Neuromuscular Disorders
Volume 19, Issue 12 , Pages 805-812 , December 2009

The PedsQL™ in pediatric patients with Spinal Muscular Atrophy: Feasibility, reliability, and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Neuromuscular Module

  • Susan T. Iannaccone

      Affiliations

    • Department of Pediatrics, UT Southwestern Medical Center, Dallas, TX, USA
    • Department of Neurology, UT Southwestern Medical Center, Dallas, TX, USA
    • Corresponding Author InformationCorresponding author. Address: Department of Pediatrics, UT Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390-9063, USA. Tel.: +1 214 559 8383.
  • ,
  • Linda S. Hynan

      Affiliations

    • Department of Clinical Sciences and Psychiatry, UT Southwestern Medical Center, Dallas, TX, USA
  • ,
  • Anne Morton

      Affiliations

    • Department of Psychology, Texas Scottish Rite Hospital for Children, Dallas, TX, USA
  • ,
  • Renee Buchanan

      Affiliations

    • Department of Psychology, Texas Scottish Rite Hospital for Children, Dallas, TX, USA
  • ,
  • Christine A. Limbers

      Affiliations

    • Department of Psychology, College of Liberal Arts, Texas A&M University, College Station, TX, USA
  • ,
  • James W. Varni

      Affiliations

    • Department of Pediatrics, College of Medicine, Texas A&M University, College Station, TX, USA
    • Department of Landscape Architecture and Urban Planning, College of Architecture, Texas A&M University, College Station, TX, USA
  • ,
  • the AmSMART Group

      Affiliations

    • AmSMART members: Mariam Andersen, Dallas, TX; Brenda Wong, MD, and Paula Morehart, Cincinnati, OH; Barry Russman, MD, and Kirsten Zilke, Portland, OR; Robert Leshner, MD, Barbara Grillo, and Angela Zimmerman, Richmond, VA and Washington, DC; Stephen Smith, MD, John Day, MD, and Heather Wendorf, St. Paul, MN; Kathy Swoboda, MD, and Sandra Reyna, Salt Lake City, UT; Richard Finkel, MD, and Kim Schadt, Philadelphia, PA; JiriVajsar, MD, and Lynn MacMillan, Toronto, CA; Anne Connolly, MD, and Charlie Wulf, St. Louis, MO; Nancy Kuntz, MD, and Wendy Korn-Peterson, Rochester, MN; Petra Kaufmann, MD, and Jessica O’Hagen, New York, NY; Basil Darras, MD, and Erica Sanborn, Boston, MA.

Received 23 April 2009 ,Revised 25 September 2009 ,Accepted 30 September 2009.

