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Review

Hematopoietic stem cell transplantation in multiple sclerosis

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Pages 567-578 | Published online: 09 Jan 2014

References

  • Frohman EM, Racke MK, Raine CS. Multiple sclerosis – the plaque and its pathogenesis. N. Engl. J. Med. 354(9), 942–955 (2006).
  • Trapp BD, Peterson J, Ransohoff RM, Rudick R, Mörk S, Bö L. Axonal transection in the lesions of multiple sclerosis. N. Engl. J. Med. 338(5), 278–285 (1998).
  • Zhang J, Markovic-Plese S, Lacet B, Raus J, Weiner HL, Hafler DA. Increased frequency of interleukin 2-responsive T cells specific for myelin basic protein and proteolipid protein in peripheral blood and cerebrospinal fluid of patients with multiple sclerosis. J. Exp. Med. 179(3), 973–984 (1994).
  • Allegretta M, Nicklas JA, Sriram S, Albertini RJ. T cells responsive to myelin basic protein in patients with multiple sclerosis. Science 247(4943), 718–721 (1990).
  • Wucherpfennig KW, Hafler DA. A review of T-cell receptors in multiple sclerosis: clonal expansion and persistence of human T-cells specific for an immunodominant myelin basic protein peptide. Ann. NY Acad. Sci. 756, 241–258 (1995).
  • Venken K, Hellings N, Hensen K, Rummens JL, Stinissen P. Memory CD4+CD127high T cells from patients with multiple sclerosis produce IL-17 in response to myelin antigens. J. Neuroimmunol. 226(1–2), 185–191 (2010).
  • Tesmer LA, Lundy SK, Sarkar S, Fox DA. Th17 cells in human disease. Immunol. Rev. 223, 87–113 (2008).
  • Korn T, Bettelli E, Oukka M, Kuchroo VK. IL-17 and Th17 cells. Annu. Rev. Immunol. 27, 485–517 (2009).
  • Kebir H, Kreymborg K, Ifergan I et al. Human Th17 lymphocytes promote blood-brain barrier disruption and central nervous system inflammation. Nat. Med. 13(10), 1173–1175 (2007).
  • Frischer JM, Bramow S, Dal-Bianco A et al. The relation between inflammation and neurodegeneration in multiple sclerosis brains. Brain 132(Pt 5), 1175–1189 (2009).
  • Magliozzi R, Howell O, Vora A et al. Meningeal B-cell follicles in secondary progressive multiple sclerosis associate with early onset of disease and severe cortical pathology. Brain 130(Pt 4), 1089–1104 (2007).
  • Lucchinetti CF, Popescu BF, Bunyan RF et al. Inflammatory cortical demyelination in early multiple sclerosis. N. Engl. J. Med. 365(23), 2188–2197 (2011).
  • Mezey E, Chandross KJ, Harta G, Maki RA, McKercher SR. Turning blood into brain: cells bearing neuronal antigens generated in vivo from bone marrow. Science 290(5497), 1779–1782 (2000).
  • Cogle CR, Yachnis AT, Laywell ED et al. Bone marrow transdifferentiation in brain after transplantation: a retrospective study. Lancet 363(9419), 1432–1437 (2004).
  • Cabanes C, Bonilla S, Tabares L, Martínez S. Neuroprotective effect of adult hematopoietic stem cells in a mouse model of motoneuron degeneration. Neurobiol. Dis. 26(2), 408–418 (2007).
  • Slavin S, Nagler A, Varadi G, Or R. Graft vs autoimmunity following allogeneic non-myeloablative blood stem cell transplantation in a patient with chronic myelogenous leukemia and severe systemic psoriasis and psoriatic polyarthritis. Exp. Hematol. 28(7), 853–857 (2000).
  • Reston JT, Uhl S, Treadwell JR, Nash RA, Schoelles K. Autologous hematopoietic cell transplantation for multiple sclerosis: a systematic review. Mult. Scler. 17(2), 204–213 (2011).
  • Takeuchi K, Inaba M, Miyashima S, Ogawa R, Ikehara S. A new strategy for treatment of autoimmune diseases in chimeric resistant MRL/lpr mice. Blood 91(12), 4616–4623 (1998).
  • Ikehara S, Ohtsuki H, Good RA et al. Prevention of Type I diabetes in nonobese diabetic mice by allogenic bone marrow transplantation. Proc. Natl Acad. Sci. USA 82(22), 7743–7747 (1985).
  • Theofilopoulos AN. Role of the thymus in murine lupus and cellular transfer of the disease. Arthritis Rheum. 25(7), 726–733 (1982).
