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Volumn 45, Issue 6, 2012, Pages 909-917

Recommendations for myasthenia gravis clinical trials

(17)  Benatar, Michael a   Sanders, Donald B b   Burns, Ted M a,c   Cutter, Gary R a,d   Guptill, Jeffrey T a,b   Baggi, Fulvio e   Kaminski, Henry J a,f   Mantegazza, Renato e   Meriggioli, Matthew N g   Quan, Joanne h   Wolfe, Gil I a,i   Barohn, Richard J a   Howard, James F a   Antegazza, R a   Merrigioli, Matthew N a   Piantadosi, Steven a   Smirnow, Alexis a  


Author keywords

All clinical trials; Clinical trials methodology study design; Myasthenia; Outcome measures; Quality of life

Indexed keywords

BIOLOGICAL MARKER; CHOLINESTERASE INHIBITOR; IMMUNOMODULATING AGENT; STEROID;

EID: 84860897376     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.23330     Document Type: Article
Times cited : (131)

References (50)
  • 1
    • 0034641222 scopus 로고    scopus 로고
    • Myasthenia gravis. Recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America
    • Jaretzki A, Barohn RB, Ernstoff RM, Kaminski HJ, Keesey JC, Penn AS, et al. Myasthenia gravis. Recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology 2000; 55: 16-23.
    • (2000) Neurology , vol.55 , pp. 16-23
    • Jaretzki, A.1    Barohn, R.B.2    Ernstoff, R.M.3    Kaminski, H.J.4    Keesey, J.C.5    Penn, A.S.6
  • 2
    • 0031747483 scopus 로고    scopus 로고
    • A randomized double-blind trial of prednisolone alone or with a zathioprine in myasthenia gravis
    • The Myasthenia Gravis Study Group
    • Palace J, Newsom-Davis J, Lecky B, The Myasthenia Gravis Study Group. A randomized double-blind trial of prednisolone alone or with a zathioprine in myasthenia gravis. Neurology 1998; 50: 1778-1783.
    • (1998) Neurology , vol.50 , pp. 1778-1783
    • Palace, J.1    Newsom-Davis, J.2    Lecky, B.3
  • 3
    • 0027363275 scopus 로고
    • A randomised clinical trial comparing prednisone and azathioprine in myasthenia gravis. Results of the second interim analysis
    • Myasthenia Gravis Clinical Study Group
    • Myasthenia Gravis Clinical Study Group. A randomised clinical trial comparing prednisone and azathioprine in myasthenia gravis. Results of the second interim analysis. J Neurol Neurosurg Psychiatry 1993; 56: 1157-1163.
    • (1993) J Neurol Neurosurg Psychiatry , vol.56 , pp. 1157-1163
  • 5
    • 49049115186 scopus 로고    scopus 로고
    • A trial of mycophenolate mofetil with prednisone as initial immunotherapy in myasthenia gravis
    • Muscle Study Group
    • Muscle Study Group. A trial of mycophenolate mofetil with prednisone as initial immunotherapy in myasthenia gravis. Neurology 2008; 71: 394-399.
    • (2008) Neurology , vol.71 , pp. 394-399
  • 6
    • 0023118848 scopus 로고
    • Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis
    • Tindall RSA, Rollins JA, Phillips JT, Greenlee RG, Wells L, Belendiuk G. Preliminary results of a double-blind, randomized, placebo-controlled trial of cyclosporine in myasthenia gravis. N Engl J Med 1987; 316: 719-724.
    • (1987) N Engl J Med , vol.316 , pp. 719-724
    • Tindall, R.S.A.1    Rollins, J.A.2    Phillips, J.T.3    Greenlee, R.G.4    Wells, L.5    Belendiuk, G.6
  • 8
    • 84860912590 scopus 로고    scopus 로고
    • Efficacy of methotrexate in myasthenia gravis
    • Bethesda (MD): National Library of Medicine (US). [Accessed Dec 23].
    • Barohn RJ. Efficacy of methotrexate in myasthenia gravis. In. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). [Accessed 2008 Dec 23].
    • (2008) ClinicalTrials.gov [Internet]
    • Barohn, R.J.1
  • 9
    • 84860908896 scopus 로고    scopus 로고
