Evidence map›Paper›PMID 39440617›Full record

ReviewAnnals of clinical and translational neurology2024

Clinical trials for Lennox-Gastaut syndrome: Challenges and priorities.

Juliet K Knowles, Aaron E L Warren, Ismail S Mohamed, Carl E Stafstrom, Hyun Yong Koh, Debopam Samanta, Renée A Shellhaas, Gita Gupta, Tracy Dixon-Salazar, Linh Tran and 4 more

Abstract readReview
In one paragraph

Review in Annals of clinical and translational neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
–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

9 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Sleep in Lennox-Gastaut Syndrome: A Scoping Review.Children (Basel, Switzerland) · 2025
    Review
  5. Article
  6. Article
  7. Review
  8. Review
  9. 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

14 authors.

Juliet K KnowlesDepartment of Neurology, Stanford University School of Medicine, Stanford, California, USA.ORCID 0000-0002-9214-2991
Aaron E L WarrenDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Ismail S MohamedDepartment of Pediatrics, University of Alabama, Birmingham, Alabama, USA.
Carl E StafstromDepartment of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Hyun Yong KohDepartment of Pediatrics, Section of Neurology and Developmental Neuroscience, Baylor College of Medicine, Houston, Texas, USA.ORCID 0000-0002-3995-4341
Debopam SamantaDepartment of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.ORCID 0000-0002-5154-8717
Renée A ShellhaasDepartment of Neurology, Washington University in St. Louis, St. Louis, Missouri, USA.
Gita GuptaDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Tracy Dixon-SalazarLennox-Gastaut Syndrome Foundation, San Diego, California, USA.
Linh TranJane and John Justin Institute for Mind Health, Cook Children's Medical Center, Fort Worth, Texas, USA.
Sonal BhatiaDivision of Pediatric Neurology, Medical University of South Carolina, Charleston, South Carolina, USA.
Jane M McCabePediatric Epilepsy Research Consortium, Niwot, Colorado, USA.
Anup D Patel *Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.
Zachary M Grinspan *Department of Pediatrics, Weill Cornell Medicine, New York, New York, USA.

Funding

CTSA K12 Program at the University of MichiganK12TR004374 · NCATS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI VICKI L ELLINGROD, Leah Elizabeth Robinson · 2023 to 2026
$6.5M
NS-PEACE Neonatal Seizures -Predicting Epilepsy and Assessing Comparative Effectiveness.R01NS130113 · NINDS · WEILL MEDICAL COLL OF CORNELL UNIV · PI GRINSPAN, ZACHARY MICHAEL · 2023 to 2025
$2.1M
Maladaptive Myelination in Pediatric EpilepsyK08NS119800 · NINDS · STANFORD UNIVERSITY · PI KNOWLES, JULIET KLASING · 2021 to 2025
$1.1M
American Epilepsy SocietyAmgenBAND foundationCitizens United for Research in EpilepsyClara InspiredDoris Duke Charitable FoundationJazz PharmaceuticalsLennox-Gastaut Syndrome FoundationMarinus PharmaceuticalsMcKnight FoundationMichigan Institute for Clinical and Health Research K12TR004374Morris and Alma Schapiro FoundationNanette Laitman Clinical Scholars ProgramNCATS NIH HHS K12 TR004374NIH HHS K08NS119800NIH HHS R01NS130113NINDS NIH HHS K08 NS119800NINDS NIH HHS R01 NS130113RTW Charitable FoundationSLC6A1 ConnectStanford Maternal and Child Health Research InstituteStanford Wu Tsai Neurosciences InstituteSTXBP1 FoundationSYNGAP Research Fund
6 · The paper itself

Abstract

objectiveLennox-Gastaut syndrome (LGS) is a severe, childhood-onset epilepsy that is typically refractory to treatment. We surveyed the current landscape of LGS treatment, aiming to identify challenges to the development of efficacious therapies, and to articulate corresponding priorities toward clinical trials that improve outcomes.

methodsThe LGS Special Interest Group of the Pediatric Epilepsy Research Consortium integrated evidence from the literature and expert opinion, into a narrative review.

resultsWe provide an overview of approved and emerging medical, dietary, surgical and neuromodulation approaches for LGS. We note that quality of care could be improved by standardizing LGS treatment based on expert consensus and empirical data. Whereas LGS natural history is incompletely understood, prospective studies and use of large retrospective datasets to understand LGS across the lifespan would enable clinical trials that address these dynamics. Recent discoveries related to LGS pathophysiology should enable development of disease-modifying therapies, which are currently lacking. Finally, clinical trials have focused chiefly on seizures involving "drops," but should incorporate additional patient-centered outcomes, using emerging measures adapted to people with LGS.

interpretationClinicians and researchers should enact these priorities, with the goal of patient-centered clinical trials that are tailored to LGS pathophysiology and natural history.

Indexed as

Clinical Trials as TopicLennox Gastaut SyndromeHumans

Identifiers

PMID39440617
PMCPMC11572735

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.