Advertisement

Haploidentical Stem Cell Transplantation

  • Narendra Agrawal
  • Dinesh Bhurani
Chapter

Abstract

Allogeneic haematopoietic stem cell transplantation (alloHCT) has emerged as a curative treatment modality for a variety of disorders including haematological malignancies, inherited and acquired bone marrow failure syndromes, congenital immunodeficiencies and errors of metabolism.

Keywords

Haploidentical transplantation 

Bibliography

  1. 1.
    Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A et al (2015) Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood 126(8):1033–1040CrossRefGoogle Scholar
  2. 2.
    Gabriel C, Fürst D, Faé I et al (2014) HLA typing by next-generation sequencing - getting closer to reality. Tissue Antigens 83:65CrossRefGoogle Scholar
  3. 3.
    Szydlo R, Goldman JM, Klein JP et al (1997) Results of allogeneic bone marrow transplants for leukemia using donors other than HLA-identical siblings. J Clin Oncol 15:1767CrossRefGoogle Scholar
  4. 4.
    Yoshihara S, Maruya E, Taniguchi K et al (2012) Risk and prevention of graft failure in patients with preexisting donor-specific HLA antibodies undergoing unmanipulated haploidentical SCT. Bone Marrow Transplant 47:508CrossRefGoogle Scholar
  5. 5.
    Ciurea SO, de Lima M, Cano P et al (2009) High risk of graft failure in patients with anti-HLA antibodies undergoing haploidentical stem-cell transplantation. Transplantation 88:1019CrossRefGoogle Scholar
  6. 6.
    Bramanti S, Nocco A, Mauro E, Milone G et al (2016) Desensitization with plasma exchange in a patient with human leukocyte antigen donor-specific antibodies before T-cell-replete haploidentical transplantation. Transfusion 56(5):1096–1100.  https://doi.org/10.1111/trf.13523CrossRefPubMedGoogle Scholar
  7. 7.
    Morin-Zorman S, Loiseau P, Taupin JL, Caillat-Zucman S (2016) Donor-Specific Anti-HLA Antibodies in Allogeneic Hematopoietic Stem Cell Transplantation. Front Immunol 7:307.  https://doi.org/10.3389/fimmu.2016.00307CrossRefPubMedPubMedCentralGoogle Scholar
  8. 8.
    Gladstone DE, Zachary AA, Fuchs EJ et al (2013) Partially mismatched transplantation and human leukocyte antigen donor specific antibodies. Biol Blood Marrow Transplant 19:647CrossRefGoogle Scholar
  9. 9.
    Kasamon YL, Luznik L, Leffell MS et al (2010) Nonmyeloablative HLA-haploidentical bone marrow transplantation with high-dose posttransplantation cyclophosphamide: effect of HLA disparity on outcome. Biol Blood Marrow Transplant 16:482CrossRefGoogle Scholar
  10. 10.
    Bashey A, Zhang X, Sizemore CA et al (2013) T-cell-replete HLA-haploidentical hematopoietic transplantation for hematologic malignancies using post-transplantation cyclophosphamide results in outcomes equivalent to those of contemporaneous HLA-matched related and unrelated donor transplantation. J Clin Oncol 31:1310CrossRefGoogle Scholar
  11. 11.
    Raiola AM, Dominietto A, di Grazia C et al (2014) Unmanipulated haploidentical transplants compared with other alternative donors and matched sibling grafts. Biol Blood Marrow Transplant 20:1573CrossRefGoogle Scholar
  12. 12.
    Lu DP, Dong L, Wu T et al (2006) Conditioning including antithymocyte globulin followed by unmanipulated HLA-mismatched/haploidentical blood and marrow transplantation can achieve comparable outcomes with HLA-identical sibling transplantation. Blood 107:3065CrossRefGoogle Scholar
  13. 13.
    Huo MR, Xu LP, Li D et al (2012) The effect of HLA disparity on clinical outcome after HLA-haploidentical blood and marrow transplantation. Clin Transpl 26:284CrossRefGoogle Scholar
  14. 14.
    Ichinohe T, Uchiyama T, Shimazaki C et al (2004) Feasibility of HLA-haploidentical hematopoietic stem cell transplantation between noninherited maternal antigen (NIMA)-mismatched family members linked with long-term fetomaternal microchimerism. Blood 104:3821CrossRefGoogle Scholar
  15. 15.
    Stern M, Ruggeri L, Mancusi A et al (2008) Survival after T cell-depleted haploidentical stem cell transplantation is improved using the mother as donor. Blood 112:2990CrossRefGoogle Scholar