  • Ikehara S, Good RA, Nakamura T et al. Rationale for bone marrow transplantation in the treatment of autoimmune diseases. Proc. Natl Acad. Sci. USA 82(8), 2483–2487 (1985).
  • Nishioka N, Toki J, Cherry et al. Repair mechanism of lupus nephritis in (NZB × NZW)F1 mice by allogeneic bone marrow transplantation. Immunobiology 192(5), 279–296 (1995).
  • Karussis D, Vourka-Karussis U, Mizrachi-Koll R, Abramsky O. Acute/relapsing experimental autoimmune encephalomyelitis: induction of long lasting, antigen-specific tolerance by syngeneic bone marrow transplantation. Mult. Scler. 5(1), 17–21 (1999).
  • Karussis DM, Slavin S, Ben-Nun A et al. Chronic-relapsing experimental autoimmune encephalomyelitis (CR-EAE): treatment and induction of tolerance, with high dose cyclophosphamide followed by syngeneic bone marrow transplantation. J. Neuroimmunol. 39(3), 201–210 (1992).
  • Karussis DM, Slavin S, Lehmann D, Mizrachi-Koll R, Abramsky O, Ben-Nun A. Prevention of experimental autoimmune encephalomyelitis and induction of tolerance with acute immunosuppression followed by syngeneic bone marrow transplantation. J. Immunol. 148(6), 1693–1698 (1992).
  • Karussis DM, Vourka-Karussis U, Lehmann D, Abramsky O, Ben-Nun A, Slavin S. Immunomodulation of autommunity in MRL/lpr mice with syngeneic bone marrow transplantation (SBMT). Clin. Exp. Immunol. 100(1), 111–117 (1995).
  • Verda L, Kim DA, Ikehara S et al. Hematopoietic mixed chimerism derived from allogeneic embryonic stem cells prevents autoimmune diabetes mellitus in NOD mice. Stem Cells 26(2), 381–386 (2008).
  • Kojima R, Kami M, Kim SW et al. Induction of graft-versus-autoimmune (GVA) disease effect against refractory psoriasis by complete donor-type chimerism and graft-versus-host disease after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 32(4), 439–442 (2003).
  • Loh Y, Oyama Y, Statkute L et al. Non-myeloablative allogeneic hematopoietic stem cell transplantation for severe systemic sclerosis: graft-versus-autoimmunity without graft-versus-host disease? Bone Marrow Transplant. 39(7), 435–437 (2007).
  • Slavin S, Aker M, Shapira MY, Resnick I, Bitan M, Or R. Reduced-intensity conditioning for the treatment of malignant and life-threatening non-malignant disorders. Clin. Transpl. 275–282 (2003).
  • Snowden JA, Kearney P, Kearney A et al. Long-term outcome of autoimmune disease following allogeneic bone marrow transplantation. Arthritis Rheum. 41(3), 453–459 (1998).
  • Yin JA, Jowitt SN. Resolution of immune-mediated diseases following allogeneic bone marrow transplantation for leukaemia. Bone Marrow Transplant. 9(1), 31–33 (1992).
  • Lowenthal RM, Cohen ML, Atkinson K, Biggs JC. Apparent cure of rheumatoid arthritis by bone marrow transplantation. J. Rheumatol. 20(1), 137–140 (1993).
  • Karussis DM, Vourka-Karussis U, Lehmann D et al. Prevention and reversal of adoptively transferred, chronic relapsing experimental autoimmune encephalomyelitis with a single high dose cytoreductive treatment followed by syngeneic bone marrow transplantation. J. Clin. Invest. 92(2), 765–772 (1993).
  • van Gelder M, Kinwel-Bohré EP, van Bekkum DW. Treatment of experimental allergic encephalomyelitis in rats with total body irradiation and syngeneic BMT. Bone Marrow Transplant. 11(3), 233–241 (1993).
  • van Gelder M, van Bekkum DW. Treatment of relapsing experimental autoimmune encephalomyelitis in rats with allogeneic bone marrow transplantation from a resistant strain. Bone Marrow Transplant. 16(3), 343–351 (1995).
  • van Bekkum DW, Bohre EP, Houben PF, Knaan-Shanzer S. Regression of adjuvant-induced arthritis in rats following bone marrow transplantation. Proc. Natl Acad. Sci. USA 86(24), 10090–10094 (1989).
  • Farge D, Labopin M, Tyndall A et al. Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica 95(2), 284–292 (2010).
  • Karussis D, Vaknin-Dembinsky A. Hematopoietic stem cell transplantation in multiple sclerosis: a review of the clinical experience and a report of an international meeting. Expert Rev. Clin. Immunol. 6(3), 347–352 (2010).
