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Stem Cell transplant: biology, review articles
- Copelan E. Hematopoietic stem-cell transplantation. NEJM 2006;354:1813
- Kolb J. Graft-versus-leukemia effects of transplantation and donor lymphocytes. Blood 2008;112:4371
- Khan et al. Is allergic disease curable or transferable with allogeneic hematopoietic cell transplantation? Blood 2009;113:279
- 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)
- Penack et al. The importance of neovascularization and its inhibition for allogeneic hematopoietic stem cell transplantation. Blood 2011;117:4181
- Sorror ML. How I assess comorbidities before hematopoietic cell transplantation. Blood 2013;121:2854
Autologous Transplant General
- Cottler-Fox et al. Stem Cell Mobilization. Hematology 2003:419-437
- 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
- 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
- Gooley et al. Reduced mortality after allogeneic hematopoieic-cell transplantation. NEJM 2010;363:2091
- Armand et al. A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood 2012;120:905
- Armand et al. Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation. Blood 2014;123:3664
- 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
- Puerta-Alcalde et al. How I perform hematopoietic stem cell transplantation on patients with a history of invasive fungal disease. Blood 2020;136:2741
- Pidala et al. Quality of life after allogeneic hematopoietic cell transplantation. Blood 2009;114:7
- 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)
- Ballen et al. Selection of optimal alternative graft source: mismatched unrelated donor, umbilical cord blood, or haploidentical transplant. Blood 2012;119:1972
- 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
- Dehn et al. Selection of unrelated donors and cord blood units for hematopoietic cell transplantation: guidelines from the NMDP/CIBMTR. Blood 2019;134:924
- 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)
- 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
- Kolb et al. Graft-versus-leukemia reactions in allogeneic chimeras. Blood 2004;103:767
- Porter DL. Allogeneic Immunotherapy to Optimize the Graft-versus-Tumor Effect: Concepts and Controversies. Hematology 2011:292
- Zeiser and Vago. Mechanisms of immune escape after allogeneic hematopoietic cell transplantation. Blood 2019;133:1290
- Laughlin et al. Outcomes after Transplantation of Cord Blood or Bone Marrow from Unrelated Donors in Adults with Leukemia. NEJM 2004;351:2265
- Rocha et al. Transplants of Umbilical-Cord Blood or Bone Marrow from Unrelated Donors in Adults with Acute Leukemia. NEJM 2004;351:2276
- 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
- Anasetti et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. NEJM 2012;367:1487(No significant difference in outcomes)
- 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)
- 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)
- Lee et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood 2007;110:4576
- 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
- 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
- Wingard et al. Long-term survival and late deaths after allogeneic hematopoietic cell transplantation. J Clin Oncol 2011;29:2230
- 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)
- 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)
- Jaspers et al. Erythropoietin therapy after allogeneic hematopoietic cell transplantation: a prospective, randomized trial. Blood 2014;124:33 (EPO started one month after HSCT hastens RBC recovery and reduces transfusion requirements)
- Chapuy et al. Daratumumab for Delayed Red-Cell Engraftment after Allogeneic Transplantation. NEJM 2018;379:1846
- Davids et al. Ipilimumab for Patients with Relapse after Allogeneic Transplantation. NEJM 2016;375:143(23% CR rate, most responses > 1 year)
- Smith et al. Posttransplant chimeric antigen receptor therapy. Blood 2018;131:1045
- Soiffer et al. Tyrosine kinase inhibitors and immune checkpoint blockade in allogeneic hematopoietic cell transplantation. Blood 2018;131:1073
- 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
- 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
- Gyurkocza and Sandmaier. Conditioning regimens for hematopoietic cell transplantation: one size does not fit all. Blood 2014;124:344
- 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
- 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)
- 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
- 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”)
- 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
- 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)
- 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
- Barker J. Umbilical Cord Blood (UCB) Transplantation: An Alternative to the Use of Unrelated Volunteer Donors? Hematology 2007:55
- Ballen K. New trends in umbilical cord blood transplantation. Blood 2005;105: 3786.
