Evidence map›Paper›PMID 32300063›Full record

ArticleNeurology2020

The CLN3 Disease Staging System: A new tool for clinical research in Batten disease.

Margaux C Masten, Justin D Williams, Jennifer Vermilion, Heather R Adams, Amy Vierhile, Alyssa Collins, Frederick J Marshall, Erika F Augustine, Jonathan W Mink

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 1 pooled it
–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

24 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Guideline
  2. Article
  3. Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Observational
  9. Article
  10. Article
  11. Observational
  12. Article
  13. Article
  14. Brain proton MR spectroscopy measurements in CLN3 disease.Molecular genetics and metabolism · 2023
    Article
  15. Review
  16. Article
  17. The Unified Batten Disease Rating Scale (UBDRS): Validation and reliability in an independent CLN3 disease sample.European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society · 2022
    Article
  18. Use of the Vineland-3, a measure of adaptive functioning, in CLN3.American journal of medical genetics. Part A · 2022
    Observational
  19. Review
  20. A diagnostic confidence scheme for CLN3 disease.Journal of inherited metabolic disease · 2021
    Observational
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

9 authors.

Margaux C MastenFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY. margaux_masten@urmc.rochester.edu.
Justin D WilliamsFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Jennifer VermilionFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Heather R AdamsFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Amy VierhileFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Alyssa CollinsFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Frederick J MarshallFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Erika F AugustineFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.
Jonathan W MinkFrom the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Rochester School of Nursing (A.V.), NY.

Funding

Training & EducationU54NS065768 · NINDS · UNIVERSITY OF MINNESOTA · PI WHITLEY, CHESTER B. · 2009 to 2024
$17.7M
Meaningful Outcomes and Multi-Site Readiness for Clinical Trials in Juvenile Neuronal Ceroid LipofuscinosisU01NS101946 · NINDS · UNIVERSITY OF ROCHESTER · PI AUGUSTINE, ERIKA · 2019 to 2024
$3.7M
Clinical and Neuropsychological Investigations in Batten DiseaseR01NS060022 · NINDS · UNIVERSITY OF ROCHESTER · PI MINK, JONATHAN W. · 2007 to 2010
$1.2M
NINDS NIH HHS R01 NS060022NINDS NIH HHS U01 NS101946NINDS NIH HHS U54 NS065768
6 · The paper itself

Abstract

objectiveTo develop a disease-specific staging system for CLN3 disease and to test the hypothesis that salient and discrete clinical features of CLN3 disease may be used to define disease stages by analyzed data from an 18-year-long natural history study.

methodsA proposed staging system, the CLN3 Staging System (CLN3SS), was based on salient and clinically meaningful endpoints. The relationships between stage and age, stage and Unified Batten Disease Rating Scale (UBDRS) physical severity score, and stage and UBDRS capability impairment subscale scores were determined. We used

resultsData were analyzed from 322 evaluations in 108 individuals. There were significant differences among the stages based on age and severity scores. For individuals with longitudinal data, no individual reverted to a less severe stage over time.

conclusionThe CLN3SS is a disease-specific staging system that can be used to classify individuals into specific strata based on age and disease severity. The CLN3SS has potential applications in clinical trials for cohort stratification.

Indexed as

AdolescentAdultChildFemaleGait Disorders, NeurologicHumansMaleMembrane GlycoproteinsMolecular ChaperonesNeuronal Ceroid-LipofuscinosesPrognosisSeizuresSeverity of Illness IndexVision DisordersCLN3 protein, humanMembrane GlycoproteinsMolecular Chaperones

Identifiers

PMID32300063
PMCPMC7455368

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Registered trials

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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.