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The amyotrophic lateral sclerosis-health index (ALS-HI): development and evaluation of a novel outcome measure.
Varma, Anika; Weinstein, Jennifer; Seabury, Jamison; Rosero, Spencer; Zizzi, Christine; Alexandrou, Danae; Wagner, Ellen; Dilek, Nuran; Heatwole, John; Wuu, Joanne; Caress, James; Bedlack, Richard; Granit, Volkan; Statland, Jeffrey; Mehta, Paul; Benatar, Michael; Kaat, Aaron; Heatwole, Chad.
Afiliación
  • Varma A; Center for Health + Technology, Rochester, NY, USA.
  • Weinstein J; Center for Health + Technology, Rochester, NY, USA.
  • Seabury J; Center for Health + Technology, Rochester, NY, USA.
  • Rosero S; Center for Health + Technology, Rochester, NY, USA.
  • Zizzi C; Center for Health + Technology, Rochester, NY, USA.
  • Alexandrou D; Center for Health + Technology, Rochester, NY, USA.
  • Wagner E; Center for Health + Technology, Rochester, NY, USA.
  • Dilek N; Department of Neurology, University of Rochester, Rochester, NY, USA.
  • Heatwole J; Pittsford Sutherland High School, Pittsford, NY, USA.
  • Wuu J; Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA.
  • Caress J; Wake Forest Baptist Health, Winston-Salem, NC, USA.
  • Bedlack R; Department of Neurology, Duke University School of Medicine, Durham, NC, USA.
  • Granit V; Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA.
  • Statland J; Department of Neurology, University of Kansas Medical Center, Kansas City, KS, USA.
  • Mehta P; Centers for Disease Control and Prevention National ALS Registry, Agency for Toxic Substances and Disease Registry, Atlanta, GA, USA, and.
  • Benatar M; Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA.
  • Kaat A; Northwestern Feinberg School of Medicine, Evanston, IL, USA.
  • Heatwole C; Center for Health + Technology, Rochester, NY, USA.
Amyotroph Lateral Scler Frontotemporal Degener ; 24(5-6): 514-522, 2023 08.
Article en En | MEDLINE | ID: mdl-37190795
Objective: The identification of effective therapeutics for ALS necessitates valid and responsive outcome measures to track disease progression and therapeutic gain in clinical trial settings. The Amyotrophic Lateral Sclerosis-Health Index (ALS-HI) is a multifaceted, disease-specific patient-reported outcome measure (PRO) designed to measure ALS symptomatic disease burden in adults with ALS. Methods: Through a national cross-sectional study of individuals with ALS, we identified the most important symptoms in ALS. These symptoms were incorporated into the ALS-HI, a measure that quantifies the multifaceted disease burden in ALS. We performed factor analysis, qualitative patient interviews, test-retest reliability assessment, and known groups analysis to evaluate and validate the ALS-HI. Results: The cross-sectional study included 497 participants with ALS who identified the most important symptoms to include in the ALS-HI. Fifteen participants beta tested the ALS-HI and found it to be clear, easy to use, and relevant. Twenty-one participants engaged in a test-retest reliability study, which indicated the reliability of the instrument (intraclass correlation coefficient = 0.952 for full instrument). The final ALS-HI and its subscales demonstrated a high internal consistency (Cronbach's α = 0.981 for full instrument) and an ability to differentiate between groups with dissimilar disease severity. Conclusions: This research supports use of the ALS-HI as a valid, sensitive, reliable, and relevant PRO to assess the multifactorial disease burden faced by adults with ALS. The ALS-HI has potential as a mechanism to track disease progression and treatment efficacy during therapeutic trials.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Esclerosis Amiotrófica Lateral Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prevalence_studies / Prognostic_studies / Qualitative_research / Risk_factors_studies Aspecto: Patient_preference Límite: Adult / Humans Idioma: En Revista: Amyotroph Lateral Scler Frontotemporal Degener Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Esclerosis Amiotrófica Lateral Tipo de estudio: Clinical_trials / Diagnostic_studies / Observational_studies / Prevalence_studies / Prognostic_studies / Qualitative_research / Risk_factors_studies Aspecto: Patient_preference Límite: Adult / Humans Idioma: En Revista: Amyotroph Lateral Scler Frontotemporal Degener Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido