Chapter 15 Clinical Trials in Muscle Disorders

Blue Books of Practical Neurology

Volumn 25, Issue C, 2001, Pages 311-325

  Mantegazza, Renato ^a   Antozzi, Carlo ^a   Cornelio, Ferdinando ^a   Di Donato, Stefano ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

EID: 77957167805     PISSN: 18773419     EISSN: None     Source Type: Book Series    
DOI: 10.1016/S1877-3419(09)70022-4     Document Type: Article

References (103)

1. Marx A., Wilisch A., Schultz A., et al. Pathogenesis of myasthenia gravis. Virchows Arch 430 (1997) 355-364
2. Engel A.G. Myasthenic Syndromes. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology. 2nd ed (1994), McGraw-Hill, New York 1798-1835
3. Sinha S.J., Newsom-Davis J., Mills K., et al. Autoimmune etiology of acquired neuromyotonia (Isaac's syndrome). Lancet 338 (1991) 75-77
4. Shillito P., Molenaar P.C., Vincent A., et al. Acquired neuromyotonia: evidence for autoantibodies directed against K+ channels of peripheral nerves. Ann Neurol 38 (1995) 714-722
5. Grob D., Arsura E., Brunner N., and Namba T. The course of myasthenia gravis and therapies affecting outcome. Ann N Y Acad Sci 505 (1987) 472-499
6. Drachman D.B. Immunotherapy in neuromuscular disorders: current and future strategies. Muscle Nerve 19 (1996) 1239-1251
7. Cornelio F., Antozzi C., Mantegazza R., et al. Immunosuppressive treatments: their efficacy on myasthenia gravis patients' outcome and on the natural course of the disease. Ann N Y Acad Sci 681 (1993) 594-602
8. Jaretski III A. Thymectomy for myasthenia gravis. Analysis of the controversies regarding technique and results. Neurology 48 5 (1997) S52-S63
9. Sghirlanzoni A., Peluchetti D., Mantegazza R., et al. Myasthenia gravis: prolonged treatment with steroids. Neurology 34 (1984) 170-174
10. Mantegazza R., Antozzi C., Peluchetti D., et al. Azathioprine as a single drug or in combination with steroids in the treatment of myasthenia gravis. J Neurol 235 (1988) 449-453
11. Hohlfeld R., Toyka K., Besinger U.A., et al. Myasthenia gravis: reactivation of clinical disease and of autoimmune factors after discontinuation of long-term azathioprine. Ann Neurol 17 (1985) 238-242
12. Palace J., Newsom-Davis J., Lecky B., and the Myasthenia Gravis Study Group. A randomized doubleblind trial of prednisolone alone or with azathioprine in myasthenia gravis. Neurology 50 (1998) 1778-1783
13. Niakan E., Harati Y., and Rolak L.A. Immunosuppressive drug therapy in myasthenia gravis. Arch Neurol 43 (1986) 155-156
14. Tindall R., Rollins J.A., Phillips J.T., et al. Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis. N Engl J Med 316 (1987) 719-724
15. Mantegazza R., Confalonieri P., and Antozzi C. Video-assisted thoracoscopic extended thymectomy (VATET) in myasthenia gravis. Two-year follow-up in 101 patients and comparison with the trans-sternal approach. Ann N Y Acad Sci 841 (1998) 749-752
16. Pinching A.J., Peters D.K., and Newsom-Davis J. Remission of myasthenia gravis following plasma exchange. Lancet (1976) 1373-1376
17. Dau P.C., Lindstrom J.M., Cassel C.K., et al. Plasmapheresis and immunosuppressive drug therapy in myasthenia gravis. N Engl J Med 297 (1977) 1134-1140
18. Antozzi C., Gemma M., Regi B., et al. A short plasma exchange protocol is effective in severe myasthenia gravis. J Neurol 238 (1991) 103-107
19. Antozzi C., Berta E., Confalonieri P., et al. Protein-A immunoadsorption in immunosuppression-resistant myasthenia gravis. Lancet 383 (1994) 124
