Evidence map›Paper›PMID 26519538›Full record

Trial reportNeurology2016

Behavioral and cognitive outcomes for clinical trials in children with neurofibromatosis type 1.

Thijs van der Vaart, André B Rietman, Ellen Plasschaert, Eric Legius, Ype Elgersma, Henriëtte A Moll, NF1-SIMCODA Study Group

Open access · greenAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 2 pooled it
1.8field-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

24 citing papers in PubMed, 2 syntheses or guidelines pooled it, 33 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Trial
  5. Trial
  6. Article
  7. Review
  8. Review
  9. Article
  10. Article
  11. Article
  12. Review
  13. Review
  14. Article
  15. Article
  16. Article
  17. Article
  18. Brain-wide structural and functional disruption in mice with oligodendrocyte-specificProceedings of the National Academy of Sciences of the United States of America · 2020
    Article
  19. Review
  20. Emerging therapeutic targets for neurofibromatosis type 1.Expert opinion on therapeutic targets · 2018
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 1 institution in 2 countries.

Thijs van der VaartFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium.
André B RietmanFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium.
Ellen PlasschaertFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium.
Eric LegiusFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium.
Ype ElgersmaFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium.
Henriëtte A MollFrom the Departments of Neuroscience (T.v.d.V., Y.E.), Pediatrics (T.v.d.V., H.A.M.), and Neurology (A.B.R.), and ENCORE-Expertise Center for Neurodevelopmental Disorders (T.v.d.V., A.B.R., Y.E., H.A.M.), Erasmus MC: University Medical Centre, Rotterdam, the Netherlands; the Department of Human Genetics (E.P., E.L.), Catholic University Leuven; and the Centre for Human Genetics at University Hospital Leuven (E.P., E.L.), Belgium. h.a.moll@erasmusmc.nl.
NF1-SIMCODA Study Group
Erasmus MC · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo evaluate the appropriateness of cognitive and behavioral outcome measures in clinical trials in neurofibromatosis type 1 (NF1) by analyzing the degree of deficits compared to reference groups, test-retest reliability, and how scores correlate between outcome measures.

methodsData were analyzed from the Simvastatin for cognitive deficits and behavioral problems in patients with neurofibromatosis type 1 (NF1-SIMCODA) trial, a randomized placebo-controlled trial of simvastatin for cognitive deficits and behavioral problems in children with NF1. Outcome measures were compared with age-specific reference groups to identify domains of dysfunction. Pearson r was computed for before and after measurements within the placebo group to assess test-retest reliability. Principal component analysis was used to identify the internal structure in the outcome data.

resultsStrongest mean score deviations from the reference groups were observed for full-scale intelligence (-1.1 SD), Rey Complex Figure Test delayed recall (-2.0 SD), attention problems (-1.2 SD), and social problems (-1.1 SD). Long-term test-retest reliability were excellent for Wechsler scales (r > 0.88), but poor to moderate for other neuropsychological tests (r range 0.52-0.81) and Child Behavioral Checklist subscales (r range 0.40-0.79). The correlation structure revealed 2 strong components in the outcome measures behavior and cognition, with no correlation between these components. Scores on psychosocial quality of life correlate strongly with behavioral problems and less with cognitive deficits.

conclusionsChildren with NF1 show distinct deficits in multiple domains. Many outcome measures showed weak test-retest correlations over the 1-year trial period. Cognitive and behavioral outcomes are complementary. This analysis demonstrates the need to include reliable outcome measures on a variety of cognitive and behavioral domains in clinical trials for NF1.

Indexed as

Quality of LifeAdolescentAttentionBehaviorChildCognitionCognition DisordersFemaleHumansIntelligenceMaleNeurofibromatosis 1Neuropsychological TestsSimvastatinSimvastatin

Identifiers

PMID26519538
PMCPMC4731686
OpenAlexW1857289517

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.