- Scaradavou et al. Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood 2013;121:752
- Wagner et al. One-Unit versus Two-Unit Cord-Blood Transplantation for Hematologic Cancers. NEJM 2014;371:1685(No advantage to two-unit transplantation)
- Brunstein et al. Umbilical cord blood transplantation after nonmyeloablative conditioning: impact on transplantation outcomes in 110 adults with hematologic disease. Blood 2007;110:3064
- 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)
- Cutler et al. Donor-specific anti-HLA antibodies predict outcome in double umbilical cord blood transplantation. Blood 2011;118:6691
- Herrera et al. Cord colitis syndrome in cord-blood stem-cell transplantation. NEJM 2011;365:815
Acute GVHD
- Jamy et al. Novel developments in the prophylaxis and treatment of acute GVHD. Blood 2023;142:1037
- Bolaños-Meade et al. Post-Transplantation Cyclophosphamide-Based Graft-versus-Host Disease Prophylaxis. NEJM 2023;388:2338
- Curtis et al. Graft-versus-Host Disease Prophylaxis with Cyclophosphamide and Cyclosporin. NEJM 2025;393:243
- Martin PJ. How I treat steroid-refractory acute graft-versus-host disease. Blood 2020;135:1630
- Zeiser and Blazar. Acute Graft-versus-Host Disease — Biologic Process, Prevention, and Therapy. NEJM 2017;377:2167
- Markey et al. The biology of graft-versus-host disease: experimental systems instructing clinical practice. Blood 2014;124:354
- Holtan et al. Acute graft-versus-host disease: a bench-to-bedside update. Blood 2014;124:363
- Zeiser and Teshima. Nonclassical manifestations of acute GVHD. Blood 2021;138:2165
- Shimabukuro-Vornhagen et al. The role of B cells in the pathogenesis of graft-versus-host disease. Blood 2009;114:4919
- Penack et al. Graft-versus-host disease: regulation by microbe-associated molecules and innate immune receptors. Blood 2010; 115:1865
- Schwab et al. Neutrophil granulocytes recruited upon translocation of intestinal bacteria enhance graft-versus-host disease via tissue damage. Nat Med 2014;20:648
- McDonald G. How I treat acute graft-versus-host disease of the gastrointestinal tract and the liver. Blood 2016;127:1544
- Paczesny S. Discovery and validation of graft-versus-host disease biomarkers. Blood 2013;121:585
- Harris et al. Plasma biomarkers of lower gastrointestinal and liver acute GVHD. Blood 2012;119:2960
- 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)
- Vander Lugt et al. ST2 as a marker for risk of therapy-resistant graft-versus-host disease and death. NEJM 2013;369:529
- MacMillan et al. Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood 2009;113:2410
- Mielcarek et al. Initial therapy of acute graft-versus-host disease with low-dose prednisone does not compromise patient outcomes. Blood 2009;113:2888
- 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
- Saliba et al. Hyperacute GVHD: risk factors, outcomes, and clinical implications. Blood 2007;109:2751
- Kean et al. Abatacept for acute graft-versus-host disease prophylaxis after unrelated donor hematopoietic cell transplantation. Blood 2024;144:1834
- 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)
- Rotta et al. Donor statin treatment protects against severe acute graft-versus-host disease after related allogeneic hematopoietic cell transplantation. Blood 2010;115:1288
- Reshef et al. Blockade of lymphocyte chemotaxis in visceral graft-versus-host disease. NEJM 2012;367:135
- 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)
- 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)
- 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)
- Zeiser et al. Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease. NEJM 2020;382:1800(With editorial)
- Jagasia et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial. Blood 2020;135:1739
- Abboud et al. Itacitinib for prevention of graft-versus-host disease and cytokine release syndrome in haploidentical transplantation. Blood 2025;145:1382
- Kornblit et al. Sirolimus with CSP and MMF as GVHD prophylaxis for allogeneic transplantation with HLA antigen–mismatched donors. Blood 2020;136:1499
- Farag et al. Dipeptidyl Peptidase 4 Inhibition for Prophylaxis of Acute Graft-versus-Host Disease. NEJM 2021;384:11(With editorial)
- Magenau et al. A phase 2 trial of CD24Fc for prevention of graft-versus-host disease. Blood 2024;143:21
- Meyer et al. Donor regulatory T-cell therapy to prevent graft-versus-host disease. Blood 2025;145:2012
Chronic GVHD
- Lee and Zeiser. FDA-approved therapies for chronic GVHD. Blood 2025;145:795
- Pidala et al.Chronic graft-versus-host disease: unresolved complication or ancient history? Blood 2024;144:1363
- MacDonald et al. Chronic graft-versus-host disease: biological insights from preclinical and clinical studies. Blood 2017;129:13
- Zeiser and Blazar. Pathophysiology of Chronic Graft-versus-Host Disease and Therapeutic Targets, NEJM 2017;377:2565
- Koyama and Hill. Alloantigen presentation and graft-versus-host disease: fuel for the fire. Blood 2016;127:2963
- Lee SJ. Classification systems for chronc graft-versus-host disease. Blood 2017;129:30
- Cutler et al. Mechanistic approaches for the prevention and treatment of chronic GVHD. Blood 2017;129:22
- Flowers and Martin. How we treat chronic graft-versus-host disease. Blood 2015;125:606
- Sarantopoulos et al. How I treat refractory chronic graft-versus-host disease. Blood 2019;133:1191
- Carpenter PA. How I conduct a comprehensive chronic graft-versus-host disease assessment. Blood 2011;118:2679
- 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
- Stein-Thoeringer et al. Lactose drives Enterococcus expansion to promote graft-versus-host disease. Science 2019;366:1143(With editorial)
- Arora et al. Chronic GVHD risk score: a Center for International Blood and Marrow Transplant Research analysis. Blood 2011;117:6714.
