Evidence map›Paper›PMID 33025730›Full record

ArticlePediatric blood & cancer2021

Phase 1 study of pomalidomide in children with recurrent, refractory, and progressive central nervous system tumors: A Pediatric Brain Tumor Consortium trial.

Jason Fangusaro, Duane A Mitchell, Mehmet Kocak, Giles W Robinson, Patricia Ann Baxter, Eugene I Hwang, Jianping Huang, Arzu Onar-Thomas, Ira J Dunkel, Maryam Fouladi and 1 more

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

Article 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 10 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 1 pooled it
1.0field-weighted citation impact, top 23% 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

10 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.

  1. Pooled it
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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

11 authors at 9 institutions in 1 country.

Jason FangusaroChildren's Healthcare of Atlanta and Emory University Medical School, Atlanta, Georgia.ORCID 0000-0003-3099-5259
Duane A MitchellPreston A. Wells, Jr. Center for Brain Tumor Therapy, University of Florida Brain Tumor Immunotherapy Program, Gainesville, Florida.
Mehmet KocakUniversity of Tennessee Health Science Center, Memphis, Tennessee.
Giles W RobinsonSt Jude Children's Research Hospital, Memphis, Tennessee.
Patricia Ann BaxterTexas Children's Cancer Center, Baylor College of Medicine, Houston, Texas.
Eugene I HwangChildren's National Health System, Washington, District of Columbia.
Jianping HuangPreston A. Wells, Jr. Center for Brain Tumor Therapy, University of Florida Brain Tumor Immunotherapy Program, Gainesville, Florida.
Arzu Onar-ThomasSt Jude Children's Research Hospital, Memphis, Tennessee.
Ira J DunkelMemorial Sloan Kettering Cancer Center, New York, New York.
Maryam FouladiNationwide Children's Hospital, Columbus, Ohio.
Katherine E WarrenDana-Farber Cancer Institute and Harvard Medical School, Boston, Massachusetts.
St. Jude Children's Research Hospital · USUniversity of Florida · USBaylor College of Medicine · USChildren's National · USEmory University · USHarvard University · USMemorial Sloan Kettering Cancer Center · USNationwide Children's Hospital · USUniversity of Tennessee Health Science Center · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4M
Pediatric Brain Tumor Consortium (PBTC)UM1CA081457 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Ira J Dunkel, Arzu Onar-Thomas · 2014 to 2026
$41.4M
NCI NIH HHS P30 CA008748NCI NIH HHS P30CA008748NCI NIH HHS UM1 CA081457NCI NIH HHS UMICA081457
6 · The paper itself

Abstract

backgroundCentral nervous system (CNS) malignancies are the most common solid tumors among children, and novel therapies are needed to help improve survival. Pomalidomide is an immunomodulatory agent that displays antiangiogenic and cytotoxic activity, making it an appropriate candidate to explore in pediatric CNS tumors.

methodsA phase 1 first in pediatric trial of pomalidomide was conducted in children with recurrent, progressive, and refractory CNS tumors. The primary objective was to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) when given orally once daily for 21 consecutive days of a 28-day cycle. Once the MTD was established, 12 additional patients were enrolled on expansion cohorts based on age and steroid use.

resultsTwenty-nine children were enrolled and 25 were evaluable for dose-limiting toxicity (DLT). The MTD was 2.6 mg/m

conclusionsThe MTD of pomalidomide is 2.6 mg/m

Indexed as

AdolescentAngiogenesis InhibitorsAntineoplastic AgentsCentral Nervous System NeoplasmsChildChild, PreschoolDose-Response Relationship, DrugFemaleHumansImmunomodulationMaleThalidomideYoung AdultAngiogenesis InhibitorsAntineoplastic AgentspomalidomideThalidomidebrain tumorcentral nervous system tumorIMiD agentpediatricpomalidomide

Identifiers

PMID33025730
PMCPMC7757731
OpenAlexW3092517018

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.