Evidence map›Paper›PMID 42420211›Full record

ArticleMuscle & nerve2026

Development and Validation of a Novel, Disease-Specific, Caregiver-Reported Outcome Measure: The Duchenne Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI).

Charlotte Engebrecht, Spencer Rosero, Jennifer Weinstein, Jamison Seabury, Anika Varma, Christine Zizzi, John Heatwole, Nuran Dilek, Aaron Kaat, Debra Guntrum and 2 more

Abstract readValidation Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Muscle & nerve, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

12 authors.

Charlotte EngebrechtCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.ORCID https://orcid.org/0009-0002-2982-8197
Spencer RoseroCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.
Jennifer WeinsteinCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.
Jamison SeaburyCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.
Anika VarmaCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.
Christine ZizziCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.
John HeatwoleCornell University, Ithaca, New York, USA.
Nuran DilekDepartment of Biostatistics and Neurology, University of Rochester Medicine, Rochester, New York, USA.
Aaron KaatDepartment of Medical Social Sciences, Northwestern University, Chicago, Illinois, USA.
Debra GuntrumDepartment of Neurology, University of Rochester Medicine, Rochester, New York, USA.
Emma CiafaloniDepartment of Neurology, University of Rochester Medicine, Rochester, New York, USA.
Chad HeatwoleCenter for Health + Technology (CHeT), University of Rochester Medicine, Rochester, New York, USA.ORCID https://orcid.org/0000-0002-2900-6612

Funding

Duchenne UKMuscular Dystrophy Association 608054Parent Project Muscular Dystrophy
6 · The paper itself

Abstract

aimRegulatory-grade outcome measures are essential for supporting future therapeutic trials. The goal of our study was to develop and validate a multifactorial, disease-specific, caregiver-reported outcome (CRO) measure for Duchenne muscular dystrophy (DMD).

methodsWe conducted qualitative interviews and a cross-sectional study with caregivers of individuals with DMD to determine the most prevalent and impactful symptoms to individuals with DMD. Symptom items were selected for the Duchenne Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI) based on their prevalence and impact to a national sample cohort of caregivers, along with their potential ability to respond to therapeutics. Subsequently, we conducted cognitive beta interviews, test-retest reliability assessments, and internal consistency analyses to evaluate and optimize the DMDCR-HI. Using known group analysis, we assessed the ability of the DMDCR-HI to differentiate between patients with differing levels of disease burden.

resultsThe DMDCR-HI is a regulatory-grade CRO that comprehensively measures 16 subscales in DMD. The instrument has a high internal consistency, strong test-retest reliability (intraclass correlation coefficient (ICC) = 0.96), and was identified by caregivers as easy to use, relevant, and comprehensive. Through known group analysis, DMDCR-HI scores demonstrated an ability to differentiate between subgroups with varying levels of symptomatic burden. DISCUSSION: The DMDCR-HI is a valid and reliable CRO designed to sensitively and reliably capture how individuals with DMD feel and function from the perspective of the caregiver. The DMDCR-HI offers a robust mechanism to longitudinally track changes in disease burden in individuals with DMD, regardless of age, cognitive ability, or ambulation status.

Indexed as

CaregiversMuscular Dystrophy, DuchennePatient Reported Outcome MeasuresAdolescentAdultChildCross-Sectional StudiesFemaleHumansMaleMiddle AgedReproducibility of ResultsYoung Adultcaregiver‐reported outcome measureclinical outcome assessmentdrug labelDuchenne muscular dystrophyneuromuscular disorder

Identifiers

PMID42420211
PMCPMC13472041

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.