Autologous stem cell transplantation in autoimmune and rheumatic diseases: from the molecular background to clinical applications

References

• Davidson A, Diamond B. General features of autoimmune disease. The autoimmune diseases4th edn, NR Rose, IR Mackay. Elsevier Academic Press, OxfordUK 2006; 25–36
   Google Scholar
• Cooper GS, Miller FW, Pandey JP. The role of genetic factors in autoimmune disease: implications for environmental research. Environ Health Perspect 1999; 107(Suppl 5)693–700
   PubMedGoogle Scholar
• Sykes M, Nikolic B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005; 435: 620–7
   PubMed Web of Science ®Google Scholar
• Burt RK, Marmont A, Oyama Y, Slavin S, Arnold R, Hiepe F, et al. Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases: the evolution from myeloablative to lymphoablative transplant regimens. Arthritis Rheum 2006; 54: 3750–60
   PubMed Web of Science ®Google Scholar
• Pavletic SZ. Nonmyeloablative allogeneic hematopoietic stem cell transplantation for autoimmune disease. Arthritis Rheum 2004; 50: 2387–90
   PubMed Web of Science ®Google Scholar
• Van Laar JM, Tyndall A. Intense immunosuppression and stem-cell transplantation for patients with severe rheumatic autoimmune disease: a review. Cancer Control 2003; 10: 57–65
   PubMedGoogle Scholar
• Gratwohl, A. Allogeneic hematopoietic stem cell transplantation for severe autoimmune diseases. Autoimmunity Oct, 2008; 28(1), DOI: 10.1080/08916930802197677
   Google Scholar
• Beilhack GF, Scheffold YC, Weissman IL, Taylor C, Jerabek L, Burge MJ, et al. Purified allogeneic hematopoietic stem cell transplantation blocks diabetes pathogenesis in NOD mice. Diabetes 2003; 52: 59–68
   PubMed Web of Science ®Google Scholar
• Salomon BL, Sudres M, Cohen JL. Regulatory T cells in graft-versus-host disease. Springer Semin Immunopathol 2006; 28: 25–9
   PubMedGoogle Scholar
• Muraro PA, Douek DC, Packer A, Chung K, Guenaga FJ, Cassiani-Ingoni R, et al. Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J Exp Med 2005; 201: 805–16
   PubMed Web of Science ®Google Scholar
• Mackall CL, Bare CV, Granger LA, Sharrow SO, Titus JA, Gress RE. Thymic-independent T cell regeneration occurs via antigen-driven expansion of peripheral T cells resulting in a repertoire that is limited in diversity and prone to skewing. J Immunol 1996; 156: 4609–16
   PubMed Web of Science ®Google Scholar
• Hirota K, Hashimoto M, Yoshitomi H, Tanaka S, Nomura T, Yamaguchi T, et al. T cell self-reactivity forms a cytokine milieu for spontaneous development of IL-17+ Th cells that cause autoimmune arthritis. J Exp Med 2007; 204: 41–7
   PubMed Web of Science ®Google Scholar
• Annacker O, Pimenta-Araujo R, Burlen-Defranoux O, Barbosa TC, Cumano A, Bandeira A. CD25+ CD4+ T cells regulate the expansion of peripheral CD4 T cells through the production of IL-10. J Immunol 2001; 166: 3008–18
   PubMed Web of Science ®Google Scholar
• Hagen KA, Moses CT, Drasler EF, Podetz-Pedersen KM, Jameson SC, Khoruts A. A role of CD28 in lymphopenia-induced proliferation of CD4 T cells. J Immunol 2004; 173: 3909–15
   PubMed Web of Science ®Google Scholar
• Herrmann MM, Gaertner S, Stadelmann C, van den Brandt J, Böscke R, Budach W, et al. Tolerance induction by bone marrow transplantation in a multiple sclerosis model. Blood 2005; 106: 1875–83
   PubMed Web of Science ®Google Scholar
• de Kleer I, Vastert B, Klein M, Teklenburg G, Arkesteijn G, Yung GP, et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 2006; 107: 1696–702
