Ex Vivo Expansion or Manipulation of Stem Cells to Improve Outcome of Umbilical Cord Blood Transplantation

详细信息    查看全文

• 作者：Mitchell E. Horwitz
• 关键词：Umbilical cord blood ; Expansion ; Manipulation ; Stem cell transplantation ; Hematopoietic recovery ; Hematopoietic progenitor cell
• 刊名：Current Hematologic Malignancy Reports
• 出版年：2016
• 出版时间：February 2016
• 年：2016
• 卷：11
• 期：1
• 页码：12-18
• 全文大小：496 KB
• 参考文献：1.Sebrango A et al. Haematopoietic transplants combining a single unrelated cord blood unit and mobilized haematopoietic stem cells from an adult HLA-mismatched third party donor. Comparable results to transplants from HLA-identical related donors in adults with acute leukaemia and myelodysplastic syndromes. Best Pract Res Clin Haematol. 2010;23(2):259–74.CrossRef PubMed
  2.Liu H et al. Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions. Blood. 2011;118(24):6438–45.CrossRef PubMed PubMedCentral
  3.Peffault de Latour R et al. Similar overall survival using sibling, unrelated donor, and cord blood grafts after reduced-intensity conditioning for older patients with acute myelogenous leukemia. Biol Blood Marrow Transplant. 2013;19(9):1355–60.CrossRef PubMed
  4.Smith AR et al. Hematopoietic cell transplantation for children with acute lymphoblastic leukemia in second complete remission: similar outcomes in recipients of unrelated marrow and umbilical cord blood versus marrow from HLA matched sibling donors. Biol Blood Marrow Transplant. 2009;15(9):1086–93.CrossRef PubMed
  5.Jaroscak J et al. Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell system. Blood. 2003;101(12):5061–7.CrossRef PubMed
  6.Scaradavou A et al. Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood. 2013;121(5):752–8.CrossRef PubMed PubMedCentral
  7.Barker JN et al. Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood. 2005;105(3):1343–7.CrossRef PubMed
  8.Milner LA et al. A human homologue of the Drosophila developmental gene, Notch, is expressed in CD34+ hematopoietic precursors. Blood. 1994;83(8):2057–62.PubMed
  9.Delaney C et al. Dose-dependent effects of the Notch ligand Delta1 on ex vivo differentiation and in vivo marrow repopulating ability of cord blood cells. Blood. 2005;106(8):2693–9.CrossRef PubMed PubMedCentral
  10.Delaney C et al. Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution. Nat Med. 2010;16(2):232–6.CrossRef PubMed PubMedCentral
  11.Gutman JA et al. Single-unit dominance after double-unit umbilical cord blood transplantation coincides with a specific CD8+ T-cell response against the nonengrafted unit. Blood. 2010;115(4):757–65.CrossRef PubMed PubMedCentral
  12.Moretta A et al. In vitro evaluation of graft-versus-graft alloreactivity as a tool to identify the predominant cord blood unit before double cord blood transplantation. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2012;18(7):1108–18.CrossRef
  13.Delaney C. Infusion of Non-HLA matched, off-the-shelf ex vivo expanded cord blood progenitor cells in patients undergoing myeloablative cord blood transplantation is safe and decreases the time to neutrophil recovery. Biol Blood Marrow Transplant. 2012;18(2):S203.CrossRef
  14.Peled T et al. Linear polyamine copper chelator tetraethylenepentamine augments long-term ex vivo expansion of cord blood-derived CD34+ cells and increases their engraftment potential in NOD/SCID mice. Exp Hematol. 2004;32(6):547–55.CrossRef PubMed
  15.Peled T et al. Pre-clinical development of cord blood-derived progenitor cell graft expanded ex vivo with cytokines and the polyamine copper chelator tetraethylenepentamine. Cytotherapy. 2004;6(4):344–55.CrossRef PubMed
  16.de Lima M et al. Transplantation of ex vivo expanded cord blood cells using the copper chelator tetraethylenepentamine: a phase I/II clinical trial. Bone Marrow Transplant. 2008.
  17.Stiff PM et al. StemEx® (copper chelation based) ex vivo expanded Umbilical Cord Blood Stem Cell Transplantation (UCBT) accelerates engraftment and improves 100 day survival in myeloablated patients compared to a registry cohort undergoing double unit UCBT: results of a multicenter study of 101 patients with hematologic malignancies. Blood. 2013;122(295):3060.
