Abstract
The recent improvements in the outcomes of severe aplastic anemia (SAA) patients who received allogeneic stem cell transplantation (SCT) from unrelated donors (URD) suggest the possibility of its alternative first-line treatment. To address this issue, results of adult SAA patients receiving allogeneic SCT were compared between the following three donor-type groups: 8/8–matched sibling (MSD; n = 153), 8/8 well-matched unrelated (WM-URD; n = 72), and 6–7/8 partially matched unrelated (PM-URD; n = 33). Proportion of patients who experienced immunosuppressive treatment failures was significantly higher in the URD groups than in the MSD group (P < 0.01). The incidences of graft failure and transplant-related mortality, and graft-vs.-host disease-free, failure-free survival rates of the MSD, WM-URD, and PM-URD groups were 14.6, 0, and 0% (P < 0.01); 6.1, 10.3, and 21.7% (P = 0.03); and 76.7, 55.5, and 51.5% (P < 0.01), respectively. The overall survival (OS) rate of the MSD group (93.9%) was higher than that of the PM-URD (78.3%; P < 0.01) group, but not to that of the WM-URD (86.2%; P = 0.18) group. Our study showed comparable OS between the MSD group and WM-URD group, which suggest that the URD-SCT can be used as a first-line treatment for adult SAA patients with WM-URD.
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References
Shin SH, Lee SE, Lee JW. Recent advances in treatment of aplastic anemia. Korean J Intern Med. 2014;29:713–26.
Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120:1185–96.
Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172:187–207.
Deeg HJ, O’Donnell M, Tolar J, Agarwal R, Harris RE, Feig SA, et al. Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy. Blood. 2006;108:1485–91.
Kojima S, Matsuyama T, Kato S, Kigasawa H, Kobayashi R, Kikuta A, et al. Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program. Blood. 2002;100:799–803.
Passweg JR, Perez WS, Eapen M, Camitta BM, Gluckman E, Hinterberger W, et al. Bone marrow transplants from mismatched related and unrelated donors for severe aplastic anemia. Bone Marrow Transpl. 2006;37:641–9.
Shin SH, Lee JW. The optimal immunosuppressive therapy for aplastic anemia. Int J Hematol. 2013;97:564–72.
Maury S, Balere-Appert ML, Chir Z, Boiron JM, Galambrun C, Yakouben K, et al. Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient. Haematologica. 2007;92:589–96.
Viollier R, Socie G, Tichelli A, Bacigalupo A, Korthof ET, Marsh J, et al. Recent improvement in outcome of unrelated donor transplantation for aplastic anemia. Bone Marrow Transpl. 2008;41:45–50.
Yagasaki H, Takahashi Y, Hama A, Kudo K, Nishio N, Muramatsu H, et al. Comparison of matched-sibling donor BMT and unrelated donor BMT in children and adolescent with acquired severe aplastic anemia. Bone Marrow Transpl. 2010;45:1508–13.
Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015;171:585–94.
Marsh JCW, Risitano AM, Mufti GJ. The case for upfront HLA-matched unrelated donor hematopoietic stem cell transplantation as a curative option for adult acquired severe aplastic anemia. Biol Blood Marrow Transpl. 2019;25:e277–84.
Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X, et al. Comparable outcomes of first-line hematopoietic stem cell transplantation from unrelated and matched sibling donors in adult patients with aplastic anemia: a retrospective single-center study. Biol Blood Marrow Transpl. 2019;25:1567–75.
Vaht K, Goransson M, Carlson K, Isaksson C, Lenhoff S, Sandstedt A, et al. High graft-versus-host disease-free, relapse/rejection-free survival and similar outcome of related and unrelated allogeneic stem cell transplantation for aplastic anemia: a nationwide swedish cohort study. Biol Blood Marrow Transpl. 2019;25:1970–4.