- 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
- Martires et al. Sclerotic-type chronic GVHD of the skin: clinical risk factors, laboratory markers, and burden of disease. Blood 2011;118:4250
- 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
- Treister et al. How we treat oral chronic graft-versus-host disease. Blood 2012;120:3407
- Kröger et al. Antilymphocyte Globulin for Prevention of Chronic Graft-versus-Host Disease. NEJM 2016;374:43(50% reduction in CGVHD rate with ATG)
- 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)
- Inamoto et al. Assessment of Joint and Fascia Manifestations in Chronic Graft-Versus-Host Disease. Arth Rheum 2014;66:1044
- Shimabukuro-Vornhagen et al. The role of B cells in the pathogenesis of graft-versus-host disease. Blood 2009;114:4919
- Lee SJ. New approaches for preventing and treating chronic graft-versus-host disease. Blood 2005;105:4200
- Martin et al. Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood 2009;113:5074(MMF ineffective)
- 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.
- Stewart et al. Duration of immunosuppressive treatment for chronic graft-versus-host disease. Blood 2004;104:3501
- 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)
- 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
- Inamoto et al. Incidence, risk factors, and outcomes of sclerosis in patients with chronic grat-versus-host disease. Blood 2013;121:5098
- Olivieri et al. Imatinib for refractory chronic graft-versus-host disease with fibrotic features. Blood 2009;114:709
- 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
- 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)
- 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
- Wall et al. Complement-mediated thrombotic microangiopathy as a link between endothelial damage and steroid-refractory GVHD. Blood Adv 2018;2:2619
- Miklos et al. Ibrutinib for chronic graft-versus-host disease after failure of prior therapy. Blood 2017;130:2243
- Zeiser et al. Ruxolitinib for Glucocorticoid-Refractory Chronic Graft-versus-Host Disease. NEJM 2021;385:228
- Koshy et al. Phase 2 clinical trial evaluating abatacept in patients with steroid-refractory chronic graft-versus-host disease. Blood 2023;141:2932
- Wolff et al. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. NEJM 2024;391:1002
GVHD and solid organ transplant
- Cooper and Abkowitz. How I diagnose and treat acute graft-versus-host disease after solid organ transplantation. Blood 2023;141:1138
Acute Myeloid Leukemia
- Cornelissen and Blaise. Hematopoietic stem cell transplantation for patients with AML in first complete remission. Blood 2016;127:62
- 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)
- Gupta et al. Allogeneic hematopoietic cell transplantation for adults with acute myeloid leukemia: myths, controversies, and unknowns. Blood 2011;117:2307
- Stone RM. Acute myeloid leukemia in first remission: to choose transplantation or not? J Clin Oncol 2013;31:1262
- 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”)
- 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)
- 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)
- 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)
- 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
- 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)
- 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
- 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)
- 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
- 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)
- 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
- 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)
- 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)
- 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
- 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)
- 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)
- 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)
- Venstrom et al. HLA-C–Dependent Prevention of Leukemia Relapse by Donor Activating KIR2DS1. NEJM 2012;367:805(With editorial)
- 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
- 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)
- Spyridonidis A. How I treat measurable (minimal) residual disease in acute leukemia after allogeneic hematopoietic cell transplantation. Blood 2020;135:1639
- Levis et al. Measurable residual disease and posttransplantation gilteritinib maintenance for patients with FLT3-ITD–mutated AML. Blood 2025;145:2138
- 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)
- 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
- 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
- Vellenga et al. Autologous peripheral blood stem cell transplantation for acute myeloid leukemia. Blood 2011;118:6037
- 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
- Goldstone and Rowe. Transplantation in adult ALL. Hematology 2009;593
- 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
- 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
