Minimal clinically important difference for the 6-min walk test: Literature review and application to Morquio A syndrome

Orphanet Journal of Rare Diseases

Volumn 12, Issue 1, 2017, Pages

  Schrover, Rudolf ^a   Evans, Kathryn ^b   Giugliani, Roberto ^c   Noble, Ian ^d   Bhattacharya, Kaustuv ^e,f  

^a SYNEVi Pty Limited   (Australia)

^b BioMarin Pharmaceutical Australia Pty Ltd   (Australia)

^c HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^d Noble Pharma Consulting   (Australia)

^e SYDNEY CHILDREN'S HOSPITAL   (Australia)

^f UNIVERSITY OF SYDNEY   (Australia)

Author keywords

Endurance; Enzyme replacement therapy; Minimal clinically important difference; Morquio A syndrome; MPS IVA; Mucopolysaccharidosis IVA; Six minute walk test

Indexed keywords

ELOSULFASE ALFA;

ANXIETY; CHRONIC OBSTRUCTIVE LUNG DISEASE; CHRONIC RESPIRATORY TRACT DISEASE; CROSS-SECTIONAL STUDY; CYSTIC FIBROSIS; DEPRESSION; ENDURANCE TRAINING; ENZYME REPLACEMENT; HEALTH ASSESSMENT QUESTIONNAIRE; HOSPITALIZATION; HUMAN; LITERATURE; LYSOSOME STORAGE DISEASE; MINIMAL RESIDUAL DISEASE; MORQUIO SYNDROME; MUSCULOSKELETAL DISEASE; OUTCOME ASSESSMENT; PUBLICATION; QUALITY OF LIFE ASSESSMENT; RESEARCH; REVIEW; SIX MINUTE WALK TEST; ADULT; MALE; MINIMAL CLINICALLY IMPORTANT DIFFERENCE; PATHOPHYSIOLOGY; PROCEDURES; WALK TEST;

ADULT; ENZYME REPLACEMENT THERAPY; HUMANS; MALE; MINIMAL CLINICALLY IMPORTANT DIFFERENCE; MUCOPOLYSACCHARIDOSIS IV; WALK TEST;

EID: 85018655036     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-017-0633-1     Document Type: Review

References (51)

