Bone marrow & stem cell transplantation

Stem Cell transplant: biology, review articles

  1. Copelan E.  Hematopoietic stem-cell transplantation. NEJM 2006;354:1813
  2. Kolb J. Graft-versus-leukemia effects of transplantation and donor lymphocytes. Blood 2008;112:4371
  3. Khan et al. Is allergic disease curable or transferable with allogeneic hematopoietic cell transplantation? Blood 2009;113:279
  4. Vago et al. Loss of mismatched HLA in leukemia after stem-cell transplantation. NEJM 2009;361:478(Loss of mismatched HLA haplotype by leukemic cells leads to relapse)
  5. Penack et al. The importance of neovascularization and its inhibition for allogeneic hematopoietic stem cell transplantation. Blood 2011;117:4181
  6. Sorror ML. How I assess comorbidities before hematopoietic cell transplantation. Blood 2013;121:2854

Autologous Transplant General

  1. Cottler-Fox et al. Stem Cell Mobilization. Hematology 2003:419-437
  2. Ford et al. Autologous Stem-Cell Transplantation Without Hematopoietic Support for the Treatment of Hematologic Malignancies in Jehovah’s Witnesses. J Clin Oncol 2015;33:1674
  3. Scala et al. Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans. Nat Med 2018;24:1683

Allogenic Transplant General

  1. Gooley et al. Reduced mortality after allogeneic hematopoieic-cell transplantation. NEJM 2010;363:2091
  2. Armand et al. A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood 2012;120:905
  3. Armand et al. Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation. Blood 2014;123:3664
  4. Zaid et al. Plasma biomarkers of risk for death in a multicenter phase 3 trial with uniform transplant characteristics post–allogeneic HCT. Blood 2017;129:162
  5. Puerta-Alcalde et al. How I perform hematopoietic stem cell transplantation on patients with a history of invasive fungal disease. Blood 2020;136:2741
  6. Pidala et al. Quality of life after allogeneic hematopoietic cell transplantation. Blood 2009;114:7
  7. Sailor et al. Hematopoietic stem cell transplantation chemotherapy causes microglia senescence and peripheral macrophage engraftment in the brain. Nat Med 2022;28:517 (With commentary)
  8. Ballen et al. Selection of optimal alternative graft source: mismatched unrelated donor, umbilical cord blood, or haploidentical transplant. Blood 2012;119:1972
  9. Kekre and Antin. 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
  10. Dehn et al. Selection of unrelated donors and cord blood units for hematopoietic cell transplantation: guidelines from the NMDP/CIBMTR. Blood 2019;134:924
  11. Kollman et al. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood 2016;127:260(Donor age and HLA match predict survival)
  12. Girgis et al. Chimerism and clinical outcomes of 110 recipients of unrelated donor bone marrow transplants who underwent conditioning with low-dose, single-exposure total body irradiation and cyclophosphamide. Blood 2005;105:3035
  13. Kolb et al.  Graft-versus-leukemia reactions in allogeneic chimeras. Blood 2004;103:767
  14. Porter DL. Allogeneic Immunotherapy to Optimize the Graft-versus-Tumor Effect: Concepts and Controversies. Hematology 2011:292
  15. Zeiser and Vago. Mechanisms of immune escape after allogeneic hematopoietic cell transplantation. Blood 2019;133:1290
  16. Laughlin et al. Outcomes after Transplantation of Cord Blood or Bone Marrow from Unrelated Donors in Adults with Leukemia. NEJM 2004;351:2265
  17. Rocha et al. Transplants of Umbilical-Cord Blood or Bone Marrow from Unrelated Donors in Adults with Acute Leukemia.  NEJM 2004;351:2276
  18. Mead et al. HLA-mismatched unrelated donors are a viable alternate graft source for allogeneic transplantation following alemtuzumab-based reduced-intensity conditioning. Blood 2010;115:5147
  19. Anasetti et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. NEJM 2012;367:1487(No significant difference in outcomes)
  20. Wang et al Who is the best donor for a related HLA heplotype-mismatched transplant? Blood 2014;124:843(Young males, noninherited maternal antigen mismatched)
  21. Alousi et al. Who is the better donor for older hematopoietic transplant recipients: an older-aged sibling or a young, matched unrelated volunteer? Blood 2013;121:2567(Related donor better to MUD for older transplant recipients)
  22. Lee et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood 2007;110:4576
  23. Zhou et al. Long-term outcome after haploidentical stem cell transplant and infusion of T cells expressing the inducible caspase 9 safety transgene. Blood 2014;123:3895
  24. Sorror et al. Hematopoietic cell transplantation–specific comorbidity index as an outcome predictor for patients with acute myeloid leukemia in first remission: combined FHCRC and MDACC experiences. Blood 2007;110:4606
  25. Wingard et al. Long-term survival and late deaths after allogeneic hematopoietic cell transplantation. J Clin Oncol 2011;29:2230
  26. Trottier et al. Association of iron overload with allogeneic hematopoietic cell transplantation outcomes: a prospective cohort study using R2-MRI–measured liver iron content. Blood 2013;122:1678 (No association between pretransplant iron overload and outcome of alloHSCT)
  27. Bredeson et al. Prospective cohort study comparing intravenous busulfan to total body irradiation in hematopoietic cell transplantation. Blood 2013;122:3871(Survival better with IV busulfan)
  28. Jaspers et al. Erythropoietin therapy after allogeneic hematopoietic cell transplantation: a prospective, randomized trial. Blood 2014;124:3(EPO started one month after HSCT hastens RBC recovery and reduces transfusion requirements)
  29. Chapuy et al. Daratumumab for Delayed Red-Cell Engraftment after Allogeneic Transplantation. NEJM 2018;379:1846
  30. Davids et al. Ipilimumab for Patients with Relapse after Allogeneic Transplantation. NEJM 2016;375:143(23% CR rate, most responses > 1 year)
  31. Smith et al. Posttransplant chimeric antigen receptor therapy. Blood 2018;131:1045
  32. Soiffer et al. Tyrosine kinase inhibitors and immune checkpoint blockade in allogeneic hematopoietic cell transplantation. Blood 2018;131:1073
  33. Romee et al. First-in-human phase 1 clinical study of the IL-15 superagonist complex ALT-803 to treat relapse after transplantation. Blood 2018;131:2515
  34. Hu et al. Sequential CD7 CAR T-Cell Therapy and Allogeneic HSCT without GVHD Prophylaxis. NEJM 2024;390:1467 (With editorial)

Nonablative/Reduced Intensity Transplant

  1. Gyurkocza and Sandmaier. Conditioning regimens for hematopoietic cell transplantation: one size does not fit all. Blood 2014;124:344
  2. Kasamon et al. Outcomes of Nonmyeloablative HLA-Haploidentical Blood or Marrow Transplantation With High-Dose Post-Transplantation Cyclophosphamide in Older Adults. J Clin Oncol 2015;33:3152
  3. Sorror et al. Long-term Outcomes Among Older Patients Following Nonmyeloablative Conditioning and Allogeneic Hematopoietic Cell Transplantation for Advanced Hematologic Malignancies. JAMA 2011;306:1874(5 year OS 35% in patients aged 60-75)
  4. Liu et al. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood 2011;118:6438
  5. Storb et al. Graft-Versus-Host Disease and Graft-Versus-Tumor Effects After Allogeneic Hematopoietic Cell Transplantation. J Clin Oncol 2013;31:1530(A “minimal-intensity” regimen. “Allogeneic HCT relying on GVT effects is feasible and results in cures of an appreciable number of malignancies”)
  6. Devine et al. Phase II Study of Allogeneic Transplantation for Older Patients With Acute Myeloid Leukemia in First Complete Remission Using a Reduced-Intensity Conditioning Regimen: Results From Cancer and Leukemia Group B 100103 (Alliance for Clinical Trials in Oncology)/Blood and Marrow Transplant Clinical Trial Network 0502. J Clin Oncol 2015;33:4167
  7. Fasslrinner et al. Long-term efficacy of reduced-intensity versus myeloablative conditioning before allogeneic haemopoietic cell transplantation in patients with acute myeloid leukaemia in first complete remission: retrospective follow-up of an open-label, randomised phase 3 trial. Lancet Haematol 2018;5:e161(Reduced intensity conditioning less toxic, does not increase relapse risk)
  8. Chang et al. Antithymocyte Globulin for Matched Sibling Donor Transplantation in Patients With Hematologic Malignancies: A Multicenter, Open-Label, Randomized Controlled Study. J Clin Oncol 20-20;38:3367 (Less cGVHD, no increase in relapse rate)

