Hematopoietic Stem-Cell Transplantation

Copelan, Edward A.

doi:10.1056/nejmra052638

Cited by 2,522Hematopoietic Stem Cell TransplantationTransplantation: Methods and OutcomesMesenchymal stem cell research

Hematopoietic Stem-Cell Transplantation

Copelan, Edward A.·2006·New England Journal of Medicine

DOI 10.1056/nejmra052638

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Abstract

Hematopoietic stem-cell transplantation, which is used to treat both malignant and nonmalignant conditions, was first conceived more than 50 years ago, but problems associated with transplanting a nonsolid organ and modulating the immune response had to be solved before the procedure could be used clinically. This review summarizes background information about hematopoietic stem-cell transplantation and discusses the current role of the procedure.

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Among stem cell therapies, only HSC transplantation is broadly accepted and used in the clinic.
“Currently, HSC transplantation is the only stem cell–based therapy widely accepted and used clinically, and it provides an effective cure for hematologic diseases such as leukemia and lymphoma135.”
From Stem cell aging: mechanisms, regulators and therapeutic opportunities (2014) · cites this paper as [135]
HSC transplantation can effectively cure blood diseases like leukemia and lymphoma.
“Currently, HSC transplantation is the only stem cell–based therapy widely accepted and used clinically, and it provides an effective cure for hematologic diseases such as leukemia and lymphoma135.”
From Stem cell aging: mechanisms, regulators and therapeutic opportunities (2014) · cites this paper as [135]

