Evidence map›Paper›PMID 32014856›Full record

ArticleCold Spring Harbor molecular case studies2020

Clinical report: one year of treatment of Proteus syndrome with miransertib (ARQ 092).

Leslie G Biesecker, Matthew Edwards, Sheridan O'Donnell, Paula Doherty, Thomas MacDougall, Kate Tith, Julia Kazakin, Brian Schwartz

Open access · diamondAbstract readCase Reports
In one paragraph

Article in Cold Spring Harbor molecular case studies, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing 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

22 citing papers in PubMed, 41 citations in OpenAlex.

  1. Trial
  2. Development and validation of an LC-MS/MS assay for the quantification of Miransertib in human plasma and clinical application.Journal of chromatography. B, Analytical technologies in the biomedical and life sciences · 2026
    Article
  3. Review
  4. Characterizing Paratesticular Neoplasms in Proteus Syndrome.The American journal of surgical pathology · 2026
    Article
  5. Review
  6. Review
  7. Article
  8. Human vascular organoids with a mosaicbioRxiv : the preprint server for biology · 2024
    Article
  9. Multimodal ocular imaging in Proteus syndrome.European journal of ophthalmology · 2023
    Article
  10. Article
  11. Review
  12. Article
  13. Article
  14. Article
  15. Review
  16. Article
  17. Article
  18. Article
  19. Article
  20. Review
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 3 institutions in 2 countries.

Leslie G BieseckerMedical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892, USA.
Matthew EdwardsHunter Genetics, Hunter-New England Local Health District, New South Wales Department of Health, Waratah, New South Wales 2298, Australia.ORCID 0000-0001-8437-8120
Sheridan O'DonnellHunter Genetics, Hunter-New England Local Health District, New South Wales Department of Health, Waratah, New South Wales 2298, Australia.
Paula DohertyPharmacy, John Hunter Hospital, Hunter-New England Local Health District, New South Wales Department of Health, New South Wales 2310, Australia.
Thomas MacDougallMedical Imaging, Paediatric Radiology, Hunter-New England Local Health District, New South Wales Department of Health, New South Wales 2310, Australia.
Kate TithArqule Inc, Burlington, Massachusetts 01803, USA.
Julia KazakinArqule Inc, Burlington, Massachusetts 01803, USA.
Brian SchwartzArqule Inc, Burlington, Massachusetts 01803, USA.ORCID 0000-0003-2632-6351
New South Wales Department of Health · AUArqule (United States) · USNational Institutes of Health · US

Funding

Rare & Mosaic Disorders - Clinical ResearchZIAHG200388 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI BIESECKER, LESLIE · 2013 to 2025
$17.3M
6 · The paper itself

Abstract

A 20-yr-old man with Proteus syndrome (PS) and somatic mosaicism of the

Indexed as

AllelesAminopyridinesDuration of TherapyHumansImage Processing, Computer-AssistedImidazolesMagnetic Resonance ImagingMaleMutationPhenotypePolymorphism, Single NucleotideProtein Kinase InhibitorsProteus SyndromeProto-Oncogene Proteins c-aktYoung AdultAminopyridinesImidazolesMiransertibProtein Kinase InhibitorsProto-Oncogene Proteins c-aktabnormal plantar dermatoglyphicsabnormal subcutaneous fat tissue distributionattention deficit hyperactivity disorderhypercoagulabilityhypertrophy of skin of solesintellectual disabilitymacrodactyly of fingermacrodactyly of toemoderateneoplasia of the male external genitaliaovarian neoplasmovergrowthpostnatal macrocephalyprominent veins on trunkproportionate tall staturetesticular neoplasmvenous malformation

Identifiers

PMID32014856
PMCPMC6996520
OpenAlexW3004718237

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.