ESPE Abstracts (2022) 95 P1-118

ESPE2022 Poster Category 1 Growth and Syndromes (85 abstracts)

A background review of several generic patient-reported outcome and clinician-reported outcome measures: Selecting measures for further evaluation in a qualitative study in achondroplasia.

Susan D. Mathias 1 , Julie Hoover-Fong 2 , Ravi Savarirayan 3 , Terry Cho 4 , Anne Lee 4 , Hilary H. Colwell 1 , Daniela Rogoff 4 & Elena Muslimova 4

22 views


1Health Outcomes Solutions, Winter Park, USA; 2Johns Hopkins University, School of Medicine, Baltimore, USA; 3Murdoch Children’s Research Institute, Melbourne, Australia; 4QED Therapeutics, San Francisco, USA


Objectives: No validated achondroplasia (ACH)-specific patient reported outcome (PRO) or clinician-reported outcome (ClinRO) measures are available. A detailed review of generic PRO and ClinRO measures was performed to select relevant tools for further evaluation in a qualitative study.

Methods: Published literature describing medical challenges and impacts among children with ACH was reviewed to identify important concepts. Based on these findings, five PROs and one ClinRO were selected for further analysis.

Results: The most common symptoms were pain, ear infections/fluid, and low stamina/tiring easily. The most common impacts were difficulty reaching objects/high places, toileting, bathing/showering, washing, grooming, and running. These concepts mapped well to PRO (CHAQ, CHQ-CF45, Pain-NRS, PedsQl, PODCI, QoLISSY) and ClinRO (WeeFIM) measures (Table 1).

Table 1: Summary of measures reviewed
Measure Assessment No. of Items Concepts Psychometric properties demonstrated Recommended Yes/No
CHAQ Change in physical functioning in children with arthritis 30 Dressing and Grooming, Arising, Eating, Walking, Hygiene, Reach, Grip, Activities Some No; covers some key concepts, but only a parent version available
CHQ-CF45/CHQ-PF50 Physical and psychosocial functioning and well-being in children/adolescents 45/50 Global Health, Physical Functioning, Social/Emotional, Social/Physical, Body Pain, Getting Along, Global Behavior, Self-Esteem, Mental and General Health, Change in Health, Family Activities, Family Cohesion Not reported No; lengthy and psychometric properties not evaluated
Pain-NRS Pain severity 1 Pain Not reported Yes; commonly used to assess pain severity
PedsQL Health related quality of life (HRQoL) in healthy children/adolescents and those with acute/chronic health conditions 23 Physical, Emotional, Social, School Functioning Some Yes; covers physical, emotional, and social functioning
PODCI Changes following pediatric orthopedic interventions for broad range of diagnoses 83-86 Upper Extremity and Physical Function, Transfer and Mobility Tasks, Sports/Physical Functioning, Pain/Comfort, Treatment Expectations, Happiness, Satisfaction with Symptoms Not reported No; lengthy and psychometric properties not evaluated
QoLISSY (Core) HRQoL in children/adolescents with short stature 22 Physical, Social, Emotional Functioning Majority Yes; covers physical, social and emotional functioning
WeeFIM Need for assistance and severity of disability in children 18 Self-care, Mobility, Cognition Some Yes; covers areas of self-care not included in other measures

Conclusion: The QoLISSY, PedsQl, Pain-NRS, and WeeFIM were identified for further evaluation in a qualitative study of children with ACH and parents of children with ACH. The content, clarity, and relevance of these measures were evaluated during combined concept elicitation/cognitive debriefing interviews (Presented elsewhere).

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

Browse other volumes

Article tools

My recent searches

No recent searches.