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HLA-matched related hematopoietic cell transplantation for chronic-phase CML using a targeted busulfan and cyclophosphamide preparative regimen
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Hyperacute graft-v-host disease in patients not given immunosuppression after allogeneic marrow transplantation
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Glutathione S-transferase M1 polymorphism: a risk factor for hepatic venoocclusive disease in bone marrow transplantation
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The DBY gene codes for an HLA-DQ5–restricted human male-specific minor histocompatibility antigen involved in graft-versus-host disease
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Hepatic injury after nonmyeloablative conditioning followed by allogeneic hematopoietic cell transplantation: a study of 193 patients
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Homografts of Bone Marrow in Dogs After Lethal Total-Body Radiation
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Peripheral blood stem cell donation: an analysis from the International Bone Marrow Transplant Registry (IBMTR) and European Group for Blood and Marrow Transplant (EBMT) databases
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Host conditioning with total lymphoid irradiation and antithymocyte globulin prevents graft-versus-host disease: the role of CD1-reactive natural killer T cells
Lan, F., Zeng, D., Higuchi, M., Higgins, J., & Strober, S. (2003) · Transplantation and Cellular Therapy · doi:10.1016/s1083-8791(03)00108-3 · Cited by 142 · see citing works
Glutathione S-Transferase M1 polymorphism – A risk factor for hepatic venoocclusive disease in bone marrow transplantation
Srivastava, A., Poonkughali, B., Shaji, R., George, B., Mathews, V., Chandy, M., Krishnamoorthy, R., & Dominic, J. (2004) · Indian Journal of Medical and Paediatric Oncology · doi:10.1055/s-0041-1733094 · Cited by 130 · see citing works
Unrelated umbilical cord blood transplants in adults: Early recoveryof neutrophils by supportive co-transplantation of a low number of highlypurified peripheral blood CD34+ cells from an HLA-haploidentical donor
Mn, F., Regidor, C., Cabrera, R., Garcia-Marco, J. A., Forés, R., Sanjuán, I., Gayoso, J., Gil, S., Ruiz, E., Little, A., et al. (2003) · Experimental Hematology · doi:10.1016/s0301-472x(03)00067-5 · Cited by 127 · see citing works
Etanercept (Enbrel) administration for idiopathic pneumonia syndrome after allogeneic hematopoietic stem cell transplantation
Yanik, G. A., Hellerstedt, B. A., Custer, J. R., Hutchinson, R. J., Kwon, D., Ferrara, J. L. M., Uberti, J. P., & Cooke, K. R. (2002) · Transplantation and Cellular Therapy · doi:10.1053/bbmt.2002.v8.pm12171486 · Cited by 125 · see citing works
Sperm banking and assisted reproduction treatment for couples following cancer treatment of the male partner
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Emergent autoimmunity in graft-versus-host disease
Tivol, E. A., KOMOROWSKI, R. A., & Drobyski, W. R. (2005) · Blood · doi:10.1182/blood-2004-12-4980 · Cited by 121 · see citing works
Acute lung injury after allogeneic stem cell transplantation: is the lung a target of acute graft-versus-host disease?
Cooke, K. R., & Yanik, G. A. (2004) · Bone Marrow Transplantation · doi:10.1038/sj.bmt.1704629 · Cited by 113 · see citing works
Embryonic Stem Cells As an Alternate Marrow Donor Source
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Final adult height of patients who received hematopoietic cell transplantation in childhood
Sanders, J. E., Guthrie, K. A., Hoffmeister, P. A., Woolfrey, A. E., Carpenter, P. A., & Appelbaum, F. R. (2004) · Blood · doi:10.1182/blood-2004-07-2528 · Cited by 104 · see citing works
Fertility and ovarian function are preserved in women treated with an intensified regimen of cyclophosphamide, adriamycin, vincristine and prednisone (Mega-CHOP) for non-Hodgkin lymphoma
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Economics, health care systems and utilization of haematopoietic stem cell transplants in Europe
