Purified Hematopoietic Stem Cell Transplantation: The Next Generation of Blood and Immune Replacement

Hematology/Oncology Clinics of North America

Volumn 25, Issue 1, 2011, Pages 75-87

  Czechowicz, Agnieszka ^a   Weissman, Irving L ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Autoimmune diseases; Hematopoeitic stem cell transplantation; Immune tolerance; Nonmalignant hematolymphoid disorders; Nonmyeloablative conditioning

Indexed keywords

ALEMTUZUMAB; CD20 ANTIBODY; CD3 ANTIBODY; CD4 ANTIBODY; PLERIXAFOR;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; BETA THALASSEMIA; BLOOD TRANSFUSION; BONE MARROW DEPRESSION; BONE MARROW TRANSPLANTATION; CHIMERA; ENGRAFTMENT; FLUORESCENCE ACTIVATED CELL SORTING; GRAFT VERSUS HOST REACTION; HEMATOLOGIC MALIGNANCY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNE DEFICIENCY; IMMUNOLOGICAL TOLERANCE; LYMPHOCYTE CLONE; MAJOR HISTOCOMPATIBILITY COMPLEX; MICROENVIRONMENT; NATURAL KILLER CELL; NONHUMAN; PRIORITY JOURNAL; REVIEW; SICKLE CELL ANEMIA; T LYMPHOCYTE;

EID: 78651232717     PISSN: 08898588     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.hoc.2010.11.006     Document Type: Review

References (72)