- 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
- 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)
- Bachanova et al. Allogeneic hematopoietic stem cell transplantation overcomes the adverse prognostic impact of CD20 expression in acute lymphoblastic leukemia. Blood 2011;117:5261
- 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
- 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
- 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)
- Wassmann et al. Early molecular response to posttransplantation imatinib determines outcome in MRD+ Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL). Blood 2005;106:458
- Mizuta et al. Pretransplant administration of imatinib for allo-HSCT in patients with BCR-ABL–positive acute lymphoblastic leukemia. Blood 2014;123:2325
- 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)
- 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
- 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
- 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)
- Spyridonidis A. How I treat measurable (minimal) residual disease in acute leukemia after allogeneic hematopoietic cell transplantation. Blood 2020;135:1639
Chronic myelogenous leukemia
- Barrett and Ito. The role of stem cell transplantation for chronic myelogenous leukemia in the 21st century. Blood 2015;125:3230
- Pavlu et al. Optimizing patient selection for myeloablative allogeneic hematopoietic cell transplantation in chronic myeloid leukemia in chronic phase. Blood 2010;115:4018
- Apperley JF. Managing the Patient with Chronic Myeloid Leukemia Through and After Allogeneic Stem Cell Transplantation. Hematology 2006;226-32
- 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)
- 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)
- 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)
- 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
- Kröger et al. How I treat transplant-eligible patients with myelofibrosis. Blood 2023;142:1683
- Mina et al. How I reduce and treat posttransplant relapse of MDS. Blood 2024;143:1344
- 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
- de Witte et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood 2017;129:1753
- Onida et al. Management of adult patients with CMML undergoing allo-HCT: recommendations from the EBMT PH&G Committee. Blood 2024;143:2227
- Robin et al. Role of allogeneic transplantation in chronic myelomonocytic leukemia: an international collaborative analysis. Blood 2022;140:1408
- 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
- 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)
- 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%)
- Takagi et al. Successful engraftment after reduced-intensity umbilical cord blood transplantation for myelofibrosis. Blood 2010;116:649
- 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
- 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)
- 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)
- 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
- Duncavage et al. Mutation Clearance after Transplantation for Myelodysplastic Syndrome. NEJM 2018;379:1028
- 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)
- Myllymäki et al. Short telomere length predicts nonrelapse mortality after stem cell transplantation for myelodysplastic syndrome. Blood 2020;136:3070
- Reilly et al. The clinical and functional effects of TERT variants in myelodysplastic syndrome. Blood 2021;140:898
Non-Hodgkins Lymphoma
- Kuruvilla J. The role of autologous and allogeneic stem cell transplantation in the management of indolent B-cell lymphoma. Blood 2016;127:2093
- van Besien et al. Comparison of autologous and allogeneic hematopoietic stem cell transplantation for follicular lymphoma. Blood 2003;102:3521-9
- 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)
- 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
- van Biesen K. Allogeneic stem cell transplantation in follicular lymphoma: recent progress and controversy. Hematology 2009;610
- 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)
- 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
- 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)
- Lambert et al. Prognostic role of PET scanning before and after reduced-intensity allogeneic stem cell transplantation for lymphoma. Blood 2010;115:2763
- 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
- 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)
- 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
- 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
- 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
- Milpied et al. Initial Treatment of Aggressive Lymphoma with High-Dose Chemotherapy and Autologous Stem-Cell Support. NEJM 2004;350:1287
- 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)
- 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)
- 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?)
- 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.
- 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.