Cord Blood Transplant

  1. Barker J. Umbilical Cord Blood (UCB) Transplantation: An Alternative to the Use of Unrelated Volunteer Donors? Hematology 2007:55
  2. Ballen K. New trends in umbilical cord blood transplantation. Blood 2005;105: 3786.
  3. Scaradavou et al. Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood 2013;121:752
  4. Wagner et al. One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers. NEJM 2014;371:1685(No advantage to two-unit transplantation)
  5. Brunstein et al. Umbilical cord blood transplantation after nonmyeloablative conditioning: impact on transplantation outcomes in 110 adults with hematologic disease. Blood 2007;110:3064
  6. Milano et al. Cord-blood transplantation in patients with minimal residual disease. NEJM 2016;375:944(Outcomes at least as good as with HLA-identical donors, better than those with HLA-mismatched donors)
  7. Cutler et al. Donor-specific anti-HLA antibodies predict outcome in double umbilical cord blood transplantation. Blood 2011;118:6691
  8. Herrera et al. Cord colitis syndrome in cord-blood stem-cell transplantation. NEJM 2011;365:815

Acute GVHD

  1. Jamy et al. Novel developments in the prophylaxis and treatment of acute GVHD. Blood 2023;142:1037
  2. Bolaños-Meade et al. Post-Transplantation Cyclophosphamide-Based Graft-versus-Host Disease Prophylaxis. NEJM 2023;388:2338
  3. Curtis et al. Graft-versus-Host Disease Prophylaxis with Cyclophosphamide and Cyclosporin. NEJM 2025;393:243
  4. Martin PJ. How I treat steroid-refractory acute graft-versus-host disease. Blood 2020;135:1630
  5. Zeiser and Blazar. Acute Graft-versus-Host Disease — Biologic Process, Prevention, and Therapy. NEJM 2017;377:2167
  6. Markey et al. The biology of graft-versus-host disease: experimental systems instructing clinical practice. Blood 2014;124:354
  7. Holtan et al. Acute graft-versus-host disease: a bench-to-bedside update. Blood 2014;124:363
  8. Zeiser and Teshima. Nonclassical manifestations of acute GVHD. Blood 2021;138:2165
  9. Shimabukuro-Vornhagen et al. The role of B cells in the pathogenesis of graft-versus-host disease. Blood 2009;114:4919
  10. Penack et al. Graft-versus-host disease: regulation by microbe-associated molecules and innate immune receptors. Blood 2010; 115:1865
  11. Schwab et al. Neutrophil granulocytes recruited upon translocation of intestinal bacteria enhance graft-versus-host disease via tissue damage. Nat Med 2014;20:648
  12. McDonald G. How I treat acute graft-versus-host disease of the gastrointestinal tract and the liver. Blood 2016;127:1544
  13. Paczesny S. Discovery and validation of graft-versus-host disease biomarkers. Blood 2013;121:585
  14. Harris et al. Plasma biomarkers of lower gastrointestinal and liver acute GVHD. Blood 2012;119:2960
  15. Legoff et al. The eukaryotic gut virome in hematopoietic stem cell transplantation: new clues in enteric graft-versus-host disease. Nat Med 2017;23:1080(Role of picobiravirus in acute GVHD of gut; with editorial)
  16. Vander Lugt et al. ST2 as a marker for risk of therapy-resistant graft-versus-host disease and death. NEJM 2013;369:529
  17. MacMillan et al. Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood 2009;113:2410
  18. Mielcarek et al. Initial therapy of acute graft-versus-host disease with low-dose prednisone does not compromise patient outcomes. Blood 2009;113:2888
  19. Kawase et al. High-risk HLA allele mismatch combinations responsible for severe acute graft-versus-host disease and implication for its molecular mechanism. Blood 2007;110:2235
  20. Saliba et al. Hyperacute GVHD: risk factors, outcomes, and clinical implications. Blood 2007;109:2751
  21. Kean et al. Abatacept for acute graft-versus-host disease prophylaxis after unrelated donor hematopoietic cell transplantation. Blood 2024;144:1834
  22. Luznik et al. High-dose cyclophosphamide as single-agent, short-course prophylaxis of graft-versus-host disease. Blood 2010;115:3224 (Lower than expected incidence of both acute and chronic GVHD in this uncontrolled study)
  23. Rotta et al. Donor statin treatment protects against severe acute graft-versus-host disease after related allogeneic hematopoietic cell transplantation. Blood 2010;115:1288
  24. Reshef et al. Blockade of lymphocyte chemotaxis in visceral graft-versus-host disease. NEJM 2012;367:135
  25. Bolaños-Meade et al. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood 2014;124:3221(No benefit from adding MMF)
  26. Battipaglia et al. Posttransplant cyclophosphamide vs antithymocyte globulin in HLA-mismatched unrelated donor transplantation. Blood 2019;134:892 (PTCY more effective in preventing acute GVHD, produced better survival in 9/10 MMUD transplants for AML)
  27. Pidala et al. Randomized multicenter trial of sirolimus vs prednisone as initial therapy for standard-risk acute GVHD: the BMT CTN 1501 trial. Blood 2020;135:97(Sirolimus as effective, less toxic)
  28. Zeiser et al. Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease. NEJM 2020;382:1800(With editorial)
  29. Jagasia et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial. Blood 2020;135:1739
  30. Abboud et al. Itacitinib for prevention of graft-versus-host disease and cytokine release syndrome in haploidentical transplantation. Blood 2025;145:1382
  31. Kornblit et al. Sirolimus with CSP and MMF as GVHD prophylaxis for allogeneic transplantation with HLA antigen–mismatched donors. Blood 2020;136:1499
  32. Farag et al. Dipeptidyl Peptidase 4 Inhibition for Prophylaxis of Acute Graft-versus-Host Disease. NEJM 2021;384:11(With editorial)
  33. Magenau et al. A phase 2 trial of CD24Fc for prevention of graft-versus-host disease.  Blood 2024;143:21
  34. Meyer et al. Donor regulatory T-cell therapy to prevent graft-versus-host disease. Blood 2025;145:2012

Chronic GVHD

  1. Lee and Zeiser. FDA-approved therapies for chronic GVHD. Blood 2025;145:795
  2. Pidala et al.Chronic graft-versus-host disease: unresolved complication or ancient history?  Blood 2024;144:1363
  3. MacDonald et al. Chronic graft-versus-host disease: biological insights from preclinical and clinical studies. Blood 2017;129:13
  4. Zeiser and Blazar. Pathophysiology of Chronic Graft-versus-Host Disease and Therapeutic Targets, NEJM 2017;377:2565
  5. Koyama and Hill. Alloantigen presentation and graft-versus-host disease: fuel for the fire. Blood 2016;127:2963
  6. Lee SJ. Classification systems for chronc graft-versus-host disease. Blood 2017;129:30
  7. Cutler et al. Mechanistic approaches for the prevention and treatment of chronic GVHD. Blood 2017;129:22
  8. Flowers and Martin. How we treat chronic graft-versus-host disease. Blood 2015;125:606
  9. Sarantopoulos et al. How I treat refractory chronic graft-versus-host disease. Blood 2019;133:1191
  10. Carpenter PA. How I conduct a comprehensive chronic graft-versus-host disease assessment. Blood 2011;118:2679
  11. Flowers et al. Comparative analysis of risk factors for acute graft-versus-host disease and for chronic graft-versus-host disease according to National Institutes of Health consensus criteria. Blood 2011;117:3214
  12. Stein-Thoeringer et al. Lactose drives Enterococcus expansion to promote graft-versus-host disease. Science 2019;366:1143(With editorial)
  13. Arora et al. Chronic GVHD risk score: a Center for International Blood and Marrow Transplant Research analysis. Blood 2011;117:6714.
  14. Jacobsohn et al. Correlation between NIH composite skin score, patient-reported skin score, and outcome: results from the Chronic GVHD Consortium. Blood 2012;120:2545
  15. Martires et al. Sclerotic-type chronic GVHD of the skin: clinical risk factors, laboratory markers, and burden of disease. Blood 2011;118:4250
  16. Palmer et al. Predictors of survival, nonrelapse mortality, and failure-free survival in patients treated for chronic graft-versus-host disease. Blood 2016;127:160
  17. Treister et al. How we treat oral chronic graft-versus-host disease. Blood 2012;120:3407
  18. Kröger et al. Antilymphocyte Globulin for Prevention of Chronic Graft-versus-Host Disease. NEJM 2016;374:43(50% reduction in CGVHD rate with ATG)
  19. Finke et al. Long-term outcomes after standard graft-versus-host disease prophylaxis with or without anti-human-T-lymphocyte immunoglobulin in haemopoietic cell transplantation from matched unrelated donors: final results of a randomised controlled trial. Lancet Haematol 2017;4:e293(ATG treatment reduced CGVHD incidence from 53% to 14%, improved OS)
  20. Inamoto et al. Assessment of Joint and Fascia Manifestations in Chronic Graft-Versus-Host Disease. Arth Rheum 2014;66:1044
  21. Shimabukuro-Vornhagen et al. The role of B cells in the pathogenesis of graft-versus-host disease. Blood 2009;114:4919
  22. Lee SJ. New approaches for preventing and treating chronic graft-versus-host disease. Blood 2005;105:4200
  23. Martin et al. Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood 2009;113:5074(MMF ineffective)
  24. Przepiorka, D, Anderlini, P, Saliba, R, Cleary, K, et al. Chronic graft-versus-host disease after allogeneic blood stem cell transplantation. Blood 2001; 98: 1695-1700.
  25. Stewart et al. Duration of immunosuppressive treatment for chronic graft-versus-host disease.  Blood 2004;104:3501
  26. Koreth et al. Interleukin-2 and regulatory T cells in graft-versus-host disease. NEJM 2011;365:2055(Low-dose IL-2 an effective steroid-sparing therapy in CGVHD)
  27. Flowers et al. A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease. Blood 2008;112:2667
  28. Inamoto et al. Incidence, risk factors, and outcomes of sclerosis in patients with chronic grat-versus-host disease. Blood 2013;121:5098
  29. Olivieri et al. Imatinib for refractory chronic graft-versus-host disease with fibrotic features. Blood 2009;114:709
  30. Marsh et al. Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia. Blood 2011;118:2351
  31. Di Stasi et al. Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy. NEJM 2011;365:1673(“Suicide gene” insertion into donor T cells allows switching off GVHD)
  32. Rachakonda et al. Single-Nucleotide Polymorphisms Within the Thrombomodulin Gene (THBD) Predict Mortality in Patients With Graft-Versus-Host Disease. J Clin Oncol 2014;32:3421
  33. Wall et al. Complement-mediated thrombotic microangiopathy as a link between endothelial damage and steroid-refractory GVHD. Blood Adv 2018;2:2619
  34. Miklos et al. Ibrutinib for chronic graft-versus-host disease after failure of prior therapy. Blood 2017;130:2243
  35. Zeiser et al. Ruxolitinib for Glucocorticoid-Refractory Chronic Graft-versus-Host Disease. NEJM 2021;385:228
  36. Koshy et al. Phase 2 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood 2023;141:2932
  37. Wolff et al. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. NEJM 2024;391:1002