Gratwohl, A. A., Passweg, J. R., Baldomero, H., Horisberger, B., & Urbano-Ispizúa, Á. (2002) · British Journal of Haematology · doi:10.1046/j.1365-2141.2002.03440.x · Cited by 66 · see citing works
Autografts of Bone Marrow in Dogs After Lethal Total-Body Radiation
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Allogeneic marrow transplants in cyclophosphamide treated mice.
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Lowered-intensity preparative regimen for allogeneic stem cell transplantation delays acute graft-versus-host disease but does not improve outcome for advanced hematologic malignancy
Levine, J. E., Uberti, J. P., Ayash, L. J., Reynolds, C., Ferrara, J. L. M., Silver, S. M., Braun, T., Yanik, G. A., Hutchinson, R. J., & Ratanatharathorn, V. (2003) · Transplantation and Cellular Therapy · doi:10.1016/s1083-8791(03)70009-3 · Cited by 54 · see citing works
CD28 costimulation and immunoaffinity-based selection efficiently generate primary gene-modified T cells for adoptive immunotherapy
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The hemochromatosis C282Y allele: a risk factor for hepatic veno-occlusive disease after hematopoietic stem cell transplantation
Kallianpur, A. R., Hall, L. D., Yadav, M., Byrne, D. W., Speroff, T., Dittus, R. S., Haines, J. L., Christman, B. W., & Summar, M. L. (2005) · Bone Marrow Transplantation · doi:10.1038/sj.bmt.1704943 · Cited by 41 · see citing works
Repifermin (keratinocyte growth factor-2) reduces the severity of graft-versus-host disease while preserving a graft-versus-leukemia effect
Clouthier, S. G., Cooke, K. R., Teshima, T., Lowler, K. P., Liu, C., Connolly, K. M., & Ferrara, J. L. M. (2003) · Transplantation and Cellular Therapy · doi:10.1016/s1083-8791(03)00230-1 · Cited by 20 · see citing works
Nonmyeloablative Therapy and Hematopoietic Cell Transplantation for Hematologic Disorders
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Risk-assessment in diffuse large cell lymphoma at first relapse. A study by the Italian Intergroup for Lymphomas.
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National estimates of the use of hematopoietic stem-cell transplantation in children with cancer in the United States
Radeva, J. I., VanScoyoc, E., Smith, F. O., Curtis, L. H., & Breitfeld, P. P. (2005) · Bone Marrow Transplantation · doi:10.1038/sj.bmt.1705077 · Cited by 11 · see citing works
Erratum: Peyer's patch is the essential site in initiating murine acute and lethal graft-versus-host reaction
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New approaches and procedures for cancer treatment: Current perspectives
Debela, D. T., Muzazu, S. G., Heraro, K. D., Ndalama, M. T., Mesele, B. W., Haile, D. C., Kitui, S. K., & Manyazewal, T. (2021) · SAGE Open Medicine · doi:10.1177/20503121211034366 · Cited by 1,597 · see citing works
Acute myeloid leukaemia
Estey, E. H., & Döhner, H. (2006) · The Lancet · doi:10.1016/s0140-6736(06)69780-8 · Cited by 1,302 · see citing works
Gene therapy returns to centre stage
Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184 · see citing works
HLA Match Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry
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The effects of intestinal tract bacterial diversity on mortality following allogeneic hematopoietic stem cell transplantation
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Stem cell aging: mechanisms, regulators and therapeutic opportunities
Oh, J., Lee, Y. D., & Wagers, A. J. (2014) · Nature Medicine · doi:10.1038/nm.3651 · Cited by 796 · see citing works
Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study
Eapen, M., Rubinstein, P., Zhang, M., Stevens, C. E., Kurtzberg, J., Scaradavou, A., Loberiza, F. R., Champlin, R. E., Klein, J. P., Horowitz, M. M., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60915-5 · Cited by 789 · see citing works
Hematopoietic Stem Cell Transplantation A Global Perspective
Gratwohl, A. A. (2010) · JAMA · doi:10.1001/jama.2010.491 · Cited by 670 · see citing works