1. Spangrude G.J., Heimfeld S., Weissman I.L. Purification and characterization of mouse hematopoietic stem cells. Science 1988, 241(4861):58-62.
2. Gatti R.A., Meuwissen H.J., Allen H.D., et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968, 2(7583):1366-1369.
3. McCulloch E.A., Till J.E. The radiation sensitivity of normal mouse bone marrow cells, determined by quantitative marrow transplantation into irradiated mice. Radiat Res 1960, 13:115-125.
4. Michlitsch J.G., Walters M.C. Recent advances in bone marrow transplantation in hemoglobinopathies. Curr Mol Med 2008, 8(7):675-689.
5. Barnes D.W., Loutit J.F., Micklem H.S. " Secondary disease" of radiation chimeras: a syndrome due to lymphoid aplasia. Ann N Y Acad Sci 1962, 99:374-385.
6. Keever C.A., Flomenberg N., Brochstein J., et al. Tolerance of engrafted donor T cells following bone marrow transplantation for severe combined immunodeficiency. Clin Immunol Immunopathol 1988, 48(3):261-276.
7. Ferrara J.L., Deeg H.J. Graft-versus-host disease. N Engl J Med 1991, 324(10):667-674.
8. Schierman L.W., Nordskog A.W. Influence of the B bloodgroup-histocompatibility locus in chickens on a graft-versus-host reaction. Nature 1963, 197:511-512.
9. Tsao G.J., Allen J.A., Logronio K.A., et al. Purified hematopoietic stem cell allografts reconstitute immunity superior to bone marrow. Proc Natl Acad Sci U S A 2009, 106(9):3288-3293.
10. Sprent J., Miller J.F. Fate of H2-activated T lymphocytes in syngeneic hosts. II. Residence in recirculating lymphocyte pool and capacity to migrate to allografts. Cell Immunol 1976, 21(2):303-313.
11. Baum C.M., Weissman I.L., Tsukamoto A.S., et al. Isolation of a candidate human hematopoietic stem-cell population. Proc Natl Acad Sci U S A 1992, 89(7):2804-2808.
12. Kiel M.J., Yilmaz O.H., Iwashita T., et al. SLAM family receptors distinguish hematopoietic stem and progenitor cells and reveal endothelial niches for stem cells. Cell 2005, 121(7):1109-1121.
13. Whitlock C.A., Tidmarsh G.F., Muller-Sieburg C., et al. Bone marrow stromal cell lines with lymphopoietic activity express high levels of a pre-B neoplasia-associated molecule. Cell 1987, 48(6):1009-1021.
14. Christensen J.L., Weissman I.L. Flk-2 is a marker in hematopoietic stem cell differentiation: a simple method to isolate long-term stem cells. Proc Natl Acad Sci U S A 2001, 98(25):14541-14546.
15. Matsuoka S., Ebihara Y., Xu M., et al. CD34 expression on long-term repopulating hematopoietic stem cells changes during developmental stages. Blood 2001, 97(2):419-425.
16. Adolfsson J., Borge O.J., Bryder D., et al. Upregulation of Flt3 expression within the bone marrow Lin(-)Sca1(+)c-kit(+) stem cell compartment is accompanied by loss of self-renewal capacity. Immunity 2001, 15(4):659-669.
17. Osawa M., Hanada K., Hamada H., et al. Long-term lymphohematopoietic reconstitution by a single CD34-low/negative hematopoietic stem cell. Science 1996, 273(5272):242-245.
18. Majeti R., Park C.Y., Weissman I.L. Identification of a hierarchy of multipotent hematopoietic progenitors in human cord blood. Cell Stem Cell 2007, 1(6):635-645.
19. Matsuzaki Y., Kinjo K., Mulligan R.C., et al. Unexpectedly efficient homing capacity of purified murine hematopoietic stem cells. Immunity 2004, 20(1):87-93.
20. Wagers A.J., Christensen J.L., Weissman I.L. Cell fate determination from stem cells. Gene Ther 2002, 9(10):606-612.
21. Camargo F.D., Chambers S.M., Drew E., et al. Hematopoietic stem cells do not engraft with absolute efficiencies. Blood 2006, 107(2):501-507.
22. Negrin R.S., Atkinson K., Leemhuis T., et al. Transplantation of highly purified CD34+Thy-1+ hematopoietic stem cells in patients with metastatic breast cancer. Biol Blood Marrow Transplant 2000, 6(3):262-271.
23. Vose J.M., Bierman P.J., Lynch J.C., et al. Transplantation of highly purified CD34+Thy-1+ hematopoietic stem cells in patients with recurrent indolent non-Hodgkin's lymphoma. Biol Blood Marrow Transplant 2001, 7(12):680-687.
24. Michallet M., Philip T., Philip I., et al. Transplantation with selected autologous peripheral blood CD34+Thy1+ hematopoietic stem cells (HSCs) in multiple myeloma: impact of HSC dose on engraftment, safety, and immune reconstitution. Exp Hematol 2000, 28(7):858-870.
