Evidence map›Paper›PMID 37877369›Full record

ArticleClinical trials (London, England)2024

Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.

Liny John, Gurbani Singh, Eva Dombi, Pamela L Wolters, Staci Martin, Andrea Baldwin, Seth M Steinberg, Jessica Bernstein, Patricia Whitcomb, Dominique C Pichard and 9 more

Registry-linked trialOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Clinical trials (London, England), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01362803 (A Phase I/II Study of the Mitogen Activated Protein Kinase), which is 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 70% 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.

NCT01362803 phase1 / phase2active not recruitingnot on this map

A Phase I/II Study of the Mitogen Activated Protein Kinase (MEK) 1 Inhibitor Selumetinib (AZD6244; Hydrogen Sulfate in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

TypeinterventionalSponsorNational Cancer Institute (NCI)Ran2011 to 2032Enrolled99ConditionsNeurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, PlexiformArmsAZD6244
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

19 authors at 5 institutions in 1 country.

Liny JohnPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0009-0003-6331-0348
Gurbani SinghPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Eva DombiPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Pamela L WoltersPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0001-9319-6281
Staci MartinPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Andrea BaldwinClinical Research Directorate (CRD), Frederick National Laboratory for Cancer Research, Frederick, MD, USA.
Seth M SteinbergBiostatistics and Data Management Section, Office of the Clinical Director, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Jessica BernsteinPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Patricia WhitcombPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Dominique C PichardPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Anne DufekPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Andy GillespiePediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Kara HeiseyClinical Research Directorate (CRD), Frederick National Laboratory for Cancer Research, Frederick, MD, USA.
Miriam BornhorstCenter for Cancer and Blood Disorders, Children's National Hospital, Washington, DC, USA.
Michael J FisherDivision of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID 0000-0003-3408-3839
Brian D WeissCancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
AeRang KimCenter for Cancer and Blood Disorders, Children's National Hospital, Washington, DC, USA.
Brigitte C WidemannPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Andrea M GrossPediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0003-1646-4531
National Institutes of Health · USCenter for Cancer and Blood Disorders · USFrederick National Laboratory for Cancer Research · USChildren's Hospital of Philadelphia · USCincinnati Children's Hospital Medical Center · US

Funding

Therapies for patients with rare tumors and genetic tumor predispositionZIASC010354 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI WIDEMANN, BRIGITTE · 2009 to 2025
$19.2M
Office of Collaborative BiostatisticsZIDSC007202 · NCI · DIVISION OF CLINICAL SCIENCES - NCI · PI CHOO-WOSOBA, HYOYOUNG · 2009 to 2025
$14.5M
Intramural NIH HHS Z99 CA999999
6 · The paper itself

Abstract

BACKGROUND/

aimsWe developed an observer disfigurement severity scale for neurofibroma-related plexiform neurofibromas to assess change in plexiform neurofibroma-related disfigurement and evaluated its feasibility, reliability, and validity.

methodsTwenty-eight raters, divided into four cohorts based on neurofibromatosis type 1 familiarity and clinical experience, were shown photographs of children in a clinical trial (NCT01362803) at baseline and 1 year on selumetinib treatment for plexiform neurofibromas (

resultsMean baseline disfigurement severity score for plexiform neurofibroma ratings were similar for the selumetinib group (6.23) and controls (6.38). Mean paired differences between pre- and on-treatment ratings was -1.01 (less disfigurement) in the selumetinib group and 0.09 in the control (

conclusionThis study demonstrates that our observer-rated disfigurement severity score for plexiform neurofibroma was feasible, reliable, and documented improvement in disfigurement in participants with plexiform neurofibroma shrinkage. Prospective studies in larger samples are needed to validate this scale further.

Indexed as

Neurofibroma, PlexiformNeurofibromatosis 1ChildHumansProspective StudiesReproducibility of ResultsDisfigurementneurofibromatosis type 1plexiform neurofibroma

Identifiers

PMID37877369
PMCPMC11003851
OpenAlexW4387931828

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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.