What question did this study set out to answer?

To identify critical measurement concepts and select appropriate clinical outcome assessments for pediatric achondroplasia clinical trials.

May 7, 2022Orphanet Journal of Rare DiseasesOpen Access

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

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Authors

NANatalie V. J. AldhouseHealth Economics and Outcomes Research (United Kingdom)HKHelen KitchenHealth Economics and Outcomes Research (United Kingdom)CJChloe JohnsonHealth Economics and Outcomes Research (United Kingdom)

Discussion

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Implication

Methodological analysis identifies outcome measures for pediatric achondroplasia trials, suggesting functional reach and well-being are vital alongside height.

Key Points

To identify critical measurement concepts and select appropriate clinical outcome assessments for pediatric achondroplasia clinical trials.
Evaluated outcome measures assessing physical functioning, daily functional activities like reach, and emotional and social well-being in pediatric achondroplasia.
Determined appropriate reporting modalities across age brackets for pediatric clinical trials (ages 0–11 and 12–17 years).
Identified that patient and caregiver treatment priorities extend beyond height and limb proportion to functional outcomes, especially physical reach.
Established the adapted Childhood Health Assessment Questionnaire (CHAQ) and QoLISSY-Brief as relevant and appropriate measures of physical and emotional well-being.
Recommended caregiver-reported outcomes for children aged 0–11 years and direct patient-reported outcomes for adolescents aged 12–17 years.

Cite This Study

Aldhouse et al. (2022) studied this question.

synapsesocial.com/papers/69d713ee8a0e2c5879bef011 https://doi.org/10.1186/s13023-022-02333-6

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Implication

Methodological analysis identifies outcome measures for pediatric achondroplasia trials, suggesting functional reach and well-being are vital alongside height.

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

Discussion

Member takes

Implication

Key Points

Cite This Study

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

Discussion

Member takes

Implication

Key Points

Cite This Study

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