GVHD and solid organ transplant

  1. Cooper and Abkowitz. How I diagnose and treat acute graft-versus-host disease after solid organ transplantation. Blood 2023;141:1138

Acute Myeloid Leukemia

  1. Cornelissen and Blaise. Hematopoietic stem cell transplantation for patients with AML in first complete remission. Blood 2016;127:62
  2. Koreth et al. Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia in First Complete Remission. JAMA 2009;301:2349(Benefits high- and intermediate-risk but not good-risk patients)
  3. Gupta et al. Allogeneic hematopoietic cell transplantation for adults with acute myeloid leukemia: myths, controversies, and unknowns. Blood 2011;117:2307
  4. Stone RM. Acute myeloid leukemia in first remission: to choose transplantation or not? J Clin Oncol 2013;31:1262
  5. Kurosawa et al. A Markov decision analysis of allogeneic hematopoietic cell transplantation versus chemotherapy in patients with acute myeloid leukemia in first remission. Blood 2011;117:2113(“Older [age 50-70] patients with a related donor and younger patients with unfavorable cytogenetics benefited the most from allo-HCT in CR1”)
  6. Stelljes et al. Allogeneic Transplantation Versus Chemotherapy as Postremission Therapy for Acute Myeloid Leukemia: A Prospective Matched Pairs Analysis. J Clin Oncol 2014;32:288(AlloSCT produces best long-term outcomes in patients < 60 yrs)
  7. Araki et al. Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia: Time to Move Toward a Minimal Residual Disease–Based Definition of Complete Remission? J Clin Oncol 2016;34:329(MRD-positive patients in morphologic remission had similar outcomes to patients with active disease at the time of transplant)
  8. Burnett et al. Curability of Patients With Acute Myeloid Leukemia Who Did Not Undergo Transplantation in First Remission. J Clin Oncol 2013;31:1293(Overall survival for intermediate-risk patients not decreased by delaying transplant until after relapse; with editorial)
  9. Lioure et al. Early matched sibling hematopoietic cell transplantation for adult AML in first remission using an age-adapted strategy: long-term results of a prospective GOELAMS study. Blood 2012;119:2943
  10. Saber et al. Outcomes after matched unrelated donor versus identical sibling hematopoietic cell transplantation in adults with acute myelogenous leukemia. Blood 2012;119:3908(Similar survival times)
  11. Gupta et al. Comparable survival after HLA-well-matched unrelated or matched sibling donor transplantation for acute myeloid leukemia in first remission with unfavorable cytogenetics at diagnosis. Blood 2010;116:1839
  12. Cornelissen et al. Results of a HOVON/SAKK donor versus no-donor analysis of myeloablative HLA-identical sibling stem cell transplantation in first remission acute myeloid leukemia in young and middle-aged adults: benefits for whom? Blood 2007;109:3658(Allotransplant benefits AML patients without favorable cytogenetics)
  13. Huang et al. The superiority of haploidentical related stem cell transplantation over chemotherapy alone as postremission treatment for patients with intermediate- or high-risk acute myeloid leukemia in first complete remission. Blood 2012;119:5584
  14. Wang et al. Haploidentical vs identical-sibling transplant for AML in remission: a multicenter, prospective study. Blood 2015;125:3956(Similar outcomes with haplo- vs fully-identical sibling transplant)
  15. Copelan et al. Better leukemia-free and overall survival in AML in first remission following cyclophosphamide in combination with busulfan compared with TBI. Blood 2013;122:3863
  16. Hourigan et al. Impact of Conditioning Intensity of Allogeneic Transplantation for Acute Myeloid Leukemia With Genomic Evidence of Residual Disease. J Clin Oncol 2020;38:1273 (6-fold higher relapse rate with reduced intensity conditioning)
  17. Fang et al. Outcome of patients with acute myeloid leukemia with monosomal karyotype who undergo hematopoietic cell transplantation. Blood 2011;118:1490(HCT improves outcomes in these patients, but those >60 still have very poor survival)
  18. Middeke et al. Outcome of high-risk acute myeloid leukemia after allogeneic hematopoietic cell transplantation: negative impact of abnl(17p) and −5/5q−. Blood 2012;120:2521
  19. Yoshizato et al. Genetic abnormalities in myelodysplasia and secondary acute myeloid leukemia: impact on outcome of stem cell transplantation. Blood 2017;129:2347(TP53 mutation plus complex karyotype, or RAS-pathway mutations in MDS, predicted poor outcome post-transplant)
  20. Röllig et al. Allogeneic Stem-Cell Transplantation in Patients With NPM1-Mutated Acute Myeloid Leukemia: Results From a Prospective Donor Versus No-Donor Analysis of Patients After Upfront HLA Typing Within the SAL-AML 2003 Trial. J Clin Oncol 2015;33:403(Allo-SCT prolonged survival in NPM1-mutated AML)
  21. Schmid et al. Treatment, risk factors, and outcome of adults with relapsed AML after reduced intensity conditioning for allogeneic stem cell transplantation. Blood 2012;119:1599(Re-induction followed by DLI or 2nd transplant most successful strategy)
  22. Venstrom et al. HLA-C–Dependent Prevention of Leukemia Relapse by Donor Activating KIR2DS1. NEJM 2012;367:805(With editorial)
  23. Cooley et al. Donor selection for natural killer cell receptor genes leads to superior survival after unrelated transplantation for acute myelogenous leukemia. Blood 2010;116:2411
  24. Christopher et al. Immune Escape of Relapsed AML Cells after Allogeneic Transplantation. NEJM 2018;379:2330(Down-regulation of MHC class II genes contributes to immune escape)
  25. Spyridonidis A. How I treat measurable (minimal) residual disease in acute leukemia after allogeneic hematopoietic cell transplantation. Blood 2020;135:1639
  26. Levis et al. Measurable residual disease and posttransplantation gilteritinib maintenance for patients with FLT3-ITD–mutated AML. Blood 2025;145:2138
  27. Schlenk et al. The value of allogeneic and autologous hematopoietic stem cell transplantation in prognostically favorable acute myeloid leukemia with double mutant CEBPA. Blood 2013;122:1576(alloHSCT in 1st remission improved relapse free, but not overall, survival)
  28. Suciu et al.  Allogeneic compared with autologous stem cell transplantation in the treatment of patients younger than 46 years with acute myeloid leukemia (AML) in first complete remission (CR1): an intention-to-treat analysis of the EORTC/GIMEMAAML-10 trial.  Blood 2003;102:1232
  29. Fernandez et al. Autologous transplantation gives encouraging results for young adults with favorable-risk acute myeloid leukemia, but is not improved with gemtuzumab ozogamicin. Blood 2011;117:5306
  30. Vellenga et al. Autologous peripheral blood stem cell transplantation for acute myeloid leukemia. Blood 2011;118:6037
  31. Sorror et al. An 8-year pragmatic observation evaluation of the benefits of allogeneic HCT in older and medically infirm patients with AML. Blood 2023;141:295 (Suggests transplantation does not benefit these patients)