1. Hendriksz CJ, Lavery C, Coker M, Ucar SK, Jain M, Bell L, et al. Burden of disease in patients with Morquio A syndrome: results from an international patient-reported outcomes survey. Orphanet J Rare Dis. 2014;9:32.
2. Bank RA, Groener JE, van Gemund JJ, Maaswinkel PD, Hoeben KA, Schut HA, et al. Deficiency in N-acetylgalactosamine-6-sulfate sulfatase results in collagen perturbations in cartilage of Morquio syndrome A patients. Mol Genet Metab. 2009;97:196-201.
3. Lavery C, Hendriksz C. Mortality in patients with Morquio Syndrome A. JIMD Rep. 2015;15:59-66.
4. Harmatz P, Mengel KE, Giugliani R, Valayannopoulos V, Lin SP, Parini R, et al. The Morquio A clinical assessment program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Mol Genet Metab. 2013;109:54-61.
5. Montaño AM, Tomatsu S, Gottesman GS, Smith M, Orii T. International Morquio A registry: clinical manifestation and natural course of Morquio A disease. J Inherit Metab Dis. 2007;30:165-74.
6. Kakkis E, Neufeld E. The Mucopolysaccharidoses. 1996.
7. Hendriksz CJ, Harmatz P, Beck M, Jones S, Wood T, Lachman R, et al. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. Mol Genet Metab. 2013;110:54-64.
8. Hendriksz CJ, Berger KI, Giugliani R, Harmatz P, Kampmann C, Mackenzie WG, et al. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet A. 2015;167A:11-25.
9. John RM, Hunter D, Swanton RH. Echocardiographic abnormalities in type IV mucopolysaccharidosis. Arch Dis Child. 1990;65:746-9.
10. Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014;37:979-90.
11. Society AT. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002;166:111-7.
12. Li AM, Yin J, Au JT, So HK, Tsang T, Wong E, et al. Standard reference for the six-minute-walk test in healthy children aged 7 to 16 years. Am J Respir Crit Care Med. 2007;176:174-80.
13. Lammers AE, Hislop AA, Flynn Y, Haworth SG. The 6-minute walk test: normal values for children of 4-11 years of age. Arch Dis Child. 2008;93:464-8.
14. Ulrich S, Hildenbrand FF, Treder U, Fischler M, Keusch S, Speich R, et al. Reference values for the 6-minute walk test in healthy children and adolescents in Switzerland. BMC Pulm Med. 2013;13:49.
15. Harmatz PR, Mengel KE, Giugliani R, Valayannopoulos V, Lin SP, Parini R, et al. Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome. Mol Genet Metab. 2015;114:186-94.
16. Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Solano Villarreal ML, et al. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. Mol Genet Metab. 2016;119:131-43.
17. Hendriksz CJ, Berger KI, Parini R, AlSayed MD, Raiman J, Giugliani R, et al. Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome. J Inherit Metab Dis. 2016;39:839-47.
18. Bittner V, Weiner DH, Yusuf S, Rogers WJ, McIntyre KM, Bangdiwala SI, et al. Prediction of mortality and morbidity with a 6-minute walk test in patients with left ventricular dysfunction. JAMA. 1993;270:1702-7.
19. Casanova C, Cote C, Marin JM, Pinto-Plata V, de Torres JP, Aguirre-Jaíme A, et al. Distance and oxygen desaturation during the 6-min walk test as predictors of long-term mortality in patients with COPD. Chest. 2008;134:746-52.
20. de Liefde II, van Domburg RT, Bax JJ, Klein J, Verhagen HJM, Poldermans D. A decline in walking distance predicts long-term outcome in patients with known or suspected peripheral artery disease. Eur J Cardiovasc Prev Rehabil. 2010;17:321-8.
21. Martin C, Chapron J, Hubert D, Kanaan R, Honoré I, Paillasseur JL, et al. Prognostic value of six minute walk test in cystic fibrosis adults. Respir Med. 2013;107:1881-7.
22. de Moraes KL, Ulisses Signori L, Antonini Ribeiro R, Vargas Silva AM, Moreira PR, Dipp T, et al. Prognostic value of the six-minute walk test in end-stage renal disease life expextancy: a prospective cohort study. Clinics. 2012;67:581-6.
23. Carey EJ, Steidley DE, Aqel BA, Byrne TJ, Mekeel KL, Rakela J, et al. Six-minute walk distance predicts mortality in liver transplant candidates. Liver Transpl. 2010;16:1373-8.
24. Golpe R, Castro-Añón O, Pérez-de-Llano LA, González-Juanatey C, Muñiz-Fernandez C, Testa-Fernández A, et al. Prognostic significance of six-minute walk test in non-group 1 pulmonary hypertension. Heart Lung. 2014;43:72-6.
25. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407-15.
26. du Bois RM, Albera C, Costabel U, Bradford WZ, Kartashov A, Noble PW, et al. 6-minute walk test distance (6MWTD) is a reliable, valid, and responsive outcome measure that predicts mortality in patients with IPF. Am J Respir Crit Care Med. 2010;181:A1103.
