Evidence map›Paper›PMID 25524464›Full record

ArticleJournal of translational medicine2014

Response of recurrent BRAFV600E mutated ganglioglioma to Vemurafenib as single agent.

Francesca del Bufalo, Andrea Carai, Lorenzo Figà-Talamanca, Benedetta Pettorini, Conor Mallucci, Felice Giangaspero, Manila Antonelli, Manuela Badiali, Loredana Moi, Giuseppe Bianco and 4 more

Open access · goldAbstract readCase Reports
In one paragraph

Article in Journal of translational medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.

0numbers the graph read from it
0cells of the map it votes in
46citing papers in PubMed
4.6field-weighted citation impact, top 4% 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.

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

46 citing papers in PubMed, 91 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Efficacy and Safety of Trametinib Monotherapy or in Combination With Dabrafenib in PediatricJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023
    Trial
  4. Trial
  5. Ganglioglioma and gangliocytoma: a review for pathologists.Journal of pathology and translational medicine · 2026
    Article
  6. Significant clinical and radiologic response to targeted therapy in pediatric cervicomedullary low-grade gliomas harboring the BRAFV600E mutation.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025
    Article
  7. Article
  8. How modern treatments have modified the role of surgery in pediatric low-grade glioma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2024
    Review
  9. Chemotherapy in pediatric low-grade gliomas (PLGG).Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2024
    Review
  10. Updates on management of gliomas in the molecular age.World journal of clinical oncology · 2024
    Review
  11. A rare tumor in the sellar region: ganglioglioma, a case report and a general overview.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2023
    Review
  12. Review
  13. Review
  14. Article
  15. Review
  16. Medical Treatment of Pediatric Low-Grade Glioma.Brain tumor research and treatment · 2022
    Review
  17. Article
  18. Review
  19. Treatment ofSurgical neurology international · 2021
    Article
  20. 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 at 6 institutions in 2 countries.

Francesca del BufaloDepartment of Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant'Onofrio 4, 00165, Rome, Italy. fradelbufalo@yahoo.it.
Andrea CaraiDepartment of Neuroscience and Neurorehabilitation, Neurosurgery Unit, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant' Onofrio 4, 00165, Rome, Italy. andrea.carai@opbg.net.
Lorenzo Figà-TalamancaDepartment of Radiology, Unit of Neuroradiology, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant' Onofrio 4, 00165, Rome, Italy. lorenzo.figatalamanca@opbg.net.
Benedetta PettoriniPaediatric Neurosurgery Department, Alder Hey Children's NHS Foundation Trust, Liverpool, UK. Benedetta.Pettorini@alderhey.nhs.uk.
Conor MallucciPaediatric Neurosurgery Department, Alder Hey Children's NHS Foundation Trust, Liverpool, UK. Conor.Mallucci@alderhey.nhs.uk.
Felice GiangasperoDepartment of Radiological, Oncological and Pathological Science, Sapienza University, Viale Regina Elena 291, 00161, Rome, Italy. felice.giangaspero@uniroma1.it.
Manila AntonelliDepartment of Radiological, Oncological and Pathological Science, Sapienza University, Viale Regina Elena 291, 00161, Rome, Italy. manila_antonelli@yahoo.it.
Manuela BadialiBone Marrow Transplantation Unit, Microcitemico Children's Hospital, Via Jenner s/n 09121, Cagliari, Italy. manuelabadiali@asl8cagliari.it.
Loredana MoiPublic Health, Clinic and Molecular Medicine Department, Microcitemico Children's Hospital, Via Jenner s/n 09121, Cagliari, Italy. lorymoi@hotmail.it.
Giuseppe BiancoPharmacy Unit, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant'Onofrio 4, 00165, Rome, Italy. giuseppe2.bianco@opbg.net.
Antonella CacchioneDepartment of Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant'Onofrio 4, 00165, Rome, Italy. antonella.cacchione@opbg.net.
Franco LocatelliDepartment of Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant'Onofrio 4, 00165, Rome, Italy. franco.locatelli@opbg.net.
Elisabetta FerrettiDepartment of Experimental Medicine, Sapienza University, Viale Regina Elena 291, 00161, Rome, Italy. elisabetta.ferretti@uniroma1.it.
Angela MastronuzziDepartment of Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, IRCCS, Piazza Sant'Onofrio 4, 00165, Rome, Italy. angela.mastronuzzi@opbg.net.
Bambino Gesù Children's Hospital · ITAlder Hey Children's NHS Foundation Trust · GBOspedale Microcitemico · ITSapienza University of Rome · ITIstituto Neurologico Mediterraneo · ITUniversity of Pavia · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundGanglioglioma (GG) and pilocytic astrocytoma (PA) represent the most frequent low-grade gliomas (LGG) occurring in paediatric age. LGGs not amenable of complete resection (CR) represent a challenging subgroup where traditional treatments often fail. Activation of the MAP Kinase (MAPK) pathway caused by the BRAFV600E mutation or the KIAA1549-BRAF fusion has been reported in pediatric GG and PA, respectively. CASE PRESENTATION: We report on a case of BRAFV600E mutated cervicomedullary GG treated with standard chemotherapy and surgery. After multiple relapse, BRAF status was analyzed by immunohistochemistry and sequencing showing a BRAFV600E mutation. Treatment with Vemurafenib as single agent was started. For the first time, a radiological and clinical response was obtained after 3 months of treatment and sustained after 6 months.

conclusionOur experience underline the importance of understanding the driver molecular alterations of LGG and suggests a role for Vemurafenib in the treatment of pediatric GG not amenable of complete surgical resection.

Indexed as

MutationAntineoplastic AgentsBrain NeoplasmsChild, PreschoolGangliogliomaHumansIndolesMagnetic Resonance ImagingMaleProto-Oncogene Proteins B-rafSulfonamidesVemurafenibAntineoplastic AgentsBRAF protein, humanIndolesProto-Oncogene Proteins B-rafSulfonamidesVemurafenib

Identifiers

PMID25524464
PMCPMC4279809
OpenAlexW2132742850

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

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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.