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
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).
| 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).
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