Peripheral Blood Stem Cell Transplantation in Multiple Sclerosis with Busulfan and Cyclophosphamide Conditioning: Report of Toxicity and Immunological Monitoring

Biology of Blood and Marrow Transplantation

Volumn 6, Issue 5 A, 2000, Pages 563-575

  Openshaw, Harry ^a   Lund, Brett T ^b   Kashyap, Ashwin ^a   Atkinson, Roscoe ^b   Sniecinski, Irena ^a   Weiner, Leslie P ^b   Forman, Stephen ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Busulfan; Cyclophosphamide; Granulocyte colony stimulating factor; Multiple sclerosis; Myelin antigens; Peripheral blood stem cell transplantation

Indexed keywords

AUTOANTIBODY; AUTOANTIGEN; BUSULFAN; CYCLOPHOSPHAMIDE; EPITOPE; GRANULOCYTE COLONY STIMULATING FACTOR; IMMUNOSUPPRESSIVE AGENT; METHYLPREDNISOLONE;

ADULT; ARTICLE; AUTOIMMUNE DISEASE; BRAIN; CELL CULTURE; CEREBROSPINAL FLUID; CYTOTOXICITY; DISEASE COURSE; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOLOGY; IMMUNOSUPPRESSIVE TREATMENT; INFECTION; LYMPHOCYTE ACTIVATION; MIDDLE AGED; MORTALITY; MULTIMODALITY CANCER THERAPY; MULTIPLE SCLEROSIS; MYELIN SHEATH; NUCLEAR MAGNETIC RESONANCE IMAGING; PATHOLOGY; STEM CELL MOBILIZATION; T LYMPHOCYTE SUBPOPULATION; TREATMENT OUTCOME;

ADULT; AUTOANTIBODIES; AUTOANTIGENS; AUTOIMMUNE DISEASES; BRAIN; BUSULFAN; CELLS, CULTURED; COMBINED MODALITY THERAPY; CYCLOPHOSPHAMIDE; CYTOTOXICITY, IMMUNOLOGIC; DISEASE PROGRESSION; FEMALE; GRANULOCYTE COLONY-STIMULATING FACTOR; HEMATOPOIETIC STEM CELL MOBILIZATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNODOMINANT EPITOPES; IMMUNOSUPPRESSION; IMMUNOSUPPRESSIVE AGENTS; INFECTION; LYMPHOCYTE ACTIVATION; MAGNETIC RESONANCE IMAGING; METHYLPREDNISOLONE; MIDDLE AGED; MULTIPLE SCLEROSIS; MYELIN SHEATH; T-LYMPHOCYTE SUBSETS; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME;

EID: 0034576841     PISSN: 10838791     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1083-8791(00)70066-8     Document Type: Article

References (65)