  16. 16.
    van Rood JJ, Loberiza FR Jr, Zhang MJ et al (2002) Effect of tolerance to noninherited maternal antigens on the occurrence of graft-versus-host disease after bone marrow transplantation from a parent or an HLA-haploidentical sibling. Blood 99:157Google Scholar
  17. 17.
    Wang Y, Chang YJ, Xu LP et al (2014) Who is the best donor for a related HLA haplotype-mismatched transplant? Blood 124:843CrossRefGoogle Scholar
  18. 18.
    Cooley S, Weisdorf DJ, Guethlein LA et al (2010) Donor selection for natural killer cell receptor genes leads to superior survival after unrelated transplantation for acute myelogenous leukemia. Blood 116:2411CrossRefGoogle Scholar
  19. 19.
    Symons HJ, Leffell MS, Rossiter ND et al (2010) Improved survival with inhibitory killer immunoglobulin receptor (KIR) gene mismatches and KIR haplotype B donors after nonmyeloablative, HLA-haploidentical bone marrow transplantation. Biol Blood Marrow Transplant 16:533CrossRefGoogle Scholar
  20. 20.
    Bachar-Lustig E, Li HW, Marcus H, Reisner Y (1998) Tolerance induction by megadose stem cell transplants: synergism between SCA-1+ Lin- cells and nonalloreactive T cells. Transplant Proc 30(8):4007–4008CrossRefGoogle Scholar
  21. 21.
    Gur H, Krauthgamer R, Berrebi A, Klein T et al (2002) Tolerance induction by megadose hematopoietic progenitor cells: expansion of veto cells by short-term culture of purified human CD34(+) cells. Blood 99(11):4174–4181CrossRefGoogle Scholar
  22. 22.
    Aversa F, Tabilio A, Terenzi A et al (1994) Successful engraftment of T-cell-depleted haploidentical “three-loci” incompatible transplants in leukemia patients by addition of recombinant human granulocyte colony-stimulating factor-mobilized peripheral blood progenitor cells to bone marrow inoculum. Blood 84:3948PubMedGoogle Scholar
  23. 23.
    Aversa F, Tabilio A, Velardi A et al (1998) Treatment of high-risk acute leukemia with T-cell-depleted stem cells from related donors with one fully mismatched HLA haplotype. N Engl J Med 339:1186CrossRefGoogle Scholar
  24. 24.
    Aversa F, Terenzi A, Tabilio A et al (2005) Full haplotype-mismatched hematopoietic stem-cell transplantation: a phase II study in patients with acute leukemia at high risk of relapse. J Clin Oncol 23:3447CrossRefGoogle Scholar
  25. 25.
    Garderet L, Snell V, Przepiorka D, Schenk T et al (1999) Effective depletion of alloreactive lymphocytes from peripheral blood mononuclear cell preparations. Transplantation 67(1):124–130CrossRefGoogle Scholar
  26. 26.
    Lang P, Greil J, Bader P et al (2004) Long-term outcome after haploidentical stem cell transplantation in children. Blood Cells Mol Dis 33:281CrossRefGoogle Scholar
  27. 27.
    Klingebiel T, Cornish J, Labopin M et al (2010) Results and factors influencing outcome after fully haploidentical hematopoietic stem cell transplantation in children with very high-risk acute lymphoblastic leukemia: impact of center size: an analysis on behalf of the Acute Leukemia and Pediatric Disease Working Parties of the European Blood and Marrow Transplant group. Blood 115:3437CrossRefGoogle Scholar
  28. 28.
    Walker I, Shehata N, Cantin G et al (2004) Canadian multicenter pilot trial of haploidentical donor transplantation. Blood Cells Mol Dis 222(39):33Google Scholar
  29. 29.
    Waller EK, Giver CR, Rosenthal H et al (2004) Facilitating T-cell immune reconstitution after haploidentical transplantation in adults. Blood Cells Mol Dis 33:233CrossRefGoogle Scholar
  30. 30.
    N N, Davison G, Abdulla R, Mowla S (2013) Definition of the variables affecting efficacy of immunodepletion ex vivo of peripheral blood progenitor cell grafts by alemtuzumab (Campath in the bag). Biol Blood Marrow Transplant 19(12):1753–1759.  https://doi.org/10.1016/j.bbmt.2013.10.001CrossRefGoogle Scholar
  31. 31.
    Bethge WA, Faul C, Bornhäuser M et al (2008) Haploidentical allogeneic hematopoietic cell transplantation in adults using CD3/CD19 depletion and reduced intensity conditioning: an update. Blood Cells Mol Dis 40:13CrossRefGoogle Scholar
  32. 32.