   PubMed Web of Science ®Google Scholar
• Sykes M, Nikolic B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005; 435: 620–7
   PubMed Web of Science ®Google Scholar
• Tyndall A, Leblanc K. Stem cells and rheumatology: update on adult stem cell therapy in autoimmune diseases. Arthritis Rheum 2006; 55: 521–5
   PubMed Web of Science ®Google Scholar
• Van Laar JM, Tyndall A. Adult stem cells in the treatment of autoimmune diseases. Rheumatology (Oxford) 2006; 45: 1187–93
   PubMed Web of Science ®Google Scholar
• Daikeler T, Tyndall A. Autoimmunity following haematopoietic stem-cell transplantation. Best Pract Res Clin Haematol 2007; 20: 349–60
   PubMed Web of Science ®Google Scholar
• Laustrup H, Voss A, Green A, Junker P. Occurrence of systemic lupus erythematosus in a Danish community: an 8-year prospective study. Scand J Rheumatol 2009; 38: 128–32
   PubMed Web of Science ®Google Scholar
• Mok CC, Ho LY, Cheung MY, Yu KL, To CH. Effect of disease activity and damage on quality of life in patients with systemic lupus erythematosus: a 2-year prospective study. Scand J Rheumatol 2009; 38: 121–7
   PubMed Web of Science ®Google Scholar
• Svenungsson E, Cederholm A, Jensen-Urstad K, Fei GZ, de Faire U, Frostegård J. Endothelial function and markers of endothelial activation in relation to cardiovascular disease in systemic lupus erythematosus. Scand J Rheumatol 2008; 37: 352–9
   PubMed Web of Science ®Google Scholar
• Jayne D. Stem cell transplantation in systemic lupus erythematosus. Best Pract Res Clin Haematol 2004; 17: 291–304
   PubMedGoogle Scholar
• de Buys P, Khanna D, Furst DE. Hemopoietic stem cell transplantation in rheumatic diseases – an update. Autoimmun Rev 2005; 4: 442–9
   PubMedGoogle Scholar
• Burt RK, Traynor A, Statkute L, Barr WG, Rosa R, Schroeder J, et al. Nonmyeloablative hematopoietic stem cell transplantation for systemic lupus erythematosus. J Am Med Assoc 2006; 295: 527–35
   PubMed Web of Science ®Google Scholar
• Illei GG, Pavletic SZ. Blood stem cells as therapy for severe lupus. Expert Opin Biol Ther 2005; 5: 1153–64
   PubMed Web of Science ®Google Scholar
• Pavletic SZ, Illei GG. The role of immune ablation and stem cell transplantation in severe SLE. Best Pract Res Clin Rheumatol 2005; 19: 869–58
   Google Scholar
• Marmont, AM, Burt, RK. Hematopoietic stem cell transplantation for systemic lupus erythematosus, the antiphospholipid syndrome and bullous skin diseases. Autoimmunity Oct, 2008; 28(1), DOI: 10.1080/08916930802197677
   Google Scholar
• Traynor AE, Schroeder J, Rosa RM, Cheng D, Stefka J, Mujais S, et al. Treatment of severe systemic lupus erythematosus with high-dose chemotherapy and haemopoietic stem-cell transplantation: a phase I study. Lancet 2000; 356: 701–7
   PubMed Web of Science ®Google Scholar
• Jayne D, Passweg J, Marmont A, Farge D, Zhao X, Arnold R, et al. European Group for Blood and Marrow Transplantation; European League Against Rheumatism Registry. Autologous stem cell transplantation for systemic lupus erythematosus. Lupus 2004; 13: 168–76
   PubMed Web of Science ®Google Scholar
• Illei GG. Hematopoietic stem cell transplantation in autoimmune diseases: is the glass half full or half empty?. Arthritis Rheum 2006; 54: 3730–4
   PubMed Web of Science ®Google Scholar
• Karim MY, Pisoni CN, Khamashta MA. Update on immunotherapy for systemic lupus erythematosus – what's hot and what's not!. Rheumatology (Oxford) 2009; 48: 332–41
   PubMed Web of Science ®Google Scholar