1. Steinman L. Multiple sclerosis: a coordinated immunological attack against myelin in the central nervous system. Cell. 1996;85: 299-302.
2. Allegretta M, Nicklas JA, Sriram S, Albertini RJ. Tcells responsive to myelin basic protein in patients with multiple sclerosis. Science. 1990;247:718-721.
3. Kerlero de Rosbo N, Milo R, Lees MB, Burger D, Bernard CC, Ben-Nun A. Reactivity to myelin antigens in multiple sclerosis: peripheral blood lymphocytes respond predominantly to myelin oligodendrocyte glycoprotein. J Clin Invest. 1993;92:2602-2608.
4. Lisak RP, Zweiman B. In vitro cell-mediated immunity of cerebrospinal-fluid lymphocytes to myelin basic protein in primary demyelinating diseases. N Engl J Med. 1977;297:850-853.
5. Martin R, McFarland H, McFarlin D. Immunological aspects of demyelinating diseases. Annu Rev Immunol. 1992;10:153-187.
6. Trotter JL, Hickey WT, van der Veen RC, Sulze L. Peripheral blood mononuclear cells from multiple sclerosis patients recognize myelin proteolipid protein and selected peptides. J Neuroimmunol. 1991;33:55-62.
7. Wekerle H. Myelin specific, autoaggressive T cell clones in the normal immune repertoire: their nature and their regulation. Int Rev Immunol. 1992;9:231-241.
8. Raine CS. Biology of disease: analysis of autoimmune demyelination: its impact upon multiple sclerosis. Lab Invest. 1984;50:608-635.
9. Baig S, Olsson T, Yu-Ping J, Hojeberg B, Cruz M, Link H. Mutiple sclerosis: cells secreting antibodies against myelin-associated glycoprotein are present in cerebrospinal fluid. Scand J Immunol. 1991;33:73-79.
10. Birnbaum G, Kotilinek L, Albrecht L. Spinal fluid lymphocytes from a subgroup of multiple sclerosis patients respond to mycobacterial antigens. Ann Neural. 1993;34:18-24.
11. Chou Y, Bordette D, Offner H. Frequency of T cells specific for myelin basic protein and myelin proteolipid protein in blood and cerebrospinal fluid in multiple sclerosis. J Neuroimmunol. 1992;38:105-113.
12. Hafler DA, Benjamin DS, Burks J, Weiner HL. Myelin basic protein and proteolipid protein reactivity of brain- and cerebrospinal fluid-derived T cell clones in multiple sclerosis and postinfectious encephalomyelitis. J Immunol. 1987;139:68-72.
13. Hafler DA, Duby AD, Lee SJ, Benjamin D, Seidman JG, Weiner HL. Oligoclonal T lymphocytes in the cerebrospinal fluid of patients with multiple sclerosis. J Exp Med. 1988;167:1313-1322.
14. Fujinami RS, Oldstone MB. Amino acid homology between the encephalitogenic site of myelin basic protein and virus: mechanism for autoimmunity. Science. 1985;230:1043-1045.
15. Kalman B, Alder H, Lublin FD. Characteristics of the T lymphocytes involved in experimental allergic encephalomyelitis. J Neuroimmunol. 1995;61:107-116.
16. Olsson T. Critical influences of the cytokine orchestration on the outcome of myelin antigen-specific T-cell autoimmunity in experimental autoimmune encephalomyelitis and multiple sclerosis. Immunol Rev. 1995;144:245-268.
17. Woodroofe MN, Cuzner ML. Cytokine mRNA expression in inflammatory multiple sclerosis lesions: detection by non-radioactive in situ hybridization. Cytokine. 1993;5:583-588.
18. Correale J, McMillan M, McCarthy K, Le T, Weiner LP. Isolation and characterization of autoreactive proteolipid protein-peptide specific T-cell clones from multiple sclerosis patients. Neurology. 1995;45:1370-1378.
19. Panitch HS, Hirsch RL, Schindler J, Johnson KP. Treatment of multiple sclerosis with gamma interferon: exacerbations associated with activation of the immune system. Neurology. 1987;37:1097-1102.
20. Becker CC, Gidal BE, Fleming JO. Immunotherapy in multiple sclerosis, part 1. Am J Health Syst Pharm. 1995;52:1985-2000.
21. Hauser SL, Dawson DM, Lehrich JR, et al. Intensive immunosuppression in progressive multiple sclerosis: a randomized, three-arm study of high-dose intravenous cyclophosphamide, plasma exchange, and ACTH. N Engl J Med. 1983;308:173-180.