    Schumm M, Lang P, Bethge W et al (2013) Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy 15:1253CrossRefGoogle Scholar
  33. 33.
    Airoldi I, Bertaina A, Prigione I et al (2015) γδ T-cell reconstitution after HLA-haploidentical hematopoietic transplantation depleted of TCR-αβ+/CD19+ lymphocytes. Blood:125, 2349Google Scholar
  34. 34.
    Federmann B, Bornhauser M, Meisner C, Kordelas L et al (2012) Haploidentical allogeneic hematopoietic cell transplantation in adults using CD3/CD19 depletion and reduced intensity conditioning: a phase II study. Haematologica 97(10):1523–1531.  https://doi.org/10.3324/haematol.2011.059378CrossRefPubMedPubMedCentralGoogle Scholar
  35. 35.
    Cavazzana-Calvo M, Fromont C, Le Deist F, Lusardi M et al (1990) Specific elimination of alloreactive T cells by an anti-interleukin-2 receptor B chain-specific immunotoxin. Transplantation 50(1):1–7CrossRefGoogle Scholar
  36. 36.
    Mavroudis DA, Jiang YZ, Hensel N, Lewalle P et al (1996) Specific depletion of alloreactivity against haplotype mismatched related individuals by a recombinant immunotoxin: a new approach to graft-versus-host disease prophylaxis in haploidentical bone marrow transplantation. Bone Marrow Transplant 17(5):793–799PubMedGoogle Scholar
  37. 37.
    Chen BJ, Cui X, Liu C, Chao NJ (2002) Prevention of graft-versus-host disease while preserving graft-versus-leukemia effect after selective depletion of host-reactive T cells by photodynamic cell purging process. Blood 99(9):3083–3088CrossRefGoogle Scholar
  38. 38.
    Andre-Schmutz I, Le Deist F, Hacein-Bey-Abina S, Vitetta E et al (2002) Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study. Lancet 360(9327):130–137CrossRefGoogle Scholar
  39. 39.
    Bastien JP, Roy J, Roy DC (2012) Selective T-cell depletion for haplotype-mismatched allogeneic stem cell transplantation. Sem Oncol 39(6):674–682CrossRefGoogle Scholar
  40. 40.
    Mielke S, Mciver ZA, Shenoy A, Fellowes V et al (2011) Selectively T cell-depleted allografts from HLA-matched sibling donors followed by low-dose posttransplantation immunosuppression to improve transplantation outcome in patients with hematologic malignancies. Biol Blood Marrow Transplant 17(12):1855–1861CrossRefGoogle Scholar
  41. 41.
    Perruccio K, Tosti A, Burchielli E et al (2005) Transferring functional immune responses to pathogens after haploidentical hematopoietic transplantation. Blood 106:4397CrossRefGoogle Scholar
  42. 42.
    Martelli MF, Di Ianni M, Ruggeri L et al (2014) “Designed” grafts for HLA-haploidentical stem cell transplantation. Blood 123:967CrossRefGoogle Scholar
  43. 43.
    Kanakry JA, Kasamon YL, Bolaños-Meade J et al (2013) Absence of post-transplantation lymphoproliferative disorder after allogeneic blood or marrow transplantation using post-transplantation cyclophosphamide as graft-versus-host disease prophylaxis. Biol Blood Marrow Transplant 19:1514CrossRefGoogle Scholar
  44. 44.
    Luznik L, O’Donnell PV, Symons HJ et al (2008) HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant 14:641CrossRefGoogle Scholar
  45. 45.
    O’Donnell PV, Luznik L, Jones RJ et al (2002) Nonmyeloablative bone marrow transplantation from partially HLA-mismatched related donors using posttransplantation cyclophosphamide. Biol Blood Marrow Transplant 8:377CrossRefGoogle Scholar
  46. 46.
    Shabbir-Moosajee M, Lombardi L, Ciurea SO (2015) An overview of conditioning regimens for haploidentical stem cell transplantation with post-transplantation cyclophosphamide. Am J Hematol 90:541CrossRefGoogle Scholar
  47. 47.
    Perruccio K, Topini F, Tosti A et al (2008) Photodynamic purging of alloreactive T cells for adoptive immunotherapy after haploidentical stem cell transplantation. Blood Cells Mol Dis 40:76CrossRefGoogle Scholar
  48. 48.
    Knight A, Madrigal AJ, Grace S et al (2010) The role of Vδ2-negative γδ T cells during cytomegalovirus reactivation in recipients of allogeneic stem cell transplantation. Blood 116:2164CrossRefGoogle Scholar
  49. 49.