27. du Bois RM, Weycker D, Albera C, Bradford WZ, Costabel U, Kartashov A, et al. Six-minute-walk test in idiopathic pulmonary fibrosis: test validation and minimal clinically important difference. Am J Respir Crit Care Med. 2011;183:1231-7.
28. Swigris JJ, Wamboldt FS, Behr J, du Bois RM, King Jr TE, Raghu G, et al. The six-minute walk in idiopathic pulmonary fibrosis: longitudinal changes and minimum important difference. Thorax. 2010;65:173-7.
29. Polkey MI, Spruit MA, Edwards LD, Watkins ML, Pinto-Plata V, Vestbo J, et al. Six-minute-walk test in chronic obstructive pulmonary disease: minimal clinically important difference for death or hospitalization. Am J Respir Crit Care Med. 2013;187:382-6.
30. Copay AG, Subach BR, Glassman SD, Polly Jr DW, Schuler TC. Understanding the minimum clinically important difference: a review of concepts and methods. Spine J. 2007;7:541-6.
31. McGlothlin AE, Lewis RJ. Minimal clinically important difference: defining what really matters to patients. JAMA. 2014;312:1342-3.
32. Lachmann R, Schoser B. The clinical relevance of outcomes used in late-onset Pompe disease: can we do better? Orphanet J Rare Dis. 2013;8:160.
33. Singh SJ, Puhan MA, Andrianopoulos V, Hernandes NA, Mitchell KE, Hill CJ, et al. An official systematic review of the European Respiratory Society/American Thoracic Society: measurement properties of field walking tests in chronic respiratory disease. Eur Respir J. 2014;44:1447-78.
34. 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 a-L-iduronidase (laronidase). J Pediatr. 2004;144:581-8.
35. Harmatz P, Giugliani R, Schwartz IVD, Guffon N, Teles EL, Miranda MCS, 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;94:469-75.
36. Schlander M, Garattini S, Holm S, Kolominsky-Rabas P, Nord E, Persson U, et al. Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. J Comp Eff Res. 2014;3:399-422.
37. Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, et al. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial. Mol Genet Metab. 2015;114:178-85.
38. Holland AE, Hill CJ, Conron M, Munro P, McDonald CF. Small changes in six-minute walk distance are important in diffuse parenchymal lung disease. Respir Med. 2009;103:1430-5.
39. Holland AE, Hill CJ, Rasekaba T, Lee A, Naughton MT, McDonald CF. Updating the minimal important difference for six-minute walk distance in patients with chronic obstructive pulmonary disease. Arch Phys Med Rehabil. 2010;91:221-5.
40. Puhan MA, Chandra D, Mosenifar Z, Ries A, Make B, Hansel NN, et al. The minimal important difference of exercise tests in severe COPD. Eur Respir J. 2011;37:784-90.
41. Redelmeier DA, Bayoumi AM, Goldstein RS, Guyatt GH. Interpreting small differences in functional status: the six minute walk test in chronic lung disease patients. Am J Respir Crit Care Med. 1997;155:1278-82.
42. Lee AL, Hill CJ, Cecins N, Jenkins S, McDonald CF, Burge AT, et al. Minimal important difference in field walking tests in non-cystic fibrosis bronchiectasis following exercise training. Respir Med. 2014;108:1303-9.
43. Gremeaux V, Troisgros O, Benaïm S, Hannequin A, Laurent Y, Casillas J-M, Benaïm C. Using field walk tests to individualize exercise training in coronary artery disease patients. Arch Phys Med Rehabil. 2011;92:1731. Abs. 137.
44. 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;92:611-9.
45. Mathai SC, Puhan MA, Lam D, Wise RA. The minimal important difference in the 6-minute walk test for patients with pulmonary arterial hypertension. Am J Respir Crit Care Med. 2012;186:428-33.
46. Kwok BC, Pua YH, Mamun K, Wong WP. The minimal clinically important difference of six-minute walk in Asian older adults. BMC Geriatr. 2013;13:23.
47. Puhan MA, Mador MJ, Held U, Goldstein R, Guyatt GH, Schünemann HJ. Interpretation of treatment changes in 6-minute walk distance in patients with COPD. Eur Respir J. 2008;32:637-43.
48. Wise RA, Brown CD. Minimal clinically important differences in the six-minute walk test and the incremental shuttle walking test. COPD. 2005;2:125-9.
49. Gilbert C, Brown MC, Cappelleri JC, Carlsson M, McKenna SP. Estimating a minimally important difference in pulmonary arterial hypertension following treatment with sildenafil. Chest. 2009;135:137-42.
50. McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, et al. 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. Muscle Nerve. 2013;48:357-68.
51. Taeger T, Wians FH, Cebola R, Froehlich H, Katus HA, Frankenstein L, et al. Biovaribility, minimal important difference and change of the 6-minute walk test in chronic heart failure. Eur J Heart Failure Suppl. 2013;S94:P820.