22. Hafler DA, Orav J, Gertz R, Stazzone L, Weiner HL. Immunologic effects of cyclophosphamide/ACTH in patients with chronic progressive multiple sclerosis. J Neuroimmunol. 1991;32:149-158.
23. Likosky WH, Fireman B, Elmore R, et al. Intense immunosuppression in chronic progressive multiple sclerosis: the Kaiser study. J Neurol Neurosurg Psychiatry. 1991;54:1055-1060.
24. Goodkin DE, Rudick RA, VanderBrug Medendorp S, et al. Low-dose (7.5 mg) oral methotrexate reduces the rate of progression in chronic progressive multiple sclerosis. Ann Neurol. 1995;37:30-40.
25. Olek MJ, Hohol MJ, Weiner HL. Methotrexate in the treatment of multiple sclerosis [letter]. Ann Neurol. 1996;39:684.
26. Beutler E, Sipe JC, Romine JS, Koziol JA, McMillan R, Zyroff J. The treatment of chronic progressive multiple sclerosis with cladaribine. Proc Natl Acad Sci U S A. 1996;93:1716-1720.
27. Kappos L, Polman C, Pozzilli C, et al. Placebo-controlled multicentre randomized trial of interferon beta-1b in treatment of secondary progressive multiple sclerosis: European Study Group on interferon beta-1b in secondary progressive MS. Lancet. 1998;352:1491-1497.
28. Hartung H, Gonsette R. Mitoxantrone in progressive multiple sclerosis: a placebo-controlled, randomized, observer-blind phase III trial: clinical results and three year follow-up [abstract]. Neurology. 1999; 52:A290.
29. 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. 1997; 20:631-638.
30. Burt RK, Traynor AE, Pope R, et al. Treatment of autoimmune disease by intense immunosuppressive conditioning and autologous hematopoietic stem cell transplantation. Blood. 1998;92:3505-3514.
31. Schumacher GA, Beebe G, Kibler RF, et al. Problems of experimental trials of therapy in multiple sclerosis: report by the panel on the evaluation of experimental trials in multiple sclerosis. Ann N Y Acad Sci. 1965;122:552.
32. Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology. 1983;33:1444-1452.
33. Somlo G, Sniecinski I, Odom-Maryon T, et al. Effect of CD34+ selection and various schedules of stem cell reinfusion and granulocyte colony-stimulating factor priming on hematopoietic recovery after high-dose chemotherapy for breast cancer. Blood. 1997;89:1521-1528.
34. Smith RE, Taylor CR, Rao NA, Young LL, Rife LL. Immuno-histochemical identification of androgen receptors in human lacrimal glands. Curr Eye Res. 1999;18:300-309.
35. Tourtellotte WW, Ma BI. Multiple sclerosis: the blood-brain-barrier and the measurement of de novo central nervous system IgG synthesis. Neurology. 1978;28:76-83.
36. Openshaw H, Stuve O, Antel JP, et al. Multiple sclerosis flares associated with recombinant granulocyte colony stimulating factor. Neurology. 2000;54:2147-2150.
37. Jain KK. Cutaneous vasculitis associated with granulocyte colony-stimulating factor. J Am Acad Dermatol. 1994;31:213-215.
38. Farhey YD, Herman JH. Vasculitis complicating granulocyte colony stimulating factor treatment of leukopenia and infection in Felty's syndrome. J Rheumatol. 1995;22:1179-1182.
39. Euler HH, Harten P, Zeuner RA, Schwab UM. Recombinant human granulocyte colony stimulating factor in patients with systemic lupus erythematosus associated neutropenia and refractory infections. J Rheumatol. 1997;24:2153-2157.
40. Xu S, Hoglund M, Venge P. The effect of granulocyte colony-stimulating factor (G-CSF) on the degranulation of secondary granule proteins from human neutrophils in vivo may be indirect. Br J Haematol. 1996;93:558-568.
41. Rieckmann P, Albrecht M, Kitze B, et al. Tumor necrosis factor-alpha messenger RNA expression in patients with relapsing-remitting multiple sclerosis is associated with disease activity. Ann Neurol. 1995;37:82-88.
42. + cells derived from granulocyte colony-stimulating factor-mobilized peripheral blood mononuclear cells. Blood. 1997;89:1629-1634.