20. Berta E., Confalonieri P., Simoncini O., et al. Removal of antiacetylcholine receptor antibodies by protein A immunoadsorption in myasthenia gravis. Int J Artif Organs 11 (1994) 603-608
21. Howard J.F. Intravenous immunoglobulins for the treatment of acquired myasthenia gravis. Neurology 51 5 (1998) S30-S36
22. Gajdos P., Chevret S., Clair B., et al. for the Myasthenia Gravis Clinical Study Group. Clinical trial of plasma exchange and high-dose intravenous immunoglobulin in myasthenia gravis. Ann Neurol 41 (1997) 789-796
23. Stricker R.B., Kwiatkowska B.J., Habis J.A., and Kiprov D.D. Myasthenia gravis: response to plasmapheresis following failure of intravenous immunoglobulins. Arch Neurol 50 (1993) 837-840
24. Dau P.C., and Denys E.H. Plasmapheresis and immunosuppressive drug therapy in the Eaton-Lambert syndrome. Ann Neurol 11 (1982) 570-575
25. Newsom-Davis J., and Murray N.M.F. Plasma exchange and immunosuppressive drug treatment in the Lambert-Eaton myasthenic syndrome. Neurology 34 (1984) 480-485
26. Bain P.G., Motomura M., Newsom-Davis J., et al. Effects of intravenous immunoglobulin on muscle weakness and calcium-channel autoantibodies in the Lambert-Eaton myasthenic syndrome. Neurology 47 (1996) 678-683
27. Cornelio F., Antozzi C., Confalonieri P., et al. Plasma treatment in disorders of the neuromuscular junction. Ann N Y Acad Sci 841 (1998) 803-810
28. Browne D.L., Gancher S.T., Nutt J.G., et al. Episodic ataxia-myokymia syndrome is associated with point mutations in the human potassium channel gene KCNA1. Nat Genet 8 (1994) 136-140
29. Newsom-Davis J., and Mills K.R. Immunological associations of acquired neuromyotonia (Isaacs' syndrome). Report of five cases and literary review. Brain 116 (1993) 453-469
30. Hohlfeld H. Immunotherapeutic Strategies. In: Zhang J., Hafler D., Hohlfeld R., and Miller A. (Eds). Immunotherapy in Neuroimmunologic Diseases (1998), Martin Dunitz, London 197-213
31. Wang Z.Y., Qiao J., and Link H. Suppression of experimental autoimmune myasthenia gravis by oral administration of acetylcholine receptor. J Neuroimmunol 44 (1993) 209-214
32. Okumura S., McIntosh K., and Drachman D.B. Oral administration of acetylcholine receptor: effects on experimental autoimmune myasthenia gravis. Ann Neurol 36 (1994) 704-713
33. Ma C.G., Zhang G.X., Xiao B.G., et al. Suppression of experimental autoimmune myasthenia gravis by nasal administration of acetylcholine receptor. J Neuroimmunol 58 (1995) 51-60
34. Shi F.D., Bai X.F., Huang Y.M., et al. Nasal tolerance in experimental autoimmune myasthenia gravis (EAMG): induction of protective tolerance in primed animals. Clin Exp Immunol 111 (1998) 506-512
35. Wu B., Deng C., Goluszko E., and Christadoss P. Tolerance to a dominant T cell epitope in the acetylcholine receptor molecule induces epitope spread and suppresses murine myasthenia gravis. J Immunol 159 (1997) 3016-3023
36. Karachunski P.I., Ostlie N.S., Okita D.K., and Conti-Fine B.M. Prevention of experimental myasthenia gravis by nasal administration of synthetic acetylcholine receptor T epitope sequences. J Clin Invest 100 (1997) 3027-3035
37. Antozzi C., Baggi F., Andreetta F., et al. Oral administration of an immunodominant TAChR epitope modulates antigen-specific T cell responses in mice. Ann N Y Acad Sci 841 (1998) 568-571
38. Barchan D., Asher O., Tzartos S., et al. Modulation of the anti-acetylcholine receptor response and experimental autoimmune myasthenia gravis by recombinant fragments of the acetylcholine receptor. Eur J Immunol 28 (1998) 616-624
