Evidence map›Paper›PMID 33140540›Full record

Trial reportPediatric blood & cancer2021

A POETIC Phase II study of continuous oral everolimus in recurrent, radiographically progressive pediatric low-grade glioma.

Karen D Wright, Xiaopan Yao, Wendy B London, Pei-Chi Kao, Lia Gore, Stephen Hunger, Russ Geyer, Kenneth J Cohen, Jeffrey C Allen, Howard M Katzenstein and 19 more

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

Trial report in Pediatric blood & cancer, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed
2.2field-weighted citation impact, top 11% 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

29 citing papers in PubMed, 39 citations in OpenAlex.

  1. Everolimus for Children With Recurrent or Progressive Low-Grade Glioma: Results From the Phase II PNOC001 Trial.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024
    Trial
  2. Article
  3. Review
  4. Critical role of cell competition in gliomagenesis.bioRxiv : the preprint server for biology · 2026
    Article
  5. Article
  6. Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Review
  12. Review
  13. Advances in the Treatment of Pediatric Low-Grade Gliomas.Current neurology and neuroscience reports · 2024
    Review
  14. Molecular-targeted therapy for childhood low-grade glial and glioneuronal tumors.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2024
    Review
  15. 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
  16. Review
  17. Review
  18. Review
  19. Article
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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

29 authors at 13 institutions in 1 country.

Karen D WrightDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.ORCID 0000-0002-9578-1545
Xiaopan YaoDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Wendy B LondonDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.ORCID 0000-0003-3571-6538
Pei-Chi KaoDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Lia GoreChildren's Hospital Colorado, 13123 E 16th Avenue, Aurora, Colorado, 80045, USA.
Stephen HungerChildren's Hospital Colorado, 13123 E 16th Avenue, Aurora, Colorado, 80045, USA.
Russ GeyerSeattle Children's Hospital, 4800 Sand Point Way NE, Seattle, Washington, 98105, USA.
Kenneth J CohenThe Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, 401 N Broadway, Baltimore, Maryland, 21231, USA.
Jeffrey C AllenNew York University Medical Center, 550 1st Avenue, New York, New York, 10016, USA.
Howard M KatzensteinChildren's Healthcare of Atlanta, 1365 Clifton Road NE, Atlanta, Georgia, 30322, USA.ORCID 0000-0002-5314-2972
Amy SmithUniversity of Florida Health Shands Cancer Hospital, 1515 SW Archer Road, Gainesville, Florida, 32608, USA.
Jessica BoklanPhoenix Children's Hospital, 1919 E Thomas Rd, Phoenix, Arizona, 85016, USA.
Kellie NazemiOHSU Doernbecher Children's Hospital, 700 SW Campus Drive, Portland, Oregon, 97239, USA.
Tanya TrippettMemorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York, 10065, USA.
Matthias KarajannisMemorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York, 10065, USA.ORCID 0000-0002-7151-6528
Cynthia HerzogMD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, Texas, 77030, USA.
Joseph DestefanoMemorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York, 10065, USA.
Jennifer DirenzoMemorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York, 10065, USA.
Jay PietrantonioDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Lianne GreenspanDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Danielle CassidyChildren's Hospital Colorado, 13123 E 16th Avenue, Aurora, Colorado, 80045, USA.
Debra SchisselChildren's Hospital Colorado, 13123 E 16th Avenue, Aurora, Colorado, 80045, USA.
John PerentesisCincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, Ohio, 45229, USA.
Mitali BasuCincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, Ohio, 45229, USA.
Tomoyuki MizunoCincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, Ohio, 45229, USA.ORCID 0000-0002-8471-9826
Alexander A VinksCincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, Ohio, 45229, USA.
Sanjay P PrabhuBoston Children's Hospital, 300 Longwood Avenue, Boston, Massachusetts, 02115, USA.ORCID 0000-0003-0871-115X
Susan N ChiDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Mark W KieranDana-Farber/Boston Children's Cancer and Blood Disorders Center, 450 Brookline Avenue, Boston, Massachusetts, 02215, USA.
Dana-Farber/Boston Children's Cancer and Blood Disorders Center · USChildren's Hospital Colorado · USCincinnati Children's Hospital Medical Center · USMemorial Sloan Kettering Cancer Center · USBoston Children's Hospital · USChildren's Healthcare of Atlanta · USColumbia University Irving Medical Center · USDoernbecher Children's Hospital · USPhoenix Children's Hospital · USSeattle Children's Hospital · USSidney Kimmel Comprehensive Cancer Center · USThe University of Texas MD Anderson Cancer Center · USUF Health Shands Hospital · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Targeting the Vasular SystemP50CA165962 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI WENDY Beth LONDON · 2013 to 2026
$33.5M
NCI NIH HHS P30 CA008748NCI NIH HHS P50 CA165962
6 · The paper itself

Abstract

backgroundTo evaluate efficacy, pharmacokinetics (PK) and pharmacodynamics of single-agent everolimus in pediatric patients with radiographically progressive low-grade glioma (LGG).

methodsEverolimus was administered at 5 mg/m

resultsTwenty-three eligible patients (median age 9.2 years) were enrolled. All patients received prior chemotherapy (median number of prior regimens two) and/or radiotherapy (two patients). By week 48, two patients had a partial response, 10 stable disease, and 11 clinical or radiographic progression; two discontinued study prior to 1 year (toxicity: 1, physician determination: 1). With a median follow up of 1.8 years (range 0.2-6.7 years), the 2-, 3-, and 5-year progression-free survivals (PFS) were 39 ± 11%, 26 ± 11%, and 26 ± 11%, respectively; two patients died of disease. The 2-, 3-, and 5-year overall survival (OS) were all 93 ± 6%. Grade 1 and 2 toxicities predominated; two definitively related grade 3 toxicities (mucositis and neutropenia) occurred. Grade 4 elevation of liver enzymes was possibly related in one patient. Predose blood levels showed substantial variability between patients with 45.5% below and 18.2% above the target range of 5-15 ng/mL. Pharmacodynamic analysis demonstrated significant inhibition in phospho-S6, 4E-BP1, and modulation of c-Myc expression.

conclusionDaily oral everolimus provides a well-tolerated, alternative treatment for multiple recurrent, radiographically progressive pediatric LGG. Based on these results, everolimus is being investigated further for this patient population.

Indexed as

AdolescentAntineoplastic AgentsBrain NeoplasmsChildChild, PreschoolEverolimusFemaleGliomaHumansMagnetic Resonance ImagingMaleNeoplasm Recurrence, LocalProgression-Free SurvivalTOR Serine-Threonine KinasesTreatment OutcomeYoung AdultAntineoplastic AgentsEverolimusMTOR protein, humanTOR Serine-Threonine Kinasesbrain tumorclinical trialeverolimuslow-grade gliomamTORphase 2

Identifiers

PMID33140540
PMCPMC9161236
OpenAlexW3097158828

What OpenQuestion holds

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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.