Outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) for hepatitis-associated aplastic anemia have not been fully evaluated. In the present study, the outcomes of 37 adult patients with hepatitis-associated aplastic anemia who underwent allogeneic HSCT were retrospectively analyzed using the registry database of Japan Society for Hematopoietic Cell Transplantation. The median age of the patients was 24 years (range, 16–61). The median period between diagnosis of hepatitis-associated aplastic anemia and HSCT was 6.0 months (range, 0.5–430.8). Stem cell sources were bone marrow (N = 19) or peripheral blood stem cells (N = 5) from an HLA-identical sibling or bone marrow (N = 11) and cord blood (N = 2) from an unrelated donor. The majority of conditioning regimens were fludarabine-based or high-dose cyclophosphamide-based. In all but 2 cases of early death, neutrophil engraftment was achieved. At the time of analysis, 32 patients were alive, with a median follow-up of 54.1 months. Five-year overall and failure-free survival rates were 86.0% (95% CI, 69.4–93.9%) and 75.0% (95% CI, 57.4–86.2%), respectively. Despite the heterogeneity in transplant procedures in a small number of patients, these results suggest that allogeneic HSCT is safe for use in hepatitis-associated aplastic anemia with a low rate of transplant-related mortality.
This is a preview of subscription content, log in to check access.
The authors would like to thank all the physicians and data managers of each transplant center and the staff member of Japanese Data Center for Hematopoietic Cell Transplantation.
Compliance with ethical standards
Conflict of interest
Mineo Kurokawa receives consulting fees from Sanofi.k.k.
Brown KE, Tisdale J, Barrett AJ, Dunbar CE, Young NS. Hepatitis-associated aplastic anemia. N Engl J Med. 1997;336:1059–64.CrossRefGoogle Scholar
Rauff B, Idrees M, Shah SA, Butt S, Butt AM, Ali L, et al. Hepatitis-associated aplastic anemia: a review. Virol J. 2011;8:87.CrossRefGoogle Scholar
Gonzalez-Casas R, Garcia-Buey L, Jones EA, Gisbert JP, Moreno-Otero R. Systematic review: hepatitis-associated aplastic anaemia—a syndrome associated with abnormal immunological function. Aliment Pharmacol Ther. 2009;30:436–43.CrossRefGoogle Scholar
Osugi Y, Yagasaki H, Sako M, Kosaka Y, Taga T, Ito T, et al. Antithymocyte globulin and cyclosporine for treatment of 44 children with hepatitis-associated aplastic anemia. Haematologica. 2007;92:1687–90.CrossRefGoogle Scholar
Hayakawa J, Kanda J, Akahoshi Y, Harada N, Kameda K, Ugai T, et al. Meta-analysis of treatment with rabbit and horse antithymocyte globulin for aplastic anemia. Int J Hematol. 2017;105:578–86.CrossRefGoogle Scholar
Wang H, Tu M, Fu R, Wu Y, Liu H, Xing L, et al. The clinical and immune characteristics of patients with hepatitis-associated aplastic anemia in China. PLoS One. 2014;9:e98142.CrossRefGoogle Scholar
Witherspoon RP, Storb R, Shulman H, Buckner CD, Deeg HJ, Clift RA, et al. Marrow transplantation in hepatitis-associated aplastic anemia. Am J Hematol. 1984;17:269–78.CrossRefGoogle Scholar
Locasciulli A, Bacigalupo A, Bruno B, Montante B, Marsh J, Tichelli A, et al. Hepatitis-associated aplastic anaemia: epidemiology and treatment results obtained in Europe. A report of The EBMT aplastic anaemia working party. Br J Haematol. 2010;149:890–5.CrossRefGoogle Scholar
Safadi R, Or R, Ilan Y, Naparstek E, Nagler A, Klein A, et al. Lack of known hepatitis virus in hepatitis-associated aplastic anemia and outcome after bone marrow transplantation. Bone Marrow Transplant. 2001;27:183–90.CrossRefGoogle Scholar
Atsuta Y. Introduction of transplant registry unified management program 2 (TRUMP2): scripts for TRUMP data analyses, part I (variables other than HLA-related data). Int J Hematol. 2016;103:3–10.CrossRefGoogle Scholar
Kanda Y. Investigation of the freely available easy-to-use software ‘EZR’ for medical statistics. Bone Marrow Transplant. 2013;48:452–8.CrossRefGoogle Scholar
Mori T, Koh H, Onishi Y, Kako S, Onizuka M, Kanamori H, et al. Impact of cyclophosphamide dose of conditioning on the outcome of allogeneic hematopoietic stem cell transplantation for aplastic anemia from human leukocyte antigen-identical sibling. Int J Hematol. 2016;103:461–8.CrossRefGoogle Scholar
Mary JY, Baumelou E, Guiguet M. Epidemiology of aplastic anemia in France: a prospective multicentric study. The French Cooperative Group for Epidemiological Study of Aplastic Anemia. Blood. 1990;75:1646–53.Google Scholar
Liang DC, Lin KH, Lin DT, Yang CP, Hung KL, Lin KS. Post-hepatitic aplastic anaemia in children in Taiwan, a hepatitis prevalent area. Br J Haematol. 1990;74:487–91.CrossRefGoogle Scholar
Chen HF, Xu BX, Shen HS, Li ZY, Jin LJ, Tang JQ, et al. Efficacy and safety of immunosuppressive therapy in the treatment of seronegative hepatitis-associated aplastic anemia. Drug Des Dev Ther. 2014;8:1299–305.CrossRefGoogle Scholar
Ohara A, Kojima S, Okamura J, Inada H, Kigasawa H, Hibi S, et al. Evolution of myelodysplastic syndrome and acute myelogenous leukaemia in children with hepatitis-associated aplastic anaemia. Br J Haematol. 2002;116:151–4.CrossRefGoogle Scholar
Pongtanakul B, Das PK, Charpentier K, Dror Y. Outcome of children with aplastic anemia treated with immunosuppressive therapy. Pediatr Blood Cancer. 2008;50:52–7.CrossRefGoogle Scholar
Maury S, Bacigalupo A, Anderlini P, Aljurf M, Marsh J, Socie G, et al. Improved outcome of patients older than 30 years receiving HLA-identical sibling hematopoietic stem cell transplantation for severe acquired aplastic anemia using fludarabine-based conditioning: a comparison with conventional conditioning regimen. Haematologica. 2009;94:1312–5.CrossRefGoogle Scholar
Bacigalupo A, Locatelli F, Lanino E, Marsh J, Socie G, Maury S, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005;36:947–50.CrossRefGoogle Scholar