Acute Lymphoblastic Leukemia

  1. Goldstone and Rowe. Transplantation in adult ALL. Hematology 2009;593
  2. Goldstone et al. In adults with standard-risk acute lymphoblastic leukemia, the greatest benefit is achieved from a matched sibling allogeneic transplantation in first complete remission, and an autologous transplantation is less effective than conventional consolidation/maintenance chemotherapy in all patients: final results of the International ALL Trial (MRC UKALL XII/ECOG E2993). Blood 2008;111:1827
  3. Hunault et al. Better outcome of adult acute lymphoblastic leukemia after early genoidentical allogeneic bone marrow transplantation (BMT) than after late high-dose therapy and autologous BMT: a GOELAMS trial. Blood 2004;104:3028
  4. Annino et al. Treatment of adult acute lymphoblastic leukemia (ALL): long-term follow-up of the GIMEMA ALL 0288 randomized study. Blood 2002;99:863
  5. Tomblyn et al. Myeloablative Hematopoietic Cell Transplantation for Acute Lymphoblastic Leukemia: Analysis of Graft Sources and Long-Term Outcome. J Clin Oncol 2009;27:3634(Allotransplant but not autotransplant results in durable disease-free survival. Cord blood appears as good as related donor stem cells as graft source)
  6. Bachanova et al. Allogeneic hematopoietic stem cell transplantation overcomes the adverse prognostic impact of CD20 expression in acute lymphoblastic leukemia. Blood 2011;117:5261
  7. Marks et al. The outcome of full-intensity and reduced-intensity conditioning matched sibling or unrelated donor transplantation in adults with Philadelphia chromosome–negative acute lymphoblastic leukemia in first and second complete remission. Blood 2010;116:366
  8. Mohty et al. Reduced-intensity versus conventional myeloablative conditioning allogeneic stem cell transplantation for patients with acute lymphoblastic leukemia: a retrospective study from the European Group for Blood and Marrow Transplantation. Blood 2010;116:4439
  9. Fieldin et al. Prospective outcome data on 267 unselected adult patients with Philadelphia chromosome–positive acute lymphoblastic leukemia confirms superiority of allogeneic transplantation over chemotherapy in the pre-imatinib era: results from the International ALL Trial MRC UKALLXII/ECOG2993. Blood 2009;113:4489(“modest but significant” benefit from allotransplant)
  10. Wassmann et al. Early molecular response to posttransplantation imatinib determines outcome in MRD+ Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL). Blood 2005;106:458
  11. Mizuta et al. Pretransplant administration of imatinib for allo-HSCT in patients with BCR-ABL–positive acute lymphoblastic leukemia. Blood 2014;123:2325
  12. Ghobadi et al. The role of allogeneic transplant for adult Ph+ ALL in CR1 with complete molecular remission: a retrospective analysis. Blood 2022;140:2101 (Does not improve survival)
  13. Atsuta et al. Disease-specific analyses of unrelated cord blood transplantation compared with unrelated bone marrow transplantation in adult patients with acute leukemia. Blood 2009;113:1631
  14. Bachanova et al. Prolonged survival in adults with acute lymphoblastic leukemia after reduced-intensity conditioning with cord blood or sibling donor transplantation. Blood 2009;113:2902
  15. Dhédin et al. Role of allogeneic stem cell transplantation in adult patients with Ph-negative acute lymphoblastic leukemia. Blood 2015;125:2486(SCT in CR1 gives 70% 3 year OS in high-risk adult ALL; poor early MRD response to chemo predicts benefit from SCT)
  16. Spyridonidis A. How I treat measurable (minimal) residual disease in acute leukemia after allogeneic hematopoietic cell transplantation. Blood 2020;135:1639

Chronic myelogenous leukemia

  1. Barrett and Ito. The role of stem cell transplantation for chronic myelogenous leukemia in the 21st century. Blood 2015;125:3230
  2. Pavlu et al. Optimizing patient selection for myeloablative allogeneic hematopoietic cell transplantation in chronic myeloid leukemia in chronic phase. Blood 2010;115:4018
  3. Apperley JF. Managing the Patient with Chronic Myeloid Leukemia Through and After Allogeneic Stem Cell Transplantation. Hematology 2006;226-32
  4. Lee et al. Impact of prior imatinib mesylate on the outcome of hematopoietic cell transplantation for chronic myeloid leukemia. Blood 2008;112:3500(No difference in outcome between imatinib-treated patients and others)
  5. Saussele et al. Allogeneic hematopoietic stem cell transplantation (allo SCT) for chronic myeloid leukemia in the imatinib era: evaluation of its impact within a subgroup of the randomized German CML Study IV. Blood 2010;115:1880 (Allotransplant may be preferred second line treatment following imatinib failure)
  6. Jabbour et al. Results of allogeneic hematopoietic stem cell transplantation for chronic myelogenous leukemia patients who failed tyrosine kinase inhibitors after developing BCR-ABL1 kinase domain mutations. Blood 2011;117:3641(Mutations associated with worse outcomes after HSCT)
  7. Warlick et al. Reduced intensity conditioning is superior to nonmyeloablative conditioning for older chronic myelogenous leukemia patients undergoing hematopoietic cell transplant during the tyrosine kinase inhibitor era. Blood 2012;119:4083

Myeloproliferative and myelodysplastic syndromes

  1. Kröger et al. How I treat transplant-eligible patients with myelofibrosis. Blood 2023;142:1683
  2. Mina et al. How I reduce and treat posttransplant relapse of MDS. Blood 2024;143:1344
  3. Gurnari et al. Clinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMT. Blood 2024;145:1987
  4. de Witte et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood 2017;129:1753
  5. Onida et al. Management of adult patients with CMML undergoing allo-HCT: recommendations from the EBMT PH&G Committee. Blood 2024;143:2227
  6. Robin et al. Role of allogeneic transplantation in chronic myelomonocytic leukemia: an international collaborative analysis. Blood 2022;140:1408
  7. Kröger et al. Monitoring of the JAK2-V617F mutation by highly sensitive quantitative real-time PCR after allogeneic stem cell transplantation in patients with myelofibrosis. Blood 2007;109:1316
  8. Gagelmann et al. Clearance of Driver Mutations after Transplantation for Myelofibrosis. NEJM 2025;392:150 (Mutation clearance at day 30 an independent predictor of response)
  9. Kröger et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood 2009;114:5264(5 year OS 67%)
  10. Takagi et al. Successful engraftment after reduced-intensity umbilical cord blood transplantation for myelofibrosis. Blood 2010;116:649
  11. Della Porta et al. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood 2014;123:2333
  12. Nakamura et al. Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome. J Clin Oncol 2021;39:3328 (HCST improved overall survival)
  13. Damaj et al. Impact of Azacitidine Before Allogeneic Stem-Cell Transplantation for Myelodysplastic Syndromes: A Study by the Société Française de Greffe de Moelle et de Thérapie-Cellulaire and the Groupe-Francophone des Myélodysplasies. J Clin Oncol 2012;30:4533(Outcomes similar with azacitidine vs standard induction chemotherapy)
  14. Deeg et al. Five-group cytogenetic risk classification, monosomal karyotype, and outcome after hematopoietic cell transplantation for MDS or acute leukemia evolving from MDS. Blood 2012;120:1398
  15. Duncavage et al. Mutation Clearance after Transplantation for Myelodysplastic Syndrome. NEJM 2018;379:1028
  16. Della Porta et al. Clinical Effects of Driver Somatic Mutations on the Outcomes of Patients With Myelodysplastic Syndromes Treated With Allogeneic Hematopoietic Stem-Cell Transplantation. J Clin Oncol 2016;34:3627(ASXL1, RUNX1, TP53 mutations associated with higher relapse risk, decreased survival)
  17. Myllymäki et al. Short telomere length predicts nonrelapse mortality after stem cell transplantation for myelodysplastic syndrome. Blood 2020;136:3070
  18. Reilly et al. The clinical and functional effects of TERT variants in myelodysplastic syndrome. Blood 2021;140:898

