Rasch analysis of clinical outcome measures in spinal muscular atrophy

Muscle and Nerve

Volumn 49, Issue 3, 2014, Pages 422-430

  Cano, Stefan J ^a,ag   Mayhew, Anna ^b,y   Glanzman, Allan M ^c   Krosschell, Kristin J ^d,x   Swoboda, Kathryn J ^e   Main, Marion ^f   Steffensen, Birgit F ^g   Bérard, Carole ^h,ac   Girardot, Françoise ^h,ac   Payan, Christine A M ^i,ab   Mercuri, Eugenio ^j,z   Mazzone, Elena ^j   Elsheikh, Bakri ^k   Florence, Julaine ^l,u   Hynan, Linda S ^m,v   Iannaccone, Susan T ^m,v   Nelson, Leslie L ⁿ   Pandya, Shree ^o,aa   Rose, Michael ^p,ad   Scott, Charles ^q,ae   Sadjadi, Reza ^r,af   Yore, Mackensie A ^s   Joyce, Cynthia ^s,w   Kissel, John T ^k,t   more..

^a PENINSULA MEDICAL SCHOOL   (United Kingdom)

^b INSTITUTE OF HUMAN GENETICS   (United Kingdom)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^d NORTHWESTERN UNIVERSITY   (United States)

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

^f GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^g Rehabiliterings Center for Muskelsvind   (Denmark)

^h CENTRE HOSPITALIER   (France)

ⁱ ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

^j FONDAZIONE POLICLINICO UNIVERSITARIO A GEMELLI IRCCS   (Italy)

^k OHIO STATE UNIVERSITY   (United States)

^l WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

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

ⁿ UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

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

^p KING S COLLEGE HOSPITAL   (United Kingdom)

^q CBS   (United States)

^r UNIVERSITY OF VIRGINIA   (United States)

^s Spinal Muscular Atrophy Foundation   (United States)

^t NATIONWIDE CHILDREN S HOSPITAL   (United States)

^u WASHINGTON UNIVERSITY   (United States)

^v UT Southwestern   (United States)

^w SMA Foundation   (United States)

^x NORTHWESTERN UNIVERSITY   (United States)

^y NEWCASTLE UNIVERSITY   (United Kingdom)

^z CATHOLIC UNIVERSITY   (Italy)

^aa UNIVERSITY OF ROCHESTER   (United States)

^ab Institut de Myologie   (France)

^ac Hopital Femme Mere Enfant   (France)

^ad KING'S COLLEGE LONDON   (United Kingdom)

^ae Project Cure   (United States)

^af British Columbia Hospital   (United States)

^ag PLYMOUTH UNIVERSITY PENINSULA SCHOOLS OF MEDICINE AND DENTISTRY   (United Kingdom)

more..

Author keywords

Clinical trials; Motor neuron disease; Outcome measures; Rasch analysis; Spinal muscular atrophy

Indexed keywords

ARTICLE; CHILDREN HOSPITAL OF PHILADELPHIA TEST FOR NEUROMUSCULAR DISORDER; CLINICAL ASSESSMENT TOOL; EGEN KLASSIFIKATION SCALE; EXPANDED HAMMERSMITH FUNCTIONAL MOTOR SCALE; GROSS MOTOR FUNCTION MEASURE; HAMMERSMITH FUNCTIONAL MOTOR SCALE; HUMAN; MODIFIED HAMMERSMITH FUNCTIONAL MOTOR SCALE EXTEND; MOTOR FUNCTION MEASURE; MOTOR PERFORMANCE; NORTH STAR AMBULATORY ASSESSMENT FOR SMA; OUTCOME ASSESSMENT; PERFORMANCE; PRIORITY JOURNAL; PSYCHOMETRY; PSYCHOMOTOR ACTIVITY; RASCH ANALYSIS; RATING SCALE; RELIABILITY; SPINAL MUSCULAR ATROPHY; TEST OF INFANT MOTOR PERFORMANCE SCREENING ITEM;

CLINICAL TRIALS; MOTOR NEURON DISEASE; OUTCOME MEASURES; RASCH ANALYSIS; SPINAL MUSCULAR ATROPHY;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; DATABASES, FACTUAL; DISABILITY EVALUATION; FEMALE; HUMANS; INFANT; MALE; MIDDLE AGED; MUSCULAR ATROPHY, SPINAL; OUTCOME ASSESSMENT (HEALTH CARE); PSYCHOMETRICS; REPRODUCIBILITY OF RESULTS; YOUNG ADULT;

EID: 84893953276     PISSN: 0148639X     EISSN: 10974598     Source Type: Journal    
DOI: 10.1002/mus.23937     Document Type: Article

References (61)