  • Saccardi R, Kozak T, Bocelli-Tyndall C et al.; Autoimmune Diseases Working Party of EBMT. Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database. Mult. Scler. 12(6), 814–823 (2006).
  • Saccardi R, Mancardi GL, Solari A et al. Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life. Blood 105(6), 2601–2607 (2005).
  • Mondria T, Lamers CH, te Boekhorst PA, Gratama JW, Hintzen RQ. Bone-marrow transplantation fails to halt intrathecal lymphocyte activation in multiple sclerosis. J. Neurol. Neurosurg. Psychiatr. 79(9), 1013–1015 (2008).
  • Fassas A, Anagnostopoulos A, Kazis A et al. Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study. Bone Marrow Transplant. 20(8), 631–638 (1997).
  • Fassas A, Kimiskidis VK, Sakellari I et al. Long-term results of stem cell transplantation for MS: a single-center experience. Neurology 76(12), 1066–1070 (2011).
  • Burt RK, Cohen BA, Russell E et al. Hematopoietic stem cell transplantation for progressive multiple sclerosis: failure of a total body irradiation-based conditioning regimen to prevent disease progression in patients with high disability scores. Blood 102(7), 2373–2378 (2003).
  • Burt RK, Loh Y, Cohen B et al. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a Phase I/II study. Lancet Neurol. 8(3), 244–253 (2009).
  • Shevchenko YL, Novik AA, Kuznetsov AN et al. High-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation as a treatment option in multiple sclerosis. Exp. Hematol. 36(8), 922–928 (2008).
  • Ni XS, Ouyang J, Zhu WH, Wang C, Chen B. Autologous hematopoietic stem cell transplantation for progressive multiple sclerosis: report of efficacy and safety at three yr of follow up in 21 patients. Clin. Transplant. 20(4), 485–489 (2006).
  • Krasulová E, Trneny M, Kozák T et al. High-dose immunoablation with autologous haematopoietic stem cell transplantation in aggressive multiple sclerosis: a single centre 10-year experience. Mult. Scler. 16(6), 685–693 (2010).
  • Mancardi GL, Sormani MP, Di Gioia M et al.; Italian BMT Study Group. Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: the Italian multicentre experience. Mult. Scler. 18(6), 835–842 (2012).
  • Metz I, Lucchinetti CF, Openshaw H et al. Autologous haematopoietic stem cell transplantation fails to stop demyelination and neurodegeneration in multiple sclerosis. Brain 130(Pt 5), 1254–1262 (2007).
  • Lu JQ, Joseph JT, Nash RA et al. Neuroinflammation and demyelination in multiple sclerosis after allogeneic hematopoietic stem cell transplantation. Arch. Neurol. 67(6), 716–722 (2010).
  • Lu JQ, Storek J, Metz L et al. Continued disease activity in a patient with multiple sclerosis after allogeneic hematopoietic cell transplantation. Arch. Neurol. 66(1), 116–120 (2009).
  • Saccardi R, Freedman MS, Sormani MP et al.; European Blood and Marrow Transplantation Group; Center for International Blood and Marrow Research; HSCT in MS International Study Group. A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Mult. Scler. 18(6), 825–834 (2012).
  • Muraro PA, Douek DC, Packer A et al. Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J. Exp. Med. 201(5), 805–816 (2005).
  • Samijn JP, te Boekhorst PA, Mondria T et al. Intense T cell depletion followed by autologous bone marrow transplantation for severe multiple sclerosis. J. Neurol. Neurosurg. Psychiatr. 77(1), 46–50 (2006).
  • Chen JT, Collins DL, Atkins HL, Freedman MS, Galal A, Arnold DL; Canadian MS BMT Study Group. Brain atrophy after immunoablation and stem cell transplantation in multiple sclerosis. Neurology 66(12), 1935–1937 (2006).
  • Rocca MA, Mondria T, Valsasina P et al. A three-year study of brain atrophy after autologous hematopoietic stem cell transplantation in rapidly evolving secondary progressive multiple sclerosis. AJNR. Am. J. Neuroradiol. 28(9), 1659–1661 (2007).
  • Roccatagliata L, Rocca M, Valsasina P et al.; Italian GITMO-NEURO Intergroup on Autologous Stem Cell Transplantation. The long-term effect of AHSCT on MRI measures of MS evolution: a five-year follow-up study. Mult. Scler. 13(8), 1068–1070 (2007).
  • Daikeler T, Hügle T, Farge D et al.; Working Party Autoimmune Diseases of the EBMT. Allogeneic hematopoietic SCT for patients with autoimmune diseases. Bone Marrow Transplant. 44(1), 27–33 (2009).