- 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)
- 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
- 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)
- 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)
- Schmitz et al. Allogeneic hematopoietic stem cell transplantation for T-cell lymphomas. Blood 2018;132:245
- 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
- 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%)
- de Masson et al. Allogeneic transplantation in advanced cutaneous T-cell lymphomas (CUTALLO): a propensity score matched controlled prospective study. Lancet 2023;401:1941
- 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)
- 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)
- 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
- Krishnan et al. Durable remissions with autologous stem cell transplantation for high-risk HIV-associated lymphomas. Blood 2005;105:874
- 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
- 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)
- 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
- 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)
- Ferreri and Illerhaus. The role of autologous stem cell transplantation in primary central nervous system lymphoma. Blood 2016;127:1642
- 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
- Shah and Moskowitz. Transplant strategies in relapsed/refractory Hodgkin lymphoma. Blood 2018;131:1689
- 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
- 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
- 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
- Alinari and Blum. How I treat relapsed classical Hodgkin lymphoma after autologous stem cell transplant. Blood 2016;127:287
- 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)
- 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
- 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
- Armand et al. PD-1 blockade with pembrolizumab for classical Hodgkin lymphoma after autologous stem cell transplantation. Blood 2019;134:22
Multiple myeloma
- van Rhee et al The future of autologous stem cell transplantation in myeloma. Blood 2014;124:328
- Kumar et al. Pros and cons of frontline autologous transplant in multiple myeloma: the debate over timing. Blood 2019;133:652
- Gertz and Dingli. How we manage autologous stem cell transplantation for patients with multiple myeloma. Blood 2014;124:882
- 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)
- 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%)
- 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)
- 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”)
- 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)
- 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
- 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
- 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.
- Attal et al. Single versus Double Autologous Stem-Cell Transplantation for Multiple Myeloma. NEJM 2003;349:2495
- 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
- Roussel et al. Bortezomib and high-dose melphalan conditioning regimen in frontline multiple myeloma: an IFM randomized phase 3 study.Blood 2022;139:2747
- 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)
- Attal et al. Lenalidomide, Bortezomib, and Dexamethasone with Transplantation for Myeloma. NEJM 2017;376:1311(RVD plus SCT superior to RVD alone; with editorial)
- 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)
- 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)
- 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)
- 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)
- 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
- 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
- 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
- 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
- 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)
- 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
- 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)
- 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
- Badros et al. Daratumumab with lenalidomide as maintenance after transplant in newly diagnosed multiple myeloma: the AURIGA study. Blood 2025;145:300
- 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)
- 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%)
- 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
- Bruno et al. Nonmyeloablative allografting for newly diagnosed multiple myeloma: the experience of the Gruppo Italiano Trapianti di Midollo. Blood 2009;113:3375
- 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)
- 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)
- Bruno et al. A comparison of allografting with autografting for newly diagnosed myeloma. NEJM 2007;356:1110
- Rotta et al. Long-term outcome of patients with multiple myeloma after autologous hematopoietic cell transplantation and nonmyeloablative allografting. Blood 2009;113:3383
- 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
- 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
- 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)
- 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
- Madan et al. High-dose melphalan and peripheral blood stem cell transplantation for light-chain amyloidosis with cardiac involvement. Blood 2012;119:1117
- Gribbben JG. How and when I do allogeneic transplant in CLL. Blood 2018;132:31
- Mato and Porter. A drive through cellular therapy for CLL in 2015: allogeneic cell transplantation and CARs. Blood 2015;126:478
- 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
- Gribben et al. Autologous and allogeneic stem cell transplantations for poor-risk chronic lymphocytic leukemia. Blood 2005;106:4389
- 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
- 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)
- 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)
- 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)
- Krämer et al. Allogeneic hematopoietic cell transplantation for high-risk CLL: 10-year follow-up of the GCLLSG CLL3X trial. Blood 2017;130:1477
- 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
- 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
- DeZern et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood 2023;141:3031
- 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)
- 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
- 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)
- 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
- High and Roncarolo. Gene therapy. NEJM 2019;381:455
- Storb et al. Hematopoietic Cell Transplantation for Benign Hematological Disorders and Solid Tumors. Hematology 2003:372-397
- Stenger et al. How I treat sickle cell disease with hematopoietic cell transplantation. Blood 2019;134:2249