Non-Hodgkins Lymphoma

  1. Kuruvilla J. The role of autologous and allogeneic stem cell transplantation in the management of indolent B-cell lymphoma. Blood 2016;127:2093
  2. van Besien et al. Comparison of autologous and allogeneic hematopoietic stem cell transplantation for follicular lymphoma. Blood 2003;102:3521-9
  3. Fenske et al. Autologous or Reduced-Intensity Conditioning Allogeneic Hematopoietic Cell Transplantation for Chemotherapy-Sensitive Mantle-Cell Lymphoma: Analysis of Transplantation Timing and Modality. J Clin Oncol 2014;32:273(Auto HCT in first remission optimal)
  4. Villa et al. Autologous and Allogeneic Stem-Cell Transplantation for Transformed Follicular Lymphoma: A Report of the Canadian Blood and Marrow Transplant Group. J Clin Oncol 2013;31:1164
  5. van Biesen K. Allogeneic stem cell transplantation in follicular lymphoma: recent progress and controversy. Hematology 2009;610
  6. Bacher et al. Conditioning regimens for allotransplants for diffuse large B-cell lymphoma: myeloablative or reduced intensity? Blood 2012;120:4256(Similar 5-year OS either way)
  7. Thompson et al. Favorable Long-Term Survival After Reduced-Intensity Allogeneic Transplantation for Multiple-Relapse Aggressive Non-Hodgkin’s Lymphoma. J Clin Oncol 2009;27:426
  8. Ghosh et al. Reduced-Intensity Transplantation for Lymphomas Using Haploidentical Related Donors Versus HLA-Matched Sibling Donors: A Center for International Blood and Marrow Transplant Research Analysis. J Clin Oncol 2016;34:3141(Survival similar to transplants using matched sibling donors, less GVHD)
  9. Lambert et al. Prognostic role of PET scanning before and after reduced-intensity allogeneic stem cell transplantation for lymphoma. Blood 2010;115:2763
  10. Kyriakou et al. Allogeneic Stem Cell Transplantation Is Able to Induce Long-Term Remissions in Angioimmunoblastic T-Cell Lymphoma: A Retrospective Study From the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol 2009;27:3951
  11. Kanate et al. Reduced-intensity transplantation for lymphomas using haploidentical related donors vs HLA-matched unrelated donors. Blood 2016;127;938(Less severe GVHD with haploidentical transplants)
  12. van Kampen et al. Allogeneic Stem-Cell Transplantation As Salvage Therapy for Patients With Diffuse Large B-Cell Non-Hodgkin’s Lymphoma Relapsing After an Autologous Stem-Cell Transplantation: An Analysis of the European Group for Blood and Marrow Transplantation Registry. J Clin Oncol 2011;29:1342
  13. Khouri et al. Eight-year experience with allogeneic stem cell transplantation for relapsed follicular lymphoma after nonmyeloablative conditioning with fludarabine, cyclophosphamide, and rituximab. Blood 2008;111:5530
  14. Khouri et al. Nonmyeloablative allogeneic transplantation with or without yttrium ibritumomab tiuxetan is potentially curative for relapsed follicular lymphoma: 12-year results. Blood 2012;119:6373
  15. Milpied et al.  Initial Treatment of Aggressive Lymphoma with High-Dose Chemotherapy and Autologous Stem-Cell Support.  NEJM 2004;350:1287
  16. Stiff et al. Autologous transplantation as consolidation for aggressive non-Hodgkin’s lymhoma. NEJM 2013;369:1681.(Improved PFS but not OS vs chemo alone; with editorial)
  17. Sebban et al. Standard chemotherapy with interferon compared with CHOP followed by high-dose therapy with autologous stem cell transplantation in untreated patients with advanced follicular lymphoma: the GELF-94 randomized study from the Groupe d’Etude des Lymphomes de l’Adulte (GELA). Blood 2006;108:2540 (No advantage to upfront ASCT)
  18. Myeloablative Therapy With Autologous Bone Marrow Transplantation for Follicular Lymphoma at the Time of Second or Subsequent Remission: Long-Term Follow-Up. J Clin Oncol 2007;25: 2554 (Long CR in many pts, some cured?)
  19. Metayer, C, Curtis, RE, Vose, J, Sobocinski, KA, et al. Myelodysplastic syndrome and acute myeloid leukemia after autotransplantation for lymphoma: a multicenter case-control study. Blood 2003; 101: 2015-2023.
  20. Vandenberghe, E, Ruiz de Elvira, C, Loberiza, FR, Conde, E, et al. Outcome of autologous transplantation for mantle cell lymphoma: a study by the European Blood and Bone Marrow Transplant and Autologous Blood and Marrow Transplant Registries. Br J Haematol 2003; 120: 793-800.
  21. Gerson et al. Survival Outcomes of Younger Patients With Mantle Cell Lymphoma Treated in the Rituximab Era. J Clin Oncol 2019;37:471(Autologous SCT prolongs PFS but not OS)
  22. Visani et al. BeEAM (bendamustine, etoposide, cytarabine, melphalan) before autologous stem cell transplantation is safe and effective for resistant/relapsed lymphoma patients. Blood 2011;118:3419
  23. Gilsselbrecht et al. Rituximab Maintenance Therapy After Autologous Stem-Cell Transplantation in Patients With Relapsed CD20+ Diffuse Large B-Cell Lymphoma: Final Analysis of the Collaborative Trial in Relapsed Aggressive Lymphoma. J Clin Oncol 2012;30:4462(No apparent benefit from maintenance rituximab)
  24. Gyan et al. High-dose therapy followed by autologous purged stem cell transplantation and doxorubicin-based chemotherapy in patients with advanced follicular lymphoma: a randomized multicenter study by the GOELAMS with final results after a median follow-up of 9 years. Blood 2009;113:995 (OS no better, more secondary malignancies after transplant than in chemo-only group)
  25. Schmitz et al. Allogeneic hematopoietic stem cell transplantation for T-cell lymphomas. Blood 2018;132:245
  26. Reimer et al. Autologous Stem-Cell Transplantation As First-Line Therapy in Peripheral T-Cell Lymphomas: Results of a Prospective Multicenter Study. J Clin Oncol 2009;27:106
  27. d’Amore et al. Up-front autologous stem-cell transplantation in peripheral T-cell lymphoma: NLG-T-01. J Clin Oncol 2012;30:3093(5-year PFS 44%)
  28. de Masson et al. Allogeneic transplantation in advanced cutaneous T-cell lymphomas (CUTALLO): a propensity score matched controlled prospective study. Lancet 2023;401:1941
  29. Schmitz et al. A randomized phase 3 trial of autologous vs allogeneic transplantation as part of first-line therapy in poor-risk peripheral T-NHL. Blood 2021;137:2646(No difference if 3-year OS; GVL benefit offset by TRM in allo patients)
  30. Deconinck et al.  High-dose therapy followed by autologous purged stem-cell transplantation and doxorubicin-based chemotherapy in patients with advanced follicular lymphoma: a randomized multicenter study by GOELAMS. Blood 2005;105:3817 (No survival advantage to ASCT vs standard chemotherapy)
  31. Soussain et al. Intensive Chemotherapy Followed by Hematopoietic Stem-Cell Rescue for Refractory and Recurrent Primary CNS and Intraocular Lymphoma: Société Française de Greffe de Moëlle Osseuse-Thérapie Cellulaire. J Clin Oncol 2008;26:2512
  32. Krishnan et al. Durable remissions with autologous stem cell transplantation for high-risk HIV-associated lymphomas. Blood 2005;105:874
  33. Re et al. High-dose therapy and autologous peripheral blood stem cell transplantation as salvage treatment for AIDS-related lymphoma: long-term results of the Italian Cooperative Group on AIDS and Tumors (GICAT) study with analysis of prognostic factors. Blood 2009; 114:1306
  34. Alvarnas et al. Autologous hematopoietic cell transplantation for HIV-related lymphoma: results of the BMT CTN 0803/AMC 071 trial. Blood 2016;128:1050(2-yr PFS about 80%, 5% one year treatment related mortality)
  35. Petrich et al. Impact of induction regimen and stem cell transplantation on outcomes in double-hit lymphoma: a multicenter retrospective analysis. Blood 2914;124:2354
  36. Kuruvilla et al. Salvage chemotherapy and autologous stem cell transplantation for transformed indolent lymphoma: a subset analysis of NCIC CTG LY12. Blood 2015;126:733(Similar outcomes with ASCT vs salvage chemotherapy)
  37. Ferreri and Illerhaus. The role of autologous stem cell transplantation in primary central nervous system lymphoma. Blood 2016;127:1642
  38. Wang et al. Phase 1 studies of central memory–derived CD19 CAR T–cell therapy following autologous HSCT in patients with B-cell NHL. Blood 2016;127:2980

