Evidence map›Paper›PMID 28838622›Full record

Trial reportPediatric neurology2017

Defining Hand Stereotypies in Rett Syndrome: A Movement Disorders Perspective.

Marisela E Dy, Jeff L Waugh, Nutan Sharma, Heather O'Leary, Kush Kapur, Alissa M D'Gama, Mustafa Sahin, David K Urion, Walter E Kaufmann

Open access · greenAbstract readClinical Trial, Phase IIRandomized Controlled Trial
In one paragraph

Trial report in Pediatric neurology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
1.3field-weighted citation impact, top 17% 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

4 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Article
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 at 2 institutions in 1 country.

Marisela E DyDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts. Electronic address: Marisela.dy@childrens.harvard.edu.
Jeff L WaughDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
Nutan SharmaDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
Heather O'LearyDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
Kush KapurDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
Alissa M D'GamaHarvard Medical School, Boston, Massachusetts.
Mustafa SahinDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
David K UrionDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
Walter E KaufmannDepartment of Neurology, Boston Children's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts; Center for Translational Research, Greenwood Genetic Center, Greenville, South Carolina.
Boston Children's Hospital · USHarvard University · US

Funding

HARVARD CLINICAL AND TRANSLATIONAL SCIENCE CENTER (UL1)UL1RR025758 · NCRR · HARVARD MEDICAL SCHOOL · PI NADLER, LEE MARSHALL · 2008 to 2011
$91.4M
Medical Scientist Training ProgramT32GM007753 · NIGMS · HARVARD UNIVERSITY (MEDICAL SCHOOL) · PI WALENSKY, LOREN DAVID · 1985 to 2021
$50.0M
SYNAPTIC MECHANISMS IN HIPPOCAMPAL EPILEPTOGENESISP30HD018655 · NICHD · CHILDREN'S HOSPITAL BOSTON · PI POMEROY, SCOTT LOREN · 1985 to 2015
$26.2M
Training and Career Development ComponentU54HD061222 · NICHD · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI PERCY, ALAN KENNETH · 2009 to 2019
$12.4M
Mouse Neurodevelopmental Behavior CoreU54HD090255 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI POMEROY, SCOTT LOREN · 2016 to 2020
$5.2M
NCRR NIH HHS UL1 RR025758NICHD NIH HHS P30 HD018655NICHD NIH HHS U54 HD061222NICHD NIH HHS U54 HD090255NIGMS NIH HHS T32 GM007753
6 · The paper itself

Abstract

introductionHand stereotypies (HS) are a primary diagnostic criterion for Rett syndrome (RTT) but are difficult to characterize and quantify systematically.

methodsWe collected video on 27 girls (2-12 years of age) with classic RTT who participated in a mecasermin trial. The present study focused exclusively on video analyses, by reviewing two five-minute windows per subject to identify the two most common HS. Three raters with expertise in movement disorders independently rated the five-minute windows using standardized terminology to determine the level of agreement. We iteratively refined the protocol in three stages to improve descriptive accuracy, categorizing HS as "central" or "peripheral," "simple" or "complex," scoring each hand separately. Inter-rater agreement was analyzed using Kappa statistics.

resultsIn the initial protocol evaluating HS by video, inter-rater agreement was 20.7%. In the final protocol, inter-rater agreement for the two most frequent HS was higher than the initial protocol at 50%.

conclusionPhenotypic variability makes standardized evaluation of HS in RTT a challenge; we achieved only 50% level of agreement and only for the most frequent HS. Therefore, objective measures are needed to evaluate HS.

Indexed as

ChildChild, PreschoolFemaleGrowth SubstancesHandHumansInsulin-Like Growth Factor IRecombinant ProteinsRett SyndromeStereotypic Movement DisorderVideo RecordingGrowth SubstancesInsulin-Like Growth Factor ImecaserminRecombinant Proteinsactigraphyhand stereotypiesoperational definitionsRett syndromevideo analysis

Identifiers

PMID28838622
PMCPMC5624791
OpenAlexW2621193640

What OpenQuestion holds

Textmetadata
LicenceTDM
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.