Evidence map›Paper›PMID 25447928›Full record

ArticlePediatric neurology2015

Composite biomarkers for assessing Duchenne muscular dystrophy: an initial assessment.

Irina Shklyar, Amy Pasternak, Kush Kapur, Basil T Darras, Seward B Rutkove

Open access · greenAbstract read
In one paragraph

Article in Pediatric neurology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
1.1field-weighted citation impact, top 22% 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

11 citing papers in PubMed, 15 citations in OpenAlex.

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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

5 authors at 3 institutions in 1 country.

Irina ShklyarDepartment of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts.
Amy PasternakDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts.
Kush KapurDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts.
Basil T DarrasDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts.
Seward B RutkoveDepartment of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts. Electronic address: srutkove@bidmc.harvard.edu.
Boston Children's Hospital · USHarvard University · USBeth Israel Deaconess Medical Center · US

Funding

HARVARD CLINICAL AND TRANSLATIONAL SCIENCE CENTER (UL1)UL1RR025758 · NCRR · HARVARD MEDICAL SCHOOL · PI NADLER, LEE MARSHALL · 2008 to 2011
$91.4M
Electrical impedance myography and quantitative ultrasound in Duchenne muscular dR01AR060850 · NIAMS · BETH ISRAEL DEACONESS MEDICAL CENTER · PI DARRAS, BASIL T, RUTKOVE, SEWARD B. · 2011 to 2014
$1.7M
Howard Hughes Medical InstituteNCRR NIH HHS UL1 RR025758NIAMS NIH HHS R01 AR060850NIAMS NIH HHS R01AR060850-02
6 · The paper itself

Abstract

backgroundCompared with individual parameters, composite biomarkers may provide a more effective means for monitoring disease progression and the effects of therapy in clinical trials than single measures. In this study, we built composite biomarkers for use in Duchenne muscular dystrophy by combining values from two objective measures of disease severity: electrical impedance myography and quantitative ultrasound and evaluating how well they correlated to standard functional measures.

methodsUsing data from an ongoing study of electrical impedance myography and quantitative ultrasound in 31 Duchenne muscular dystrophy and 26 healthy boys aged 2-14 years, we combined data sets by first creating z scores based on the normal subject data and then using simple mathematical operations (addition and multiplication) to create composite measures. These composite scores were then correlated to age and standard measures of function including the 6-minute walk test, the North Star Ambulatory Assessment, and handheld dynamometry.

resultsCombining data sets resulted in stronger correlations with all four outcomes than for either electrical impedance myography or quantitative ultrasound alone in six of eight instances. These improvements reached statistical significance (P < 0.05) in several cases. For example, the correlation coefficient for the composite measure with the North Star Ambulatory Assessment was 0.79 but was only 0.66 and 0.67 (respectively) for gray scale level and electrical impedance myography separately.

conclusionsArithmetically derived composite scores can provide stronger correlations to functional measures than isolated biomarkers. Longitudinal study of such composite markers in Duchenne muscular dystrophy clinical trials is warranted.

Indexed as

AdolescentBiomarkersChildChild, PreschoolDisease ProgressionElectric ImpedanceFemaleHumansMaleMuscle, SkeletalMuscle Strength DynamometerMuscular Dystrophy, DuchenneMyographyOutcome Assessment, Health CareStatistics as TopicBiomarkersbiomarkercompositeDuchenne muscular dystrophyelectrical impedance myographyoutcome measurequantitative ultrasound

Identifiers

PMID25447928
PMCPMC4336219
OpenAlexW2132187306

What OpenQuestion holds

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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.