1. Sugarman E, Nagan N, Zhu H, Akmaev VR, Zhou Z, et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72, 400 specimens. Eur J Hum Genet 2012;20:27-32.
2. Monani U. Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease. Neuron 2005;48:885-896.
3. Zerres K, Rudnick-Schoneborn S. Natural history in proximal spinal muscular atrophy. Arch Neurol 1995;52:518-523.
4. Bosboom W, Vrancken A, van den Berg L, Wokke J, Iannaccone S. Drug treatment for spinal muscular atrophy Type I. Cochrane Database Syst Rev 2009;1:1-17.
5. Bosboom W, Vrancken A, van den Berg L, Wokke J, Iannaccone S. Drug treatment for spinal muscular atrophy types II and III. Cochrane Database Syst Rev 2009;1:1-34.
6. Kolb S, Kissel J. Spinal muscular atrophy: a timely review. Arch Neurol 2011;68:979-984.
7. Food and Drug Administration.Patient reported outcome measures: use in medical product development to support labelling claims [online]. Available at: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf.
8. Food and Drug Administration.Qualification process for drug development tools [online]. Available at: www.fda.gov/cber/gdlns/prolbl.pdf. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf.
9. Hobart J, Cano S, Zajicek J, Thompson A. Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations. Lancet Neurol 2007;6:1094-1105.
10. Hobart J, Cano S. Improving the evaluation of therapeutic intervention in MS: the role of new psychometric methods. Monograph for the UK Health Technology Assessment Programme 2009;13:1-200.
11. Kaufmann P, McDermott M, Darras B, Finkel R, et al.Prospective cohort study of spinal muscular atrophy type II and III. Neurology 2012;79(18):1889-1897.
12. Main M, Kairon H, Mercuri E, Muntoni F. The Hammersmith Functional Motor Scale for Children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation. Eur J Paediatr Neurol 2003;7:155-159.
13. Mercuri E, Messina S, Battini R, Berardinelli A, Boffi P, et al. Reliability of the Hammersmith functional motor scale for spinal muscular atrophy in a multicentric study. Neuromuscul Disord 2006;16:93-98.
14. Glanzman A, O'Hagen J, McDermott M, Martens WB, Flickinger J, et al. Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III. J Child Neurol 2011;26:1499-1507.
15. Krosschell K, Maczulski J, Crawford T, Scott C, Swoboda K. A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy. Neuromuscul Disord 2006;16:417-426.
16. Krosschell K, Scott C, Maczulski J, Lewelt A, Reyna S, et al. Reliability of the Modified Hammersmith Functional Motor Scale in young children with spinal muscular atrophy. Muscle Nerve 2011;44:246-251.
17. Bérard C, Payan C, group Ms. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord 2005;15:463-470.
18. Nelson L, Owens H, Hynan L, Iannaccone S, Group A. The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy. Neuromuscul Disord 2006;16:374-380.
19. Iannaccone S, Hynan L. Reliability of 4 outcome measures in pediatric spinal muscular atrophy. Arch Neurol 2003;60:1130-1136.
20. Scott E, Eagle M, Main M, Sheehan J. The North Star Ambulatory Assessment. In: 32nd Annual Meeting of British Paediatric Neurology Association, Bristol, UK, 2006.
21. Mazzone E, Messina S, Vasco G. Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscul Disord 2009;19:458-461.
22. Steffensen B, Hyde S, Lyager S, Mattsson E. Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy. Physiother Res Int 2001;6:119-134.
23. Glanzman A, Mazzone E, Main M, Pelliccioni M, Wood J, et al. The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord 2010;20:155-161.
24. Glanzman A, McDermott M, Montes J, Martens WB, Flickinger J, et al. Validation of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Pediatr Phys Ther 2011;23:322-326.
25. Finkel R, Hynan L, Glanzman A, Owens H, Nelson L, et al. The test of infant motor performance: reliability in spinal muscular atrophy type I. Pediatr Phys Ther 2008;20:242-246.
26. O'Hagen J, Glanzman A, McDermott M, Ryan PA, Flickinger J, et al. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscul Disord 2007;17:693-697.
27. Russell D, Rosenbaum P, Avery L, Lane M. Gross motor function measure user's manual. London: Mac Keith Press; 2002.
28. Steffensen B, Lyager S, Werge B, Rahbek J, Mattsson E. Physical capacity in non-ambulatory people with Duchenne muscular dystrophy or spinal muscular atrophy: a longitudinal study. Dev Med Child Neurol 2002;44:623-632.
29. Campbell S, Swanlund A, Smith E, Liao P, Zawacki L. Validity of the TIMPSI for estimating concurrent performance on the test of infant motor performance. Pediatr Phys Ther 2008;20:3-10.
30. Andrich D. Rasch models for measurement. Beverley Hills, CA: Sage Publications; 1988.
31. Cano SJ, Warner TT, Linacre JM, Bhatia KP, Thompson AJ, et al. Capturing the true burden of dystonia on patients: the Cervical Dystonia Impact Profile (CDIP-58). Neurology 2004;63:1629-1633.
