The 6‐minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

Abstract

Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6‐minute walk test (6MWT) and other endpoints. Methods: Screening and baseline evaluations included the 6‐minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate–determined energy expenditure index, and other exploratory endpoints. Results: The 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods. Conclusions: The ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression. Muscle Nerve 48 : 357–368, 2013

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References 78

1. 1.

   Hoffman EP, Brown RH, Kunkel LM. Dystrophin: the protein product of the Duchennemuscular dystrophy locus. Cell. 1987;51:919-928.

   Not in our library10.1016/0092-8674(87)90579-4

2. 2.

   Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71:304-313.

   Not in our library10.1002/ana.23528

3. 3.

   2009;58:1119-1122.

   Not in our library

4. 4.

   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.

   Not in our library10.1016/s1474-4422(09)70271-6

5. 5.

   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.

   Not in our library10.1016/s1474-4422(09)70272-8

6. 6.

   Dent KM, Dunn DM, von Niederhausern AC, Aoyagi AT, Kerr L, Bromberg MB, et al. Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort. Am J Med Genet A. 2005;134:295-298.

   Not in our library10.1002/ajmg.a.30617

7. 7.

   Hirawat S, Elfring GL, Leonard EM.

   Not in our library

8. 8.

   Hirawat S, Elfring GL, Leonard EM.

   Not in our library

9. 9.

   Chamberlain JS, Rando TA. Duchenne muscular dystrophy: advances in therapeutics. 2006.

   Not in our library

10. 10.

    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.

    Not in our library10.1038/nature05756

11. 11.

    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.

    Not in our library10.1056/nejmoa1011367

12. 12.
    Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study · 2009

    No notice
13. 13.

    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.

    Not in our library

14. 14.

    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.

    Not in our library10.1212/wnl.39.4.475

15. 15.

    McDonald CM, Fowler WM. The role of the neuromuscular medicine and physiatry specialists in the multidisciplinary management of neuromuscular disease. Phys Med Rehabil Clin N Am. 2012;23:475-493.

    Not in our library10.1016/j.pmr.2012.06.010

16. 16.

    McDonald CM, Abresch RT, Carter GT, Fowler WM, Johnson ER, Kilmer DD, et al. Profiles of neuromuscular diseases. Duchenne muscular dystrophy. Am J Phys Med Rehabil. 1995;74(suppl):S70-92.

    Not in our library10.1097/00002060-199509001-00003

17. 17.

    Guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002;166:111-117.

    Not in our library10.1164/ajrccm.166.1.at1102

18. 18.

    Takeuchi Y, Katsuno M, Banno H, Suzuki K, Kawashima M, Atsuta N, et al. Walking capacity evaluated by the 6-minute walk test in spinal and bulbar muscular atrophy. Muscle Nerve. 2008;38:964-971.

    Not in our library10.1002/mus.21077

19. 19.

    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.

    Not in our library10.1056/nejmoa012212

20. 20.

    Dumas HM, Fragala MA, Haley SM, Skrinar AM, Wraith JE, Cox GF. Physical performance testing in mucopolysaccharidosis I: a pilot study. Pediatr Rehabil. 2004;7:125-131.

    Not in our library10.1080/13638490310001654763

21. 21.

    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.

    Not in our library10.1097/01.gim.0000232477.37660.fb

22. 22.

    Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007;90:329-337.

    Not in our library10.1016/j.ymgme.2006.09.001

23. 23.

    Harmatz P, Giugliani R, Schwartz, Guffon N, Teles EL, Miranda MC, et al. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008;94:469-475.

    Not in our library10.1016/j.ymgme.2008.04.001

24. 24.

    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.

    Not in our library10.1016/j.jpeds.2004.01.046

25. 25.

    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.

    Not in our library10.1056/nejmoa0909859

26. 26.

    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.

    Not in our library10.1002/mus.21544

27. 27.

    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.

    Not in our library10.1002/mus.21808

28. 28.

    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.

    Not in our library10.1016/j.nmd.2010.06.014

29. 29.

    Mazzone E, 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.

    Not in our library10.1212/wnl.0b013e318225ab2e

30. 30.

    Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407-415.

    Not in our library10.1016/0197-2456(89)90005-6

31. 31.

    Beaton DE, Bombardier C, Katz JN, Wright JG, Wells G, Boers M, et al. Looking for important change/differences in studies of responsiveness. OMERACT MCID Working Group. Outcome measures in rheumatology. Minimal clinically important difference. J Rheumatol. 2001;28:400-405.

    Not in our library

32. 32.
    Minimal changes in health status questionnaires: distinction between minimally detectable change and minimally important change · 2006

    No notice
33. 33.

    Hays RD, Woolley JM. The concept of clinically meaningful difference in health-related quality-of-life research. How meaningful is it? Pharmacoeconomics. 2000;18:419-423.

    Not in our library10.2165/00019053-200018050-00001

34. 34.

    Wells G, Anderson J, Beaton D, Bellamy N, Boers M, Bombardier C, et al. Minimal clinically important difference module: summary, recommendations, and research agenda. J Rheumatol. 2001;28:452-454.

    Not in our library

35. 35.

    Jacobson N, Follette WC, Revenstorf D. Toward a standard definition of clinically significant change. Behav Ther. 1986;17:308-311.

    Not in our library

36. 36.

    Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol. 2003;56:395-407.

    Not in our library10.1016/s0895-4356(03)00044-1

37. 37.

    Crosby RD, Kolotkin RL, Williams GR. An integrated method to determine meaningful changes in health-related quality of life. J Clin Epidemiol. 2004;57:1153-1160.

    Not in our library10.1016/j.jclinepi.2004.04.004

38. 38.

    Jacobson NS, Truax P. Clinical significance: a statistical approach to defining meaningful change in psychotherapy research. J Consult Clin Psychol. 1991;59:12-19.

    Not in our library10.1037//0022-006x.59.1.12

39. 39.

    Deyo RA, Diehr P, Patrick DL. Reproducibility and responsiveness of health status measures. Statistics and strategies for evaluation. Control Clin Trials. 1991;12(suppl):142S-158S.

    Not in our library10.1016/s0197-2456(05)80019-4

40. 40.

    Deyo RA, Centor RM. Assessing the responsiveness of functional scales to clinical change: an analogy to diagnostic test performance. J Chronic Dis. 1986;39:897-906.

    Not in our library10.1016/0021-9681(86)90038-x

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• THE 6‐minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study · 2013
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