Hodgkins Disease

  1. Shah and Moskowitz. Transplant strategies in relapsed/refractory Hodgkin lymphoma. Blood 2018;131:1689
  2. Moskowitz et al. Five-year PFS from the AETHERA trial of brentuximab vedotin for Hodgkin lymphoma at high risk of progression or relapse. Blood 2018;132:2639
  3. Lavoie et al. High-dose chemotherapy and autologous stem cell transplantation for primary refractory or relapsed Hodgkin lymphoma: long-term outcome in the first 100 patients treated in Vancouver. Blood 2005;106:1473
  4. Sarina et al. Allogeneic transplantation improves the overall and progression-free survival of Hodgkin lymphoma patients relapsing after autologous transplantation: a retrospective study based on the time of HLA typing and donor availability. Blood 2010;115:3671
  5. Alinari and Blum. How I treat relapsed classical Hodgkin lymphoma after autologous stem cell transplant. Blood 2016;127:287
  6. Herbaux et al. Efficacy and tolerability of nivolumab after allogeneic transplantation for relapsed Hodgkin lymphoma. Blood 2017;129:2471(Treatment considered effective with acceptable safety; 30% got GVHD)
  7. Armand et al. Nivolumab for Relapsed/Refractory Classic Hodgkin Lymphoma After Failure of Autologous Hematopoietic Cell Transplantation: Extended Follow-Up of the Multicohort Single-Arm Phase II CheckMate 205 Trial. J Clin Oncol 2018;36:1428
  8. Herbaux et al. Recommendations for managing PD-1 blockade in the context of allogeneic HCT in Hodgkin lymphoma: taming a necessary evil. Blood 2018;132:9
  9. Armand et al. PD-1 blockade with pembrolizumab for classical Hodgkin lymphoma after autologous stem cell transplantation. Blood 2019;134:22

Multiple myeloma

  1. van Rhee et al The future of autologous stem cell transplantation in myeloma. Blood 2014;124:328
  2. Kumar et al. Pros and cons of frontline autologous transplant in multiple myeloma: the debate over timing. Blood 2019;133:652
  3. Gertz and Dingli. How we manage autologous stem cell transplantation for patients with multiple myeloma. Blood 2014;124:882
  4. Palumbo et al. Autologous Transplantation and Maintenance Therapy in Multiple Myeloma. NEJM 2014;371:895(HSCT significantly prolonged PFS and OS; lenalidomide maintenance prolonged PFS but not OS)
  5. Gertz et al. Safety Outcomes for Autologous Stem Cell Transplant in Multiple Myeloma. Mayo Clin Proc 2018;93: 56(100 day all-cause mortality 0.3%)
  6. Martinez-Lopez et al. Long-term prognostic significance of response in multiple myeloma after stem cell transplantation. Blood 2011;118:529(Plateau in survival curve after 11 yrs; up to 35% of patients who achieve CR after autologous SCT may be cured)
  7. de Tute et al. Minimal Residual Disease After Autologous Stem-Cell Transplant for Patients With Myeloma: Prognostic Significance and the Impact of Lenalidomide Maintenance and Molecular Risk. J Clin Oncol 2022;40:2889 (“MRD status at both ASCT + 3 and ASCT + 9 is a powerful predictor of PFS and OS”)
  8. Costa et al. Daratumumab, Carfilzomib, Lenalidomide, and Dexamethasone With Minimal Residual Disease Response-Adapted Therapy in Newly Diagnosed Multiple Myeloma. J Clin Oncol 2022;140:2901 (Safe to stop chemo after ASCT if MRD negative on two consecutive tests)
  9. Attal, M, Harousseau, JL, Stoppa, AM, Sotto, JJ, et al. A prospective, randomized trial of autologous bone marrow transplantation and chemotherapy in multiple myeloma. Intergroupe Francais du Myelome.. N Engl J Med 1996; 335: 91-97
  10. Lenhoff et al. Impact on survival of high-dose therapy with autologous stem cell support in patients younger than 60 years with newly diagnosed multiple myeloma: a population-based study. Blood 2000;95:7
  11. Child, JA, Morgan, GJ, Davies, FE, Owen, RG, et al. High-dose chemotherapy with hematopoietic stem-cell rescue for multiple myeloma. N Engl J Med 2003; 348: 1875-1883.
  12. Attal et al.  Single versus Double Autologous Stem-Cell Transplantation for Multiple Myeloma.  NEJM 2003;349:2495
  13. Roussel et al. Bortezomib and high-dose melphalan as conditioning regimen before autologous stem cell transplantation in patients with de novo multiple myeloma: a phase 2 study of the Intergroupe Francophone du Myélome (IFM). Blood 2010;115:32
  14. Roussel et al. Bortezomib and high-dose melphalan conditioning regimen in frontline multiple myeloma: an IFM randomized phase 3 study.Blood 2022;139:2747
  15. Palumbo et al. Melphalan 200 mg/m2 versus melphalan 100 mg/m2 in newly diagnosed myeloma patients: a prospective, multicenter phase 3 study. Blood 2010; 115: 1873(Higher dose melphalan gave superior results)
  16. Attal et al. Lenalidomide, Bortezomib, and Dexamethasone with Transplantation for Myeloma. NEJM 2017;376:1311(RVD plus SCT superior to RVD alone; with editorial)
  17. Lokhorst et al. A randomized phase 3 study on the effect of thalidomide combined with adriamycin, dexamethasone, and high-dose melphalan, followed by thalidomide maintenance in patients with multiple myeloma. Blood 2010;115:1113 (Addition of thalidomide to induction therapy improved response rates and event-free survival)
  18. McCarthy et al. Lenalidomide after stem-cell transplantation for multiple myeloma. NEJM 2012;366:1770(More toxicity, more 2nd cancers, better overall survival with lenalidomide)
  19. Attal et al. Lenalidomide maintanance after stem-cell transplantation for multiple myeloma. NEJM 2012;366:1782(Better EFS and PFS, similar OS with lenalidomide after 4 y)
  20. Rosiñol et al. Lenalidomide and dexamethasone maintenance with or without ixazomib, tailored by residual disease status in myeloma. Blood 2023;142:1518 (Post-transplant maintenance; no benefit from ixazomib, safe to stop at 2 yrs if MRD neg)
  21. Cavo et al.Bortezomib-thalidomide-dexamethasone is superior to thalidomide-dexamethasone as consolidation therapy after autologous hematopoietic stem cell transplantation in patients with newly diagnosed multiple myeloma. Blood 2012;120:9
  22. Moreau et al. Bortezomib, thalidomide, and dexamethasone with or without daratumumab before and after autologous stem-cell transplantation for newly diagnosed multiple myeloma (CASSIOPEIA): a randomised, open-label, phase 3 study. Lancet 2019;394:29
  23. Mellqvist et al. Bortezomib consolidation after autologous stem cell transplantation in multiple myeloma: a Nordic Myeloma Study Group randomized phase 3 trial. Blood 2013;121:4647
  24. Moreau et al. Tandem autologous stem cell transplantation in high-risk de novo multiple myeloma: final results of the prospective and randomized IFM 99-04 protocol. Blood 2006;107:397
  25. Cavo et al. Prospective, Randomized Study of Single Compared With Double Autologous Stem-Cell Transplantation for Multiple Myeloma: Bologna 96 Clinical Study. J Clin Oncol 2007;25:2434(Double ASCT gave superior CR and EFS, but did not improve OS)
  26. Abdelkefi et al. Single autologous stem-cell transplantation followed by maintenance therapy with thalidomide is superior to double autologous transplantation in multiple myeloma: results of a multicenter randomized clinical trial. Blood 2008;111:1805
  27. Stadtmauer et al. Autologous Transplantation, Consolidation, and Maintenance Therapy in Multiple Myeloma: Results of the BMT CTN 0702 Trial. J Clin Oncol 2019;37:589(Best outcomes with single autotransplant + lenalidomide maintenance)
  28. Dimopoulos et al.Oral ixazomib maintenance following autologous stem cell transplantation (TOURMALINE-MM3): a double-blind, randomised, placebo-controlled phase 3 trial. Lancet 2019;393:19
  29. Badros et al. Daratumumab with lenalidomide as maintenance after transplant in newly diagnosed multiple myeloma: the AURIGA study. Blood 2025;145:300
  30. Rosiñol et al. A prospective PETHEMA study of tandem autologous transplantation versus autograft followed by reduced-intensity conditioning allogeneic transplantation in newly diagnosed multiple myeloma. Blood 2008;112:3591(allotransplant gave no overall survival benefit vs autotransplant)
  31. D’Souza et al. Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience. Blood 2012;120:56(5 yr PFS 75%)
  32. Jasielec et al. Carfilzomib, lenalidomide, and dexamethasone plus transplant in newly diagnosed multiple myeloma. Blood 2020;136:2513(Stringent CR in 76%; 5 yr PFS 72%)

