TY - JOUR
T1 - Meaningful and Measurable Health Domains in Huntington's Disease
T2 - Large-Scale Validation of the Huntington's Disease Health-Related Quality of Life Questionnaire Across Severity Stages
AU - European Huntington's Disease Network
AU - Sue Beevers and team
AU - Catherine Paradise and team
AU - Ho, Aileen K.
AU - Horton, Mike C.
AU - Landwehrmeyer, G. Bernhard
AU - Burgunder, Jean Marc
AU - Tennant, Alan
AU - Downie, Lorna
AU - Jack, Roisin
AU - Matheson, Kirsty
AU - Miedzybrodzka, Zosia
AU - Rae, Daniela
AU - Simpson, Sheila A.
AU - Summers, Fiona
AU - Ure, Alexandra
AU - Vaughan, Vivien
AU - Akhtar, Shahbana
AU - Crooks, Jenny
AU - Curtis, Adrienne
AU - de Souza (Keylock), Jenny
AU - Piedad, John
AU - Rickards, Hugh
AU - Wright, Jan
AU - Coulthard, Elizabeth
AU - Gethin, Louise
AU - Hayward, Beverley
AU - Sieradzan, Kasia
AU - Wright, Abigail
AU - Barker, Roger A.
AU - O'Keefe, Deidre
AU - Gerrtiz (nee Di Pietro), Anna
AU - Fisher, Kate
AU - Goodman, Anna
AU - Hill, Susan
AU - Mason, Sarah
AU - Swain, Rachel
AU - Guzman, Natalie Valle
AU - Busse, Monica
AU - Butcher, Cynthia
AU - Callaghan, Jenny
AU - Dunnett, Stephen
AU - Clenaghan, Catherine
AU - Fullam, Ruth
AU - Hunt, Sarah
AU - Jones, Lesley
AU - Jones, Una
AU - Khalil, Hanan
AU - Minster, Sara
AU - Owen, Michael
AU - Price, Kathleen
AU - Townhill, Jenny
AU - Rosser, Anne
N1 - Publisher Copyright:
© 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research
PY - 2019/6
Y1 - 2019/6
N2 - Background: Although health-related quality of life is key for patients with long-term neurodegenerative conditions, measuring this is less straightforward and complex in Huntington's disease (HD). Objectives: To refine and validate a fully patient-derived instrument, the Huntington's Disease health-related Quality of Life questionnaire (HDQoL), and to elucidate health domains that are meaningful to patients’ lived experience. Methods: Five-hundred forty-one participants, from premanifest to end-stage disease, completed the HDQoL, together with generic quality-of-life measures and in-person motor, cognitive, and behavioral assessments. The psychometric properties of the HDQoL were examined using factor analysis and Rasch analysis. Results: Four HDQoL domains emerged, reflecting the classical triad of HD features; they were Physical-Functional, Cognitive, and 2 different behavioral aspects, that is, the Mood-Self domain and a distinct Worries domain. These domains clarify the behavioral sequelae as experienced by patients, and all showed good to excellent internal consistency. Known-groups analyses illustrated significant and graded changes in clinical assessments and corresponding HDQoL domains across disease severity levels. Convergent and discriminant validity was demonstrated by the expected pattern of correlations between specific HDQoL domains and corresponding domain-relevant clinical assessments as well as patient-reported measures. The data demonstrate robust support for the refined HDQoL across disease stages. Conclusions: The HDQoL, with its 2 distinct behavioral domains of Mood-Self and Worries as well as the Physical-Functional and Cognitive domains, is a relevant, reliable, and valid patient-derived instrument to measure the impact of HD across all severity stages.
AB - Background: Although health-related quality of life is key for patients with long-term neurodegenerative conditions, measuring this is less straightforward and complex in Huntington's disease (HD). Objectives: To refine and validate a fully patient-derived instrument, the Huntington's Disease health-related Quality of Life questionnaire (HDQoL), and to elucidate health domains that are meaningful to patients’ lived experience. Methods: Five-hundred forty-one participants, from premanifest to end-stage disease, completed the HDQoL, together with generic quality-of-life measures and in-person motor, cognitive, and behavioral assessments. The psychometric properties of the HDQoL were examined using factor analysis and Rasch analysis. Results: Four HDQoL domains emerged, reflecting the classical triad of HD features; they were Physical-Functional, Cognitive, and 2 different behavioral aspects, that is, the Mood-Self domain and a distinct Worries domain. These domains clarify the behavioral sequelae as experienced by patients, and all showed good to excellent internal consistency. Known-groups analyses illustrated significant and graded changes in clinical assessments and corresponding HDQoL domains across disease severity levels. Convergent and discriminant validity was demonstrated by the expected pattern of correlations between specific HDQoL domains and corresponding domain-relevant clinical assessments as well as patient-reported measures. The data demonstrate robust support for the refined HDQoL across disease stages. Conclusions: The HDQoL, with its 2 distinct behavioral domains of Mood-Self and Worries as well as the Physical-Functional and Cognitive domains, is a relevant, reliable, and valid patient-derived instrument to measure the impact of HD across all severity stages.
KW - Huntington's disease
KW - patient-reported outcome
KW - presymptomatic/preclinical Huntington's disease
KW - quality of life
KW - questionnaire
UR - http://www.scopus.com/inward/record.url?scp=85067025295&partnerID=8YFLogxK
U2 - 10.1016/j.jval.2019.01.016
DO - 10.1016/j.jval.2019.01.016
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C2 - 31198189
AN - SCOPUS:85067025295
SN - 1098-3015
VL - 22
SP - 712
EP - 720
JO - Value in Health
JF - Value in Health
IS - 6
ER -