Autologous hemopoietic stem cell transplantation for multiple sclerosis: Is it worthwile?

Autoimmunity

Volumn 41, Issue 8, 2008, Pages 601-610

  Fassas, Athanasios ^a   Mancardi, Gian Luigi ^b  

^a G PAPANICOLAOU HOSPITAL   (Greece)

^b UNIVERSITY OF GENOA   (Italy)

Author keywords

Multiple sclerosis; Stem cell transplantation

Indexed keywords

CARMUSTINE; CYCLOPHOSPHAMIDE; CYTARABINE; ETOPOSIDE; GADOLINIUM; GRANULOCYTE COLONY STIMULATING FACTOR RECEPTOR; IMMUNOMODULATING AGENT; IMMUNOSUPPRESSIVE AGENT; MELPHALAN; METHYLPREDNISOLONE; MITOXANTRONE; THYMOCYTE ANTIBODY;

ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BONE MARROW TRANSPLANTATION; CLINICAL TRIAL; DRUG FEVER; DRUG MEGADOSE; HUMAN; IMMUNOLOGICAL TOLERANCE; IMMUNOSUPPRESSIVE TREATMENT; LIFESPAN; LYMPHOPROLIFERATIVE DISEASE; MORBIDITY; MORTALITY; MULTIPLE SCLEROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; PATIENT SELECTION; QUALITATIVE ANALYSIS; RISK FACTOR; STEM CELL MOBILIZATION; TREATMENT INDICATION; WHOLE BODY RADIATION;

DISEASE PROGRESSION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MULTIPLE SCLEROSIS; TRANSPLANTATION CONDITIONING;

EID: 55149089450     PISSN: 08916934     EISSN: 1607842X     Source Type: Journal    
DOI: 10.1080/08916930802197347     Document Type: Article

References (64)

1. Compston, A. and Coles, A. (2002) Multiple sclerosis. Lancet, 359:9313, pp. 1221-1231.
2. Goebels, N., Hofstetter, H. Schmidt, S. et al. (2000) Repertoire dynamics of autoreactive T cells in multiple sclerosis patients and healthy subjects: Epitope spreading versus clonal persistence. Brain, 123, pp. 508-518.
3. Lassmann, H. (2003) Brain damage when multiple sclerosis is diagnosed clinically. Lancet, 361:9366, pp. 1317-1318.
4. Bronnum-Hansen, H., Koch-Henriksen, N. and Stenager, E. (2004) Trends in survival and cause of death in Danish patients with multiple sclerosis. Brain, 127, pp. 844-850.
5. Daumer, M., Griffith, LM Meister, W. et al. (2006) Survival and time to an advanced disease state or progression, of untreated patients with moderately severe multiple sclerosis in a multicenter observational database: Relevance for design of a clinical trial for high dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation. Mult Scler, 12:2, pp. 174-179.
6. Filippini, G., Munari, L. Incorvaia, B. et al. (2003) Interferons in relapsing remitting multiple sclerosis: A systematic review. Lancet, 361:9357, pp. 545-552.
7. Martinelli Boneschi, F, Rovaris, M. Johnson, KP et al. (2003) Effects of glatiramer acetate on relapse rate and accumulated disability in multiple sclerosis: Meta-analysis of three double-blind, randomized, placebo-controlled clinical trials. Mult Scler, 9:4, pp. 349-355.
8. Polman, CH, O'Connor, PW Havrdova, E. et al. (2006) A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med, 354:9, pp. 899-910.
9. Hartung, HP, Gonsette, R. Konig, N. et al. (2002) Mitoxantrone in progressive multiple sclerosis: A placebo-controlled, double-blind, randomised, multicentre trial. Lancet, 360:9350, pp. 2018-2025.
10. Weiner, HL and Cohen, JA (2002) Treatment of multiple sclerosis with cyclophosphamide: Critical review of clinical and immunologic effects. Mult Scler, 8:2, pp. 142-154.
11. Coles, AJ, Wing, MG Molyneux, P. et al. (1999) Monoclonal antibody treatment exposes three mechanisms underlying the clinical course of multiple sclerosis. Ann Neurol, 46:3, pp. 296-304.
12. Barnett, MH and Prineas, JW (2004) Relapsing and remitting multiple sclerosis: Pathology of the newly forming lesion. Ann Neurol, 55:4, pp. 458-468.
13. Filippi, M., Bozzali, M. Rovaris, M. et al. (2003) Evidence for widespread axonal damage at the earliest clinical stage of multiple sclerosis. Brain, 126, pp. 433-437.
14. Confavreux, C., Vukusic, S., Moreau, T. and Adeleine, P. (2000) Relapses and progression of disability in multiple sclerosis. N Engl J Med, 343:20, pp. 1430-1438.
15. Coles, AJ, Cox, A. Le Page, E et al. (2006) The window of therapeutic opportunity in multiple sclerosis: Evidence from monoclonal antibody therapy. J Neurol, 253:1, pp. 98-108.
16. Ikehara, S., Good, RA Nakamura, T. et al. (1985) Rationale for bone marrow transplantation in the treatment of autoimmune diseases. Proc Natl Acad Sci U S A, 82:8, pp. 2483-2487.
17. Marmont, AM (1994) Immune ablation followed by allogeneic or autologous bone marrow transplantation: A new treatment for severe autoimmune diseases?. Stem Cells, 12:1, pp. 125-135.
18. van Bekkum, DW (2004) Stem cell transplantation for autoimmune disorders. Preclinical experiments. Best Pract Res Clin Haematol, 17:2, pp. 201-222.
19. Fassas, A., Anagnostopoulos, A. Kazis, A. et al. (1997) Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: First results of a pilot study. Bone Marrow Transplant, 20:8, pp. 631-638.
20. Fassas, A. and Nash, R. (2004) Stem cell transplantation for autoimmune disorders. Multiple sclerosis. Best Pract Res Clin Haematol, 17:2, pp. 247-262.
21. Fassas, A., Passweg, JR Anagnostopoulos, A. et al. (2002) Hematopoietic stem cell transplantation for multiple sclerosis. A retrospective multicenter study. J Neurol, 249:8, pp. 1088-1097.
22. Saccardi, R., Kozak, T. Bocelli-Tyndall, C. et al. (2006) 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, pp. 814-823.
23. Fassas, A., Anagnostopoulos, A. Kazis, A. et al. (2000) Autologous stem cell transplantation in progressive multiple sclerosis-an interim analysis of efficacy. J Clin Immunol, 20:1, pp. 24-30.
24. Kozak, T., Havrdova, E. Pit'ha, J. et al. (2000) High-dose immunosuppressive therapy with PBPC support in the treatment of poor risk multiple sclerosis. Bone Marrow Transplant, 25:5, pp. 525-531.
25. Mancardi, GL, Saccardi, R. Filippi, M. et al. (2001) Autologous hematopoietic stem cell transplantation suppresses Gd-enhanced MRI activity in MS. Neurology, 57:1, pp. 62-68.
26. Carreras, E., Saiz, A. Marin, P. et al. (2003) 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, pp. 306-314.
27. Saccardi, R., Mancardi, GL Solari, A. et al. (2005) Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: Impact on disease activity and quality of life. Blood, 105:6, pp. 2601-2607.
28. Su, L., Xu, J. Ji, BX et al. (2006) Autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Int J Hematol, 84:3, pp. 276-281.
29. Ni, XS, Ouyang, J. Zhu, WH et al. (2006) 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, pp. 485-489.
30. Nash, RA, Bowen, JD McSweeney, PA et al. (2003) High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Blood, 102:7, pp. 2364-2372.
31. Mancardi, GL, Murialdo, A. Rossi, P. et al. (2005) Autologous stem cell transplantation as rescue therapy in malignant forms of multiple sclerosis. Mult Scler, 11:3, pp. 367-371.
32. Havrdova, E. (2005) Aggressive multiple sclerosis-is there a role for stem cell transplantation?. J Neurol, 252, pp. III34-III37.
33. Portaccio, E., Amato, MP Siracusa, G. et al. (2007) Autologous hematopoietic stem cell transplantation for very active relapsing-remitting multiple sclerosis: Report of two cases. Mult Scler, 13:5, pp. 676-678.
34. Kimiskidis, V., Sakellari, I. Tsimourtou, V. et al. (2008) Treatment of a malignant form of multiple sclerosis with immune ablation and autologous stem cell transplantation. Mult Scler, 14:2, pp. 278-283.