NKAML: A pilot study to determine the safety and feasibility of haploidentical natural killer cell transplantation in childhood acute myeloid leukemia
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Cancer treatment therapies: traditional to modern approaches to combat cancers
Kaur, R., Bhardwaj, A., & Gupta, S. K. (2023) · Molecular Biology Reports · doi:10.1007/s11033-023-08809-3 · Cited by 602 · see citing works
Microbial translocation augments the function of adoptively transferred self/tumor-specific CD8+ T cells via TLR4 signaling
Paulos, C. M., Wrzesinski, C., Kaiser, Hinrichs, C. S., Chieppa, M., Cassard, L., Palmer, D. C., Boni, A., Muranski, P., Yu, Z., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci32205 · Cited by 586 · see citing works
Effect of graft source on unrelated donor haemopoietic stem-cell transplantation in adults with acute leukaemia: a retrospective analysis
Eapen, M., Rocha, V., Sanz, G. F., Scaradavou, A., Zhang, M., Arcese, W., Sîrvent, A., Champlin, R. E., Chao, N. J. A., Gee, A. P., et al. (2010) · The Lancet Oncology · doi:10.1016/s1470-2045(10)70127-3 · Cited by 583 · see citing works
Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation
Leen, A. M., Bollard, C. M., Mendizabal, A. M., Shpall, E. J., Szabolcs, P., Antin, J. H., Kapoor, N., Pai, S., Rowley, S. D., Kebriaei, P., et al. (2013) · Blood · doi:10.1182/blood-2013-02-486324 · Cited by 552 · see citing works
Overview of the mucopolysaccharidoses
Muenzer, J. (2011) · Rheumatology · doi:10.1093/rheumatology/ker394 · Cited by 495 · see citing works
Hypoxic mesenchymal stem cell-derived exosomes promote bone fracture healing by the transfer of miR-126
Liu, W., Li, L., Rong, Y., Qian, D., Chen, J., Zhou, Z. Y., Luo, Y., Jiang, D., Cheng, L., Zhao, S., et al. (2019) · Acta Biomaterialia · doi:10.1016/j.actbio.2019.12.020 · Cited by 473 · see citing works
Mucopolysaccharidosis I: Management and Treatment Guidelines
Muenzer, J., Wraith, J. E., Clarke, L. A., & and the International Consensus Panel on the Management and Treatment of Mucopolysaccharidosis, I. (2008) · PEDIATRICS · doi:10.1542/peds.2008-0416 · Cited by 462 · see citing works
Fully functional bioengineered tooth replacement as an organ replacement therapy
Ikeda, E., Morita, R., Nakao, K., Ishida, K., Nakamura, T., Takano-Yamamoto, T., Ogawa, M., Mizuno, M., Kasugai, S., & Tsuji, T. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0902944106 · Cited by 447 · see citing works
One million haemopoietic stem-cell transplants: a retrospective observational study
Gratwohl, A. A., Pasquini, M. C., Aljurf, M. D., Atsuta, Y., Baldomero, H., Foeken, L., Gratwohl, M., Bouzas, L. F., Confer, D. L., Frauendorfer, K., et al. (2015) · The Lancet Haematology · doi:10.1016/s2352-3026(15)00028-9 · Cited by 444 · see citing works
HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders
Bertaina, A., Merli, P., Rutella, S., Pagliara, D., Bernardo, M. E., Masetti, R., Pende, D., Falco, M., Handgretinger, R., Moretta, F., et al. (2014) · Blood · doi:10.1182/blood-2014-03-563817 · Cited by 435 · see citing works
Long-term ex vivo haematopoietic-stem-cell expansion allows nonconditioned transplantation
Wilkinson, A. C., Ishida, R., Kikuchi, M., Sudo, K., Morita, M., Crisostomo, R. V., Yamamoto, R., Loh, K. M., Nakamura, Y., Watanabe, M., et al. (2019) · Nature · doi:10.1038/s41586-019-1244-x · Cited by 411 · see citing works
Spermatogonial Stem Cell Transplantation into Rhesus Testes Regenerates Spermatogenesis Producing Functional Sperm
Hermann, B. P., Sukhwani, M., Winkler, F., Pascarella, J. N., Peters, K. A., Sheng, Y., Pulaski, H., Rodriguez, M., Ezzelarab, M. B., Dargo, G., et al. (2012) · Cell stem cell · doi:10.1016/j.stem.2012.07.017 · Cited by 431 · see citing works
Optimizing Autologous Stem Cell Mobilization Strategies to Improve Patient Outcomes: Consensus Guidelines and Recommendations
Giralt, S. A., Costa, L. J., Schriber, J. R., DiPersio, J. F., Maziarz, R. T., McCarty, J. M., Shaughnessy, P. J., Snyder, E. L., Bensinger, W., Copelan, E. A., et al. (2013) · Transplantation and Cellular Therapy · doi:10.1016/j.bbmt.2013.10.013 · Cited by 408 · see citing works