Allotransplant in myeloma

  1. Bruno et al. Nonmyeloablative allografting for newly diagnosed multiple myeloma: the experience of the Gruppo Italiano Trapianti di Midollo. Blood 2009;113:3375
  2. Giaccone et al. Long-term follow-up of a comparison of nonmyeloablative allografting with autografting for newly diagnosed myeloma. Blood 2011;117:6721(Long-term followup of above study; allograft receipients had better survival)
  3. Kumar et al. Trends in allogeneic stem cell transplantation for multiple myeloma: a CIBMTR analysis. Blood 2011;118:1979(Increasing age, longer time from dx to transplant, and unrelated donor grafts adversely affect survival)
  4. Bruno et al. A comparison of allografting with autografting for newly diagnosed myeloma. NEJM 2007;356:1110
  5. Rotta et al. Long-term outcome of patients with multiple myeloma after autologous hematopoietic cell transplantation and nonmyeloablative allografting. Blood 2009;113:3383
  6. Lokhorst et al. Donor versus no-donor comparison of newly diagnosed myeloma patients included in the HOVON-50 multiple myeloma study. Blood 2012;119:6219(No apparent overall survival benefit to allotransplant)

Amyloidosis

  1. D’Souza et al. Improved Outcomes After Autologous Hematopoietic Cell Transplantation for Light Chain Amyloidosis: A Center for International Blood and Marrow Transplant Research Study. J Clin Oncol 2015;33:3741
  2. Jaccard et al. High-Dose Melphalan versus Melphalan plus Dexamethasone for AL Amyloidosis. NEJM 2007;357:1083(High dose therapy not superior to standard treatment, trend toward shorter survival with high dose therapy)
  3. Cibeira et al. Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients. Blood 2011;118:4346
  4. Madan et al. High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement. Blood 2012;119:1117

Chronic lymphocytic leukemia

  1. Gribbben JG. How and when I do allogeneic transplant in CLL. Blood 2018;132:31
  2. Mato and Porter. A drive through cellular therapy for CLL in 2015: allogeneic cell transplantation and CARs. Blood 2015;126:478
  3. Dreger et al. Allogeneic stem cell transplantation provides durable disease control in poor-risk chronic lymphocytic leukemia: long-term clinical and MRD results of the German CLL Study Group CLL3X trial. Blood 2010;116:2438
  4. Gribben et al. Autologous and allogeneic stem cell transplantations for poor-risk chronic lymphocytic leukemia. Blood 2005;106:4389
  5. Milligan et al. High incidence of myelodysplasia and secondary leukaemia in the UK Medical Research Council Pilot of autografting in chronic lymphocytic leukaemia. Br J Haematol 2006;133:173
  6. Michallet et al. Autologous hematopoietic stem cell transplantation in chronic lymphocytic leukemia: results of European intergroup randomized trial comparing autografting versus observation. Blood 2011;117:1516(No survival benefit from autotransplant)
  7. Dreger et al. Early autologous stem cell transplantation for chronic lymphocytic leukemia: long-term follow-up of the German CLL Study Group CLL3 trial. Blood 2012;119:4851(Overall outcome similar to that in patients treated with FCR without transplant)
  8. Cwynarski et al. Autologous and Allogeneic Stem-Cell Transplantation for Transformed Chronic Lymphocytic Leukemia (Richter’s Syndrome): A Retrospective Analysis From the Chronic Lymphocytic Leukemia Subcommittee of the Chronic Leukemia Working Party and Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol 2012;30:2211(Transplantation may benefit patients who respond to induction therapy)
  9. Krämer et al. Allogeneic hematopoietic cell transplantation for high-risk CLL: 10-year follow-up of the GCLLSG CLL3X trial. Blood 2017;130:1477
  10. Ryan et al. Ibrutinib efficacy and tolerability in patients with relapsed chronic lymphocytic leukemia following allogeneic HCT. Blood 2016;128:2899(Treatment appears to enhance GVL effect)

Waldenström macroglobulinemia

  1. Kyriakou et al. Allogeneic Stem-Cell Transplantation in Patients With Waldenström Macroglobulinemia: Report From the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol 2010;28:4926

Aplastic anemia

  1. DeZern et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood 2023;141:3031
  2. McReynolds et al. Genetic testing in severe aplastic anemia is required for optimal hematopoietic cell transplant outcomes. Blood 2022;140:909 (7% of patients had an unrecognized inherited marrow failure syndrome, with worse outcomes)
  3. Deeg et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood 2006;108:1485
  4. Eapen et al. Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia. Blood 2011;118:2618(Bone marrow stem cells associated withless acute GVHD and lower mortality than peripheral blood stem cells)
  5. Gadalla et al. Association Between Donor Leukocyte Telomere Length and Survival After Unrelated Allogeneic Hematopoietic Cell Transplantation for Severe Aplastic Anemia. JAMA 2015;313:594

Other disorders; gene therapy

  1. High and Roncarolo. Gene therapy. NEJM 2019;381:455
  2. Storb et al. Hematopoietic Cell Transplantation for Benign Hematological Disorders and Solid Tumors. Hematology 2003:372-397
  3. Stenger et al. How I treat sickle cell disease with hematopoietic cell transplantation. Blood 2019;134:2249
  4. Gluckman et al. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood 2017;129:1548(5-year OS 93%)
  5. Hsieh et al. Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype. JAMA 2014;312:48(Adolescent and adult patients; fairly good short-term outcomes)
  6. Brazauskas et al. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood 2020;136:623
  7. Wu et al. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med 2019;25:776 (Editing HSCs to increase HbF in sickle dz)
  8. Hulbert et al. Normalization of cerebral hemodynamics after hematopoietic stem cell transplant in children with sickle cell disease. Blood 2023;141:335
  9. Li et al. A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation. Blood 2012;120:3875
  10. Locatelli et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood 2013;122:1072
  11. Fitzhugh et al. At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT. Blood 2017;130:1946
  12. King and Shenoy. Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia. Blood 2014;123:3089
  13. Burt et al. Nonmyeloablative Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus. JAMA 2006;295:527
  14. Burt et al. Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis. Lancet 2013;381:1116
  15. van Laar et al. Autologous Hematopoietic Stem Cell Transplantation vs Intravenous Pulse Cyclophosphamide in Diffuse Cutaneous Systemic Sclerosis. A Randomized Clinical Trial. JAMA 2014;311:2490(Higher short-term mortality, better long-term EFS with HCST)
  16. Sullivan et al. Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma. NEJM 2018;378:35
  17. Daley et al. Realistic Prospects for Stem Cell Therapeutics. Hematology 2003:398-418
  18. Boztug et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. NEJM 2010;363:1918
  19. Allers et al. Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation. Blood 2011;117:2791
  20. Daikeler et al. Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party. Blood 2011;118:1693
  21. Cuellar-Rodriguez et al. Successful allogeneic hematopoietic stem cell transplantation for GATA2 deficiency. Blood 2011;118:3715
  22. Roos-Weil et al. Stem cell transplantation can provide durable disease control in blastic plasmacytoid dendritic cell neoplasm: a retrospective study from the European Group for Blood and Marrow Transplantation. Blood 2013;121:440
  23. Ayas et al. Allogeneic Hematopoietic Cell Transplantation for Fanconi Anemia in Patients With Pretransplantation Cytogenetic Abnormalities, Myelodysplastic Syndrome, or Acute Leukemia. J Clin Oncol 2013;31:1669
  24. Río et al. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nat Med 2019;25:1396
  25. de Latour et al. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience. Blood 2013;122:4279
  26. Burt et al. Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis. A Randomized Clinical Trial. JAMA 2019;321:165
  27. Albert et al. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study. Blood 2022;140:1635