39. Mantegazza R., Bernasconi P., Confalonieri P., and Cornelio F. Inflammatory myopathies and systemic disorders: a review of immunopathogenetic mechanisms and clinical features. J Neurol 244 (1997) 177-287
40. Mantegazza R., Andreetta F., Bernasconi P., et al. Analysis of T cell receptor repertoire of muscle-infiltrating T lymphocytes in polymyositis. Restricted V alpha/beta rearrangements may indicate antigen-driven selection. J Clin Invest 91 (1993) 2880-2886
41. Dalakas M. Current treatment of inflammatory myopathies. Curr Opin Rheumatol 6 (1994) 595-601
42. Barohn R.J., Amato A.A., Sahenk Z., et al. Inclusion body myositis: explanation for poor response to immunosuppressive therapies. Neurology 45 (1995) 1302-1304
43. Bennington J.L., and Dau P.C. Patients with polymyositis and dermatomyositis who undergo plasma-pheresis therapy. Arch Neurol 38 (1981) 553-560
44. Clarke C.R., Dyall-Smith T., and Mackay I.R. Plasma exchange in dermatomyositis/polymyositis: beneficial effects in three cases. J Clin Lab Immunol 27 (1988) 149-152
45. Miller F.W., Leitman S.F., Cronin M.E., et al. Controlled trial of plasma exchange and leukapheresis in polymyositis and dermatomyositis. N Engl J Med 326 (1992) 1380-1384
46. Sussman G.L., and Pruzanski W. Treatment of inflammatory myopathy with intravenous gamma globulin. Curr Opin Rheumatol 7 (1995) 510-515
47. Mastaglia F.L., Phillips B.A., and Zilko P.J. Immunoglobulin therapy in inflammatory myopathy. J Neurol Neurosurg Psychiatry 65 (1998) 107-110
48. Dalakas M.C., Illa I., Dambrosia J.M., et al. A controlled trial of high-dose intravenous immune globulin infusions as treatment for dermatomyositis. N Engl J Med 329 (1993) 1993-2000
49. Dalakas M.C. Controlled studies with high-dose intravenous immunoglobulin in the treatment of dermatomyositis, inclusion body myositis and polymyositis. Neurology 51 5 (1998) S37-S45
50. Soueidan S.A., and Dalakas M.C. Treatment of inclusion-body myositis with high-dose intravenous immunoglobulin. Neurology 43 (1993) 876-879
51. Amato A.A., Barohn R.J., Jackson C.E., et al. Inclusion body myositis: treatment with intravenous immunoglobulins. Neurology 43 (1994) 876-879
52. Dalakas M.C., Sonies B., Dambrosia J., et al. Treatment of inclusion body myositis with IVIG: a double-blind, placebo-controlled study. Neurology 48 (1997) 712-716
53. Lundberg I.E., and Nyberg P. New developments in the role of cytokines and chemokines in inflammatory myopathies. Curr Opin Rheumatol 10 (1998) 521-529
54. Emery A. Duchenne Muscular Dystrophy. In: Harper P., and Bobrow M. (Eds). Oxford Monographs on Medical Genetics (1987), Oxford University Press, Oxford, UK 71-91
55. Engel A.G., Yamamoto M., and Fischbeck K.H. Dystrophinopathies. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology (1994), McGraw-Hill, New York 1133-1187
56. Koenig M., Beggs A.H., Moyer M., et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet 45 (1989) 498-506
57. Hoffman E.P., Brown R.H., and Kunkel L.M. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51 (1987) 919-928
58. Dubowitz V. The Muscular Dystrophy. Muscle Biopsy: A Practical Approach (1985), Baillière Tindall, East Sussex, UK 1289-1404
59. McDouall R.M., Dunn M.J., and Dubowitz V. Nature of the mononuclear infiltrate and the mechanism of muscle damage in juvenile dermatomyositis and Duchenne muscular dystrophy. J Neurol Sci 99 (1990) 199-217