    • Safety and efficacy study of eculizumab in patients with refractory generalized myasthenia gravis
    • Alexion Pharmaceuticals. In. Bethesda (MD): National Library of Medicine (US). [Accessed July 30].
    • Alexion Pharmaceuticals. Safety and efficacy study of eculizumab in patients with refractory generalized myasthenia gravis. In. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). [Accessed 2008 July 30].
    • (2008) ClinicalTrials.gov [Internet]
  • 10
    • 51649110864 scopus 로고    scopus 로고
    • The MGTX experience: challenges in planning and executing an international, multicenter clinical trial
    • Aban IB, Wolfe GI, Cutter GR, Kaminski HJ, Jaretzki A, Minisman G, et al. The MGTX experience: challenges in planning and executing an international, multicenter clinical trial. J Neuroimmunol 2008; 201: 80-84.
    • (2008) J Neuroimmunol , vol.201 , pp. 80-84
    • Aban, I.B.1    Wolfe, G.I.2    Cutter, G.R.3    Kaminski, H.J.4    Jaretzki, A.5    Minisman, G.6
  • 11
    • 84860909206 scopus 로고    scopus 로고
    • Efficacy of prednisone in the treatment of ocular myasthenia (EPITOME)
    • Bethesda (MD): National Library of Medicine (US). [Accessed Oct 14].
    • Benatar M. Efficacy of prednisone in the treatment of ocular myasthenia (EPITOME). In. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). [Accessed 2009 Oct 14].
    • (2009) ClinicalTrials.gov [Internet]
    • Benatar, M.1
  • 13
    • 27744586691 scopus 로고    scopus 로고
    • Treatment of myasthenia gravis exacerbation with intravenous immunoglobulin. A randomized double-blind clinical trial
    • Gajdos P, Tranchant C, Clair B, Bolgert F, Eymard B, Stojkovic T, et al. Treatment of myasthenia gravis exacerbation with intravenous immunoglobulin. A randomized double-blind clinical trial. Arch Neurol 2005; 62: 1689-1693.
    • (2005) Arch Neurol , vol.62 , pp. 1689-1693
    • Gajdos, P.1    Tranchant, C.2    Clair, B.3    Bolgert, F.4    Eymard, B.5    Stojkovic, T.6
  • 14
    • 34047230056 scopus 로고    scopus 로고
    • IV immunoglobulin in patients with myasthenia gravis: a randomized controlled trial
    • Zinman L, Ng E, Bril V. IV immunoglobulin in patients with myasthenia gravis: a randomized controlled trial. Neurology 2007; 68: 837-841.
    • (2007) Neurology , vol.68 , pp. 837-841
    • Zinman, L.1    Ng, E.2    Bril, V.3
  • 15
    • 0035698077 scopus 로고    scopus 로고
    • Immunoglobulin treatment versus plasma exchange in patients with chronic moderate to severe myasthenia gravis
    • Ronager J, Ravnborg M, Hermansen I, Vorstrup S. Immunoglobulin treatment versus plasma exchange in patients with chronic moderate to severe myasthenia gravis. Artif Organs 2001; 25: 967-973.
    • (2001) Artif Organs , vol.25 , pp. 967-973
    • Ronager, J.1    Ravnborg, M.2    Hermansen, I.3    Vorstrup, S.4
  • 16
    • 0031001759 scopus 로고    scopus 로고
    • Clinical trial of plasma exchange and high-dose intravenous immunoglobulin in myasthenia gravis. Myasthenia Gravis Clinical Study Group
    • Gajdos P, Chevret S, Clair B, Tranchant C, Chastang C. Clinical trial of plasma exchange and high-dose intravenous immunoglobulin in myasthenia gravis. Myasthenia Gravis Clinical Study Group. Ann Neurol 1997; 41: 789-796.
    • (1997) Ann Neurol , vol.41 , pp. 789-796
    • Gajdos, P.1    Chevret, S.2    Clair, B.3    Tranchant, C.4    Chastang, C.5
  • 17
    • 79958765623 scopus 로고    scopus 로고
    • Comparison of IVIg and PLEX in patients with myasthenia gravis
    • Barth D, Nabavi Nouri M, Ng E, Nwe P, Bril V. Comparison of IVIg and PLEX in patients with myasthenia gravis. Neurology 2011; 76: 2017-2023.
    • (2011) Neurology , vol.76 , pp. 2017-2023
