disease-related indications — Historically, allo-HCT was offered to patients with hematologic malignancies who had exhausted other treatment modalities, and eligibility was largely based upon whether the patient was in adequate physical condition to tolerate the anticipated toxicity. Currently, a decision to perform allo-HCT must include an assessment of the underlying disease state and whether allo-HCT is likely to offer results superior to those achieved with nontransplant options. The risks of morbidity and mortality associated with allo-HCT must be compared with those of other treatment approaches.

The role of allo-HCT in specific diseases is discussed in more detail separately. In general, allo-HCT may be considered in the following settings:

Acute myeloid leukemia – As post-remission therapy for patients in first or subsequent remission. (See "Post-remission therapy for acute myeloid leukemia in younger adults", section on 'Myeloablative allogeneic transplantation' and "Treatment of relapsed or refractory acute myeloid leukemia", section on 'Hematopoietic cell transplantation'.)

To continue reading this article, you must log in with your personal, hospital, or group practice subscription. For more information on subscription options, click below on the option that best describes you:

Subscribers log in here

Literature review current through: Nov 2017. | This topic last updated: Apr 19, 2017.
The content on the UpToDate website is not intended nor recommended as a substitute for medical advice, diagnosis, or treatment. Always seek the advice of your own physician or other qualified health care professional regarding any medical questions or conditions. The use of this website is governed by the UpToDate Terms of Use ©2017 UpToDate, Inc.
  1. Lee SJ, Joffe S, Artz AS, et al. Individual physician practice variation in hematopoietic cell transplantation. J Clin Oncol 2008; 26:2162.
  2. Bacigalupo A, Ballen K, Rizzo D, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant 2009; 15:1628.
  3. Martino R, Iacobelli S, Brand R, et al. Retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic hematopoietic stem cell transplantation using HLA-identical sibling donors in myelodysplastic syndromes. Blood 2006; 108:836.
  4. de Lima M, Anagnostopoulos A, Munsell M, et al. Nonablative versus reduced-intensity conditioning regimens in the treatment of acute myeloid leukemia and high-risk myelodysplastic syndrome: dose is relevant for long-term disease control after allogeneic hematopoietic stem cell transplantation. Blood 2004; 104:865.
  5. Scott BL, Sandmaier BM, Storer B, et al. Myeloablative vs nonmyeloablative allogeneic transplantation for patients with myelodysplastic syndrome or acute myelogenous leukemia with multilineage dysplasia: a retrospective analysis. Leukemia 2006; 20:128.
  6. Scott BL, Pasquini MC, Logan B, et al. Results of a Phase III Randomized, Multi-Center Study of Allogeneic Stem Cell Transplantation after High Versus Reduced Intensity Conditioning in Patients with Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML): Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0901 (LBA #8). Blood (ASH Annual Meeting Abstracts) 2015.
  7. Baron F, Storb R, Storer BE, et al. Factors associated with outcomes in allogeneic hematopoietic cell transplantation with nonmyeloablative conditioning after failed myeloablative hematopoietic cell transplantation. J Clin Oncol 2006; 24:4150.
  8. Martino R, Caballero MD, de la Serna J, et al. Low transplant-related mortality after second allogeneic peripheral blood stem cell transplant with reduced-intensity conditioning in adult patients who have failed a prior autologous transplant. Bone Marrow Transplant 2002; 30:63.
  9. Armand P, Gibson CJ, Cutler C, et al. A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood 2012; 120:905.
  10. Armand P, Kim HT, Logan BR, et al. Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation. Blood 2014; 123:3664.
  11. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 1997; 89:2079.
  12. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood 2012; 120:2454.
  13. Passamonti F, Cervantes F, Vannucchi AM, et al. Dynamic International Prognostic Scoring System (DIPSS) predicts progression to acute myeloid leukemia in primary myelofibrosis. Blood 2010; 116:2857.
  14. Gangat N, Caramazza D, Vaidya R, et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol 2011; 29:392.
  15. Kröger N, Giorgino T, Scott BL, et al. Impact of allogeneic stem cell transplantation on survival of patients less than 65 years of age with primary myelofibrosis. Blood 2015; 125:3347.