60. Arahata K., and Engel A.G. Monoclonal antibody analysis of mononuclear cells in myopathies. I: Quantitation of subsets according to diagnosis and sites of accumulation and demonstration and counts of muscle fibers invaded by T cells. Ann Neurol 16 (1984) 193-208
61. Gorospe J.R.M., Tharp M.D., Hinckley J., et al. A role for mast cells in the progression of Duchenne muscular dystrophy? Correlations in dystrophin-deficient humans, dogs and mice. J Neurol Sci 122 (1994) 44-56
62. Gorospe J.R.M., Tharp M.D., Demitsu T., and Hoffman E.P. Dystrophin-deficient myofibers are vulnerable to mast cell granule-induced necrosis. Neuromusc Disord 4 (1994) 325-333
63. Bernasconi P., Torchiana E., Confalonieri P., et al. Expression of transforming growth factor-β1 in dystrophic patient muscles correlates with fibrosis: pathogenetic role of a fibrogenic cytokine. J Clin Invest 96 (1995) 1137-1144
64. Siegel I.M., Miller J.E., and Ray R.D. Failure of corticosteroid in the treatment of Duchenne (pseudohypertrophic) muscular dystrophy: report on a clinically matched three year double-blind study. Ill Med J 145 (1974) 32-33
65. Griggs R.C., Moxley III R.T., Mendell J.R., et al. Prednisone in Duchenne dystrophy. A randomized, controlled study defining the time course and dose response. Arch Neurol 84 (1991) 383-388
66. Griggs R.C., Moxley III R.T., Mendell J.R., et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 43 (1993) 520-527
67. Sansome A., Royston P., and Dubowitz V. Steroids in Duchenne muscular dystrophy; pilot study of a new low-dosage schedule. Neuromusc Disord 3 (1993) 567-569
68. Mesa L.E., Dubrovski A.L., Corderi J., et al. Steroids in Duchenne muscular dystrophy-Deflazacort trial. Neuromusc Disord 1 (1991) 261-266
69. Mendel J.R., Moxley R.T., Griggs R.C., et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 320 (1989) 1592-1597
70. Fenichel G.M., Florence J., Pestronk A., et al. Prednisone slows strength decline in Duchenne muscular dystrophy: two-year observation. Neurology 41 (1991) 166
71. Backman E., and Henriksson K.G. Low dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromusc Disord 5 (1995) 233-241
72. Angelini C., Pegoraro E., Turella E., et al. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve 17 (1994) 386-391
73. Gussoni E., Pavlath G., Lanctot A.M., et al. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 356 (1992) 435-438
74. Karpati G., Ajdukovich D., Arnold D., et al. Myoblast transfer in Duchenne muscular dystrophy. Ann Neurol 34 (1993) 8-17
75. Huard J., Bouchard J.P., Roy R., et al. Human myoblast transplantation: preliminary results of 4 cases. Muscle Nerve 15 (1992) 550-560
76. Tremblay J.P., Malouin F., Roy R., et al. Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 2 (1993) 99-112
77. Law P.K., Goodwin T.G., Fang Q., et al. Feasibility, safety and efficacy of myoblast transfer therapy on Duchenne muscular dystrophy boys. Cell Transplant 1 (1992) 235-244
78. Morandi L., Bernasconi P., Gebbia M., et al. Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer. Neuromusc Disord 5 (1995) 291-295
79. Mendell J.R., Kissel J.T., Amato A.A., et al. Myoblast transfer in the treatment of Duchenne's muscular dystrophy. N Engl J Med 333 (1995) 832-838
80. Neumeyer A.M., Cros D., McKenna-Yasek D., et al. Pilot study of myoblast transfer in the treatment of Becker muscular dystrophy. Neurology 51 (1998) 589-592
81. Huard J., Roy R., Bouchard J.P., et al. Human myoblast transplantation between immunohistocompatible donor and recipients produces immune reactions. Transplant Proc 24 (1992) 3049-3051
