Evidence map›Paper›PMID 38358393›Full record

ArticleClinical cancer research : an official journal of the American Association for Cancer Research2024

Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium.

Jason Fangusaro, Robert A Avery, Michael J Fisher, Roger J Packer, Karin S Walsh, Antoinette Schouten-van Meeteren, Dominik Karres, Diana Bradford, Vishal Bhatnagar, Harpreet Singh and 3 more

Abstract read
In one paragraph

Article in Clinical cancer research : an official journal of the American Association for Cancer Research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing 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

8 citing papers in PubMed.

  1. Trial
  2. Review
  3. RAS/MAPK inhibition in pediatric gliomas.Neuro-oncology advances · 2026
    Review
  4. Review
  5. Review
  6. Article
  7. Molecular-targeted therapy for childhood low-grade glial and glioneuronal tumors.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2024
    Review
  8. 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

13 authors.

Jason FangusaroChildren's Hospital of Atlanta, Emory University and the Aflac Cancer Center, Atlanta, Georgia.ORCID 0000-0003-3099-5259
Robert A AveryThe Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.ORCID 0000-0003-1453-7282
Michael J FisherThe Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.ORCID 0000-0003-3408-3839
Roger J PackerCenter for Neuroscience and Behavioral Medicine and Brain Tumor Institute, Children's National Hospital, and The George Washington University School of Medicine, Washington, DC.ORCID 0000-0001-9413-7531
Karin S WalshCenter for Neuroscience and Behavioral Medicine and Brain Tumor Institute, Children's National Hospital, and The George Washington University School of Medicine, Washington, DC.ORCID 0000-0001-9637-2164
Antoinette Schouten-van MeeterenPrincess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.ORCID 0000-0002-1089-8091
Dominik KarresPediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, the Netherlands.ORCID 0009-0008-6773-622X
Diana BradfordCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-5208-8222
Vishal BhatnagarCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-4650-0513
Harpreet SinghCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-1526-0916
Paul G KluetzCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-9796-4791
Martha DonoghueCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-4493-6399
Elizabeth S DukeCenter for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-3361-1436

Funding

Biomarkers of Vision Loss in Children with Optic Pathway GliomasR01EY029687 · NEI · CHILDREN'S HOSP OF PHILADELPHIA · PI AVERY, ROBERT ANDREW · 2019 to 2023
$3.3M
Intramural FDA HHS FD999999NEI NIH HHS R01 EY029687U.S. Food and Drug Administration (FDA)
6 · The paper itself

Abstract

In October 2022, the FDA Oncology Center of Excellence hosted an educational symposium entitled, "Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma (pLGG) Clinical Trials." The symposium brought together patient advocates, regulators from the FDA and the European Medicines Agency (EMA), and an international group of academic thought leaders in the field of pediatric neuro-oncology to discuss the potential role of functional outcomes, including visual acuity, motor function, and neurocognitive performance, as endpoints in clinical trials enrolling patients with pLGG. The panel discussed challenges and opportunities regarding the selection, implementation, and evaluation of clinical outcome assessments in these functional domains and outlined key considerations for their inclusion in future clinical trial design and role in new drug development.

Indexed as

Brain NeoplasmsClinical Trials as TopicGliomaUnited States Food and Drug AdministrationChildHumansNeoplasm GradingOutcome Assessment, Health CareTreatment OutcomeUnited States

Identifiers

PMID38358393
PMCPMC11147731

What Socratic 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 Socratic graph.