  16. Mielcarek M, Martin PJ, Leisenring W, et al. Graft-versus-host disease after nonmyeloablative versus conventional hematopoietic stem cell transplantation. Blood 2003; 102:756.
  17. http://www.cibmtr.org/ReferenceCenter/SlidesReports/SummarySlides/pages/index.aspx (Accessed on November 18, 2011).
  18. Lim Z, Brand R, Martino R, et al. Allogeneic hematopoietic stem-cell transplantation for patients 50 years or older with myelodysplastic syndromes or secondary acute myeloid leukemia. J Clin Oncol 2010; 28:405.
  19. McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with myelodysplastic syndrome. J Clin Oncol 2010; 28:1878.
  20. Sorror ML, Sandmaier BM, Storer BE, et al. Long-term outcomes among older patients following nonmyeloablative conditioning and allogeneic hematopoietic cell transplantation for advanced hematologic malignancies. JAMA 2011; 306:1874.
  21. Yee GC, Lennon TP, Gmur DJ, et al. Age-dependent cyclosporine: pharmacokinetics in marrow transplant recipients. Clin Pharmacol Ther 1986; 40:438.
  22. Esposito C, Plati A, Mazzullo T, et al. Renal function and functional reserve in healthy elderly individuals. J Nephrol 2007; 20:617.
  23. Glezerman IG, Jhaveri KD, Watson TH, et al. Chronic kidney disease, thrombotic microangiopathy, and hypertension following T cell-depleted hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2010; 16:976.
  24. Chien JW, Sullivan KM. Carbon monoxide diffusion capacity: how low can you go for hematopoietic cell transplantation eligibility? Biol Blood Marrow Transplant 2009; 15:447.
  25. Deeg HJ, Seidel K, Bruemmer B, et al. Impact of patient weight on non-relapse mortality after marrow transplantation. Bone Marrow Transplant 1995; 15:461.
  26. Morton AJ, Gooley T, Hansen JA, et al. Association between pretransplant interferon-alpha and outcome after unrelated donor marrow transplantation for chronic myelogenous leukemia in chronic phase. Blood 1998; 92:394.
  27. Fleming DR, Rayens MK, Garrison J. Impact of obesity on allogeneic stem cell transplant patients: a matched case-controlled study. Am J Med 1997; 102:265.
  28. Deeg HJ, Seidel K, Sullivan KM. Body weight and outcome of hematopoietic stem cell transplantation. Am J Med 1998; 104:607.
  29. Hansen JA, Gooley TA, Martin PJ, et al. Bone marrow transplants from unrelated donors for patients with chronic myeloid leukemia. N Engl J Med 1998; 338:962.
  30. Nakao M, Chihara D, Niimi A, et al. Impact of being overweight on outcomes of hematopoietic SCT: a meta-analysis. Bone Marrow Transplant 2014; 49:66.
  31. Yee GC, Lennon TP, Gmur DJ, et al. Effect of obesity on cyclosporine disposition. Transplantation 1988; 45:649.
  32. Gratwohl A, Hermans J, Goldman JM, et al. Risk assessment for patients with chronic myeloid leukaemia before allogeneic blood or marrow transplantation. Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Lancet 1998; 352:1087.
  33. Parimon T, Au DH, Martin PJ, Chien JW. A risk score for mortality after allogeneic hematopoietic cell transplantation. Ann Intern Med 2006; 144:407.
  34. Sorror ML, Maris MB, Storb R, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood 2005; 106:2912.
  35. Kataoka K, Nannya Y, Ueda K, et al. Differential prognostic impact of pretransplant comorbidity on transplant outcomes by disease status and time from transplant: a single Japanese transplant centre study. Bone Marrow Transplant 2010; 45:513.
  36. Gooley TA, Chien JW, Pergam SA, et al. Reduced mortality after allogeneic hematopoietic-cell transplantation. N Engl J Med 2010; 363:2091.
  37. Horan JT, Logan BR, Agovi-Johnson MA, et al. Reducing the risk for transplantation-related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made? J Clin Oncol 2011; 29:805.
  38. Barba P, Piñana JL, Martino R, et al. Comparison of two pretransplant predictive models and a flexible HCT-CI using different cut off points to determine low-, intermediate-, and high-risk groups: the flexible HCT-CI Is the best predictor of NRM and OS in a population of patients undergoing allo-RIC. Biol Blood Marrow Transplant 2010; 16:413.
  39. Xhaard A, Porcher R, Chien JW, et al. Impact of comorbidity indexes on non-relapse mortality. Leukemia 2008; 22:2062.
  40. Charlson M, Szatrowski TP, Peterson J, Gold J. Validation of a combined comorbidity index. J Clin Epidemiol 1994; 47:1245.
  41. Wood WA, Le-Rademacher J, Syrjala KL, et al. Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902). Cancer 2016; 122:91.
  42. Sorror M, Storer B, Sandmaier BM, et al. Hematopoietic cell transplantation-comorbidity index and Karnofsky performance status are independent predictors of morbidity and mortality after allogeneic nonmyeloablative hematopoietic cell transplantation. Cancer 2008; 112:1992.
