Evidence map›Paper›PMID 19430883›Full record

ReviewJournal of neuro-oncology2009

Suggested response criteria for phase II antitumor drug studies for neurofibromatosis type 2 related vestibular schwannoma.

Scott R Plotkin, Chris Halpin, Jaishri O Blakeley, William H Slattery, D Bradley Welling, Susan M Chang, Jay S Loeffler, Gordon J Harris, A Gregory Sorensen, Michael J McKenna and 1 more

Open access · greenAbstract readReview
In one paragraph

Review in Journal of neuro-oncology, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 2 pooled it
2.7field-weighted citation impact, top 10% 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

31 citing papers in PubMed, 2 syntheses or guidelines pooled it, 50 citations in OpenAlex.

  1. The application of radiomics in vestibular schwannomas.The Journal of laryngology and otology · 2025
    Pooled it
  2. Pooled it
  3. Trial
  4. Trial
  5. Efficacy and Biomarker Study of Bevacizumab for Hearing Loss Resulting From Neurofibromatosis Type 2-Associated Vestibular Schwannomas.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2016
    Trial
  6. Trial
  7. Trial
  8. Development and Initial Validation of the Quality of life Evaluation inmedRxiv : the preprint server for health sciences · 2026
    Article
  9. [BMJ open · 2026
    Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Article
  17. Article
  18. Reduced dosage of bevacizumab in treatment of vestibular schwannomas in patients with neurofibromatosis type 2.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2015
    Article
  19. The effect of bevacizumab on vestibular schwannoma tumour size and hearing in patients with neurofibromatosis type 2.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2015
    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

11 authors at 7 institutions in 1 country.

Scott R PlotkinDepartment of Neurology and Cancer Center, Massachusetts General Hospital, Boston, MA 02114, USA.
Chris Halpin
Jaishri O Blakeley
William H Slattery
D Bradley Welling
Susan M Chang
Jay S Loeffler
Gordon J Harris
A Gregory Sorensen
Michael J McKenna
Fred G Barker
Massachusetts General Hospital · USMassachusetts Eye and Ear Infirmary · USCenter for Neuro-Oncology · USHouse Clinic · USJohns Hopkins University · USThe Ohio State University · USUniversity of California, San Francisco · US

Funding

Translational Research Central ServicesP30CA006973 · NCI · JOHNS HOPKINS UNIVERSITY · PI Rosalie Wollett · 1985 to 2026
$208.6M
NCI NIH HHS P30 CA006973
6 · The paper itself

Abstract

Neurofibromatosis type 2 (NF2) is a tumor suppressor gene syndrome characterized by multiple schwannomas, especially vestibular schwannomas (VS), and meningiomas. Anticancer drug trials are now being explored, but there are no standardized endpoints in NF2. We review the challenges of NF2 clinical trials and suggest possible response criteria for use in initial phase II studies. We suggest two main response criteria in such trials. Objective radiographic response is defined as a durable 20% or greater reduction in VS volume based on post-contrast T1-weighted MRI images collected with 3 mm or finer cuts through the internal auditory canal. Hearing response is defined as a statistically significant improvement in word recognition scores using 50-word recorded lists in audiology. A possible composite endpoint incorporating both radiographic response and hearing response is outlined. We emphasize pitfalls in response assessment and suggest guidelines to minimize misinterpretations of response. We also identify research goals in NF2 to facilitate future trial conduct, such as identifying the expectations for time to tumor progression and time to measurable hearing loss in untreated NF2-related VS, and the relation of both endpoints to patient prognostic factors (such as age, baseline tumor volume, and measures of disease severity). These data would facilitate future use of endpoints based on stability of tumor size and hearing, which might be more appropriate for testing certain drugs. We encourage adoption of standardized endpoints early in the development of phase II trials for this population to facilitate comparison of results across trials of different agents.

Indexed as

Antineoplastic AgentsClinical Trials, Phase II as TopicEar, InnerHearing LossHumansNeurofibromatosis 2Neuroma, AcousticResearch DesignAntineoplastic Agents

Identifiers

PMID19430883
PMCPMC4036446
OpenAlexW1990309105

What OpenQuestion 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 OpenQuestion graph.