32. Cano SJ, Riazi A, Schapira AH, Cooper JM, Hobart JC. Friedreich's ataxia impact scale: a new measure striving to provide the flexibility required by today's studies. Mov Disord 2009;24:984-992.
33. Hobart J, Cano S. Improving the evaluation of therapeutic interventions in multiple sclerosis: the role of new psychometric methods. Health Technol Assess 2009;13:iii, ix-x, 1-177.
34. Andrich D. Rating scales and Rasch measurement. Expert Rev Pharmacoecon Outcomes Res 2011;11:571-585.
35. Rasch G. Probabilistic models for some intelligence and attainment tests. Copenhagen: Danish Institute for Education Research (Expanded edition (1980) with foreword and afterword by B.D. Wright, Chicago: The University of Chicago Press, 1980. Reprinted Chicago: MESA Press, 1993. Available from www.rasch.org/books.htm); 1960.
36. Cano S, Warner T, Thompson A, Bhatia K, Fitzpatrick R, et al. The cervical dystonia impact profile (CDIP-58): can a Rasch developed patient reported outcome measure satisfy traditional psychometric criteria? Health Qual Life Outcomes 2008;6:58.
37. Mayhew A, Cano S, Scott E, Eagle M, Bushby K, et al. Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol 2011;53:535-542.
38. International Coordinating Committee for SMA Clinical Trials. Meeting of the FDA SEALD Division and Representatives of the International Coordinating Committee for SMA Clinical Trials. New York: International Coordinating Committee for SMA Clinical Trials; 2010.
39. Massof R. Understanding Rasch and item response theory models: applications to the estimation and validation of interval latent trait measures from responses to rating scale questionnaires. Ophthalmic Epidemiol 2011;18:1-19.
40. 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.
41. Ware JEJ, Harris WJ, Gandek B, Rogers BW, Reese PR. MAP-R for windows: multitrait / multi-item analysis program - revised user's guide. Boston, MA: Health Assessment Lab; 1997.
42. RUMM 2030 [computer program]. Perth, WA: RUMM Laboratory Pty Ltd; 1997-2012.
43. Andrich D. A rating formulation for ordered response categories. Psychometrika 1978;43:561-573.
44. Andrich D. An index of person separation in latent trait theory, the traditional KR20 index, and the Guttman scale response pattern. Educ Psychol Res 1982;9:95-104.
45. Andrich D. An elaboration of Guttman scaling with Rasch models for measurement. In: Tuma NB, editor. Social methodology, Chap 2. San Francisco: Jossey-Bass, 1985. p 33-80.
46. Andrich D, de Jong JHAL, Sheridan BE. Diagnostic opportunities with the Rasch model for ordered response categories. In: Rost J, Langeheine R, editors. Applications of latent trait and latent class models in the saocial sciences. Munster, Germany: Waxmann Verlag GmbH; 1997. p 59-70.
47. Hagquist C, Andrich D. Is the sense of coherence instrument applicable on adolescents? A latent trait analysis using Rasch modelling. Pers Individ Dif 2004;36:955-968.
48. Wright BD, Masters G. Rating scale analysis: Rasch measurement. Chicago: MESA; 1982.
49. Marais I, Andrich D. Formalising dimension and response violations of local independence in the unidimensional Rasch model. J Appl Meas 2008;9:200-215.
50. Cronbach LJ. Coefficient alpha and the internal structure of tests. Psychometrika 1951;16:297-334.
51. Pallant J, Tennant A. An introduction to the Rasch measurement model: an example using the Hospital Anxiety and Depression Scale (HADS). Br J Clin Psychol 2007;46:1-18.
52. Andrich D. Implication and applications of modern test theory in the context of outcomes based research. Stud Educ Eval 2002;28:103-121.
53. Cano S, Hobart J. The problem with health measurement. Patient Prefer Adherence 2011;5:279-290
54. Michener L, McClure P, Sennett B. American Shoulder and Elbow Surgeons Standardized Shoulder Assessment Form, patient self-report section: reliability, validity, and responsiveness. J Shoulder Elbow Surg 2002;11:587-594.
55. Andrich D, Hagquist C. Real and artificial differential item functioning. J Educ Behav Stat 2012;37:387-416.
56. Stenner AJ, Smith M. Testing construct theories. Percept Mot Skills 1982;55:415-426.
57. Andrich D. Controversy and the Rasch model: a characteristic of incompatible paradigms? Med Care 2004;42:I7-I16.
58. Revicki D. FDA draft guidance and health-outcomes research. Lancet 2007;369:540-542.
59. Stenner AJ, Burdick H, Sandford EE, Burdick DS. How accurate are lexile text measures? J Appl Meas 2006;7:307-322.
60. Choppin B. An item bank using sample free calibration. Nature 1968;219:870-872.
61. White S, Clement J. Assessing the Lexile Framework: results of a panel meeting NCES Working Paper Series, Working Paper No. 2001-08. Washington, DC: US Department of Education, Office of Educational Research and Improvement; 2001.