Self-citation
Immune signature drives leukemia escape and relapse after hematopoietic cell transplantation
Toffalori, C., Zito, L., Gambacorta, V., Riba, M., Oliveira, G., Bucci, G., Barcella, M., Spinelli, O., Greco, R., Crucitti, L., et al. (2019) · Nature Medicine · doi:10.1038/s41591-019-0400-z · Cited by 405 · see citing works
Hematopoietic stem cell transplantation in Europe 2014: more than 40 000 transplants annually
for the European Society for Blood and Marrow Transplantation (EBMT), Passweg, J. R., Baldomero, H., Bader, P., Bonini, C., Cesaro, S., Dreger, P., Duarte, R. F., Dufour, C., Kuball, J. H. E., et al. (2016) · Bone Marrow Transplantation · doi:10.1038/bmt.2016.20 · Cited by 402 · see citing works
Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report
for the European Society for Blood and Marrow Transplantation (EBMT), Passweg, J. R., Baldomero, H., Bader, P., Bonini, C., Duarte, R. F., Dufour, C., Gennery, A. R., Kröger, N. M., Kuball, J. H. E., et al. (2017) · Bone Marrow Transplantation · doi:10.1038/bmt.2017.34 · Cited by 391 · see citing works
NK cells mediate reduction of GVHD by inhibiting activated, alloreactive T cells while retaining GVT effects
Olson, J. A., Leveson-Gower, D. B., Gill, S. I., Baker, J. B., Beilhack, A., & Negrin, R. S. (2010) · Blood · doi:10.1182/blood-2009-05-222190 · Cited by 387 · see citing works
Ex vivo gene transfer and correction for cell-based therapies
Naldini, L. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2985 · Cited by 386 · see citing works
Risk score for outcome after allogeneic hematopoietic stem cell transplantation
Gratwohl, A. A., Stern, M., Brand, R., Apperley, J. F., Baldomero, H., de Witte, T., Dini, G., Rocha, V., Passweg, J. R., Sureda, A. M., et al. (2009) · Cancer · doi:10.1002/cncr.24531 · Cited by 380 · see citing works
Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial
Corbacioglu, S., Cesaro, S., Faraci, M., Valteau‐Couanet, D., Gruhn, B., Rovelli, A., Boelens, J. J., Hewitt, A., Schrum, J., Schulz, A., et al. (2012) · The Lancet · doi:10.1016/s0140-6736(11)61938-7 · Cited by 373 · see citing works
Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases
Farge, D., Labopin, M., Tyndall, A., Fassas, Α., Mancardi, G., van Laar, J. M., Ouyang, J., Kozák, T., Moore, J. J., Kötter, I., et al. (2009) · Haematologica · doi:10.3324/haematol.2009.013458 · Cited by 360 · see citing works
Easing of Suffering in Children With Cancer at the End of Life: Is Care Changing?
Wolfe, J., Hammel, J. F., Edwards, K. E., Duncan, J., Comeau, M., Breyer, J., Aldridge, S. A., Grier, H. E., Berde, C., Dussel, V., et al. (2008) · Journal of Clinical Oncology · doi:10.1200/jco.2007.14.0277 · Cited by 344 · see citing works
Mesenchymal stromal cell therapies: immunomodulatory properties and clinical progress
Wu, X., Jiang, J., Gu, Z., Zhang, J., Chen, Y., & Liu, X. (2020) · Stem Cell Research & Therapy · doi:10.1186/s13287-020-01855-9 · Cited by 343 · see citing works
Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response
Schiroli, G., Conti, A., Ferrari, S., della Volpe, L., Jacob, A., Albano, L., Beretta, S., Calabria, A., Vavassori, V., Gasparini, P., et al. (2019) · Cell Stem Cell · doi:10.1016/j.stem.2019.02.019 · Cited by 342 · see citing works
Phase 1 clinical trial using mbIL21 ex vivo–expanded donor-derived NK cells after haploidentical transplantation
Ciurea, S. O., Schafer, J. R., Bassett, R. L., Denman, C. J., Cao, K., Willis, D. B., Rondón, G., Chen, J. J., Soebbing, D., Kaur, I., et al. (2017) · Blood · doi:10.1182/blood-2017-05-785659 · Cited by 342 · see citing works
The Transcription Factor EGR1 Controls Both the Proliferation and Localization of Hematopoietic Stem Cells
Min, I. M., Pietramaggiori, G., Kim, F. S., Passegue, E. A., Stevenson, K. E., & Wagers, A. J. (2008) · Cell stem cell · doi:10.1016/j.stem.2008.01.015 · Cited by 327 · see citing works
Physical, psychological, and social sequelae following hematopoietic stem cell transplantation: a review of the literature
