Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

Natalie V. J. Aldhouse; Helen Kitchen; Chloe Johnson; Chris Marshall; Hannah Pegram; Sheryl Pease; Sam Collins; Christine L. Baker; Katherine Beaverson; Chandler Crews; Jill Massey; Kathleen W. Wyrwich

doi:10.1186/s13023-022-02333-6

Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

Natalie V. J. Aldhouse, Helen Kitchen, Chloe Johnson, Chris Marshall, Hannah Pegram, Sheryl Pease, Sam Collins, Christine L. Baker, Katherine Beaverson, Chandler Crews, Jill Massey, Kathleen W. Wyrwich

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4 Citations (Scopus)

Abstract

This study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia. Qualitative interviews lasting up to 90 min were conducted in the US with children/adolescents with achondroplasia and/or their caregivers. Interviews utilized concept elicitation methodology to explore experiences and priorities for treatment outcomes. Cognitive debriefing methodology explored relevance and understanding of selected COAs.

Original language	English
Journal	Orphanet Journal of Rare Diseases
Volume	17
Issue number	1
DOIs	https://doi.org/10.1186/s13023-022-02333-6
Publication status	Published - 7 May 2022

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Aldhouse, N. V. J., Kitchen, H., Johnson, C., Marshall, C., Pegram, H., Pease, S., Collins, S., Baker, C. L., Beaverson, K., Crews, C., Massey, J., & Wyrwich, K. W. (2022). Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials. Orphanet Journal of Rare Diseases, 17(1). https://doi.org/10.1186/s13023-022-02333-6

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AU - Aldhouse, Natalie V. J.

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AU - Pease, Sheryl

AU - Collins, Sam

AU - Baker, Christine L.

AU - Beaverson, Katherine

AU - Crews, Chandler

AU - Massey, Jill

AU - Wyrwich, Kathleen W.

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Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

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