25. Ito M., Shizuru J.A. Graft-vs.-lymphoma effect in an allogeneic hematopoietic stem cell transplantation model. Biol Blood Marrow Transplant 1999, 5(6):357-368.
26. Shizuru J.A., Jerabek L., Edwards C.T., et al. Transplantation of purified hematopoietic stem cells: requirements for overcoming the barriers of allogeneic engraftment. Biol Blood Marrow Transplant 1996, 2(1):3-14.
27. Barth E., Malorgio C., Tamaro P. Allogeneic bone marrow transplantation in hematologic disorders of childhood: new trends and controversies. Haematologica 2000, 85(11 Suppl):2-8.
28. Main J.M., Prehn R.T. Successful skin homografts after the administration of high dosage X radiation and homologous bone marrow. J Natl Cancer Inst 1955, 15(4):1023-1029.
29. Starzl T.E., Demetris A.J., Murase N., et al. Cell migration, chimerism, and graft acceptance. Lancet 1992, 339(8809):1579-1582.
30. Scandling J.D., Busque S., Dejbakhsh-Jones S., et al. Tolerance and chimerism after renal and hematopoietic-cell transplantation. N Engl J Med 2008, 358(4):362-368.
31. Millan M.T., Shizuru J.A., Hoffmann P., et al. Mixed chimerism and immunosuppressive drug withdrawal after HLA-mismatched kidney and hematopoietic progenitor transplantation. Transplantation 2002, 73(9):1386-1391.
32. Alexander S.I., Smith N., Hu M., et al. Chimerism and tolerance in a recipient of a deceased-donor liver transplant. N Engl J Med 2008, 358(4):369-374.
33. Shizuru J.A., Weissman I.L., Kernoff R., et al. Purified hematopoietic stem cell grafts induce tolerance to alloantigens and can mediate positive and negative T cell selection. Proc Natl Acad Sci U S A 2000, 97(17):9555-9560.
34. Gandy K.L., Weissman I.L. Tolerance of allogeneic heart grafts in mice simultaneously reconstituted with purified allogeneic hematopoietic stem cells. Transplantation 1998, 65(3):295-304.
35. Kawai T., Cosimi A.B., Spitzer T.R., et al. HLA-mismatched renal transplantation without maintenance immunosuppression. N Engl J Med 2008, 358(4):353-361.
36. Nikolic B., Takeuchi Y., Leykin I., et al. Mixed hematopoietic chimerism allows cure of autoimmune diabetes through allogeneic tolerance and reversal of autoimmunity. Diabetes 2004, 53(2):376-383.
37. van Gelder M., Kinwel-Bohre E.P., van Bekkum D.W. Treatment of experimental allergic encephalomyelitis in rats with total body irradiation and syngeneic BMT. Bone Marrow Transplant 1993, 11(3):233-241.
38. Traynor A.E., Schroeder J., Rosa R.M., et al. Treatment of severe systemic lupus erythematosus with high-dose chemotherapy and haemopoietic stem-cell transplantation: a phase I study. Lancet 2000, 356(9231):701-707.
39. Smith-Berdan S., Gille D., Weissman I.L., et al. Reversal of autoimmune disease in lupus-prone New Zealand black/New Zealand white mice by nonmyeloablative transplantation of purified allogeneic hematopoietic stem cells. Blood 2007, 110(4):1370-1378.
40. Todd J.A., Bell J.I., McDevitt H.O. HLA-DQ beta gene contributes to susceptibility and resistance to insulin-dependent diabetes mellitus. Nature 1987, 329(6140):599-604.
41. Beilhack G.F., Scheffold Y.C., Weissman I.L., et al. Purified allogeneic hematopoietic stem cell transplantation blocks diabetes pathogenesis in NOD mice. Diabetes 2003, 52(1):59-68.
42. Krauss A.C., Kamani N.R. Hematopoietic stem cell transplantation for pediatric autoimmune disease: where we stand and where we need to go. Bone Marrow Transplant 2009, 44(3):137-143.
43. McCormack M.P., Rabbitts T.H. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2004, 350(9):913-922.
44. Hutter G., Nowak D., Mossner M., et al. Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med 2009, 360(7):692-698.
45. van Griensven J., De Clercq E., Debyser Z. Hematopoietic stem cell-based gene therapy against HIV infection: promises and caveats. AIDS Rev 2005, 7(1):44-55.
46. Ferry C., Socie G. Busulfan-cyclophosphamide versus total body irradiation-cyclophosphamide as preparative regimen before allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia: what have we learned?. Exp Hematol 2003, 31(12):1182-1186.
47. Walters M.C., Patience M., Leisenring W., et al. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant 2001, 7(12):665-673.
48. Iannone R., Luznik L., Engstrom L.W., et al. Effects of mixed hematopoietic chimerism in a mouse model of bone marrow transplantation for sickle cell anemia. Blood 2001, 97(12):3960-3965.