- 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%)
- 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)
- Brazauskas et al. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood 2020;136:623
- 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)
- Hulbert et al. Normalization of cerebral hemodynamics after hematopoietic stem cell transplant in children with sickle cell disease. Blood 2023;141:335
- 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
- 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
- Fitzhugh et al. At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT. Blood 2017;130:1946
- 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
- Burt et al. Nonmyeloablative Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus. JAMA 2006;295:527
- 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
- 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)
- Sullivan et al. Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma. NEJM 2018;378:35
- Daley et al. Realistic Prospects for Stem Cell Therapeutics. Hematology 2003:398-418
- Boztug et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. NEJM 2010;363:1918
- Allers et al. Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation. Blood 2011;117:2791
- 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
- Cuellar-Rodriguez et al. Successful allogeneic hematopoietic stem cell transplantation for GATA2 deficiency. Blood 2011;118:3715
- 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
- 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
- Río et al. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nat Med 2019;25:1396
- 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
- 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
- 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
- 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
- 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
- Laskin et al. Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation–associated thrombotic microangiopathy. Blood 2011;118:1452
- 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
- Gloude et al. Circulating dsDNA, endothelial injury, and complement activation in thrombotic microangiopathy and GVHD. Blood 2017;130:1259
- Jodele et al. A prospective multi-institutional study of eculizumab to treat high-risk stem cell transplantation–associated TMA. Blood 2024;143:1112
- 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
- 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)
- Baur et al. Immune cytopenia after allogeneic haematopoietic stem-cell transplantation: challenges, approaches, and future directions. Lancet Haematol 2021;8:e229
- 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)
- 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)
- Chao N. How I treat sinusoidal obstruction syndrome. Blood 2014;123:4023
- 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)
- Hingorani S. Renal complications of hematopoietic-cell transplantation. NEJM 2016;374:2256
- 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
- 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
- Herrera et al. Cord colitis syndrome in cord-blood stem-cell transplantation. NEJM 2011;365:815
Late complications
- 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
- 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)
- 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)
- Socie, G, Salooja, N, Cohen, A, Rovelli, A, et al. Nonmalignant late effects after allogeneic stem cell transplantation. Blood 2003; 101: 3373-3385.
- 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
- Khera et al. Nonmalignant Late Effects and Compromised Functional Status in Survivors of Hematopoietic Cell Transplantation. J Clin Oncol 2011;30:71
- Syrjala et al. Recovery and Long-term Function After Hematopoietic Cell Transplantation for Leukemia or Lymphoma. JAMA 2004;291:2335-2343.
- 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.
- Savani et al. How I treat late effects in adults after allogeneic stem cell transplantation. Blood 2011;117:3002
- Wong et al. Long-term recovery after hematopoietic cell transplantation: predictors of quality-of-life concerns. Blood 2010;115:2508
- 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
- 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.
- 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
- Williams KM. How I treat bronchiolitis obliterans syndrome after hematopoietic stem cell transplantation. Blood 2017;129:448
- Santo Tomas et al. Risk Factors for Bronchiolitis Obliterans in Allogeneic Hematopoietic Stem-Cell Transplantation for Leukemia. Chest 2005;128:153
- 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
- Lai et al. How I diagnose and treat organizing pneumonia in hematopoietic cell transplant recipients. Blood 2024;144:1048
- Schulte and Beelen. Bone loss following hematopoietic stem cell transplantation: a long-term follow-up. Blood 2004;103:3635
- 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)
- Baker et al. Total body irradiation dose and risk of subsequent neoplasms following allogeneic hematopoietic cell transplantation. Blood 2019;133:2790
- Landgren et al. Risk factors for lymphoproliferative disorders after allogeneic hematopoietic cell transplantation. Blood 2009;113:4992
- 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
- 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
- Sanders et al. Thyroid function following hematopoietic cell transplantation in children: 30 years’ experience. Blood 2009;113:306
- Griffith et al. Dyslipidemia after allogeneic hematopoietic stem cell transplantation: evaluation and management. Blood 2010;116:1197
- Chow et al. Cardiovascular Hospitalizations and Mortality Among Recipients of Hematopoietic Stem Cell Transplantation. Ann Intern Med 2011;155:21
- Armenian et al. Cardiovascular risk factors in hematopoietic cell transplantation survivors: role in development of subsequent cardiovascular disease. Blood 2012;120:4505
- 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
- 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
- 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
- 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)
- 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
- Antin, JH. Clinical practice. Long-term care after hematopoietic-cell transplantation in adults. N Engl J Med 2002; 347: 36-42.
- Gajewski et al. A review of transfusion practice before, during, and after hematopoietic progenitor cell transplantation. Blood 2008;112:3036
- 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)
- Spielberger et al. Palifermin for Oral Mucositis after Intensive Therapy for Hematologic Cancers. NEJM 2004;351:2590
- 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