Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

Muscle and Nerve

Volumn 35, Issue 1, 2007, Pages 36-42

  Mayhew, Jill E ^a   Florence, Julaine M ^b   Mayhew, Thomas P ^c   Henricson, Erik K ^a   Leshner, Robert T ^a   McCarter, Robert J ^a   Escolar, Diana M ^a  

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c VIRGINIA COMMONWEALTH UNIVERSITY   (United States)

Author keywords

Duchenne muscular dystrophy; Interrater reliability; Pediatric neuromuscular testing; Quantitative muscle testing (QMT)

Indexed keywords

ADOLESCENT; ARTICLE; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CORRELATION COEFFICIENT; DISEASE MARKER; DUCHENNE MUSCULAR DYSTROPHY; EVALUATION; FORCED EXPIRATORY VOLUME; FUNCTION TEST; HUMAN; MALE; MULTICENTER STUDY; MUSCLE FUNCTION; MUSCLE STRENGTH; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; RELIABILITY; SCORING SYSTEM; SENSITIVITY ANALYSIS;

ADOLESCENT; BIOLOGICAL MARKERS; CHILD; CHILD, PRESCHOOL; CLINICAL TRIALS; DISABILITY EVALUATION; HUMANS; MALE; MUSCLE WEAKNESS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; NEUROLOGIC EXAMINATION; OBSERVER VARIATION; OUTCOME ASSESSMENT (HEALTH CARE); PREDICTIVE VALUE OF TESTS; REPRODUCIBILITY OF RESULTS; TREATMENT OUTCOME;

EID: 33845989867     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.20654     Document Type: Article

References (27)

1. Aitkens S, Lord J, Bernauer E, Fowler WM Jr, Lieberman JS, Berck P. Relationship of manual muscle testing to objective strength measurements. Muscle Nerve 1989;12:173-177.
2. American Thoracic Society. Standardization of spirometry. Am J Respir Crit Care Med 1995;152:1107-1136.
3. Andres PL, Finison LJ, Conlon T, Thibodean LM, Munsat TL. Use of composite scores (megascores) to measure deficit in amyotrophic lateral sclerosis. Neurology 1988;38:405-408.
4. Andres PL, Hedlund W, Finison L, Conlon T, Fehnus M, Munsat TL. Quantitative motor assessment in amyotrophic lateral sclerosis. Neurology 1986;36:937-941.
5. Andres PL, Skerry LM, Thornell H, Portney LG, Finison LJ, Munsat TL. A comparison of three measures of disease progression in ALS. J Neurol Sci l996;139(suppl):64-70.
6. Boiteau M, Malouin F, Richards CL. Use of a hand-held dynamometer and a Kin-Cotn dynamometer for evaluating spastic hypertonia in children: a reliability study. Phys Ther 1995;75:796-802.
7. Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology 1989;39:475-481.
8. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shinnate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981;4:186-197.
9. Brussock CM, Haley SM, Munsat TL, Bernhardt DB. Measurement of isometric force in children with and without Duchenne's muscular dystrophy. Phys Ther 1992;72:105-114.
10. Escolar DM, Buyse G, Henricson L, Leshner R, Florence J, Mayhew J, et al. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol 2005;58:151-155.
11. Escolar DM, Henricson EK, Mayhew J, Florence J, Leshner R, Patel KM, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve 2001;24:787-791.
12. Escolar DM, Henricson FK, Pasquali L, Gorni K, Hoffman EP. (Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG). Neuromuscul Disord 2002;12(suppl 1):S147-S154.
13. Florence JM, Pandya S, King WM, Robison JD, Signore LC, Wentzell M, et al. Clinical trials in Duchenne dystrophy. Standardization and reliability of evaluation procedures. Phys Ther 1984;64:41-45.
14. Fowler WM Jr, Gardner GW. Quantitative strength measurements in muscular dystrophy. Arch Phys Med Rehabil 1967;48:629-644.
15. Hoffman EP. Dystrophinopathies. In: Karpati G, Hilton-Jones D, Griggs RC, editors. Disorders of voluntary muscle. Cambridge, UK: Cambridge University Press; 2001. p 385-432.
16. Iannaccone ST. Outcome measures for pediatric spinal muscular atrophy. Arch Neurol 2002;59:1445-1450.
17. Iannaccone ST, Hynan LS. Reliability of 4 outcome measures in pediatric spinal muscular atrophy. Arch Neurol 2003;60:1130-1136.
18. Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989;320:1592-1597.
19. Munsat TL. Clinical trials in neuromuscular disease. Muscle Nerve 1990;13(suppl):S3-S6.
20. Munsat TL. Development of measurement techniques. Neurology 1996;47(suppl):S83-S85.
21. Personius KE, Pandya S, King WM, Tawil R, McDermott MP. Facioscapulohumeral dystrophy natural history study: standardization of testing procedures and reliability of measurements. The FSH DY Group. Phys Ther 1994;74:253-263.
22. Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med 2001;164:2191-2194.
23. Russman BS, Buncher CR, White M, Samaha FJ, Iannaccone ST. Function changes in spinal muscular atrophy II and III. The DCN/SMA Group. Neurology 1996;47:973-976.
24. Scott OM, Hyde SA, Goddard C. Quantification of muscle function in children: a prospective study in Duchenne muscular dystrophy. Muscle Nerve 1982;5:291-301.
25. Shrake K. AARC Clinical practice guideline. Respir Care 1996;41:629-636.
26. Shrout PE, Fleiss JL. Intraclass correlations: uses in assessing rater reliability. Psychol Bull 1979;86:420-428.
27. van der Ploeg RJ, Oosterhuis HJ, Reuvekamp J. Measuring muscle strength. J Neurol 1984;231:200-203.