Evidence map›Paper›PMID 39697056›Full record

ArticleDevelopmental medicine and child neurology2025

Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy.

Linda Pax Lowes, Corinne M Le Reun, Lindsay N Alfano, Natalie F Reash, Megan A Iammarino, Shivangi Patel, Ivana F Audhya

Abstract read
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Article in Developmental medicine and child neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

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3citing papers in PubMed
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1 · What the graph read from it

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3 · Its place in the literature

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3 citing papers in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

7 authors.

Linda Pax LowesCenter for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0003-4206-0557
Corinne M Le ReunBiostatistics, Sainte-Anne, Guadeloupe, France.
Lindsay N AlfanoCenter for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0002-2263-7569
Natalie F ReashCenter for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Megan A IammarinoCenter for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0003-2800-3855
Shivangi PatelPatient-Centered Outcomes, Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Ivana F AudhyaPatient-Centered Outcomes, Sarepta Therapeutics, Inc., Cambridge, MA, USA.

Funding

Sarepta Therapeutics, Inc N/A
6 · The paper itself

Abstract

aimTo evaluate the psychometric properties and measurement quality of the Patient-Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility item bank (v1.0, 23 items) for children with Duchenne muscular dystrophy (DMD), through Rasch statistical analysis.

methodDe-identified PROMIS PP Mobility items were completed by the caregivers of male patients with DMD, aged 4 to 12 years, as part of standard clinical care at the Nationwide Children's Hospital clinic; data were mined retrospectively from electronic health records. Rasch analysis was used to assess the internal functioning of the measure and items.

resultsOverall, 151 observations were available for the Rasch analysis, equally split between patients aged 4 to 7 years and 8 to 12 years. After removing clinically irrelevant items and regrouping response options for specific items, the resulting 19-item measure demonstrated overall good fit to Rasch model expectations and the ability to discriminate between respondents with different mobility levels (Person Separation Index = 0.95, excellent reliability).

interpretationThe customized PROMIS PP Mobility measure demonstrated good fit and may be a reliable option for mobility assessment in children with DMD. Rasch analysis can be used by other researchers to improve the sensitivity of patient-reported outcomes in their field of interest.

Indexed as

Mobility LimitationMuscular Dystrophy, DuchenneParentsPatient Reported Outcome MeasuresPsychometricsChildChild, PreschoolHumansMaleProxyReproducibility of ResultsRetrospective Studies

Identifiers

PMID39697056
PMCPMC12134438

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