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Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in
Chaya N Murali1,2, David Cuthbertson3, Brady Slater1
1Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Insights
The Pediatric Outcomes Data Collection Instrument (PODCI) reliably measures physical functioning in children with osteogenesis imperfecta (OI). This patient-reported outcome measure provides valuable health status insights for intervention studies.
Area of Science:
- Pediatric Orthopedics
- Clinical Trial Endpoints
- Patient-Reported Outcome Measures (PROMs)
Background:
- PROMs are crucial for assessing patient health status in clinical research.
- The Pediatric Outcomes Data Collection Instrument (PODCI) is a PROM for pediatric musculoskeletal disorders.
- Osteogenesis imperfecta (OI) is a genetic disorder affecting bone fragility.
Purpose of the Study:
- To evaluate the validity and reliability of the PODCI in children diagnosed with osteogenesis imperfecta (OI).
- To assess physical functioning and psychological well-being using PODCI in a large OI cohort.
Main Methods:
- A multicenter study enrolled 417 children with OI types I, III, and IV.
- PODCI assessed physical functioning and psychological well-being.
- PODCI physical function scores were correlated with the Brief Assessment of Motor Function (BAMF) and psychological well-being scores.
Main Results:
- Children with OI type III exhibited significantly lower physical function scores compared to types I and IV.
- No significant differences in psychological well-being were observed across OI types.
- PODCI demonstrated moderate-to-strong correlations with BAMF, indicating good validity for physical function.
Conclusions:
- The PODCI is a reliable instrument for measuring physical functioning in pediatric OI patients.
- PODCI offers valuable insights into patient-reported health status for OI.
- This PROM can inform the evaluation of interventions for children with OI.
Purpose:
Patient-reported outcome measures (PROMs) are increasingly recognized as valuable endpoints in clinical trials. The Pediatric Outcomes Data Collection Instrument (PODCI) is a PROM utilized in children with musculoskeletal disorders. We evaluated the validity and reliability of PODCI in children with osteogenesis imperfecta (OI).
Methods:
Physical functioning and psychological well-being were assessed using PODCI in a large cohort of children enrolled in a multicenter study conducted by the Brittle Bone Disorders Consortium. Physical function scores were correlated with a validated, observer-rated scale, Brief Assessment of Motor Function (BAMF), and with psychological well-being scores. We calculated sample sizes required to detect clinically meaningful differences in physical function.
Results:
Four hundred seventeen children with OI types I, III, and IV were enrolled. Physical function scores in OI type III were significantly lower than those in OI types I and IV. There were no significant differences in psychological well-being. PODCI physical function scores showed moderate-to-strong correlation with BAMF. The Global Functioning Scale, a composite of physical function, did not consistently correlate with psychological well-being.
Conclusion:
PODCI can be a reliable measure of physical functioning in children with OI and offers valuable information about patient-reported health status and new ways to examine the utility of interventions in this population.