82. Gussoni E., Blau H.M., and Kunkel L.M. The fate of individual myoblasts after transplantation into muscles of DMD patients. Nature Med 3 (1997) 970-977
83. Bates D., Stevens J.C., and Hodgson P. "Polymyositis" with involvement of facial and distal musculature. One form of the facioscapulohumeral syndrome?. J Neurol Sci 19 (1973) 105-108
84. Munsat T.L., Piper D., Cancilla P., et al. Inflammatory myopathy with facioscapulohumeral distribution. Neurology 22 (1972) 335-347
85. Tawil R., McDermott M.P., Pandya S., et al. A pilot trial of prednisone in facioscapulohumeral muscular dystrophy. Neurology 48 (1997) 46-49
86. Kissel J.T., McDermott M.P., Natarajan R., et al. Pilot trial of albuterol in facioscapulohumeral muscular dystrophy. Neurology 50 (1998) 1402-1406
87. Harper P.S., and Rudel R. Myotonic Dystrophy. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology (1994), McGraw-Hill, New York 1192-1219
88. Aslanidis C., Jansen G., Amemiya C., et al. Cloning of the essential myotonic dystrophy region and mapping of the putative defect. Nature 355 (1992) 548-551
89. Brook J.D., McCurrach M.E., Harley H.G., et al. Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3' end of a transcript encoding a protein kinase family member. Cell 68 (1992) 799-808
90. Buxton J., Shelbourne P., Davies J., et al. Detection of an unstable fragment of DNA specific to individuals with myotonic dystrophy. Nature 355 (1992) 547-548
91. Harley H.G., Brook J.D., Rundle S.A., et al. Expansion of an unstable DNA region and phenotypic variation in myotonic dystrophy. Nature 355 (1992) 545-546
92. Imbert G., Kretz C., Johnson K., and Mandel J.L. Origin of the expansion mutation in myotonic dystrophy. Nat Genet 4 (1993) 72-76
93. Mahadevan M., Tsilfidis C., Sabourin L., et al. Myotonic dystrophy mutation: An unstable CTG repeat in the 3' untranslated region of the gene. Science 255 (1992) 1253-1255
94. Fu Y.H., Pizzuti A., Fenwick R.G., et al. An unstable triplet repeat in a gene related to myotonic muscular dystrophy, myotonin protein kinase. Science 255 (1992) 1256-1258
95. Munsat T.L. Therapy of myotonia. A double-blind evaluation of diphenylhydantoin, procainamide, and placebo. Neurology 17 (1967) 359-367
96. Durelli L., Mutani R., and Fassio F. The treatment of myotonia: evaluation of chronic oral taurine therapy. Neurology 33 (1983) 599-603
97. Pouget J., and Serratrice G. Myotonie avec faiblesse musculaire corrigée par l'exercice. Effet thérapeutique de la mexilétine. Rev Neurol 139 (1983) 665-672
98. Rennie M.J. Muscle protein turnover and wasting due to injury and disease. Br Med Bull 41 (1985) 257-264
99. Griggs R.C., Jorefowicz R., Kingstone W., et al. Mechanism of muscle wasting in myotonic dystrophy. Ann Neurol 27 (1990) 505-512
100. Vlachopapadopoulou E., Zachwieja J.J., Gertner J.M., et al. Metabolic and clinical response to recombinant human insulin-like growth factor I in myotonic dystrophy-A clinical research center study. J Clin Endocrinol Metab 80 (1995) 3715-3723
101. Carter J.N., and Steinbeck K.S. Reduced adrenal androgens in patients with myotonic dystrophy. J Clin Endocrinol Metab 60 (1985) 611-614
102. Griggs R.C., Pandya S., Florence J.M., et al. Randomized controlled trial of testosterone in myotonic dystrophy. Neurology 39 (1989) 219-222
103. Sugino M., Ohsawa N., Ito T., et al. A pilot study of dehydroepiandrosterone sulfate in myotonic dystrophy. Neurology 51 (1998) 586-589