Evidence map›Paper›PMID 34499753›Full record

ArticleJournal of inherited metabolic disease2021

Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy.

Ashish O Gupta, David R Nascene, Ryan Shanley, Daniel L Kenney-Jung, Julie B Eisengart, Troy C Lund, Paul J Orchard, Elizabeth I Pierpont

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of inherited metabolic disease, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
0.9field-weighted citation impact, top 27% 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

6 citing papers in PubMed, 13 citations in OpenAlex.

  1. Article
  2. Observational
  3. Article
  4. Article
  5. Review
  6. 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

8 authors at 1 institution in 1 country.

Ashish O GuptaDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
David R NasceneDepartment of Radiology, University of Minnesota, Minneapolis, Minnesota, USA.
Ryan ShanleyBioinformatics and Biostatistics Core, University of Minnesota, Minneapolis, Minnesota, USA.
Daniel L Kenney-JungDepartment of Neurology, University of Minnesota, Minneapolis, Minnesota, USA.
Julie B EisengartDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
Troy C LundDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
Paul J OrchardDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
Elizabeth I PierpontDepartment of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.ORCID 0000-0001-7555-8613
University of Minnesota · US

Funding

University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UL1TR002494 · NCATS · UNIVERSITY OF MINNESOTA · PI BLAZAR, BRUCE R, WEISDORF, DANIEL J · 2018 to 2022
$34.9M
Institutional Career Development CoreKL2TR002492 · NCATS · UNIVERSITY OF MINNESOTA · PI INGBAR, DAVID H · 2018 to 2022
$6.3M
NCATS NIH HHS KL2 TR002492NCATS NIH HHS UL1 TR002494
6 · The paper itself

Abstract

In the most common variant of childhood cerebral adrenoleukodystrophy (cALD), demyelinating brain lesions are distributed predominately in parieto-occipital white matter. Less frequently, lesions first develop in frontal white matter. This matched cohort study examined whether outcomes after standard treatment with hematopoietic cell transplantation (HCT) differ in patients with early stage frontal lesions as compared to parieto-occipital lesions. Retrospective chart review identified seven pediatric patients with frontal cALD lesions and MRI severity score < 10 who underwent a single HCT at our center between 1990 and 2019. Concurrent MRI, neurocognitive and psychiatric outcomes at last comprehensive follow-up (mean 1.2 years; range 0.5-2.1 years) were compared with a group of seven boys with the parieto-occipital variant matched on pre-HCT MRI severity score. Both groups showed similar rates of transplant complications and radiographic disease advancement. Neurocognitive outcomes were broadly similar, with more frequent working memory deficits among individuals with frontal lesions. Psychiatric problems (hyperactivity, aggression, and atypical behavior) were considerably more common and severe among patients with frontal lesions. Aligned with the critical role of the frontal lobes in emotional and behavioral regulation, functional disruption of self-regulation skills is widely observed among patients with frontal lesions. Comprehensive care for cALD should address needs for psychiatric care and management.

Indexed as

Hematopoietic Stem Cell TransplantationAdolescentAdrenoleukodystrophyChildChild, PreschoolDemyelinating DiseasesEmotionsFrontal LobeHumansMagnetic Resonance ImagingMaleMental DisordersNeuropsychological TestsRetrospective StudiesSeverity of Illness IndexTreatment OutcomeadrenoleukodystrophycerebralcognitionMRIpsychiatricstem cell transplantation

Identifiers

PMID34499753
PMCPMC8578392
OpenAlexW3197793335

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.