    • Barth, D.1    Nabavi Nouri, M.2    Ng, E.3    Nwe, P.4    Bril, V.5
  • 18
    • 45249104185 scopus 로고    scopus 로고
    • Lessons from two trials of mycophenolate mofetil in myasthenia gravis
    • Sanders DB, Siddiqi ZA. Lessons from two trials of mycophenolate mofetil in myasthenia gravis. Ann N Y Acad Sci 2008; 1132: 249-253.
    • (2008) Ann N Y Acad Sci , vol.1132 , pp. 249-253
    • Sanders, D.B.1    Siddiqi, Z.A.2
  • 19
  • 21
    • 0141838996 scopus 로고    scopus 로고
    • A simple manual muscle test for myasthenia gravis: validation and comparison with the QMG score
    • Sanders DB, Tucker-Lipscomb B, Massey JM. A simple manual muscle test for myasthenia gravis: validation and comparison with the QMG score. Ann N Y Acad Sci 2003; 998: 440-444.
    • (2003) Ann N Y Acad Sci , vol.998 , pp. 440-444
    • Sanders, D.B.1    Tucker-Lipscomb, B.2    Massey, J.M.3
  • 23
    • 0036126222 scopus 로고    scopus 로고
    • Health-related quality of life in patients with myasthenia gravis and the relationship between patient-oriented assessment and conventional measurements
    • Padua L, Evoli A, Aprile I, Caliandro P, Mazza S, Padua R, et al. Health-related quality of life in patients with myasthenia gravis and the relationship between patient-oriented assessment and conventional measurements. Neurol Sci 2001; 22: 363-369.
    • (2001) Neurol Sci , vol.22 , pp. 363-369
    • Padua, L.1    Evoli, A.2    Aprile, I.3    Caliandro, P.4    Mazza, S.5    Padua, R.6
  • 24
    • 49449087743 scopus 로고    scopus 로고
    • A disease-specific measure of quality of life for myasthenia gravis
    • Muscle Study Group
    • Mullins LL, Carpentier MY, Paul RH, Sanders DB; Muscle Study Group. A disease-specific measure of quality of life for myasthenia gravis. Muscle Nerve 2008; 38: 947-956.
    • (2008) Muscle Nerve , vol.38 , pp. 947-956
    • Mullins, L.L.1    Carpentier, M.Y.2    Paul, R.H.3    Sanders, D.B.4
  • 25
    • 49449083294 scopus 로고    scopus 로고
    • Less is more, or almost as much: a 15-item quality-of-life instrument for myasthenia gravis
    • Muscle Study Group
    • Burns TM, Conaway MR, Cutter GR, Sanders DB; Muscle Study Group. Less is more, or almost as much: a 15-item quality-of-life instrument for myasthenia gravis. Muscle Nerve 2008; 38: 957-963.
    • (2008) Muscle Nerve , vol.38 , pp. 957-963
    • Burns, T.M.1    Conaway, M.R.2    Cutter, G.R.3    Sanders, D.B.4
  • 26
    • 56749163489 scopus 로고    scopus 로고
    • Construction of an efficient evaluative instrument for myasthenia gravis: the MG composite
    • Muscle Study Group
    • Burns TM, Conaway M, Cutter GR, Sanders DB; Muscle Study Group. Construction of an efficient evaluative instrument for myasthenia gravis: the MG composite. Muscle Nerve 2008; 38: 1553-1562.
    • (2008) Muscle Nerve , vol.38 , pp. 1553-1562
    • Burns, T.M.1    Conaway, M.2    Cutter, G.R.3    Sanders, D.B.4
  • 27
    • 77951810213 scopus 로고    scopus 로고
    • The MG composite: a valid and reliable outcome measure for myasthenia gravis patient care
    • MG Composite and MG-QOL, 15 Study Group
    • Burns TM, Conaway M, Sanders DB; MG Composite and MG-QOL, 15 Study Group. The MG composite: a valid and reliable outcome measure for myasthenia gravis patient care. Neurology 2010; 74: 1434-1440.
    • (2010) Neurology , vol.74 , pp. 1434-1440
    • Burns, T.M.1    Conaway, M.2    Sanders, D.B.3
  • 28
    • 0023696691 scopus 로고
    • Azathioprine as a single drug or in combination with steroids in the treatment of myasthenia gravis
    • Mantegazza R, Antozzi C, Peluchetti D, Sghirlanzoni A, Cornelio F. Azathioprine as a single drug or in combination with steroids in the treatment of myasthenia gravis. J Neurol 1988; 235: 449-453.