35. Fagius, J. and Oberg, G. (2007) Haematopoietic stem cell transplantation in early highly aggressive multiple sclerosis. Mult Scler, 13
36. Burt, RK, Cohen, BA Russell, E. et al. (2003) 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, pp. 2373-2378.
37. Samijn, JP, te Boekhorst, PA Mondria, T. et al. (2006) Intense T-cell depletion followed by autologous bone marrow transplantation for severe multiple sclerosis. J Neurol Neurosurg Psychiatry, 77:1, pp. 46-50.
38. Saiz, A., Blanco, Y. Carreras, E. et al. (2004) Clinical and MRI outcome after autologous hematopoietic stem cell transplantation in MS. Neurology, 62:2, pp. 282-284.
39. Inglese, M., Mancardi, GL Pagani, E. et al. (2004) Brain tissue loss occurs after suppression of enhancement in patients with multiple sclerosis treated with autologous haematopoietic stem cell transplantation. J Neurol Neurosurg Psychiatry, 75:4, pp. 643-644.
40. Filippi, M., Rovaris, M. Capra, R. et al. (1998) A multi-centre longitudinal study comparing the sensitivity of monthly MRI after standard and triple dose gadolinium-DTPA for monitoring disease activity in multiple sclerosis. Implications for phase II clinical trials. Brain, 121, pp. 2011-2020.
41. Gratwohl, A., Passweg, J. Bocelli-Tyndall, C. et al. (2005) Autologous hematopoietic stem cell transplantation for autoimmune diseases. Bone Marrow Transplant, 35:9, pp. 869-879.
42. Burt, RK, Marmont, A. Oyama, Y. et al. (2006) Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases: The evolution from myeloablative to lymphoablative transplant regimens. Arthritis Rheum, 54:12, pp. 3750-3760.
43. Comi, G., Kappos, L. Clanet, M. et al. (2000) Guidelines for autologous blood and marrow stem cell transplantation in multiple sclerosis: A consensus report written on behalf of the European Group for Blood and Marrow Transplantation and the European Charcot Foundation. BMT-MS Study Group. J Neurol, 247:5, pp. 376-382.
44. Loh, Y., Oyama, Y. Statkute, L. et al. (2007) Development of a secondary autoimmune disorder after hematopoietic stem cell transplantation for autoimmune diseases: Role of conditioning regimen used. Blood, 109:6
45. Muraro, PA, Douek, DC Packer, A. et al. (2005) Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J Exp Med, 201:5, pp. 805-816.
46. De Kleer, I., Vastert, B. Klein, M. et al. (2006) Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood, 107:4, pp. 1696-1702.
47. Abrahamsson, S., Packer, A. Oh, U. et al. (2007) Effects of immunosopressive conditioning regimens on immune reconstitution after haematopoietic stem cell transplantation in patients with MS. Mult Scler, 13
48. Burt, RK, Fassas, A. Snowden, J. et al. (2001) Collection of hematopoietic stem cells from patients with autoimmune diseases. Bone Marrow Transplant, 28:1, pp. 1-12.
49. Statkute, L., Verda, L. Oyama, Y. et al. (2007) Mobilization, harvesting and selection of peripheral blood stem cells in patients with autoimmune diseases undergoing autologous hematopoietic stem cell transplantation. Bone Marrow Transplant, 39:6, pp. 317-329.
50. Snir, O., Lavie, G. Achiron, A. et al. (2006) G-CSF enhances the adhesion of encephalitogenic T cells to extracellular matrix components: A possible mechanism for exacerbation of multiple sclerosis. J Neuroimmunol, 172:1, pp. 145-155.
51. Openshaw, H., Lund, BT Kashyap, A. et al. (2000) Peripheral blood stem cell transplantation in multiple sclerosis with busulfan and cyclophosphamide conditioning: Report of toxicity and immunological monitoring. Biol Blood Marrow Transplant, 6:5, pp. 563-575.
52. Saiz, A., Carreras, E. Berenguer, J. et al. (2001) MRI and CSF oligoclonal bands after autologous hematopoietic stem cell transplantation in MS. Neurology, 56:8, pp. 1084-1089.
53. Metz, I., Lucchinetti, CF Openshaw, H. et al. (2007) Autologous haematopoietic stem cell transplantation fails to stop demyelination and neurodegeneration in multiple sclerosis. Brain, 130, pp. 1254-1262.
54. Nash, R., Stuve, O. Bowen, J. et al. (2008) Autologous HSCT for advanced MS: Is the glass half-empty or really half-full?. Brain, 131, pp. e89-e90.
55. Jager, HR, Williams, EJ Savage, DG et al. (1996) Assessment of brain changes with registered MR before and after bone marrow transplantation for chronic myeloid leukemia. AJNR Am J Neuroradiol, 17:7, pp. 1275-1282.
56. Chen, JT, Collins, DL Atkins, HL et al. (2006) Brain atrophy after immunoablation and stem cell transplantation in multiple sclerosis. Neurology, 66:12, pp. 1935-1937.
57. Roccatagliata, L., Rocca, M. Valsasina, P. et al. (2007) The long-term effect of AHSCT on MRI measures of MS evolution: A five-year follow-up study. Mult Scler, 13:8, pp. 1068-1070.
58. Rocca, MA, Mondria, T. Valsasina, P. et al. (2007) 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, pp. 1659-1661.
59. Sun, W., Popat, U. Hutton, G. et al. (2004) Characteristics of T-cell receptor repertoire and myelin-reactive T cells reconstituted from autologous haematopoietic stem-cell grafts in multiple sclerosis. Brain, 127, pp. 996-1008.
60. Kaloyannidis, P., Sakellari, I. Fassas, A. et al. (2004) Acquired hemophilia-A in a patient with multiple sclerosis treated with autologous hematopoietic stem cell transplantation and interferon beta-1a. Bone Marrow Transplant, 34:2, pp. 187-188.
61. McDonald, WI, Compston, A. Edan, G. et al. (2001) Recommended diagnostic criteria for multiple sclerosis: Guidelines from the International Panel on the diagnosis of multiple sclerosis. Ann Neurol, 50:1, pp. 121-127.
62. Edan, G., Miller, D. Clanet, M. et al. (1997) Therapeutic effect of mitoxantrone combined with methylprednisolone in multiple sclerosis: A randomised multicentre study of active disease using MRI and clinical criteria. J Neurol Neurosurg Psychiatry, 62:2, pp. 112-118.
63. Sormani, MP, Bruzzi, P. Rovaris, M. et al. (2001) Modelling new enhancing MRI lesion counts in multiple sclerosis. Mult Scler, 7:5, pp. 298-304.
64. Mancardi, GL (2007) Present status of HSCT in MS. Mult Scler, 13