49. Storb R., Yu C., Sandmaier B.M., et al. Mixed hematopoietic chimerism after marrow allografts. Transplantation in the ambulatory care setting. Ann N Y Acad Sci 1999, 872:372-375. [discussion: 375-6].
50. Gandy K.L., Domen J., Aguila H., et al. CD8+TCR+ and CD8+TCR- cells in whole bone marrow facilitate the engraftment of hematopoietic stem cells across allogeneic barriers. Immunity 1999, 11(5):579-590.
51. Kernan N.A., Bordignon C., Heller G., et al. Graft failure after T-cell-depleted human leukocyte antigen identical marrow transplants for leukemia: I. Analysis of risk factors and results of secondary transplants. Blood 1989, 74(6):2227-2236.
52. Marmont A.M., Horowitz M.M., Gale R.P., et al. T-cell depletion of HLA-identical transplants in leukemia. Blood 1991, 78(8):2120-2130.
53. Maloney D.G., Sandmaier B.M., Mackinnon S., et al. Non-myeloablative transplantation. Hematology Am Soc Hematol Educ Program 2002, 392-421.
54. Tomita Y., Sachs D.H., Sykes M. Myelosuppressive conditioning is required to achieve engraftment of pluripotent stem cells contained in moderate doses of syngeneic bone marrow. Blood 1994, 83(4):939-948.
55. Schofield R. The relationship between the spleen colony-forming cell and the haemopoietic stem cell. Blood Cells 1978, 4(1-2):7-25.
56. Morrison S.J., Spradling A.C. Stem cells and niches: mechanisms that promote stem cell maintenance throughout life. Cell 2008, 132(4):598-611.
57. Micklem H.S., Clarke C.M., Evans E.P., et al. Fate of chromosome-marked mouse bone marrow cells transfused into normal syngeneic recipients. Transplantation 1968, 6(2):299-302.
58. Saxe D.F., Boggs S.S., Boggs D.R. Transplantation of chromosomally marked syngeneic marrow cells into mice not subjected to hematopoietic stem cell depletion. Exp Hematol 1984, 12(4):277-283.
59. Stewart F.M., Crittenden R.B., Lowry P.A., et al. Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice. Blood 1993, 81(10):2566-2571.
60. Wu D.D., Keating A. Hematopoietic stem cells engraft in untreated transplant recipients. Exp Hematol 1993, 21(2):251-256.
61. Bhattacharya D., Rossi D.J., Bryder D., et al. Purified hematopoietic stem cell engraftment of rare niches corrects severe lymphoid deficiencies without host conditioning. J Exp Med 2006, 203(1):73-85.
62. Czechowicz A., Kraft D., Weissman I.L., et al. Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches. Science 2007, 318(5854):1296-1299.
63. Muller S.M., Kohn T., Schulz A.S., et al. Similar pattern of thymic-dependent T-cell reconstitution in infants with severe combined immunodeficiency after human leukocyte antigen (HLA)-identical and HLA-nonidentical stem cell transplantation. Blood 2000, 96(13):4344-4349.
64. Cavazzana-Calvo M., Carlier F., Le Deist F., et al. Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype. Blood 2007, 109(10):4575-4581.
65. Wright D.E., Wagers A.J., Gulati A.P., et al. Physiological migration of hematopoietic stem and progenitor cells. Science 2001, 294(5548):1933-1936.
66. Bhattacharya D., Czechowicz A., Ooi A.G., et al. Niche recycling through division-independent egress of hematopoietic stem cells. J Exp Med 2009, 206(12):2837-2850.
67. Broxmeyer H.E., Orschell C.M., Clapp D.W., et al. Rapid mobilization of murine and human hematopoietic stem and progenitor cells with AMD3100, a CXCR4 antagonist. J Exp Med 2005, 201(8):1307-1318.
68. Chen J., Larochelle A., Fricker S., et al. Mobilization as a preparative regimen for hematopoietic stem cell transplantation. Blood 2006, 107(9):3764-3771.
69. Domen J., Weissman I.L. Hematopoietic stem cells need two signals to prevent apoptosis; BCL-2 can provide one of these, Kitl/c-Kit signaling the other. J Exp Med 2000, 192(12):1707-1718.
70. Ogawa M., Matsuzaki Y., Nishikawa S., et al. Expression and function of c-kit in hemopoietic progenitor cells. J Exp Med 1991, 174(1):63-71.
71. Murphy W.J., Kumar V., Bennett M. Acute rejection of murine bone marrow allografts by natural killer cells and T cells. Differences in kinetics and target antigens recognized. J Exp Med 1987, 166(5):1499-1509.
72. Thomas E.D., Lochte H.L., Lu W.C., et al. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N Engl J Med 1957, 257(11):491-496.