  43. Passweg JR, Walker I, Sobocinski KA, et al. Validation and extension of the EBMT Risk Score for patients with chronic myeloid leukaemia (CML) receiving allogeneic haematopoietic stem cell transplants. Br J Haematol 2004; 125:613.
  44. Shouval R, Bonifazi F, Fein J, et al. Validation of the acute leukemia-EBMT score for prediction of mortality following allogeneic stem cell transplantation in a multi-center GITMO cohort. Am J Hematol 2017; 92:429.
  45. Gratwohl A, Stern M, Brand R, et al. Risk score for outcome after allogeneic hematopoietic stem cell transplantation: a retrospective analysis. Cancer 2009; 115:4715.
  46. Au BK, Gooley TA, Armand P, et al. Reevaluation of the pretransplant assessment of mortality score after allogeneic hematopoietic transplantation. Biol Blood Marrow Transplant 2015; 21:848.
  47. Sorror ML. How I assess comorbidities before hematopoietic cell transplantation. Blood 2013; 121:2854.
  48. Sorror ML, Martin PJ, Storb RF, et al. Pretransplant comorbidities predict severity of acute graft-versus-host disease and subsequent mortality. Blood 2014; 124:287.
  49. Smith AR, Majhail NS, MacMillan ML, et al. Hematopoietic cell transplantation comorbidity index predicts transplantation outcomes in pediatric patients. Blood 2011; 117:2728.
  50. Kerbauy DM, Chyou F, Gooley T, et al. Allogeneic hematopoietic cell transplantation for chronic myelomonocytic leukemia. Biol Blood Marrow Transplant 2005; 11:713.
  51. Sorror ML, Sandmaier BM, Storer BE, et al. Comorbidity and disease status based risk stratification of outcomes among patients with acute myeloid leukemia or myelodysplasia receiving allogeneic hematopoietic cell transplantation. J Clin Oncol 2007; 25:4246.
  52. Raimondi R, Tosetto A, Oneto R, et al. Validation of the Hematopoietic Cell Transplantation-Specific Comorbidity Index: a prospective, multicenter GITMO study. Blood 2012; 120:1327.
  53. Deschler B, Binek K, Ihorst G, et al. Prognostic factor and quality of life analysis in 160 patients aged > or =60 years with hematologic neoplasias treated with allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 2010; 16:967.
  54. Sorror ML, Storb RF, Sandmaier BM, et al. Comorbidity-age index: a clinical measure of biologic age before allogeneic hematopoietic cell transplantation. J Clin Oncol 2014; 32:3249.
  55. Baker KS, Davies SM, Majhail NS, et al. Race and socioeconomic status influence outcomes of unrelated donor hematopoietic cell transplantation. Biol Blood Marrow Transplant 2009; 15:1543.
  56. Mielcarek M, Gooley T, Martin PJ, et al. Effects of race on survival after stem cell transplantation. Biol Blood Marrow Transplant 2005; 11:231.
  57. Majhail NS, Omondi NA, Denzen E, et al. Access to hematopoietic cell transplantation in the United States. Biol Blood Marrow Transplant 2010; 16:1070.
  58. Unequal Treatment: Confronting Racial and Ethnic Disparities in Health Care, National Academies Press, Washington, DC 2003.
  59. Kreklau EL, Pollok KE, Bailey BJ, et al. Hematopoietic expression of O(6)-methylguanine DNA methyltransferase-P140K allows intensive treatment of human glioma xenografts with combination O(6)-benzylguanine and 1,3-bis-(2-chloroethyl)-1-nitrosourea. Mol Cancer Ther 2003; 2:1321.
  60. Mitchell JM, Meehan KR, Kong J, Schulman KA. Access to bone marrow transplantation for leukemia and lymphoma: the role of sociodemographic factors. J Clin Oncol 1997; 15:2644.
  61. Joshua TV, Rizzo JD, Zhang MJ, Horowitz MM. Access to hematopoietic stem cell transplantation: effect of race and gender [abstract]. Biol Blood Marrow Transplant 2007; 13[suppl 1]:22.
  62. Hwang JP, Lam TP, Cohen DS, et al. Hematopoietic stem cell transplantation among patients with leukemia of all ages in Texas. Cancer 2004; 101:2230.
  63. Klingemann HG, Deeg HJ, Self S, et al. Is race a risk factor for allogeneic marrow transplantation? Bone Marrow Transplant 1986; 1:87.
  64. Deeg HJ, Sandmaier BM. Who is fit for allogeneic transplantation? Blood 2010; 116:4762.
  65. Shlomchik WD, Lee SJ, Couriel D, Pavletic SZ. Transplantation's greatest challenges: advances in chronic graft-versus-host disease. Biol Blood Marrow Transplant 2007; 13:2.
  66. Stewart BL, Storer B, Storek J, et al. Duration of immunosuppressive treatment for chronic graft-versus-host disease. Blood 2004; 104:3501.
  67. Storb R, Gyurkocza B, Storer BE, et al. Graft-versus-host disease and graft-versus-tumor effects after allogeneic hematopoietic cell transplantation. J Clin Oncol 2013; 31:1530.
  68. Sun CL, Francisco L, Kawashima T, et al. Prevalence and predictors of chronic health conditions after hematopoietic cell transplantation: a report from the Bone Marrow Transplant Survivor Study. Blood 2010; 116:3129.