Evidence map›Paper›PMID 33085768›Full record

Trial reportNeuro-oncology2021

A phase II study of dose-dense temozolomide and lapatinib for recurrent low-grade and anaplastic supratentorial, infratentorial, and spinal cord ependymoma.

Mark R Gilbert, Ying Yuan, Jimin Wu, Tito Mendoza, Elizabeth Vera, Antonio Omuro, Frank Lieberman, H Ian Robins, Elizabeth R Gerstner, Jing Wu and 4 more

Open access · bronzeAbstract readClinical Trial, Phase II
In one paragraph

Trial report in Neuro-oncology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed, 1 pooled it
3.9field-weighted citation impact, top 6% 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

42 citing papers in PubMed, 1 synthesis or guideline pooled it, 59 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Article
  5. A brief history of ependymoma.Neuro-oncology · 2026
    Review
  6. Article
  7. Article
  8. Article
  9. Article
  10. Review
  11. Article
  12. Ependymoma: Advances in Systems Treatment Strategies.Current neurology and neuroscience reports · 2025
    Review
  13. Article
  14. Article
  15. Review
  16. Review
  17. Spinal ependymal tumors.Neuro-oncology advances · 2024
    Review
  18. Review
  19. Review
  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

14 authors at 8 institutions in 1 country.

Mark R GilbertCenter for Cancer Research, National Cancer Institute, Bethesda, Maryland.
Ying YuanThe University of Texas MD Anderson Cancer Center, Houston, Texas.
Jimin WuThe University of Texas MD Anderson Cancer Center, Houston, Texas.
Tito MendozaThe University of Texas MD Anderson Cancer Center, Houston, Texas.
Elizabeth VeraCenter for Cancer Research, National Cancer Institute, Bethesda, Maryland.
Antonio OmuroYale University, New Haven, Connecticut.
Frank LiebermanUniversity of Pittsburgh, Pittsburgh, Pennsylvania.
H Ian RobinsUniversity of Wisconsin-Madison, Madison, Wisconsin.
Elizabeth R GerstnerMassachusetts General Hospital, Boston, Massachusetts.
Jing WuCenter for Cancer Research, National Cancer Institute, Bethesda, Maryland.
Patrick Y WenDana Farber Cancer Institute, Boston, Massachusetts.
Tom MikkelsenHenry Ford Hospital, Detroit, Michigan.
Kenneth AldapeCenter for Cancer Research, National Cancer Institute, Bethesda, Maryland.
Terri S ArmstrongCenter for Cancer Research, National Cancer Institute, Bethesda, Maryland.
National Cancer Institute · USThe University of Texas MD Anderson Cancer Center · USDana-Farber Cancer Institute · USHenry Ford Hospital · USMassachusetts General Hospital · USUniversity of Pittsburgh · USUniversity of Wisconsin–Madison · USYale University · US

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
CONNECT: Comprehensive Oncology Network Evaluating Rare CNS Tumors - CuresZIABC011853 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI GILBERT, MARK · 2020 to 2024
$28.4M
Discovering novel therapies for glioma patientsZIABC011840 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI WU, JING · 2018 to 2025
$4.6M
NCATS NIH HHS UL1 TR001863
6 · The paper itself

Abstract

backgroundNo standard medical treatment exists for adult patients with recurrent ependymoma, and prospective clinical trials in this population have not succeeded because of its rarity and challenges in accruing patients. The Collaborative Ependymoma Research Network conducted a prospective phase II clinical trial of dose-dense temozolomide (TMZ) and lapatinib, targeting the unmethylated O6-methylguanine-DNA methyltransferase (MGMT) promoter status and increased expression of ErbB2 (human epidermal growth factor receptor 2) and ErbB1 (epidermal growth factor receptor) in ependymomas.

methodsPatients age 18 or older with histologically proven and progressive ependymoma or anaplastic ependymoma were eligible and received dose-dense TMZ and daily lapatinib. The primary outcome measure was median progression-free survival (PFS). Landmark 6- and 12-month PFS and objective response were measured. Serial assessments of symptom burden using the MD Anderson Symptom Inventory Brain Tumor (MDASI-BT)/MDASI-Spine Tumor modules were collected.

resultsThe 50 patients enrolled had a median age of 43.5 years, median Karnofsky performance status of 90, and a median of 2 prior relapses. Twenty patients had grade III, 16 grade II, and 8 grade I ependymoma. Half had spinal cord tumors; 15 had a supratentorial tumor, 8 infratentorial, and 2 had disseminated disease. Treatment was well tolerated. The median PFS was 7.8 months (95% CI: 5.5,12.2); the 6- and 12-month PFS rates were 55% and 38%, with 2 complete and 6 partial responses. Measures of symptom burden showed reduction in moderate-severe pain and other disease-related symptoms in most patients.

conclusionsThis treatment, with demonstrated clinical activity with objective responses and prolonged disease control associated with disease-related symptom improvements, is an option as a salvage regimen for adult patients with recurrent ependymoma.

Indexed as

Brain NeoplasmsEpendymomaSpinal Cord NeoplasmsAdolescentAdultDacarbazineDisease-Free SurvivalHumansLapatinibProspective StudiesTemozolomideDacarbazineLapatinibTemozolomidecombination chemotherapyependymomalapatinibprogression-free survivaltemozolomide

Identifiers

PMID33085768
PMCPMC7992893
OpenAlexW3094388587

What OpenQuestion holds

Textmetadata
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.