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Quality of Life Research
Gepubliceerd in: Quality of Life Research 9/2019

15-05-2019

Cross-cultural selection and validation of instruments to assess patient-reported outcomes in children and adolescents with achondroplasia

Auteurs: Janika Bloemeke, Rachel Sommer, Stefanie Witt, Monika Bullinger, Clementine Nordon, Francisco Javier Badia, Felipe Luna González, Antonio Leiva-Gea, Francisco de Borja Delgado Rufino, Fermín Mayoral-Cleries, Pablo Romero-Sanchiz, Verónica Clamagirand Saiz, Raquel Nogueira-Arjona, Klaus Mohnike, Julia Quitmann

Gepubliceerd in: Quality of Life Research | Uitgave 9/2019

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Abstract

Purpose

Achondroplasia, as the most common form of disproportionate short stature, potentially impacts the health-related quality of life (HRQOL) and functioning of people with this condition. Because there are no psychometrically validated patient-reported outcome (PRO) condition-specific instruments for achondroplasia, this study selected and tested available generic, disease-specific and under development questionnaires for possible use in multinational clinical research.

Methods

A three-step approach was applied. First, a literature review and clinician/expert opinions were used to select relevant PRO questionnaires. Second, focus group discussions, including a group cognitive debriefing for piloting of the questionnaires with children/adolescents with achondroplasia and their parents, were performed in Spain and Germany. Third, a field-test study was conducted to test the psychometric properties of these instruments.

Results

Six questionnaires were identified as potentially relevant in children with achondroplasia. In each country, five focus groups including a cognitive debriefing were conducted, and the results narrowed the possibilities to three instruments as most appropriate to assess HRQOL (the generic PedsQL, the height-specific QoLISSY, and the achondroplasia-specific APLES). Results of the field study indicate the QoLISSY and the PedsQL questionnaires to be most appropriate for use in clinical research at this time.

Conclusion

This selection study is a step forward in assessing the impact of achondroplasia on HRQOL. Of the instruments examined, the QoLISSY and the PedsQL both capture items relevant to children with achondroplasia and have met the psychometric validation criteria needed for use in research. The APLES instrument is a promising tool that should be revisited upon psychometric validation.
vorige artikel Psychometric properties and cross-language equivalence of the revised Bristol Rheumatoid Arthritis Fatigue and the Rheumatoid Arthritis Impact of Disease scales in rheumatoid arthritis
volgende artikel Use of an expert panel to identify domains and indicators of delirium severity
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Metagegevens
Titel
Cross-cultural selection and validation of instruments to assess patient-reported outcomes in children and adolescents with achondroplasia
Auteurs
Janika Bloemeke
Rachel Sommer
Stefanie Witt
Monika Bullinger
Clementine Nordon
Francisco Javier Badia
Felipe Luna González
Antonio Leiva-Gea
Francisco de Borja Delgado Rufino
Fermín Mayoral-Cleries
Pablo Romero-Sanchiz
Verónica Clamagirand Saiz
Raquel Nogueira-Arjona
Klaus Mohnike
Julia Quitmann
Publicatiedatum
15-05-2019
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 9/2019
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-019-02210-z

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