• Hügle T, van Laar JM. Stem cell transplantation for rheumatic autoimmune diseases. Arthritis Res Ther 2008; 10: 217
   PubMed Web of Science ®Google Scholar
• Scheja, A, Bartosik, I, Wuttge, DM, Hesselstrand, R. Renal function is mostly preserved in patients with systemic sclerosis. Scand J Rheumatol Feb, 2009; 11: 1–4, DOI: 10.1080/03009740802629424
   Google Scholar
• Kampolis C, Plastiras S, Vlachoyiannopoulos P, Moyssakis I, Tzelepis G. The presence of anti-centromere antibodies may predict progression of estimated pulmonary arterial systolic pressure in systemic sclerosis. Scand J Rheumatol 2008; 37: 278–83
   PubMed Web of Science ®Google Scholar
• Farge D, Henegar C, Carmagnat M, Daneshpouy M, Marjanovic Z, Rabian C, et al. Analysis of immune reconstitution after autologous bone marrow transplantation in systemic sclerosis. Arthritis Rheum 2005; 52: 1555–63
   PubMed Web of Science ®Google Scholar
• Farge D, Passweg J, van Laar JM, Marjanovic Z, Besenthal C, Finke J, et al. EBMT/EULAR Registry. Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry. Ann Rheum Dis 2004; 63: 974–81
   PubMed Web of Science ®Google Scholar
• Nikolov NP, Pavletic SZ. Technology Insight: hematopoietic stem cell transplantation for systemic rheumatic disease. Nat Clin Pract Rheumatol 2008; 4: 184–91
   PubMedGoogle Scholar
• van Laar JM, Farge D, Tyndall A. Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial: hope on the horizon for patients with severe systemic sclerosis. Ann Rheum Dis 2005; 64: 1515
   PubMed Web of Science ®Google Scholar
• Tyndall A, Furst DE. Adult stem cell treatment of scleroderma. Curr Opin Rheumatol 2007; 19: 604–10
   PubMed Web of Science ®Google Scholar
• van Laar, JM, Farge, D, Tyndall, A. Stem cell transplantation: a treatment option for severe systemic sclerosis?. Ann Rheum Dis 2008;67(Suppl 3)iii35–iii38
   PubMedGoogle Scholar
• Fassas A, Anagnostopoulos A, Kazis A, Kapinas K, Sakellari I, Kimiskidis V, et al. Autologous stem cell transplantation in progressive multiple sclerosis – an interim analysis of efficacy. J Clin Immunol 2000; 20: 24–30
   PubMed Web of Science ®Google Scholar
• Su L, Xu J, Ji BX, Wan SG, Lu CY, Dong HQ, et al. Autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Int J Hematol 2006; 84: 276–81
   PubMed Web of Science ®Google Scholar
• Kozak T, Havrdova E, Pit'ha J, Gregora E, Pytlík R, Maaloufova J, et al. High-dose immunosuppressive therapy with PBPC support in the treatment of poor risk multiple sclerosis. Bone Marrow Transplant 2000; 25: 525–31
   PubMedGoogle Scholar
• Mancardi G, Saccardi R. Autologous haematopoietic stem cell transplantation in multiple sclerosis. Lancet Neurol 2008; 7: 626–36
   PubMed Web of Science ®Google Scholar
• Saccardi R, Mancardi GL, Solari A, Bosi A, Bruzzi P, Di Bartolomeo P, et al. Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life. Blood 2005; 105: 2601–7
   PubMed Web of Science ®Google Scholar
• Saiz A, Blanco Y, Carreras E, Berenguer J, Rovira M, Pujol T, et al. Clinical and MRI outcome after autologous hematopoietic stem cell transplantation in MS. Neurology 2004; 62: 282–4
   PubMed Web of Science ®Google Scholar
• Mancardi, GL. Autologous Stem-cell Transplantation International MS trial. EudraCT number, 2007-000064-24. Available online at: www.astims.org (accessed 29 May 2008).
   Google Scholar
• Bowen, J. High-Dose Immunosuppression and Autologous Transplantation for Multiple Sclerosis (HALT MS) Study, Available online at: http://clinicaltrials.gov/ct2/show/NCT00288626.