  • Griffith LM, Pavletic SZ, Tyndall A et al.; Workshop participants. Feasibility of allogeneic hematopoietic stem cell transplantation for autoimmune disease: position statement from a National Institute of Allergy and Infectious Diseases and National Cancer Institute-Sponsored International Workshop, Bethesda, MD, March 12 and 13, 2005. Biol. Blood Marrow Transplant. 11(11), 862–870 (2005).
  • Matsuo Y, Kamezaki K, Takeishi S et al. Encephalomyelitis mimicking multiple sclerosis associated with chronic graft-versus-host disease after allogeneic bone marrow transplantation. Intern. Med. 48(16), 1453–1456 (2009).
  • Rouquette-Gally AM, Boyeldieu D, Gluckman E, Abuaf N, Combrisson A. Autoimmunity in 28 patients after allogeneic bone marrow transplantation: comparison with Sjögren syndrome and scleroderma. Br. J. Haematol. 66(1), 45–47 (1987).
  • Bacigalupo A, Ballen K, Rizzo D et al. Defining the intensity of conditioning regimens: working definitions. Biol. Blood Marrow Transplant. 15(12), 1628–1633 (2009).
  • Carella AM, Giralt S, Slavin S. Low intensity regimens with allogeneic hematopoietic stem cell transplantation as treatment of hematologic neoplasia. Haematologica 85(3), 304–313 (2000).
  • Carella AM, Champlin R, Slavin S, McSweeney P, Storb R. Mini-allografts: ongoing trials in humans. Bone Marrow Transplant. 25(4), 345–350 (2000).
  • Kozák T, Havrdová E, Pit’ha J et al. Immunoablative therapy with autologous stem cell transplantation in the treatment of poor risk multiple sclerosis. Transplant. Proc. 33(3), 2179–2181 (2001).
  • Fassas A. Autologous stem cell transplants in treatment of multiple sclerosis: where we stand and future prospects. Int. J. Hematol. 76(Suppl. 1), 223–225 (2002).
  • Nash RA, Bowen JD, McSweeney PA et al. High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Blood 102(7), 2364–2372 (2003).
  • Carreras E, Saiz A, Marín P et al. CD34+ selected autologous peripheral blood stem cell transplantation for multiple sclerosis: report of toxicity and treatment results at one year of follow-up in 15 patients. Haematologica 88(3), 306–314 (2003).
  • Inglese M, Mancardi GL, Pagani E et al.; Italian GITMO-NEURO Group on Autologous Hematopoietic Stem Cell Transplantation. Brain tissue loss occurs after suppression of enhancement in patients with multiple sclerosis treated with autologous haematopoietic stem cell transplantation. J. Neurol. Neurosurg. Psychiatr. 75(4), 643–644 (2004).
  • Xu J, Ji BX, Su L, Dong HQ, Sun XJ, Liu CY. Clinical outcomes after autologous haematopoietic stem cell transplantation in patients with progressive multiple sclerosis. Chin. Med. J. 119(22), 1851–1855 (2006).
  • Freedman MS Atkins HL, Arnold DL, Bar-Or A. Immune ablation and autologous stem cell transplantation for aggressive multiple sclerosis: interim 5-year report. Mult. Scler. 13, Abstract Supp.22 (2007).
  • Saiz A, Blanco Y, Berenguer J et al. [Clinical outcome 6 years after autologous hematopoietic stem cell transplantation in multiple sclerosis]. Neurologia. 23(7), 405–407 (2008).
  • Openshaw H, Lund BT, Kashyap A et al. Peripheral blood stem cell transplantation in multiple sclerosis with busulfan and cyclophosphamide conditioning: report of toxicity and immunological monitoring. Biol. Blood Marrow Transplant. 6(5A), 563–575 (2000).
  • Fagius J, Lundgren J, Oberg G. Early highly aggressive MS successfully treated by hematopoietic stem cell transplantation. Mult. Scler. 15(2), 229–237 (2009).
  • Hamerschlak N, Rodrigues M, Moraes DA et al. Brazilian experience with two conditioning regimens in patients with multiple sclerosis: BEAM/horse ATG and CY/rabbit ATG. Bone Marrow Transplant. 45(2), 239–248 (2010).
  • Xu J, Ji BX, Su L et al. Clinical outcome of autologous peripheral blood stem cell transplantation in opticospinal and conventional forms of secondary progressive multiple sclerosis in a Chinese population. Ann. Hematol. 90(3), 343–348 (2011).
  • Bowen JD, Kraft GH, Wundes A et al. Autologous hematopoietic cell transplantation following high-dose immunosuppressive therapy for advanced multiple sclerosis: long-term results. Bone Marrow Transplant. 47(7), 946–951 (2012).

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