43. Openshaw H, Hinton DR, Slatkin NE, Biennan PJ, Hoffman FM, Snyder DS. Exacerbation of inflammatory demyelinating polyneuropathy after bone marrow transplantation. Bone Marrow Transplant. 1991;7:411-414.
44. Openshaw H. Peripheral neuropathy after bone marrow transplantation. Biol Blood Marrow Transplant. 1997;3:202-209.
45. 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. 1995;16:343-351.
46. Chiang CS, McBride WH. Radiation enhances tumor necrosis factor alpha production by murine brain cells. Brain Res. 1991;566:265-269.
47. Openshaw H, Slatkin NE, Stein AS, Hinton DR, Forman SJ. Acute polyneuropathy after high dose cytosine arabinoside in patients with leukemia. Cancer. 1996;78:1899-1905.
48. Johnson NT, Crawford SW, Sargur M. Acute acquired demyelinating polyneuropathy with respiratory failure following high-dose systemic cytosine arabinoside and marrow transplantation. Bone Marrow Transplant. 1987;2:203-207.
49. Hassan M, Ehrsson H, Smedmyr B, et al. Cerebrospinal fluid and plasma concentrations of busulfan during high-dose therapy. Bone Marrow Transplant. 1989;4:113-114.
50. Santos GW. The development of busulfan/cyclophosphamide preparative regimens. Semin Oncol. 1993;20:12-16.
51. Elson LA, Galton DAG, Till M. The action of chlorambucil (CB. 1348) and busulfan (Myleran) on the haemopoietic organs of the rat. Br J Haematol. 1958;4:355.
52. Hobbs JR, Hugh-Jones K, Shaw PJ, Downie CJ, Williamson S. Engraftment rates related to busulphan and cyclophosphamide dosages for displacement bone marrow transplants in fifty children. Bone Marrow Transplant. 1986;1:201-208.
53. Slattery JT, Sanders JE, Buckner CD, et al. Graft-rejection and toxicity following bone marrow transplantation in relation to busulfan pharmacokinetics. Bone Marrow Transplant. 1995;16:31-42.
54. Hauser SL, Bhan AK, Gilles F, Kemp M, Kerr C, Weiner HL. Immunohistochemical analysis of the cellular infiltrate in multiple sclerosis lesions. Ann Neurol. 1986;19:578-587.
55. Burt RK, Bus W, Ruvolo P, et al. Syngeneic bone marrow transplantation eliminates V beta 8.2 T lymphocytes from the spinal cord of Lewis rats with experimental allergic encephalomyelitis. J Neurosci Res. 1995;41:526-531.
56. Azzaralli B, Bruck, Koler O. Bone marrow transplantation (BMT) in multiple sclerosis (MS) [abstract]. J Neuropath Exp Neurol. 1998;57:489.
57. + multiple sclerosis. Eur J Immunol. 1998;28:3329-3335.
58. Bieganowska KD, Ausubel LJ, Modabber Y, Slovik E, Messersmith W, Hafler DA. Direct ex vivo analysis of activated, Fas-sensitive autoreactive T cells in human autoimmune disease. J Exp Med. 1997;185:1585-1594.
59. Liblau R, Tournier-Lasserve E, Maciazek J, et al. T cell response to myelin basic protein epitopes in multiple sclerosis patients and healthy subjects. Eur J Immunol. 1991;21:1391-1395.
60. McFarland HF. The multiple sclerosis lesion [comment]. Ann Neurol. 1995;37:419-421.
61. Rudick R, Antel J, Confavreux C, et al. Recommendations from the National Multiple Sclerosis Society Clinical Outcomes Assessment Task Force. Ann Neurol. 1997;42:379-382.
62. Trapp BD, Peterson J, Ransohoff RM, Rudick R, Mork S, Bo L. Axonal transection in the lesions of multiple sclerosis. N Engl J Med. 1998;338:278-285.
63. Weaver CH, Schwartzberg LS, Hainsworth J, et al. Treatment-related mortality in 1000 consecutive patients receiving high-dose chemotherapy and peripheral blood progenitor cell transplantation in community cancer centers. Bone Marrow Transplant. 1997;19: 671-678.
64. Weinshenker BG, Rice GP, Noseworthy JH, Carriere W, Baskerville J, Ebers GC. The natural history of multiple sclerosis: a geographically based study, 4: applications to planning and interpretation of clinical therapeutic trials. Brain. 1991;114:1057-1067.
65. Myers L, Ellison G, Leake B, Mickey M. Selection of patients for therapeutic clinical trials in multiple sclerosis. In Siva A, Kesselring J, Thompson A, eds. Frontiers in Multiple Sclerosis. Vol. 2. London: Martin Duntz; 1999:151-160.