Marsh JC, Ball SE, Cavenagh J, Darbyshire P, Dokal I, Gordon-Smith EC, et al. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009;147(1):43–70.

Article  PubMed  CAS  Google Scholar 

Yoshida N, Kojima S. Updated guidelines for the treatment of acquired aplastic anemia in children. Curr Oncol Rep. 2018;20(9):67.

Article  PubMed  Google Scholar 

Qin X, Zhu YP, Luo CJ, Zhou M, Huang K, Chen C, et al. Optimizing conditioning regimen with low-dose irradiation or busulfan enables the outcome of transplantation from a 6–7/8 HLA-matched donor comparable to that from an 8/8 HLA-matched unrelated donor in severe aplastic anemia patients under 40 years. Ann Hematol. 2021;100(9):2363–73.

Article  PubMed  CAS  Google Scholar 

Young NS. Aplastic anemia. N Engl J Med. 2018;379(17):1643–56.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120(6):1185–96.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376(16):1540–50.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Kahl C, Leisenring W, Deeg HJ, Chauncey TR, Flowers ME, Martin PJ, et al. Cyclophosphamide and antithymocyte globulin as a conditioning regimen for allogeneic marrow transplantation in patients with aplastic anaemia: a long-term follow-up. Br J Haematol. 2005;130(5):747–51.

Article  PubMed  CAS  Google Scholar 

Bacigalupo A, Socie G, Lanino E, Prete A, Locatelli F, Locasciulli A, et al. Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA Working Party. Haematologica. 2010;95(6):976–82.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Samarasinghe S, Webb DK. How I manage aplastic anaemia in children. Br J Haematol. 2012;157(1):26–40.

Article  PubMed  CAS  Google Scholar 

Bejanyan N, Kim S, Hebert KM, Kekre N, Abdel-Azim H, Ahmed I, et al. Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Adv. 2019;3(20):3123–31.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Lawler M, McCann SR, Marsh JC, Ljungman P, Hows J, Vandenberghe E, et al. Serial chimerism analyses indicate that mixed haemopoietic chimerism influences the probability of graft rejection and disease recurrence following allogeneic stem cell transplantation (SCT) for severe aplastic anaemia (SAA): indication for routine assessment of chimerism post SCT for SAA. Br J Haematol. 2009;144(6):933–45.

Article  PubMed  CAS  Google Scholar 

Ashizawa M, Akahoshi Y, Nakano H, Ugai T, Wada H, Yamasaki R, et al. A combination of fludarabine, half-dose cyclophosphamide, and anti-thymocyte globulin is an effective conditioning regimen before allogeneic stem cell transplantation for aplastic anemia. Int J Hematol. 2014;99(3):311–7.

Article  PubMed  CAS  Google Scholar 

Haines HL, Bleesing JJ, Davies SM, Hornung L, Jordan MB, Marsh RA, et al. Outcomes of donor lymphocyte infusion for treatment of mixed donor chimerism after a reduced-intensity preparative regimen for pediatric patients with nonmalignant diseases. Biol Blood Marrow Transplant. 2015;21(2):288–92.

Article  PubMed  Google Scholar 

Muskens KF, Lindemans CA, Dandis R, Nierkens S, Belderbos ME. Definitions, incidence and outcome of poor graft function after hematopoietic cell transplantation: A systematic review and meta-analysis. Blood Rev. 2023;60: 101076.

Article  PubMed  Google Scholar 

Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995;15(6):825–8.

PubMed  CAS  Google Scholar 

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 Transplant. 2005;11(12):945–56.

Article  PubMed  Google Scholar 

ElGohary G, El Fakih R, de Latour R, Risitano A, Marsh J, Schrezenmeier H, et al. Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant. 2020;55(10):1906–17.

Article  PubMed  Google Scholar 

Dufour C, Pillon M, Socie G, Rovo A, Carraro E, Bacigalupo A, et al. Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant. Br J Haematol. 2015;169(4):565–73.

Article  PubMed  Google Scholar 

Yoshida N. Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia. Int J Hematol. 2023;119:240–7.

Article  PubMed  Google Scholar 

Kikuchi A, Yabe H, Kato K, Koh K, Inagaki J, Sasahara Y, et al. Long-term outcome of childhood aplastic anemia patients who underwent allogeneic hematopoietic SCT from an HLA-matched sibling donor in Japan. Bone Marrow Transplant. 2013;48(5):657–60.

Article  PubMed  CAS  Google Scholar 

Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica. 2019;104(10):1974–83.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Zhang Y, Chen X, Li L, Li Y, Lin L, Cao Y, et al. Retrospective comparison of efficacy and safety of rabbit anti-thymocyte globulin and porcine anti-lymphocyte globulin in patients with acquired aplastic anemia undergoing hematopoietic stem cell transplantation from matched sibling donors. Front Immunol. 2022;13: 889784.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Chorao P, Montoro J, Balaguer-Rosello A, Guerreiro M, Villalba M, Facal A, et al. T Cell-Depleted Peripheral Blood versus Unmanipulated Bone Marrow in Matched Sibling Transplantation for Aplastic Anemia. Transplant Cell Ther. 2023;29(5):322.e1-322.e5.

Article  PubMed  CAS  Google Scholar 

Storb R, Etzioni R, Anasetti C, Appelbaum FR, Buckner CD, Bensinger W, et al. Cyclophosphamide Combined With Antithymocyte Globulin in Preparation for Allogeneic Marrow Transplants in Patients With Aplastic Anemia. Blood. 1994;1(3):941–9.

Article  Google Scholar 

Kang HJ, Shin HY, Choi HS, Ahn HS. Fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia. Bone Marrow Transplant. 2004;34(11):939–43.

Article  PubMed  CAS  Google Scholar 

Gottdiener JS, Appelbaum FR, Ferrans VJ, Deisseroth A, Ziegler J. Cardiotoxicity associated with high-dose cyclophosphamide therapy. Arch Intern Med. 1981;141(6):758–63.

Article  PubMed  CAS  Google Scholar 

Tolar J, Deeg HJ, Arai S, Horwitz M, Antin JH, McCarty JM, et al. Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels. Biol Blood Marrow Transplant. 2012;18(7):1007–11.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Anderlini P, Wu J, Gersten I, Ewell M, Tolar J, Antin JH, et al. Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1–2 dose de-escalation study. Lancet Haematol. 2015;2(9):e367–75.

Article  PubMed  PubMed Central  Google Scholar 

Yoshida N, Takahashi Y, Yabe H, Kobayashi R, Watanabe K, Kudo K, et al. Conditioning regimen for allogeneic bone marrow transplantation in children with acquired bone marrow failure: fludarabine/melphalan vs. fludarabine/cyclophosphamide. Bone Marrow Transplant. 2020;55(7):1272–81.

Article  PubMed  CAS