References 

  1. Oskoui M, Levy G, Garland CJ, et al. The changing natural history of spinal muscular atrophy type 1. Neurology. 2007;69:1931–1936
  2. Emery AE. Population frequencies of inherited neuromuscular diseases – a world survey. Neuromuscul Disord. 1991;1:19–29
  3. Lefebvre S, Burlet P, Liu Q, et al. Correlation between severity and SMN protein level in spinal muscular atrophy. Nat Genet. 1997;16:265–269
  4. Vrbova G, Melki J. 103rd ENMC international workshop: designing rational therapy of SMA based on the understanding of its pathophysiology, 18–20 January, 2002, Naarden, The Netherlands. Neuromuscul Disord. 2003;13:173–178
  5. Oskoui M, Kaufmann P. Spinal muscular atrophy. Neurotherapeutics. 2008;5:499–506
  6. Kaufmann P, Finkel R. Learning to walk: challenges for spinal muscular atrophy clinical trials. Neurology. 2007;68:11–12
  7. Wong BL, Hynan LS, Iannaccone ST. A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy. J Clin Neuromuscul Dis. 2007;8:101–110
  8. Iannaccone ST. Outcome measures for pediatric spinal muscular atrophy. Arch Neurol. 2002;59:1445–1450
  9. Iannaccone ST, Hynan LS. Reliability of 4 outcome measures in pediatric spinal muscular atrophy. Arch Neurol. 2003;60:1130–1136
  10. Nelson L, Owens H, Hynan LS, Iannaccone ST. The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy. Neuromuscul Disord. 2006;16:374–380
  11. Young HK, Lowe A, Fitzgerald DA, et al. Outcome of noninvasive ventilation in children with neuromuscular disease. Neurology. 2007;68:198–201
  12. Mah JK, Thannhauser JE, Kolski H, Dewey D. Parental stress and quality of life in children with neuromuscular disease. Pediatr Neurol. 2008;39:102–107
  13. World Health Organization. Constitution of the World Health Organization: basic document. Geneva, Switzerland: World Health Organization; 1948.
  14. FDA. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Rockville, MD: Center for Drug Evaluation and Research, Food and Drug Administration; 2006.
  15. Kushner RF, Foster GD. Obesity and quality of life. Nutrition. 2000;16:947–952
  16. Sprangers MAG, Cull A, Bjordal K, Groenvold M, Aaronson NK. The European Organization for Research and Treatment of Cancer approach to quality of life assessment: guidelines for developing questionnaire modules. Qual Life Res. 1993;2:287–295
  17. Patrick DL, Deyo RA. Generic and disease-specific measures in assessing health status and quality of life. Med Care. 1989;27:S217–S233
  18. Uzark K, Jones K, Burwinkle TM, Varni JW. The Pediatric Quality of Life Inventory in children with heart disease. Prog Pediatr Cardiol. 2003;18:141–148
  19. Palmer SN, Meeske KA, Katz ER, Burwinkle TM, Varni JW. The PedsQL™ Brain Tumor Module: initial reliability and validity. Pediatr Blood Cancer. 2007;49:287–293
  20. Varni JW, Burwinkle TM, Berrin SJ, et al. The PedsQL™ in pediatric cerebral palsy: reliability, validity, and sensitivity of the Generic Core Scales and Cerebral Palsy Module. Dev Med Child Neurol. 2006;48:442–449
  21. Varni JW, Burwinkle TM, Jacobs JR, Gottschalk M, Kaufman F, Jones KL. The PedsQL™ in Type 1 and Type 2 diabetes: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Type 1 Diabetes Module. Diabetes Care. 2003;26:631–637
  22. Varni JW, Burwinkle TM, Rapoff MA, Kamps JL, Olson N. The PedsQL™ in pediatric asthma: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales and Asthma Module. J Behav Med. 2004;27:297–318
  23. Varni JW, Burwinkle TM, Katz ER, Meeske K, Dickinson P. The PedsQL™ in pediatric cancer: reliability and validity of the Pediatric Quality of Life Inventory™ Generic Core Scales, Multidimensional Fatigue Scale, and Cancer Module. Cancer. 2002;94:2090–2106
  24. Varni JW, Seid M, Rode CA. The PedsQL™: measurement model for the Pediatric Quality of Life Inventory. Med Care. 1999;37:126–139
  25. Varni JW, Seid M, Kurtin PS. PedsQL™ 4.0: reliability and validity of the Pediatric Quality of Life Inventory™ Version 4.0 Generic Core Scales in healthy and patient populations. Med Care. 2001;39:800–812
  26. Varni JW, Limbers CA. The pediatric quality of life inventory: measuring pediatric health-related quality of life from the perspective of children and their parents. Pediatr Clin North Am. 2009;56:843–863
  27. Burd L, Short SK, Martsolf JT, Nelson RA. Prevalence of type I spinal muscular atrophy in North Dakota. Am J Med Genet. 1991;41:212–215
