title: Measuring quality of life in muscular dystrophy.
abstract: OBJECTIVE
  The objectives of this study were to develop a conceptual model of quality of life (QOL) in muscular dystrophies (MDs) and review existing QOL measures for use in the MD population.
  
  
  METHODS
  Our model for QOL among individuals with MD was developed based on a modified Delphi process, literature review, and input from patients and patient advocacy organizations. Scales that have been used to measure QOL among patients with MD were identified through a literature review and evaluated using the COSMIN (Consensus-Based Standards for the Selection of Health Measurement Instruments) checklist.
  
  
  RESULTS
  The Comprehensive Model of QOL in MD (CMQM) captures 3 broad domains of QOL (physical, psychological, and social), includes factors influencing self-reported QOL (disease-related factors, support/resources, and expectations/aspirations), and places these concepts within the context of the life course. The literature review identified 15 QOL scales (9 adult and 6 pediatric) that have been applied to patients with MD. Very few studies reported reliability data, and none included data on responsiveness of the measures to change in disease progression, a necessary psychometric property for measures included in treatment and intervention studies. No scales captured all QOL domains identified in the CMQM model.
  
  
  CONCLUSIONS
  Additional scale development research is needed to enhance assessment of QOL for individuals with MD. Item banking and computerized adaptive assessment would be particularly beneficial by allowing the scale to be tailored to each individual, thereby minimizing respondent burden.
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA. cmb@rti.org.
authors: Carla M
authors: CM
authors: Bann
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Richard T
authors: RT
authors: Abresch
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Barbara
authors: B
authors: Biesecker
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Kristin Caspers
authors: KC
authors: Conway
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Chad
authors: C
authors: Heatwole
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Holly
authors: H
authors: Peay
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Peter
authors: P
authors: Scal
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Jonathan
authors: J
authors: Strober
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Karen
authors: K
authors: Uzark
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Jodi
authors: J
authors: Wolff
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Marjorie
authors: M
authors: Margolis
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Angela
authors: A
authors: Blackwell
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Natalie
authors: N
authors: Street
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Angela
authors: A
authors: Montesanti
authors: From RTI International (C.M.B.), Research Triangle Park, NC; University of California, Davis (R.T.A.); National Human Genome Research Institute (B.B.), Bethesda, MD; University of Iowa (K.C.C.), Iowa City; University of Rochester (C.H.), NY; Parent Project Muscular Dystrophy (H.P.), Hackensack, NJ; University of Minnesota (P.S.), Minneapolis; UCSF Benioff Children's Hospital (J.S.), San Francisco, CA; University of Michigan (K.U.), Ann Arbor; Muscular Dystrophy Association (J.W.), Chicago, IL; University of North Carolina (M.M.), Chapel Hill; RTI International (A.B.), Atlanta; and Centers for Disease Control and Prevention (N.S., A.M., J.B.), Atlanta, GA.
authors: Julie
authors: J
authors: Bolen
mesh_terms: D006801:Humans; D009136:Muscular Dystrophies / Q000523:psychology*; D011594:Psychometrics / Q000295:instrumentation*; D011788:Quality of Life / Q000523:psychology*; D011795:Surveys and Questionnaires / Q000592:standards*