THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

Muscle and Nerve

Volumn 48, Issue 3, 2013, Pages 343-356

  Mcdonald, Craig M ^a   Henricson, Erik K ^a   Abresch, R Ted ^a   Florence, Julaine M ^b   Eagle, Michelle ^c   Gappmaier, Eduard ^d   Glanzman, Allan M ^e   Spiegel, Robert ^f   Barth, Jay ^f   Elfring, Gary ^f   Reha, Allen ^f   Peltz, Stuart ^f  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c NEWCASTLE UNIVERSITY   (United Kingdom)

^d UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^e CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^f PTC THERAPEUTICS   (United States)

Author keywords

6 minute walk test; Ambulation; Duchenne muscular dystrophy; Dystrophinopathy; Myometry; Natural history; Prediction of loss of function; Timed function tests

Indexed keywords

ATALUREN; PLACEBO;

ADOLESCENT; ARTICLE; CHILD; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; FUNCTION TEST; HUMAN; LONGITUDINAL STUDY; MAJOR CLINICAL STUDY; MALE; MOBILIZATION; MULTICENTER STUDY; NONSENSE MUTATION; OBSERVATIONAL STUDY; PHASE 2 CLINICAL TRIAL; PRESCHOOL CHILD; PRIORITY JOURNAL; QUANTITATIVE STUDY; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD;

6-MINUTE WALK TEST; AMBULATION; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHINOPATHY; MYOMETRY; NATURAL HISTORY; PREDICTION OF LOSS OF FUNCTION; TIMED FUNCTION TESTS;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; DOUBLE-BLIND METHOD; ELECTROMYOGRAPHY; EXERCISE TEST; GLUCOCORTICOIDS; HAND STRENGTH; HUMANS; LONGITUDINAL STUDIES; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OBSERVATION; OUTCOME ASSESSMENT (HEALTH CARE); OXADIAZOLES; PREDICTIVE VALUE OF TESTS; TIME FACTORS; WALKING;

EID: 84883055833     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.23902     Document Type: Article

References (41)

1. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010;9:77-93.
2. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010;9:177-189.
3. Crespo CJ, Keteyian SJ, Heath GW, Sempos CT. Leisure-time physical activity among US adults. Results from the Third National Health and Nutrition Examination Survey. Arch Intern Med 1996;156:93-98.
4. McDonald CM, Henricson EK, Han JJ, Nicorici A, Abresch RT, Atkinson LA, et al. The 6-minute walk test (6MWT) as a clinical trial outcome measure in Duchenne/Becker muscular dystrophy (DMD/BMD). Neuromuscul Disord 2008;18:739.
5. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, et al. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve 2010;41:500-510.
6. McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Atkinson L, et al. The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations. Muscle Nerve 2010;42:966-974.
7. Temple R. Are surrogate markers adequate to assess cardiovascular disease drugs? JAMA 1999;282:790-795.
8. Mazzone E, Martinelli D, Berardinelli A, Messina S, D'Amico A, Vasco G, et al. North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy. Neuromuscul Disord 2010;20:712-716.
9. Mazzone EG, Vasco G, Sormani MP, Torrente Y, Berardinelli A, Messina S, et al. Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study. Neurology 2011;77:250-256.
10. Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, Elfring G, et al. Percent predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences. Version 2. PLoS Curr. 2012 Jan 25 [revised 2012 Feb 2];4:RRN1297.
11. McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, et al. for the for the PTC124 GD 007 DMD Study Group. The 6-minute walk test and other clinical endpoints in severe dystrophinopathy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve (in press).
12. PTC Therapeutics. Phase 2b study of PTC124 in Duchenne/Becker muscular dystrophy (DMD/BMD) [online]. Available at: http://clinicaltrials.gov/ct2/show/NCT00592553. Accessed July 11, 2011.
13. Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007;447:87-91.
14. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981;4:186-197.
15. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB. The natural history of Duchenne muscular dystrophy: a caveat for therapeutic trials. Trans Am Neurol Assoc 1981;106:195-199.
16. Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve 1983;6:91-103.
17. Mendell JR, Province MA, Moxley RT III, Griggs RC, Brooke MH, Fenichel GM, et al. Clinical investigation of Duchenne muscular dystrophy. A methodology for therapeutic trials based on natural history controls. Arch Neurol 1987;44:808-811.
18. 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.
19. Griggs RC, Moxley RT III, Mendell JR, Fenichel GM, Brooke MH, Pestronk A, et al. Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Arch Neurol 1991;48:383-388.
20. Fowler WM Jr, Abresch RT, Aitkens S, Carter GT, Johnson ER, Kilmer DD, et al. Profiles of neuromuscular diseases. Design of the protocol. Am J Phys Med Rehabil 1995;74(suppl):S62-69.
21. Beenakker EA, Maurits NM, Fock JM, Brouwer OF, van der Hoeven JH. Functional ability and muscle force in healthy children and ambulant Duchenne muscular dystrophy patients. Eur J Paediatr Neurol 2005;9:387-393.
22. Pradhan S, Ghosh D, Srivastava NK, Kumar A, Mittal B, Pandey CM, et al. Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation. J Neurol 2006;253:1309-1316.
23. Mayhew JE, Florence JM, Mayhew TP, Henricson EK, Leshner RT, McCarter RJ, et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve 2007;35:36-42.
24. Beenakker EA, van der Hoeven JH, Fock JM, Maurits NM. Reference values of maximum isometric muscle force obtained in 270 children aged 4-16 years by hand-held dynamometry. Neuromuscul Disord 2001;11:441-446.
25. Hyde SA, Steffensen BF, Floytrup I, Glent S, Kroksmark AK, Salling B, et al. Longitudinal data analysis: an application to construction of a natural history profile of Duchenne muscular dystrophy. Neuromuscul Disord 2001;11:165-170.
26. Geiger R, Strasak A, Treml B, Gasser K, Kleinsasser A, Fischer V, et al. Six-minute walk test in children and adolescents. J Pediatr 2007;150:395-399.
27. Bello L, Piva L, Barp A, Taglia A, Picillo E, Vasco G, et al. Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy. Neurology 2012;79:159-162.
28. 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.
29. McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, et al. The CINRG Duchenne Natural History Study-a longitudinal natural history study in the era of glucocorticoid therapy: design of the protocol and methods. Muscle Nerve (in press).
30. Matthews DJ, James KA, Miller LA, Pandya S, Campbell KA, Ciafaloni E, et al. Use of corticosteroids in a population-based cohort of boys with Duchenne and Becker muscular dystrophy. J Child Neurol 2010;25:1319-1324.
31. McDonald CM, Abresch RT, Carter GT, Fowler WM Jr, Johnson ER, Kilmer DD, et al. Profiles of neuromuscular diseases. Duchenne muscular dystrophy. Am J Phys Med Rehabil 1995;74(suppl):S70-92.
32. McDonald CM. CINRG Duchenne natural history study overview and future plans; ambulatory clinical endpoints in DMD. In: NIDRR State of the Science Meeting on Outcome Measures in Duchenne Muscular Dystrophy, Crystal City, VA, 2012.
33. Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, et al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 2011;364:1513-1522.
34. Mendell JR, Rodino-Klapac L, Sahenik Z, Roush K, Bird L, Lowes LP, et al. Results at 48 weeks of a phase IIB Extension study of exon-skipping drug Eteplirsen in patients with Duchenne muscular dystrophy (DMD). In: World Muscle Society Congress, Perth, Australia, 2012.
35. Wyrwich KW, Nienaber NA, Tierney WM, Wolinsky FD. Linking clinical relevance and statistical significance in evaluating intra-individual changes in health-related quality of life. Med Care 1999;37:469-478.
36. Yost KJ, Eton DT. Combining distribution- and anchor-based approaches to determine minimally important differences: the FACIT experience. Eval Health Prof 2005;28:172-191.
37. Rubin LJ, Badesch DB, Barst RJ, Galie N, Black CM, Keogh A, et al. Bosentan therapy for pulmonary arterial hypertension. N Engl J Med 2002;346:896-903.
38. Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr 2004;144:581-588.
39. Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 2006;8:465-473.
40. van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, et al. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med 2010;362:1396-1406.
41. Gremeaux V, Troisgros O, Benaïm S, Hannequin A, Laurent Y, Casillas JM, et al. Determining the minimal clinically important difference for the six-minute walk test and the 200-meter fast-walk test during cardiac rehabilitation program in coronary artery disease patients after acute coronary syndrome. Arch Phys Med Rehabil 2011;92:611-619.