Optimizing outcomes for haploidentical hematopoietic stem cell transplantation in severe aplastic anemia with intensive GVHD prophylaxis: a review of current findings

References

• Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017;129(11):1428–1436.
   PubMed Web of Science ®Google Scholar
• Georges GE, Doney K, Storb R. Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment. Blood Adv. 2018;2(15):2020–2028.
   PubMed Web of Science ®Google Scholar
• Wagner JL, Deeg HJ, Seidel K, et al. Bone marrow transplantation for severe aplastic anemia from genotypically HLA-nonidentical relatives. An update of the Seattle experience. Transplantation. 1996;61(1):54–61.
   PubMed Web of Science ®Google Scholar
• Xu ZL, Huang XJ. Haploidentical stem cell transplantation for aplastic anemia: the current advances and future challenges. Bone Marrow Transplant. 2020;56(4):779–785.
   Web of Science ®Google Scholar
• Kanakry CG, Fuchs EJ, Luznik L. Modern approaches to HLA-haploidentical blood or marrow transplantation. Nat Rev Clin Oncol. 2016;13(1):10–24.
   PubMed Web of Science ®Google Scholar
• Xu LP, Liu KY, Liu DH, et al. A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia. Bone Marrow Transplant. 2012;47(12):1507–1512.
   PubMed Web of Science ®Google Scholar
• Clay J, Kulasekararaj AG, Potter V, et al. Nonmyeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2014;20(11):1711–1716.
   PubMed Web of Science ®Google Scholar
• Xu LP, Wang SQ, Wu DP, et al., Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study. Br J Haematol. 175(2): 265–274. 2016.
   PubMed Web of Science ®Google Scholar
• Xu LP, Jin S, Wang SQ, et al., Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant. J Hematol Oncol. 10(1): 25. 2017.
   PubMed Web of Science ®Google Scholar
• Chang YJ, Zhao XY, Huang XJ. Granulocyte colony-stimulating factor-primed unmanipulated haploidentical blood and marrow transplantation. Front Immunol. 2019;10:2516.
   PubMed Web of Science ®Google Scholar
• Siddiqui S, Cox J, Herzig R, et al. Anti-thymocyte globulin in haematology: recent developments. Indian J Med Res. 2019;150(3):221–227.
   PubMed Web of Science ®Google Scholar
• Nishihori T, Al-Kadhimi Z, Hamadani M, et al. Antithymocyte globulin in allogeneic hematopoietic cell transplantation: benefits and limitations. Immunotherapy. 2016;8(4):435–447.
   PubMed Web of Science ®Google Scholar
• Yang JZ, Zhang JQ, Sun LX. Mechanisms for T cell tolerance induced with granulocyte colony-stimulating factor. Mol Immunol. 2016;70:56–62.
   PubMed Web of Science ®Google Scholar
• Mielcarek M, Martin PJ, Torok-Storb B. Suppression of alloantigen-induced T-cell proliferation by CD14+ cells derived from granulocyte colony-stimulating factor-mobilized peripheral blood mononuclear cells. Blood. 1997;89(5):1629–1634.
   PubMed Web of Science ®Google Scholar
• Arpinati M, Green CL, Heimfeld S, et al. Granulocyte-colony stimulating factor mobilizes T helper 2-inducing dendritic cells. Blood. 2000;95(8):2484–2490.
   PubMed Web of Science ®Google Scholar
• MacDonald KP, Le Texier L, Zhang P, et al. Modification of T cell responses by stem cell mobilization requires direct signaling of the T cell by G-CSF and IL-10. J Immunol. 2014;192(7):3180–3189.
   PubMed Web of Science ®Google Scholar
• Xu LP, Zhang XH, Wang FR, et al. Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia. Bone Marrow Transplant. 2017;52(3):381–387.
   PubMed Web of Science ®Google Scholar
• Xu LP, Xu ZL, Wang FR, et al. Unmanipulated haploidentical transplantation conditioning with busulfan, cyclophosphamide and anti-thymoglobulin for adult severe aplastic anaemia. Bone Marrow Transplant. 2018;53(2):188–192.
   PubMed Web of Science ®Google Scholar
• DeZern AE, Brodsky RA. Haploidentical donor bone marrow transplantation for severe aplastic anemia. Hematol Oncol Clin North Am. 2018;32(4):629–642.
   PubMed Web of Science ®Google Scholar
• Esteves I, Bonfim C, Pasquini R, et al. Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study. Bone Marrow Transplant. 2015;50(5):685–689.
   PubMed Web of Science ®Google Scholar
• Bacigalupo A, Sica S. HLA haplotype mismatch transplants and posttransplant cyclophosphamide. Adv Hematol. 2016;2016:7802967.
   PubMedGoogle Scholar
• Nunes NS, Kanakry CG. Mechanisms of graft-versus-host disease prevention by post-transplantation cyclophosphamide: an evolving understanding. Front Immunol. 2019;10:2668.
   PubMed Web of Science ®Google Scholar
• Kanakry CG, Ganguly S, Zahurak M, et al. Aldehyde dehydrogenase expression drives human regulatory T cell resistance to posttransplantation cyclophosphamide. Sci Transl Med. 2013;5(211):211ra157.
   PubMed Web of Science ®Google Scholar
• Prata PH, Eikema DJ, Afansyev B, et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT severe aplastic anemia working party. Bone Marrow Transplant. 2020;55(6):1050–1058.
