Comparison of outcome measures from a trial of mycophenolate mofetil in myasthenia gravis

Muscle and Nerve

Volumn 38, Issue 5, 2008, Pages 1429-1433

  Wolfe, Gil I ^a   Barohn, Richard J ^b,e   Sanders, Donald B ^c   McDermott, Michel P ^d,e   Thornton, Charles ^e   Tawil, Rabi ^e  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^b UNIVERSITY OF KANSAS SCHOOL OF MEDICINE   (United States)

^c DUKE UNIVERSITY MEDICAL CENTER   (United States)

^d UNIVERSITY OF ROCHESTER SCHOOL OF MEDICINE AND DENTISTRY   (United States)

^e NONE

Author keywords

Clinical trials; Myasthenia; Outcome measures

Indexed keywords

MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PLACEBO; PREDNISONE;

ADULT; AGED; ANALYTIC METHOD; ARTICLE; CLINICAL EVALUATION; CLINICAL TRIAL; COMPARATIVE STUDY; CONTROLLED STUDY; CORRELATION ANALYSIS; DAILY LIFE ACTIVITY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; EQUIPMENT; FEMALE; HUMAN; INTERMETHOD COMPARISON; MALE; MONOTHERAPY; MULTICENTER STUDY; MUSCLE; MYASTHENIA GRAVIS; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; SCORING SYSTEM; TREATMENT OUTCOME; TREATMENT RESPONSE;

ACTIVITIES OF DAILY LIVING; ADULT; AGED; ANTI-INFLAMMATORY AGENTS, NON-STEROIDAL; DOUBLE-BLIND METHOD; FEMALE; FOLLOW-UP STUDIES; HUMANS; MALE; MIDDLE AGED; MYASTHENIA GRAVIS; MYCOPHENOLIC ACID; OUTCOME ASSESSMENT (HEALTH CARE); PSYCHOMOTOR PERFORMANCE; RETROSPECTIVE STUDIES; SEVESO ACCIDENTAL RELEASE; TIME FACTORS;

EID: 55749101723     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.21142     Document Type: Article

References (19)

1. Barohn RJ. Standards of measurement in myasthenia gravis. Ann NY Acad Sci 2003;998:432-439.
2. Barohn RJ, McIntire D, Herbelin L, Wolfe GI, Nations S, Bryan, WW. Reliability testing of the Quantitative Myasthenia Gravis Score. Ann NY Acad Sci 1998;841:769-772.
3. Bedlack RS, Simel DL, Bosworth H, Samsa G, Tucker-Lipscomb B, Sanders DB. Quantitative myasthenia gravis score: assessment of responsiveness and longitudinal validity. Neurology 2005;64:1968-1970.
4. Cohen JJ. Statistical power analysis for the behavioral sciences. Hillsdale, NJ: Erlbaum; 1988.
5. Guyatt G, Walter S, Norman G. Measuring change over time: assessing the usefulness of evaluative instruments. J Chron Dis 1987;40:171-178.
6. Iannaccone ST, Hynan LS, American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group. Reliability of 4 outcome measures in pediatric spinal muscular atrophy. Arch Neurol 2003;60:1130-1136.
7. Jaretzki A III, Barohn RJ, Ernstoff RM, Kaminski HJ, Keesey JC, Penn AS, et al. Myasthenia gravis: recommendations for clinical research standards. Neurology 2000;55:16-23.
8. Kaufmann P, Levy G, Montes J, Buchsbaum R, Barsdorf AI, Battista V, et al. Excellent inter-rater, intra-rater, and telephone-administered reliability of the ALSFRS-R in a multicenter clinical trial. Amyotroph Lateral Scler 2007;8:42-46.
9. Kaufmann P, Muntoni F. Issues in SMA clinical trial design: the International Coordinating Committee (ICC) for SMA subcommittee on SMA clinical trial design. Neuromuscul Disord 2007;17:499-505.
10. Meriggioli MN, Ciafaloni E, Al-Hayk KA, Rowin J, Tucker-Lipscomb B, Massey JM, et al. Mycophenolate mofetil for myasthenia gravis: an analysis of efficacy, safety, and tolerability. Neurology 2003;61:1438-1440.
11. Muscle Study Group. A trial of mycophenolate mofetil with prednisone as initial immunotherapy in myasthenia gravis. Neurology (in press, available in epub).
12. Nagane Y, Utsugisawa K, Obara D, Kondoh R, Terayama Y. Efficacy of low-dose FK506 in the treatment of myasthenia gravis - a randomized pilot study. Eur Neurol 2005;53:146-150.
13. Ponseti JM, Azem J, Fort JM, Codina A, Montoro JB, Armengol M. Benefits of FK506 (tacrolimus) for residual, cyclosporin-and prednisone-resistant myasthenia gravis: one-year follow-up of an open-label study. Clin Neurol Neurosurg 2005;107:187-190.
14. Rowin J, Meriggioli MN, Tüzün E, Leurgans S, Christadoss P. Etanercept treatment in cortocosteroid-dependent myasthenia gravis. Neurology 2004;63:2390-2392.
15. Sanders DB, Tucker-Lipscomb B, Massey JM. A simple manual muscle test for myasthenia gravis: validation and comparison with the QMG score. Ann NY Acad Sci 2003;998:440-444.
16. Sharshar T, Chevret S, Mazighi M, Chillet P, Huberfeld G, Berreotta C, et al. Validity and reliability of two muscle strength scores commonly used as endpoints in assessing treatment of myasthenia gravis. J Neurol 2000;246:286-289.
17. Traynor BJ, Zhang H, Shefner JM, Schoenfeld D, Cudkowicz ME. Functional outcome measures as clinical trial endpoints in ALS. Neurology 2004;63:1933-1935.
18. Vincent A, Palace J, Hilton-Jones D. Myasthenia gravis. Lancet 2001;357:2122-2128.
19. Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.