    • (1988) J Neurol , vol.235 , pp. 449-453
    • Mantegazza, R.1    Antozzi, C.2    Peluchetti, D.3    Sghirlanzoni, A.4    Cornelio, F.5
  • 29
    • 77954130816 scopus 로고    scopus 로고
    • History of outcome measures for myasthenia gravis
    • Burns TM. History of outcome measures for myasthenia gravis. Muscle Nerve 2010; 42: 5-13.
    • (2010) Muscle Nerve , vol.42 , pp. 5-13
    • Burns, T.M.1
  • 30
    • 84860912589 scopus 로고    scopus 로고
    • US Food and Drug Administration. Innovation or stagnation: Challenge and opportunity on the critical path to new medicinal products. In: FDA.gov/ScienceResearch/SpecialTopics/CriticalPath Initiative/CriticalPathOpportunitiesReports/ucm077262.htm. [Internet] [Accessed Apr 20].
    • US Food and Drug Administration. Innovation or stagnation: Challenge and opportunity on the critical path to new medicinal products. In: FDA.gov/ScienceResearch/SpecialTopics/CriticalPath Initiative/CriticalPathOpportunitiesReports/ucm077262.htm. [Internet] [Accessed 2011 Apr 20].
    • (2011)
  • 31
    • 84860908890 scopus 로고    scopus 로고
    • US Food and Drug Administration. Innovation or stagnation: critical path opportunities list. In: FDA.gov/ScienceResearch/SpecialTopics/ CriticalPathInitiative/CriticalPathOpportunitiesReports/default.htm. [Internet] [Accessed Apr 20].
    • US Food and Drug Administration. Innovation or stagnation: critical path opportunities list. In: FDA.gov/ScienceResearch/SpecialTopics/ CriticalPathInitiative/CriticalPathOpportunitiesReports/default.htm. [Internet] [Accessed 2011 Apr 20].
    • (2011)
  • 32
    • 0035100888 scopus 로고    scopus 로고
    • Biomarkers and surrogate endpoints: preferred definitions and conceptual framework
    • Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001; 69: 89-95.
    • (2001) Clin Pharmacol Ther , vol.69 , pp. 89-95
  • 33
    • 0034065330 scopus 로고    scopus 로고
    • Acetylcholine receptors and myasthenia
    • Lindstrom JM. Acetylcholine receptors and myasthenia. Muscle Nerve 2000; 23: 453-477.
    • (2000) Muscle Nerve , vol.23 , pp. 453-477
    • Lindstrom, J.M.1
  • 36
    • 33746849034 scopus 로고    scopus 로고
    • Neuroprotective agents for clinical trials in ALS: a systematic assessment
    • Traynor BJ, Bruijn L, Conwit R, Beal F, O'Neill G, Fagan SC, et al. Neuroprotective agents for clinical trials in ALS: a systematic assessment. Neurology 2006; 67: 20-27.
    • (2006) Neurology , vol.67 , pp. 20-27
    • Traynor, B.J.1    Bruijn, L.2    Conwit, R.3    Beal, F.4    O'Neill, G.5    Fagan, S.C.6
  • 37
    • 84860908893 scopus 로고    scopus 로고
    • TACT: the TREAT-NMD Advisory Committee for Therapeutics. In. TREAT-NMD.eu/resources/TACT/introduction/ [Internet]. [Accessed Aug 31].
    • TACT: the TREAT-NMD Advisory Committee for Therapeutics. In. TREAT-NMD.eu/resources/TACT/introduction/ [Internet]. [Accessed 2011 Aug 31].
    • (2011)
  • 38
    • 45549096108 scopus 로고    scopus 로고
    • Etanercept treatment in myasthenia gravis
    • Rowin J. Etanercept treatment in myasthenia gravis. Ann N Y Acad Sci 2008; 1132: 300-304.
    • (2008) Ann N Y Acad Sci , vol.1132 , pp. 300-304
    • Rowin, J.1
  • 39
    • 51849128170 scopus 로고    scopus 로고
    • Immunosuppression of experimental autoimmune myasthenia gravis by mycophenolate mofetil
    • Janssen SPM, Phernambucq M, Martinez P, De Baets MH, Losen M. Immunosuppression of experimental autoimmune myasthenia gravis by mycophenolate mofetil. J Neuroimmunol 2008; 201: 111-120.
    • (2008) J Neuroimmunol , vol.201 , pp. 111-120
    • Janssen, S.P.M.1    Phernambucq, M.2    Martinez, P.3    De Baets, M.H.4    Losen, M.5