Early complications

  1. Mullighan et al. Mannose-binding lectin status is associated with risk of major infection following myeloablative sibling allogeneic hematopoietic stem cell transplantation. Blood 2008;112:2120
  2. Marty et al. Sirolimus-based graft-versus-host disease prophylaxis protects against cytomegalovirus reactivation after allogeneic hematopoietic stem cell transplantation: a cohort analysis. Blood 2007;110:490
  3. Laskin et al. Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation–associated thrombotic microangiopathy. Blood 2011;118:1452
  4. Vasu et al. High incidence of severe TA-TMA increases mortality in adult allogeneic transplant recipients: a prospective MIDAS Consortium study. Blood 2025;146:638
  5. Gloude et al. Circulating dsDNA, endothelial injury, and complement activation in thrombotic microangiopathy and GVHD. Blood 2017;130:1259
  6. Jodele et al. A prospective multi-institutional study of eculizumab to treat high-risk stem cell transplantation–associated TMA. Blood 2024;143:1112
  7. Khaled et al. Narsoplimab, a Mannan-Binding Lectin-Associated Serine Protease-2 Inhibitor, for the Treatment of Adult Hematopoietic Stem-Cell Transplantation–Associated Thrombotic Microangiopathy. J Clin Oncol 2022;40:2447
  8. Gerber et al. The incidence of and risk factors for venous thromboembolism (VTE) and bleeding among 1514 patients undergoing hematopoietic stem cell transplantation: implications for VTE prevention. Blood 2008;112:504(4.6% incidence, most episodes catheter-related)
  9. Baur et al. Immune cytopenia after allogeneic haematopoietic stem-cell transplantation: challenges, approaches, and future directions. Lancet Haematol 2021;8:e229
  10. Cutler et al. Sirolimus is associated with veno-occlusive disease of the liver after myeloablative allogeneic stem cell transplantation. Blood 2008; 112:4425(sirolimus plus MTX for GVHD prophylaxis associated with 3-fold increase in VOD incidence; busulfan conditioning + sirolimus assocated with 8-fold increased risk)
  11. Maradei et al. Serum ferritin as risk factor for sinusoidal obstruction syndrome of the liver in patients undergoing hematopoietic stem cell transplantation. Blood 2009;114:1270(Serum ferritin >1000 is independent risk factor for SOS/VOD)
  12. Chao N. How I treat sinusoidal obstruction syndrome. Blood 2014;123:4023
  13. Richardson et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood 2016;127:1656(Treatment improves survival vs historical controls)
  14. Hingorani S. Renal complications of hematopoietic-cell transplantation. NEJM 2016;374:2256
  15. Erard et al. BK DNA viral load in plasma: evidence for an association with hemorrhagic cystitis in allogeneic hematopoietic cell transplant recipients. Blood 2005;106:1130
  16. Martino et al. Impact of the intensity of the pretransplantation conditioning regimen in patients with prior invasive aspergillosis undergoing allogeneic hematopoietic stem cell transplantation: a retrospective survey of the Infectious Diseases Working Party of the European Group for Blood and Marrow Transplantation. Blood 2006;108:2928
  17. Herrera et al. Cord colitis syndrome in cord-blood stem-cell transplantation. NEJM 2011;365:815

Late complications

  1. Armenian et al. Burden of Long-Term Morbidity Borne by Survivors of Acute Myeloid Leukemia Treated With Blood or Marrow Transplantation: The Results of the BMT Survivor Study. J Clin Oncol 2022;40:3278
  2. Bhatia et al.Late mortality after allogeneic hematopoietic cell transplantation and functional status of long-term survivors: report from the Bone Marrow Transplant Survivor Study. Blood 2007;110:3784 (Mortality rates among 15-year survivors of HCT twice as high as general population)
  3. Chow et al. Morbidity and Mortality Differences Between Hematopoietic Cell Transplantation Survivors and Other Cancer Survivors. J Clin Oncol 2017;35:306(Relatively high rates of infectious and respiratory complications in transplant survivors)
  4. Socie, G, Salooja, N, Cohen, A, Rovelli, A, et al. Nonmalignant late effects after allogeneic stem cell transplantation. Blood 2003; 101: 3373-3385.
  5. Sun 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
  6. Khera et al. Nonmalignant Late Effects and Compromised Functional Status in Survivors of Hematopoietic Cell Transplantation. J Clin Oncol 2011;30:71
  7. Syrjala et al. Recovery and Long-term Function After Hematopoietic Cell Transplantation for Leukemia or Lymphoma.  JAMA 2004;291:2335-2343.
  8. Baker et al.  Late effects in survivors of chronic myeloid leukemia treated with hematopoietic cell transplantation: results from the Bone Marrow Transplant Survivor Study. Blood 2004;104:1898.
  9. Savani et al. How I treat late effects in adults after allogeneic stem cell transplantation. Blood 2011;117:3002
  10. Wong et al. Long-term recovery after hematopoietic cell transplantation: predictors of quality-of-life concerns. Blood 2010;115:2508
  11. Friedman et al. Increased risk of breast cancer among survivors of allogeneic hematopoietic cell transplantation: a report from the FHCRC and the EBMT-Late Effect Working Party. Blood 2008;111:939
  12. Curtis, RE, Travis, LB, Rowlings, PA, Socie, G, et al. Risk of lymphoproliferative disorders after bone marrow transplantation: a multi-institutional study. Blood 1999; 94: 2208-2216.
  13. Sakaida et al. Late-onset noninfectious pulmonary complications after allogeneic stem cell transplantation are significantly associated with chronic graft-versus-host disease and with the graft-versus-leukemia effect. Blood 2003;102:4236
  14. Williams KM. How I treat bronchiolitis obliterans syndrome after hematopoietic stem cell transplantation. Blood 2017;129:448
  15. Santo Tomas et al. Risk Factors for Bronchiolitis Obliterans in Allogeneic Hematopoietic Stem-Cell Transplantation for Leukemia. Chest 2005;128:153
  16. Freudenberger et al.  Association between acute and chronic graft-versus-host disease and bronchiolitis obliterans organizing pneumonia in recipients of hematopoietic stem cell transplants. Blood 2003;102:3822
  17. Lai et al. How I diagnose and treat organizing pneumonia in hematopoietic cell transplant recipients. Blood 2024;144:1048
  18. Schulte and Beelen.  Bone loss following hematopoietic stem cell transplantation: a long-term follow-up. Blood 2004;103:3635
  19. Rizzo et al. Solid cancers after allogeneic hematopoietic cell transplantation. Blood 2009;113:1175 (Risk higher in patients exposed to conditioning radiation at younger age; lifelong surveillance recommended)
  20. Baker et al. Total body irradiation dose and risk of subsequent neoplasms following allogeneic hematopoietic cell transplantation. Blood 2019;133:2790
  21. Landgren et al. Risk factors for lymphoproliferative disorders after allogeneic hematopoietic cell transplantation. Blood 2009;113:4992
  22. Doubrovina et al. Adoptive immunotherapy with unselected or EBV-specific T cells for biopsy-proven EBV+ lymphomas after allogeneic hematopoietic cell transplantation. Blood 2012;119:2644
  23. Syrjala et al. Sexual function changes during the 5 years after high-dose treatment and hematopoietic cell transplantation for malignancy, with case-matched controls at 5 years. Blood 2007;111:989
  24. Sanders et al. Thyroid function following hematopoietic cell transplantation in children: 30 years’ experience. Blood 2009;113:306
  25. Griffith et al. Dyslipidemia after allogeneic hematopoietic stem cell transplantation: evaluation and management. Blood 2010;116:1197
  26. Chow et al. Cardiovascular Hospitalizations and Mortality Among Recipients of Hematopoietic Stem Cell Transplantation. Ann Intern Med 2011;155:21
  27. Armenian et al. Cardiovascular risk factors in hematopoietic cell transplantation survivors: role in development of subsequent cardiovascular disease. Blood 2012;120:4505
  28. Sharafeldin et al. Cognitive Functioning After Hematopoietic Cell Transplantation for Hematologic Malignancy: Results From a Prospective Longitudinal Study. J Clin Oncol 2018;36:463(Allotransplant but not autotransplant associated with cognitive decline)

Risks of stem cell donation

  1. Pulsipher et al. Acute toxicities of unrelated bone marrow versus peripheral blood stem cell donation: results of a prospective trial from the National Marrow Donor Program. Blood 2013;121:197
  2. Hölig et al. Safety and efficacy of hematopoietic stem cell collection from mobilized peripheral blood in unrelated volunteers: 12 years of single-center experience in 3928 donors. Blood 2009;114:3757
  3. Hirsch et al. Effects of granulocyte-colony stimulating factor on chromosome aneuploidy and replication asynchrony in healthy peripheral blood stem cell donors. Blood 2011;118:2602(No evidence that G-CSF, as given to stem cell donors, causes chromosomal instability)
  4. Pulsipher et al. Lower risk for serious adverse events and no increased risk for cancer after PBSC vs BM donation. Blood 2014;123:3655

Supportive Care

  1. Antin, JH. Clinical practice. Long-term care after hematopoietic-cell transplantation in adults. N Engl J Med 2002; 347: 36-42.
  2. Gajewski et al. A review of transfusion practice before, during, and after hematopoietic progenitor cell transplantation. Blood 2008;112:3036
  3. Tay et al. Liberal Versus Restrictive Red Blood Cell Transfusion Thresholds in Hematopoietic Cell Transplantation: A Randomized, Open Label, Phase III, Noninferiority Trial. J Clin Oncol 2020;38:1463 (No apparent benefit to transfusion threshold of 9 g/dL vs 7 g/dL)
  4. Spielberger et al.  Palifermin for Oral Mucositis after Intensive Therapy for Hematologic Cancers.  NEJM 2004;351:2590
  5. Boeckh et al. Long-term acyclovir for prevention of varicella zoster virus disease after allogeneic hematopoietic cell transplantation: a randomized double-blind placebo-controlled study. Blood 2006;107:1800