Evidence map›Paper›PMID 36461157›Full record

ArticleAmerican journal of medical genetics. Part A2023

Anesthesia outcomes in lysosomal disorders: CLN3 and GM1 gangliosidosis.

Amelia Luckett, Muhammad Yousef, Cynthia Tifft, Kisha Jenkins, Andrew Smith, Andrea Munoz, Rachel Quimby, Forbes D Porter, An Ngoc Dang Do

2 registry-linked trialsOpen access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in American journal of medical genetics. Part A, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact, top 79% 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.

NCT00029965 recruitingnot on this map

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

TypeobservationalSponsorNational Human Genome Research Institute (NHGRI)Ran2002Enrolled200ConditionsNeurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
NCT03307304 recruitingnot on this map

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) and CLN3-Related Conditions

TypeobservationalSponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Ran2017 to 2050Enrolled300ConditionsJuvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
3 · Its place in the literature

Who cites it

0 citing papers in PubMed, 0 citations in OpenAlex.

No citing paper in PubMed yet.

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.

Amelia LuckettDepartment of Anesthesia and Surgical Services, NIH Clinical Center, National Institutes of Health, Bethesda, Maryland, USA.
Muhammad YousefDepartment of Anesthesia and Surgical Services, NIH Clinical Center, National Institutes of Health, Bethesda, Maryland, USA.
Cynthia TifftNational Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
Kisha JenkinsEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Andrew SmithEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Andrea MunozEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Rachel QuimbyNational Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
Forbes D PorterEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
An Ngoc Dang DoEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.ORCID 0000-0002-4171-0493
National Institutes of Health · USNational Institutes of Health Clinical Center · US

Funding

Clinical Investigations of Inborn Errors of MetabolismZIAHD008989 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI PORTER, FORBES · 2020 to 2025
$5.1M
Intramural NIH HHS ZIA HD008989
6 · The paper itself

Abstract

Natural history studies of pediatric rare neurometabolic diseases are important to understand disease pathophysiology and to inform clinical trial outcome measures. Some data collections require sedation given participants' age and neurocognitive impairment. To evaluate the safety of sedation for research procedures, we reviewed medical records between April 2017 and October 2019 from a natural history study for CLN3 (NCT03307304) and one for GM1 gangliosidosis (NCT00029965). Twenty-two CLN3 individuals underwent 28 anesthetic events (age median 11.0, IQR 8.4-15.3 years). Fifteen GM1 individuals had 19 anesthetic events (9.8, 7.1-14.7). All participants had the American Society of Anesthesiology classification of II (8/47) or III (39/47). Mean sedation durations were 186 (SD = 54; CLN3) and 291 (SD = 33; GM1) min. Individuals with GM1 (6/19, 31%) were more frequently prospectively intubated for sedation (CLN3 3/28, 11%). Minor adverse events associated with sedation occurred in 8/28 (28%, CLN3) and 6/19 (32%, GM1) individuals, frequencies within previously reported ranges. No major adverse clinical outcomes occurred in 47 anesthetic events in pediatric participants with either CLN3 or GM1 gangliosidosis undergoing research procedures. Sedation of pediatric individuals with rare neurometabolic diseases for research procedures is safe and allows for the collection of data integral to furthering their understanding and treatment.

Indexed as

AnesthesiaAnesthesiologyGangliosidosis, GM1Adolescentbeta-GalactosidaseChildG(M1) GangliosideHumansLysosomesMembrane GlycoproteinsMolecular ChaperonesRare DiseasesRetrospective Studiesbeta-GalactosidaseCLN3 protein, humanG(M1) GangliosideMembrane GlycoproteinsMolecular Chaperonesbattenemergence agitationinstitutional review boardsperi-anesthesia

Identifiers

PMID36461157
PMCPMC9928896
OpenAlexW4310645469

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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.