Evidence map›Paper›PMID 31772349›Full record

ArticleGenetics in medicine : official journal of the American College of Medical Genetics2020

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

Chaya N Murali, David Cuthbertson, Brady Slater, Dianne Nguyen, Alicia Turner, Gerald Harris, V Reid Sutton, Brendan Lee, Members of the BBD Consortium, Sandesh C S Nagamani

Open access · bronzeAbstract read
In one paragraph

Article in Genetics in medicine : official journal of the American College of Medical Genetics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
1.4field-weighted citation impact, top 16% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 26 citations in OpenAlex.

  1. Trial
  2. Use of Dermal Substitute in Pediatric Traumatic Fingertip Amputation Tissue Loss.Plastic and reconstructive surgery. Global open · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 16 institutions in 2 countries.

Chaya N MuraliDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
David CuthbertsonCollege of Medicine, University of South Florida, Tampa, FL, USA.
Brady SlaterDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Dianne NguyenDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Alicia TurnerDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Gerald HarrisMarquette University and Medical College of Wisconsin, Wisconsin, USA.
V Reid SuttonDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Brendan LeeDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Members of the BBD Consortium
Sandesh C S NagamaniDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA. nagamani@bcm.edu.ORCID http://orcid.org/0000-0001-6818-7367
Baylor College of Medicine · USMontreal Children's Hospital · CAUniversity of Nebraska Medical Center · USUniversity of South Florida · USAlfred I. duPont Hospital for Children · USChildren's National · USHospital for Special Surgery · USKennedy Krieger Institute · USMarquette University · USMcGill University · CAOregon Health & Science University · USOsteogenesis Imperfecta Foundation · USShriners Hospitals for Children - Chicago · USShriners Hospitals for Children - Tampa · USUniversity of California, Los Angeles · USUniversity of Washington · US

Funding

The Impact of COVID-19 on People Living with Rare Diseases and Their FamiliesU2CTR002818 · NCATS · CINCINNATI CHILDRENS HOSP MED CTR · PI Maurizio Macaluso · 2019 to 2026
$51.2M
Project 3: 3D ImagingU54AR068069 · NIAMS · BAYLOR COLLEGE OF MEDICINE · PI Sandesh Chakravarthy Sreenath Nagamani · 2014 to 2026
$19.3M
MEDICAL GENETICS RESEARCH FELLOWSHIP PROGRAMT32GM007526 · NIGMS · BAYLOR COLLEGE OF MEDICINE · PI Brendan Lee · 1985 to 2026
$11.4M
Preclinical and Clincial OutcomesP50HD103555 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI MIRJANA MALETIC-SAVATIC · 2020 to 2026
$9.9M
Steps towards a paternal gene activation therapy for Angelman syndromeU54HD083092 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI NELSON, DAVID LOREN, SAMACO, RODNEY C · 2014 to 2019
$7.8M
NCATS NIH HHS U2C TR002818NIAMS NIH HHS U54 AR068069NICHD NIH HHS P50 HD103555NICHD NIH HHS U54 HD083092NIGMS NIH HHS T32 GM007526
6 · The paper itself

Abstract

purposePatient-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).

methodsPhysical 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.

resultsFour 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.

conclusionPODCI 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.

Indexed as

Patient Reported Outcome MeasuresPediatricsAdolescentChildChild, PreschoolCohort StudiesFemaleHumansMaleMotor ActivityOsteogenesis ImperfectaOutcome Assessment, Health CareQuality of LifeSeverity of Illness IndexSurveys and Questionnairesclinical trial readinesshealth-related quality of lifeosteogenesis imperfectapatient-reported outcome measurePediatric Outcomes Data Collection Instrument

Identifiers

PMID31772349
PMCPMC7060104
OpenAlexW2990433723

What OpenQuestion holds

Textmetadata
LicenceTDM
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.