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; received in revised form 25 September 2009; accepted 30 September 2009.

Abstract 

For Phase II and III clinical trials in children with Spinal Muscular Atrophy (SMA), reliable and valid outcome measures are necessary. Since 2000, the American Spinal Muscular Atrophy Randomized Trials (AmSMART) group has established reliability and validity for measures of strength, lung function, and motor function in the population from age 2years to 18years. The PedsQL™ (Pediatric Quality of Life Inventory™) Measurement Model was designed to integrate the relative merits of generic and disease-specific approaches, with disease-specific modules. The PedsQL™ 3.0 Neuromuscular Module was designed to measure HRQOL dimensions specific to children ages 2–18years with neuromuscular disorders, including SMA. One hundred seventy-six children with SMA and their parents completed the PedsQL™ 4.0 Generic Core Scales and PedsQL™ 3.0 Neuromuscular Module. The PedsQL™ demonstrated feasibility, reliability, and validity in the SMA population. Consistent with the conceptualization of disease-specific symptoms as causal indicators of generic HRQOL, the majority of intercorrelations among the Neuromuscular Module Scales and the Generic Core Scales were in the medium to large range, supporting construct validity. For the purposes of a clinical trial, the PedsQL™ Neuromuscular Module and Generic Core Scales provide an integrated measurement model with the advantages of both generic and condition-specific instruments.

Keywords: PedsQL™ (Pediatric Quality of Life Inventory™), Health-related quality of life, Neuromuscular Module, Spinal Muscular Atrophy, Outcome measures

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☆ 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

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