   Google Scholar
• Carli C, Bridges JF, Ask J, Lindblad S. Swedish Rheumatoid Arthritis Register. Charting the possible impact of national guidelines on the management of rheumatoid arthritis. Scand J Rheumatol 2008; 37: 188–93
   PubMed Web of Science ®Google Scholar
• Snowden JA, Passweg J, Moore JJ, Milliken S, Cannell P, Van Laar J, et al. Autologous hematopoietic stem cell transplantation in severe rheumatoid arthritis. J. Rheumatol 2004; 31: 482–8
   PubMed Web of Science ®Google Scholar
• Tyndall A, Saccardi R. Haematopoietic stem cell transplantation in the treatment of severe autoimmune disease: results from phase I/II studies, prospective randomized trials and future directions. Clin Exp Immunol 2005; 141: 1–9
   PubMed Web of Science ®Google Scholar
• Verburg RJ, Kruize AA, van den Hoogen FH, Fibbe WE, Petersen EJ, Preijers F, et al. High dose chemotherapy and autologous stem cell transplantation in patients with rheumatoid arthritis: results of an open study to assess feasibility, safety and efficacy. Arthritis Rheum 2001; 44: 754–60
   PubMed Web of Science ®Google Scholar
• Moore J, Brooks P, Milliken S, Biggs J, Ma D, Handel M, et al. A pilot randomized trial comparing CD34-selected versus unmanipulated hemopoietic stem cell transplantation for severe, refractory rheumatoid arthritis. Arthritis Rheum 2002; 46: 2301–9
   PubMed Web of Science ®Google Scholar
• Passweg J, Tyndall A. Autologous stem cell transplantation in autoimmune diseases. Semin Hematol 2007; 44: 278–85
   PubMed Web of Science ®Google Scholar
• Verburg RJ, Flierman R, Sont JK, Ponchel F, van Dreunen L, Levarht EW, et al. Outcome of intensive immunosuppression and autologous stem cell transplantation in patients with severe rheumatoid arthritis is associated with the composition of synovial T cell infiltration. Ann Rheum Dis 2005; 64: 1397–405
   PubMed Web of Science ®Google Scholar
• Teng YK, Verburg RJ, Sont JK, van den Hout WB, Breedveld FC, van Laar JM. Long-term followup of health status in patients with severe rheumatoid arthritis after high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Arthritis Rheum 2005; 52: 2272–6
   PubMed Web of Science ®Google Scholar
• Barron KS, Wallace C, Woolfrey CEA, Laxer RM, Hirsch R, Horwitz M, et al. Autologous stem cell transplantation for pediatric rheumatic diseases. J Rheumatol 2001; 28: 2337–58
   PubMedGoogle Scholar
• Wulffraat NM, de Kleer IM, Prakken BJ, Kuis W. Stem cell transplantation for autoimmune disorders. Refractory juvenile idiopathic arthritis. Best Pract Res Clin Haematol 2004; 17: 277–89
   PubMed Web of Science ®Google Scholar
• Wallace CA. Current management of juvenile idiopathic arthritis. Best Pract Res Clin Rheumatol 2006; 20: 279–300
   PubMed Web of Science ®Google Scholar
• Miltenyi S. CD34+ selection: the basic component for graft engineering. Oncologist 1997; 2: 410–13
   PubMedGoogle Scholar
• Wulffraat NM, Rijkers GT, Elst E, Brooimans R, Kuis W. Reduced perforin expression in systemic juvenile idiopathic arthritis is restored by autologous stem-cell transplantation. Rheumatology (Oxford) 2003; 42: 375–9
   PubMed Web of Science ®Google Scholar
• Verda, L, Oyama, Y, Craig, R, Statkute, L, Krosnjar, N, Quigley, K, , et al. Restoration of the CD4+CD25bright regulatory cell subset in patients with Crohn's disease following autologous non-myeloablative stem cell transplantation (NST). Blood 2005; 106, Abstract 2193
   PubMedGoogle Scholar
• Váróczy L, Illés A, Kiss E, Szekanecz Z, Soltész P, Sipka S, et al. High-dose chemotherapy followed by autologous CD34+ stem cell transplantation in the treatment of severe refractory multisystem autoimmune diseases of adults – first experiences in Hungary. Orv Hetil 2008; 149: 1405–12
   PubMedGoogle Scholar
• Zeher M, Szegedi G. The treatment of autoimmune patients with autologous stem cell transplantation. Lege Artis Medicinae 2007; 17: 853–8
   Google Scholar
• de Kleer I, Vastert B, Klein M, Teklenburg G, Arkesteijn G, Yung GP, et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 2006; 107: 1696–702
   PubMed Web of Science ®Google Scholar
• Braun-Moscivivi Y, Furst DE. Stem cell therapy in scleroderma. Curr Opin Rheumatol 2002; 14: 711–16
   PubMedGoogle Scholar
• Bohgaki T, Atsumi T, Koike T. Autoimmune disease after autologous hematopoietic stem cell transplantation. Autoimmun Rev 2008; 7: 198–203
   PubMed Web of Science ®Google Scholar
• Imamura R, Inoue H, Kato K, Kobayashi S, Tsukamoto H, Nagafuji K, et al. Development of rheumatoid arthritis following autologous peripheral blood stem cell transplantation. Bone Marrow Transplant 2002; 30: 527–9
   PubMed Web of Science ®Google Scholar
• Müller I, Lymperi S, Dazzi F. Mesenchymal stem cell therapy for degenerative inflammatory disorders. Curr Opin Organ Transplant 2008; 13: 639–44
   PubMed Web of Science ®Google Scholar