  • 40
    • 40049103666 scopus 로고    scopus 로고
    • Anti-c5 antibody treatment ameliorates weakness in experimentally acquired myasthenia gravis
    • Zhou Y, Gong B, Lin F, Rother RP, Medof ME, Kaminski HJ. Anti-c5 antibody treatment ameliorates weakness in experimentally acquired myasthenia gravis. J Immunol 2007; 179: 8562-8567.
    • (2007) J Immunol , vol.179 , pp. 8562-8567
    • Zhou, Y.1    Gong, B.2    Lin, F.3    Rother, R.P.4    Medof, M.E.5    Kaminski, H.J.6
  • 41
    • 34548253969 scopus 로고    scopus 로고
    • Prevention of experimental autoimmune myasthenia gravis by rat Crry-Ig: a model agent for long-term complement inhibition in vivo
    • Hepburn NJ, Chamberlain-Banoub JL, Williams AS, Morgan BP, Harris CL. Prevention of experimental autoimmune myasthenia gravis by rat Crry-Ig: a model agent for long-term complement inhibition in vivo. Mol Immunol 2008; 45: 395-405.
    • (2008) Mol Immunol , vol.45 , pp. 395-405
    • Hepburn, N.J.1    Chamberlain-Banoub, J.L.2    Williams, A.S.3    Morgan, B.P.4    Harris, C.L.5
  • 43
    • 84860907609 scopus 로고    scopus 로고
    • US Food and Drug Administration. Guidance for industry adaptive design clinical trials for drugs and biologics. In: FDA.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. [Internet] [Accessed Apr 20].
    • US Food and Drug Administration. Guidance for industry adaptive design clinical trials for drugs and biologics. In: FDA.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. [Internet] [Accessed 2011 Apr 20].
    • (2011)
  • 45
    • 84860907610 scopus 로고    scopus 로고
    • Myasthenia Gravis Foundation of America. In: myasthenia.org [Internet] [Accessed Aug 31].
    • Myasthenia Gravis Foundation of America. In: myasthenia.org [Internet] [Accessed 2011 Aug 31].
    • (2011)
  • 46
    • 0033933798 scopus 로고    scopus 로고
    • Endovascular and surgical treatment of unruptured cerebral aneurysms: comparison of risks
    • Johnston SC, Wilson CB, Halback VV, et al. Endovascular and surgical treatment of unruptured cerebral aneurysms: comparison of risks. Ann Neurol 2000; 48: 11-19.
    • (2000) Ann Neurol , vol.48 , pp. 11-19
    • Johnston, S.C.1    Wilson, C.B.2    Halback, V.V.3
  • 47
    • 3543135271 scopus 로고    scopus 로고
    • Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group
    • D'Agostini RB. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group. Stat Med 1998; 17: 2265-2281.
    • (1998) Stat Med , vol.17 , pp. 2265-2281
    • D'Agostini, R.B.1
  • 49
    • 11244291391 scopus 로고    scopus 로고
    • Functional defect of regulatory CD4+CD25+ T cells in the thymus of patients with autoimmune myasthenia gravis
    • Ballandina A, Sandrine L, Dartevelle P, Saoudi A, Berrih-Aknin S. Functional defect of regulatory CD4+CD25+ T cells in the thymus of patients with autoimmune myasthenia gravis. Blood 2005; 105: 735-741.
    • (2005) Blood , vol.105 , pp. 735-741
    • Ballandina, A.1    Sandrine, L.2    Dartevelle, P.3    Saoudi, A.4    Berrih-Aknin, S.5
  • 50
    • 51649105337 scopus 로고    scopus 로고
    • The ageing and myasthenic thymus: a morphometric study validating a standard procedure in the histological workup of thymic specimens
    • Strobel P, Moritz R, Leite MI, Willcox N, Chuang WY, Gold R, et al. The ageing and myasthenic thymus: a morphometric study validating a standard procedure in the histological workup of thymic specimens. J Neuroimmunol 2008; 201-202: 64-73.
    • (2008) J Neuroimmunol , vol.201-202 , pp. 64-73
    • Strobel, P.1    Moritz, R.2    Leite, M.I.3    Willcox, N.4    Chuang, W.Y.5    Gold, R.6


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