  18.Peled T et al. Nicotinamide, a SIRT1 inhibitor, inhibits differentiation and facilitates expansion of hematopoietic progenitor cells with enhanced bone marrow homing and engraftment. Exp Hematol. 2012;40(4):342–55.e1.CrossRef PubMed
  19.Horwitz ME et al. Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. J Clin Invest. 2014;124(7):3121–8.CrossRef PubMed PubMedCentral
  20.Briddell RA et al. Recombinant rat stem cell factor synergizes with recombinant human granulocyte colony-stimulating factor in vivo in mice to mobilize peripheral blood progenitor cells that have enhanced repopulating potential. Blood. 1993;82(6):1720–3.PubMed
  21.McNiece I et al. Ex vivo expansion of cord blood mononuclear cells on mesenchymal stem cells. Cytotherapy. 2004;6(4):311–7.CrossRef PubMed
  22.de Lima M et al. Cord-blood engraftment with ex vivo mesenchymal-cell coculture. N Engl J Med. 2012;367(24):2305–15.CrossRef PubMed PubMedCentral
  23.Boitano AE et al. Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells. Science. 2010;329(5997):1345–8.CrossRef PubMed PubMedCentral
  24.Wagner JE et al. StemRegenin-1 (SR1) Expansion Culture Abrogates the Engraftment Barrier Associated with Umbilical Cord Bood Transplantation. Abstract to be presented at the 56th American Society of Hematology Meeting and Exposition. 2014.
  25.North TE et al. Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis. Nature. 2007;447(7147):1007–11.CrossRef PubMed PubMedCentral
  26.Cutler C et al. Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation. Blood. 2013;122(17):3074–81.CrossRef PubMed PubMedCentral
  27.Ratajczak J et al. Mobilization studies in mice deficient in either C3 or C3a receptor (C3aR) reveal a novel role for complement in retention of hematopoietic stem/progenitor cells in bone marrow. Blood. 2004;103(6):2071–8.CrossRef PubMed
  28.Reca R et al. Functional receptor for C3a anaphylatoxin is expressed by normal hematopoietic stem/progenitor cells, and C3a enhances their homing-related responses to SDF-1. Blood. 2003;101(10):3784–93.CrossRef PubMed
  29.Brunstein CG et al. Complement fragment 3a priming of umbilical cord blood progenitors: safety profile. Biol Blood Marrow Transplant. 2013;19(10):1474–9.CrossRef PubMed PubMedCentral
  30.Hidalgo A et al. Functional selectin ligands mediating human CD34(+) cell interactions with bone marrow endothelium are enhanced postnatally. J Clin Invest. 2002;110(4):559–69.CrossRef PubMed PubMedCentral
  31.Robinson SN et al. Fucosylation with fucosyltransferase VI or fucosyltransferase VII improves cord blood engraftment. Cytotherapy. 2014;16(1):84–9.CrossRef PubMed
  32.Xia L et al. Surface fucosylation of human cord blood cells augments binding to P-selectin and E-selectin and enhances engraftment in bone marrow. Blood. 2004;104(10):3091–6.CrossRef PubMed
  33.Popat U et al. Enforced fucosylation of cord blood hematopoietic cells accelerates neutrophil and platelet engraftment after transplantation. Blood. 2015;125(19):2885–92.CrossRef PubMed
  34.Robinson SN et al. Ex vivo fucosylation improves human cord blood engraftment in NOD-SCID IL-2Rgamma(null) mice. Exp Hematol. 2012;40(6):445–56.CrossRef PubMed PubMedCentral
  
• 作者单位：Mitchell E. Horwitz (1)
  
  1. Adult Blood and Marrow Transplant Program, Duke University School of Medicine, 2400 Pratt St. DUMC 3961, Durham, NC, 27710, USA
  
• 刊物主题：Hematology; Oncology; Geriatrics/Gerontology;
• 出版者：Springer US
• ISSN：1558-822X

文摘

The outcome of umbilical cord blood transplantation for adult patients with hematologic malignancies now rivals that of matched unrelated donor transplantation. However, delayed hematopoietic and immunologic recovery remains a source of significant morbidity and mortality. Multiple strategies are now being studied to overcome these limitations. One strategy involves ex vivo expansion of the umbilical cord blood unit prior to transplantation. A second strategy involves exposure of the umbilical cord blood graft to compounds aimed at improving homing and engraftment following transplantation. Such a strategy may also address the problem of slow hematopoietic recovery as well as the increased risk of graft failure. Many of these strategies are now being tested in late phase multi-center clinical trials. If proven cost-effective and efficacious, they may alter the landscape of donor options for allogeneic stem cell transplantation.