Mosher, C. E., Redd, W. H., Rini, C. M., Burkhalter, J. E., & DuHamel, K. N. (2009) · Psycho-Oncology · doi:10.1002/pon.1399 · Cited by 315 · see citing works
Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey
Niederwieser, D. W., Baldomero, H., Szer, J., Gratwohl, M., Aljurf, M. D., Atsuta, Y., Bouzas, L. F. S., Confer, D. L., Greinix, H. T., Horowitz, M. M., et al. (2016) · Bone Marrow Transplantation · doi:10.1038/bmt.2016.18 · Cited by 312 · see citing works
Rapid mobilization of functional donor hematopoietic cells without G-CSF using AMD3100, an antagonist of the CXCR4/SDF-1 interaction
Devine, S. M., Vij, R., Rettig, M. P., Todt, L. M., McGlauchlen, K., Fisher, N. M., Devine, H., Link, D. C., Calandra, G., Bridger, G. J., et al. (2008) · Blood · doi:10.1182/blood-2007-12-130179 · Cited by 307 · see citing works
The immune system and cancer evasion strategies: therapeutic concepts
Muenst, S., Läubli, H., Soysal, S. D., Zippelius, A., Tzankov, A., & Hoeller, S. (2016) · Journal of Internal Medicine · doi:10.1111/joim.12470 · Cited by 304 · see citing works
Adherence and biofilm formation of non-Candida albicans Candida species
Silva, S., Negri, M., Henriques, M., Oliveira, R., Williams, D. W., & Azeredo, J. (2011) · Trends in Microbiology · doi:10.1016/j.tim.2011.02.003 · Cited by 304 · see citing works
Inhibition of the prostaglandin-degrading enzyme 15-PGDH potentiates tissue regeneration
Zhang, Y., Desai, A. B., Yang, S. Y., Bae, K. B., Antczak, M. I., Fink, S. P., Tiwari, S. R., Willis, J. E., Williams, N. S., Dawson, D. M., et al. (2015) · Science · doi:10.1126/science.aaa2340 · Cited by 297 · see citing works
Acute graft-versus-host disease
Malard, F., Holler, E., Sandmaier, B. M., Huang, H., & Mohty, M. (2023) · Nature Reviews Disease Primers · doi:10.1038/s41572-023-00438-1 · Cited by 290 · see citing works
High Risk of Graft Failure in Patients With Anti-HLA Antibodies Undergoing Haploidentical Stem-Cell Transplantation
Ciurea, S. O., Lima, M. J. G. D., Cano, P., Korbling, M. J., Giralt, S. A., Shpall, E. J., Wang, X., Thall, P. F., Champlin, R. E., & Fernández-Viña, M. (2009) · Transplantation · doi:10.1097/tp.0b013e3181b9d710 · Cited by 287 · see citing works
Remyelination therapies: a new direction and challenge in multiple sclerosis
Plemel, J. R., Liu, W., & Yong, V. W. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2017.115 · Cited by 281 · see citing works
Effects of a partly self-administered exercise program before, during, and after allogeneic stem cell transplantation
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Authors: Copelan, E. A.
Affiliations: The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (US)
Corresponding author: Copelan, Edward A.
ORCID: Copelan, Edward A.
Venue: New England Journal of Medicine
Publisher: Massachusetts Medical Society
Published: 2006-04-27
Volume: 354
Issue: 17
Pages: 1813-1826
ISSN: 0028-4793 (ISSN-L)
1533-4406
DOI: 10.1056/nejmra052638
OpenAlex: W2160666689
Cited by: 2,522
References: 102
Citations by year: 2026: 89 · 2025: 99 · 2024: 121 · 2023: 143 · 2022: 163 · 2021: 149 · 2020: 128 · 2019: 126 · 2018: 128 · 2017: 161 · 2016: 123 · 2015: 162 · 2014: 142 · 2013: 142 · 2012: 120
FWCI: 10.56
Type: journal article
Language: en
Open access: closed
Locations: New England Journal of Medicine (publishedVersion)
PubMed (publishedVersion)
DigitalGeorgetown (Georgetown University Library) (submittedVersion)
Topics: Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
Health Sciences › Medicine › Surgery › Transplantation: Methods and Outcomes
Health Sciences › Medicine › Genetics › Mesenchymal stem cell research
Keywords: hematopoietic stem cell transplantation, hematopoietic stem cells, immunomodulation, history of transplantation
MeSH terms: Graft vs Host Disease, Humans, Major Histocompatibility Complex, Transplantation Immunology, Transplantation, Autologous, Transplantation, Homologous, Hematopoietic Stem Cell Transplantation, Transplantation Conditioning, Cord Blood Stem Cell Transplantation

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