  28. Varni JW, Burwinkle TM, Seid M, Skarr D. The PedsQL™ 4.0 as a pediatric population health measure: feasibility, reliability, and validity. Ambul Pediatr. 2003;3:329–341
  29. SPSS. SPSS 16.0 for Windows. Chicago: SPSS, Inc.; 2008.
  30. Varni JW, Seid M, Knight TS, Burwinkle TM, Brown J, Szer IS. The PedsQL™ in pediatric rheumatology: reliability, validity, and responsiveness of the Pediatric Quality of Life Inventory™ Generic Core Scales and Rheumatology Module. Arthritis Rheum. 2002;46:714–725
  31. In:  Schwarz N,  Sudman N editor. Answering questions: methodology for determining cognitive and communicative processes in survey research. San Francisco: Jossey-Bass; 1996;
  32. Fowler FJ. Improving survey questions: design and evaluation. Thousand Oaks, CA: Sage; 1995;
  33. Aday LA. Designing and conducting health surveys: a comprehensive guide. 2nd ed.. San Francisco: Jossey-Bass; 1996;
  34. McHorney CA, Ware JE, Lu JFR, Sherbourne CD. The MOS 36-item short-form health survey (SF-36): III. Tests of data quality, scaling assumptions, and reliability across diverse patient groups. Med Care. 1994;32:40–66
  35. Hays RD, Anderson R, Revicki D. Psychometric considerations in evaluating health-related quality of life measures. Qual Life Res. 1993;2:441–449
  36. Cronbach LJ. Coefficient alpha and the internal structure of tests. Psychometrika. 1951;16:297–334
  37. Pedhazur EJ, Schmelkin LP. Measurement, design, and analysis: an integrated approach. Hillsdale, NJ: Erlbaum; 1991;
  38. Nunnally JC, Bernstein IR. Psychometric theory. 3rd ed.. New York: McGraw-Hill; 1994;
  39. Chan LFP, Chow SMK, Lo SK. Preliminary validation of the Chinese version of the Pediatric Quality of Life Inventory. Int J Rehabil Res. 2005;28:219–227
  40. Wilson KA, Dowling AJ, Abdolell M, Tannock IF. Perception of quality of life by patients, partners and treating physicians. Qual Life Res. 2001;9:1041–1052
  41. Bartko JJ. The intraclass correlation coefficient as a measure of reliability. Psychol Rep. 1966;19:3–11
  42. Fayers PM, Machin D. Quality of life: assessment, analysis, and interpretation. New York: Wiley; 2000;
  43. Cohen J. Statistical power analysis for the behavioral sciences. 2nd ed.. Hillsdale, NJ: Erlbaum; 1988;
  44. Fayers PM, Hand DJ. Factor analysis, causal indicators and quality of life. Qual Life Res. 1997;6:139–150
  45. McGraw KO, Wong SP. Forming inferences about some intraclass correlation coefficients. Psychol Methods. 1996;1:30–46
  46. Cremeens J, Eiser C, Blades M. Factors influencing agreement between child self-report and parent proxy-reports on the Pediatric Quality of Life Inventory™ 4.0 (PedsQL™) Generic Core Scales. Health Qual Life Outcomes. 2006;4:1–8
  47. Novick M, Lewis G. Coefficient alpha and the reliability of composite measurements. Psychometrika. 1967;32:1–13
  48. Sprangers MAG, Aaronson NK. The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: a review. J Clin Epidemiol. 1992;45:743–760
  49. Achenbach TM, McConaughy SH, Howell CT. Child/adolescent behavioral and emotional problems: implications of cross-informant correlations for situational specificity. Psychol Bull. 1987;101:213–232
  50. Upton P, Lawford J, Eiser C. Parent–child agreement across child health-related quality of life instruments: a review of the literature. Qual Life Res. 2008;17:895–913
  51. Eiser C, Morse R. Can parents rate their child’s health-related quality of life? Results from a systematic review. Qual Life Res. 2001;10:347–357
  52. Comrey AL, Lee HB. A first course in factor analysis. 2nd ed.. Hillsdale, NJ: Lawrence Erlbaum Associates; 1992;
  53. Tabachnick BG, Fidell LS. Using multivariate analysis. 4th ed.. Boston, MA: Allyn and Bacon; 2001;
  54. Palermo TM, Long AC, Lewandowski AS, Drotar D, Quittner AL, Walker LS. Evidence-based assessment of health-related quality of life and functional impairment in pediatric psychology. J Pediatr Psychol. 2008;33:983–996

 Competing interests: Dr. Varni holds the copyright and the trademark for the PedsQL™ and receives financial compensation from the Mapi Research Trust, which is a non-profit research institute that charges distribution fees to for-profit companies that use the Pediatric Quality of Life Inventory™.

PII: S0960-8966(09)00623-3

doi: 10.1016/j.nmd.2009.09.009

Neuromuscular Disorders
Volume 19, Issue 12 , Pages 805-812 , December 2009