Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta

Members of the BBD Consortium

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish (US)
JournalGenetics in Medicine
DOIs
StateAccepted/In press - Jan 1 2019

Fingerprint

Osteogenesis Imperfecta
Pediatrics
Psychology
Reproducibility of Results
Sample Size
Health Status
Multicenter Studies
Patient Reported Outcome Measures
Clinical Trials
Population

Keywords

  • Pediatric Outcomes Data Collection Instrument
  • clinical trial readiness
  • health-related quality of life
  • osteogenesis imperfecta
  • patient-reported outcome measure

ASJC Scopus subject areas

  • Genetics(clinical)

Cite this

@article{6838b395725e4acd885f93b59391ec5e,
title = "Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta",
abstract = "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.",
keywords = "Pediatric Outcomes Data Collection Instrument, clinical trial readiness, health-related quality of life, osteogenesis imperfecta, patient-reported outcome measure",
author = "{Members of the BBD Consortium} and Murali, {Chaya N.} and David Cuthbertson and Brady Slater and Dianne Nguyen and Alicia Turner and Gerald Harris and Sutton, {V. Reid} and Brendan Lee and Frank Rauch and Francis Glorieux and Retrouvey, {Jean Marc} and Paul Esposito and Eric Rush and Michael Bober and David Eyre and Danielle Gomez and Tracy Hart and Mahim Jain and Deborah Krakow and Jeffrey Krischer and Eric Orwoll and Cathleen Raggio and Peter Smith and Laura Tosi and Nagamani, {Sandesh C.S.}",
year = "2019",
month = "1",
day = "1",
doi = "10.1038/s41436-019-0688-6",
language = "English (US)",
journal = "Genetics in Medicine",
issn = "1098-3600",
publisher = "Lippincott Williams and Wilkins",

}

TY - JOUR

T1 - Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta

AU - Members of the BBD Consortium

AU - Murali, Chaya N.

AU - Cuthbertson, David

AU - Slater, Brady

AU - Nguyen, Dianne

AU - Turner, Alicia

AU - Harris, Gerald

AU - Sutton, V. Reid

AU - Lee, Brendan

AU - Rauch, Frank

AU - Glorieux, Francis

AU - Retrouvey, Jean Marc

AU - Esposito, Paul

AU - Rush, Eric

AU - Bober, Michael

AU - Eyre, David

AU - Gomez, Danielle

AU - Hart, Tracy

AU - Jain, Mahim

AU - Krakow, Deborah

AU - Krischer, Jeffrey

AU - Orwoll, Eric

AU - Raggio, Cathleen

AU - Smith, Peter

AU - Tosi, Laura

AU - Nagamani, Sandesh C.S.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - 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.

AB - 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.

KW - Pediatric Outcomes Data Collection Instrument

KW - clinical trial readiness

KW - health-related quality of life

KW - osteogenesis imperfecta

KW - patient-reported outcome measure

UR - http://www.scopus.com/inward/record.url?scp=85075585053&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85075585053&partnerID=8YFLogxK

U2 - 10.1038/s41436-019-0688-6

DO - 10.1038/s41436-019-0688-6

M3 - Article

C2 - 31772349

AN - SCOPUS:85075585053

JO - Genetics in Medicine

JF - Genetics in Medicine

SN - 1098-3600

ER -