Evidence map›Paper›PMID 25567352›Full record

Trial reportJournal of neuro-oncology2015

Phase II study of mTORC1 inhibition by everolimus in neurofibromatosis type 2 patients with growing vestibular schwannomas.

Stéphane Goutagny, Eric Raymond, Marina Esposito-Farese, Stéphanie Trunet, Christian Mawrin, Daniele Bernardeschi, Béatrice Larroque, Olivier Sterkers, Marco Giovannini, Michel Kalamarides

2 registry-linked trialsAbstract readClinical Trial, Phase II
PubMed Publisher
In one paragraph

Trial report in Journal of neuro-oncology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 71 papers.

0numbers the graph read from it
0cells of the map it votes in
71citing 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.

NCT01345136 phase2terminatednot on this map

A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

TypeinterventionalSponsorJonsson Comprehensive Cancer CenterRan2015 to 2024Enrolled4ConditionsNeurofibromatosis Type 2, Neuroma, AcousticArmsRAD001, everolimus
NCT01490476 phase2completednot on this map

A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

TypeinterventionalSponsorAssistance Publique - Hôpitaux de ParisRan2012 to 2017Enrolled10ConditionsNeurofibromatosis 2ArmsRAD001
3 · Its place in the literature

Who cites it

71 citing papers in PubMed.

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  14. Update on Cancer and Central Nervous System Tumor Surveillance in Pediatric NF2-, SMARCB1-, and LZTR1-Related Schwannomatosis.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025
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11 more citing papers are in PubMed but not listed here.

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

10 authors.

Stéphane GoutagnyNeurochirurgie, Hôpital Beaujon, Assistance Publique Hôpitaux de Paris, 100 boulevard du General Leclerc, 92100, Clichy, France.
Eric Raymond
Marina Esposito-Farese
Stéphanie Trunet
Christian Mawrin
Daniele Bernardeschi
Béatrice Larroque
Olivier Sterkers
Marco Giovannini
Michel Kalamarides

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Neurofibromatosis type 2 (NF2) is a genetic disorder with bilateral vestibular schwannomas (VS) as the most frequent manifestation. Merlin, the NF2 tumor suppressor, was identified as a negative regulator of mammalian target of rapamycin complex 1. Pre-clinical data in mice showed that mTORC1 inhibition delayed growth of NF2-schwannomas. We conducted a prospective single-institution open-label phase II study to evaluate the effects of everolimus in ten NF2 patients with progressive VS. Drug activity was monitored every 3 months. Everolimus was administered orally for 12 months and, if the decrease in tumor volume was >20 % from baseline, treatment was continued for 12 additional months. Other patients stopped when completed 12 months of everolimus but were allowed to resume treatment when VS volume was >20 % during 1 year follow-up. Nine patients were evaluable. Safety was evaluated using CTCAE 3.0 criteria. After 12 months of everolimus, no reduction in volume ≥20 % was observed. Four patients had progressive disease, and five patients had stable disease with a median annual growth rate decreasing from 67 %/year before treatment to 0.5 %/year during treatment. In these patients, tumor growth resumed within 3-6 months after treatment discontinuation. Everolimus was then reintroduced and VS decreased by a median 6.8 % at 24 months. Time to tumor progression increased threefold from 4.2 months before treatment to > 12 months. Hearing was stable under treatment. The safety of everolimus was manageable. Although the primary endpoint was not reached, further studies are required to confirm the potential for stabilization of everolimus.

Indexed as

AdolescentAdultAntineoplastic AgentsBiomarkers, TumorCranial Nerve NeoplasmsDisease-Free SurvivalDisease ProgressionEverolimusFemaleFollow-Up StudiesHumansMaleMechanistic Target of Rapamycin Complex 1Multiprotein ComplexesNeurilemmomaNeurofibromatosis 2Antineoplastic AgentsBiomarkers, TumorEverolimusMechanistic Target of Rapamycin Complex 1Multiprotein ComplexesTOR Serine-Threonine Kinases

Identifiers

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.