Park SS, Kwak DH, Jeon YW, Yoon JH, Lee SE, Cho BS, et al. Beneficial role of low-dose antithymocyte globulin in unrelated stem cell transplantation for adult patients with acquired severe aplastic anemia: reduction of graft-versus-host disease and improvement of graft-versus-host disease-free, failure-free survival rate. Biol Blood Marrow Transpl. 2017;23:1498–508.
Shin SH, Jeon YW, Yoon JH, Yahng SA, Lee SE, Cho BS, et al. Comparable outcomes between younger (40 years) and older (>40 years) adult patients with severe aplastic anemia after HLA-matched sibling stem cell transplantation using fludarabine-based conditioning. Bone Marrow Transpl. 2016;51:1456–63.
Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N. Engl J Med. 1982;306:645–52.
Sorror ML, Maris MB, Storb R, Baron F, Sandmaier BM, Maloney DG, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106:2912–9.
Champlin RE, Perez WS, Passweg JR, Klein JP, Camitta BM, Gluckman E, et al. Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens. Blood. 2007;109:4582–5.
Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transpl. 1995;15:825–8.
Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transpl. 2005;11:945–56.
McDonald GB, Hinds MS, Fisher LD, Schoch HG, Wolford JL, Banaji M, et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118:255–67.
Silva Lde P, Patah PA, Saliba RM, Szewczyk NA, Gilman L, Neumann J, et al. Hemorrhagic cystitis after allogeneic hematopoietic stem cell transplants is the complex result of BK virus infection, preparative regimen intensity and donor type. Haematologica. 2010;95:1183–90.
The National Cancer Institute Common Terminology Criteria for adverse events version 3.0. Available at: Common Terminology Criteria for Adverse Events v3.0. http://ctep.cancer.gov/protocolDevelopment/electronic_applications/docs/ctcaev3.pdf. Accessed 17 January 2020.
Holtan SG, DeFor TE, Lazaryan A, Bejanyan N, Arora M, Brunstein CG, et al. Composite end point of graft-versus-host disease-free, relapse-free survival after allogeneic hematopoietic cell transplantation. Blood. 2015;125:1333–8.
Austin PC. An introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivar Behav Res. 2011;46:399–424.
Bacigalupo A, Socie G, Hamladji RM, Aljurf M, Maschan A, Kyrcz-Krzemien S, et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica. 2015;100:696–702.
Bacigalupo A, Socie G, Schrezenmeier H, Tichelli A, Locasciulli A, Fuehrer M, et al. Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: survival advantage for bone marrow in all age groups. Haematologica. 2012;97:1142–8.
Schrezenmeier H, Passweg JR, Marsh JC, Bacigalupo A, Bredeson CN, Bullorsky E, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007;110:1397–400.
Eapen M, Le Rademacher J, Antin JH, Champlin RE, Carreras J, Fay J, et al. Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia. Blood. 2011;118:2618–21.
MacMillan ML, Robin M, Harris AC, DeFor TE, Martin PJ, Alousi A, et al. A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biol Blood Marrow Transpl. 2015;21:761–7.
Fiuza-Luces C, Simpson RJ, Ramirez M, Lucia A, Berger NA. Physical function and quality of life in patients with chronic GvHD: a summary of preclinical and clinical studies and a call for exercise intervention trials in patients. Bone Marrow Transpl. 2016;51:13–26.
Servais S, Beguin Y, Delens L, Ehx G, Fransolet G, Hannon M, et al. Novel approaches for preventing acute graft-versus-host disease after allogeneic hematopoietic stem cell transplantation. Expert Opin Investig Drugs. 2016;25:957–72.
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Shin, S.H., Park, S.S., Yoon, J.H. et al. Comparison of HLA-matched sibling and unrelated donor transplantation in adult patients with acquired severe aplastic anemia. Bone Marrow Transplant 55, 1570–1579 (2020). https://doi.org/10.1038/s41409-020-0820-x
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DOI: https://doi.org/10.1038/s41409-020-0820-x
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