    Huang XJ, Liu DH, Liu KY et al (2006) Haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion for the treatment of hematological malignancies. Bone Marrow Transplant 38:291CrossRefGoogle Scholar
  50. 50.
    Liu D, Huang X, Liu K et al (2008) Haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for treatment of hematological malignancies in children. Biol Blood Marrow Transplant 14:469CrossRefGoogle Scholar
  51. 51.
    Wang Y, Liu QF, Xu LP et al (2015) Haploidentical vs identical-sibling transplant for AML in remission: a multicenter, prospective study. Blood 125:3956CrossRefGoogle Scholar
  52. 52.
    Di Bartolomeo P, Santarone S, De Angelis G et al (2013) Haploidentical, unmanipulated, G-CSF-primed bone marrow transplantation for patients with high-risk hematologic malignancies. Blood 121:849CrossRefGoogle Scholar
  53. 53.
    Chang YJ, Zhao XY, Huo MR et al (2012) Immune reconstitution following unmanipulated HLA-mismatched/haploidentical transplantation compared with HLA-identical sibling transplantation. J Clin Immunol 32:268CrossRefGoogle Scholar
  54. 54.
    Zeidan AM, Forde PM, Symons H et al (2014) HLA-haploidentical donor lymphocyte infusions for patients with relapsed hematologic malignancies after related HLA-haploidentical bone marrow transplantation. Biol Blood Marrow Transplant 20:314CrossRefGoogle Scholar
  55. 55.
    Ghiso A, Raiola AM, Gualandi F et al (2015) DLI after haploidentical BMT with post-transplant CY. Bone Marrow Transplant 50:56CrossRefGoogle Scholar
  56. 56.
    Blaise D, Fürst S, Crocchiolo R, El-Cheikh J, et al, Haploidentical T cell-replete transplantation with post-transplantation cyclophosphamide for patients in or above the sixth decade of age compared with allogeneic hematopoietic stem cell transplantation from an human leukocyte Antigen-matched related or unrelated donor. Biol Blood Marrow Transplant. 2015. pii: S1083-8791(15)00577-7. doi: https://doi.org/10.1016/j.bbmt.2015.08.029
  57. 57.
    Armand P, Kim HT, Logan BR et al (2014) Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation. Blood 123(23):3664–3671CrossRefGoogle Scholar
  58. 58.
    McCurdy SR, Kanakry JA, Showel MM, Tsai HL et al (2015) Risk-stratified outcomes of nonmyeloablative HLA-haploidentical BMT with high-dose posttransplantation cyclophosphamide. Blood 125(19):3024–3031.  https://doi.org/10.1182/blood-2015-01-623991CrossRefPubMedPubMedCentralGoogle Scholar
  59. 59.
    Wang Y, Liu Q-F, Lan-Ping X, Liu K-Y et al (2015) Haploidentical vs identical-sibling transplant for AML in remission: a multicenter, prospective study. Blood 125(25):3956–3962CrossRefGoogle Scholar
  60. 60.
    Mo XD, Xu LP, Zhang XH, Liu DH et al (2015) Haploidentical hematopoietic stem cell transplantation in adults with Philadelphia-negative acute lymphoblastic leukemia: no difference in the high- and low-risk groups. Int J Cancer 136(7):1697–1707.  https://doi.org/10.1002/ijc.29146CrossRefPubMedGoogle Scholar
  61. 61.
    Bolanos-Meade J, Fuchs EJ, Luznik L, Lanzkron SM, Gamper CJ, Jones RJ et al (2012) HLA haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood 120:4285–4291CrossRefGoogle Scholar
  62. 62.
    Sodani P, Isgro A, Gaziev J, Polchi P et al (2010) Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia. Blood 115:1296–1302CrossRefGoogle Scholar
  63. 63.
    Anurathapan U, Hongeng S, Pakakasama S, Sirachainan N et al (2016) Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors. Bone Marrow Transplant 51:813–818CrossRefGoogle Scholar
  64. 64.
    Georges GE, Storb R (2016) Hematopoietic stem cell transplantation for acquired aplastic anemia. Curr Opin Hematol 23(6):495–500CrossRefGoogle Scholar
  65. 65.
    Xu LP, Wang SQ, Wu DP, Wang JM et al (2016) Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study. Br J Haematol 175(2):265–274.  https://doi.org/10.1111/bjh.14225CrossRefPubMedGoogle Scholar

Copyright information

© Springer Nature Singapore Pte Ltd. 2018

Authors and Affiliations

  • Narendra Agrawal
    • 1
  • Dinesh Bhurani
    • 1
  1. 1.Department of HematologyRajiv Gandhi Cancer Institute & Research CentreDelhiIndia

Personalised recommendations