   PubMed Web of Science ®Google Scholar
• Arcuri LJ, Nabhan SK, Cunha R, et al., Impact of CD34 cell dose and conditioning regimen on outcomes after haploidentical donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for relapsed/refractory severe aplastic anemia. Biol Blood Marrow Transplant. 26(12): 2311–2317. 2020.
   PubMed Web of Science ®Google Scholar
• Arcuri LJ, Nabhan SK, Loth G, et al. A case series of post-transplantation cyclophosphamide in unrelated donor hematopoietic cell transplantation for aplastic anemia. Biol Blood Marrow Transplant. 2020;26(9):e222–e6.
   PubMed Web of Science ®Google Scholar
• Champlin RE, Perez WS, Passweg JR, et al. Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens. Blood. 2007;109(10):4582–4585.
   PubMed Web of Science ®Google Scholar
• Marsh JC, Gupta V, Lim Z, 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(8):2351–2357.
   PubMed Web of Science ®Google Scholar
• DeZern AE, Zahurak M, Symons H, et al. Alternative donor transplantation with high-dose post-transplantation cyclophosphamide for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2017;23(3):498–504.
   PubMed Web of Science ®Google Scholar
• DeZern AE, Zahurak ML, Symons HJ, et al., Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide. Blood Adv. 4(8): 1770–1779. 2020.
   PubMed Web of Science ®Google Scholar
• Xu L, Fu B, Wang W, et al. Haploidentical hematopoietic cell transplantation for severe acquired aplastic anemia: a case-control study of post-transplant cyclophosphamide included regimen vs. anti-thymocyte globulin & colony-stimulating factor-based regimen. Sci China Life Sci. 2020;63(6):940–942.
   PubMed Web of Science ®Google Scholar
• Wang Y, Chang YJ, Xu LP, et al. Who is the best donor for a related HLA haplotype-mismatched transplant? Blood. 2014;124(6):843–850.
   PubMed Web of Science ®Google Scholar
• Wang Y, Chang YJ, Chen L, et al. Low-dose post-transplant cyclophosphamide can mitigate GVHD and enhance the G-CSF/ATG induced GVHD protective activity and improve haploidentical transplant outcomes. Oncoimmunology. 2017;6(11):e1356152.
   PubMed Web of Science ®Google Scholar
• Wang Y, Wu DP, Liu QF, et al. Low-dose post-transplant cyclophosphamide and anti-thymocyte globulin as an effective strategy for GVHD prevention in haploidentical patients. J Hematol Oncol. 2019;12(1):88.
   PubMed Web of Science ®Google Scholar
• Guo Z, Gao HY, Zhang TY, et al. Analysis of allogeneic hematopoietic stem cell transplantation with high-dose cyclophosphamide-induced immune tolerance for severe aplastic anemia. Int J Hematol. 2016;104(6):720–728.
   PubMed Web of Science ®Google Scholar
• Mariotti J, Raiola AM, Evangelista A, et al. Impact of donor age and kinship on clinical outcomes after T-cell-replete haploidentical transplantation with PT-Cy. Blood Adv. 2020;4(16):3900–3912.
   PubMed Web of Science ®Google Scholar
• DeZern AE, Franklin C, Tsai HL, et al. Relationship of donor age and relationship to outcomes of haploidentical transplantation with posttransplant cyclophosphamide. Blood Adv. 2021;5(5):1360–1368.
   PubMed Web of Science ®Google Scholar
• Xu LP, Wang SQ, Ma YR, et al. Who is the best haploidentical donor for acquired severe aplastic anemia? Experience from a multicenter study. J Hematol Oncol. 2019;12(1):87.
   PubMed Web of Science ®Google Scholar
• Ma YR, Wang WJ, Cheng YF, et al. Impact of ABO incompatibility on outcomes after haploidentical hematopoietic stem cell transplantation for severe aplastic anemia. Bone Marrow Transplant. 2020;55(6):1068–1075.
   PubMed Web of Science ®Google Scholar
• Cheuk DK. Optimal stem cell source for allogeneic stem cell transplantation for hematological malignancies. World J Transplant. 2013;3(4):99–112.
   PubMedGoogle Scholar
• Li XH, Gao CJ, Da WM, et al. Reduced intensity conditioning, combined transplantation of haploidentical hematopoietic stem cells and mesenchymal stem cells in patients with severe aplastic anemia. PLoS One. 2014;9(3):e89666.
   PubMed Web of Science ®Google Scholar
• Yamei W, Rongmu L, Yongbin C, et al. Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning. Oncotarget. 2017;8(48):83817–83830.
   PubMedGoogle Scholar
• Liu L, Zhang Y, Jiao W, et al. Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia. Leukemia. 2020;34(12):3359–3369.
   PubMed Web of Science ®Google Scholar
• Luo XH, Chang YJ, Xu LP, et al. The impact of graft composition on clinical outcomes in unmanipulated HLA-mismatched/haploidentical hematopoietic SCT. Bone Marrow Transplant. 2009;43(1):29–36.
   PubMed Web of Science ®Google Scholar
• Chang YJ, Xu LP, Wang Y, et al. Controlled, randomized, open-label trial of risk-stratified corticosteroid prevention of acute graft-versus-host disease after haploidentical transplantation. J Clin Oncol. 2016;34(16):1855–1863.
   PubMed Web of Science ®Google Scholar