Development and psychometric evaluation of the MetabQoL 1.0

• Verfasst von: Zeltner, Nina Amélie [VerfasserIn]
• Verfasst von: Bondarenko, Aljona [VerfasserIn]
• Verfasst von: Kölker, Stefan [VerfasserIn]
• Verfasst von: Burgard, Peter [VerfasserIn]
• Titel: Development and psychometric evaluation of the MetabQoL 1.0
• Titelzusatz: a quality of life questionnaire for paediatric patients with intoxication-type inborn errors of metabolism
• Verf.angabe: Nina A. Zeltner, Matthias R. Baumgartner, Aljona Bondarenko, Regina Ensenauer, Daniela Karall, Stefan Kölker, Chris Mühlhausen, Sabine Scholl-Bürgi, Eva Thimm, Julia Quitmann, Peter Burgard, Markus A. Landolt, Martina Huemer
• E-Jahr: 2017
• Jahr: 01 March 2017
• Umfang: 9 S.
• Teil: volume:37
• Teil: year:2017
• Teil: pages:27-35
• Teil: extent:9
• Fussnoten: Gesehen am 24.07.2018
• Titel Quelle: Enthalten in: JIMD reports
• Ort Quelle: Hoboken, NJ : Wiley, 2011
• Jahr Quelle: 2017
• Band/Heft Quelle: 37(2017), Seite 27-35
• ISSN Quelle: 2192-8312
• DOI: doi:10.1007/8904_2017_11
• Datenträger: Online-Ressource
• Sprache: eng
• K10plus-PPN: 1577897595

Abstract

Introduction: This study is part of the “European network and registry for intoxication type metabolic diseases” (E-IMD) project. Intoxication-type inborn errors of metabolism (IT-IEM) such as urea cycle disorders (UCD) and organic acidurias (OA) have a major impact on patients’ lives. Patients have to adhere to strict diet and medication and may suffer from metabolic crises and neurocognitive impairment. Disease-specific health-related quality of life (HrQoL) assessment questionnaires are the method of choice to estimate the subjective burden of a disease. To date, no such instrument is available for IT-IEM. Methods: Disease-specific patient- and parent-reported HrQoL questions were constructed in German based on focus group interviews with patients and parents. Questionnaires for patients from 8 to 18 years were piloted with 14 participants (n = 9 children and adolescents, n = 5 parents) by cognitive debriefing and tested psychometrically with 80 participants (n = 38 patients, n = 42 parents) for item characteristics, validity, and reliability to construct the first version of a disease-specific HrQoL questionnaire. Results: Twenty-eight questions were selected based on item descriptives. Scales of self- and proxy questionnaires demonstrated acceptable to excellent reliability in terms of internal consistency (Cronbach’s α = 0.70-0.93). Scales and total scores correlated with those of generic HrQoL questionnaires, showing convergent validity. Discussion: The MetabQoL 1.0 questionnaire exhibits sound psychometric properties and is a promising step towards assessing patient-reported outcomes in research and clinical practice. It provides a solid basis for translation into other languages and further elaboration and psychometric exploration in larger populations.