Evidence map›Paper›PMID 37526549›Full record

ArticleNeuro-oncology2023

Phase I trial of panobinostat in children with diffuse intrinsic pontine glioma: A report from the Pediatric Brain Tumor Consortium (PBTC-047).

Michelle Monje, Tabitha Cooney, John Glod, Jie Huang, Cody J Peer, Damien Faury, Patricia Baxter, Kim Kramer, Alicia Lenzen, Nathan J Robison and 9 more

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

Article in Neuro-oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed, 1 pooled it
15.5field-weighted citation impact, top 1% 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

48 citing papers in PubMed, 1 synthesis or guideline pooled it, 56 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Article
  5. Article
  6. Article
  7. Review
  8. Article
  9. Review
  10. Review
  11. The functional imperative in high-grade glioma.Experimental & molecular medicine · 2026
    Review
  12. Review
  13. Article
  14. Article
  15. Review
  16. Review
  17. Integrating clinical features withBMC medical imaging · 2025
    Article
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  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

19 authors at 11 institutions in 2 countries.

Michelle MonjeDepartment of Neurology, Stanford University and Lucile Packard Children's Hospital, Palo Alto, CA, USA.
Tabitha CooneyDepartment of Pediatric Oncology, Dana Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.
John GlodPediatric Oncology, Pediatric Oncology Branch, National Cancer Institute, Bethesda, MDUS.ORCID 0000-0002-3071-7876
Jie HuangDepartment of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Cody J PeerCenter for Cancer Research, Clinical Pharmacology Program, National Cancer Institute, Bethesda, Maryland, USA.
Damien FauryResearch Institute of the McGill University Health Center, Montreal, QuebecCANADA.
Patricia BaxterPediatric Oncology, Texas Children's Cancer Center, Houston, TX, USA.ORCID 0000-0003-3703-8742
Kim KramerDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, USA.
Alicia LenzenPediatric Hematology Oncology, Lurie Children's Hospital, Chicago, IL, USA.
Nathan J RobisonDepartment of Pediatrics, Children's Hospital, Los Angeles, CA, USA.
Lindsay KilburnDepartment of Oncology, Children's National Hospital, Washington, DC, USA.
Anna VinitskyDepartment of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.
William D FiggCenter for Cancer Research, Clinical Pharmacology Program, National Cancer Institute, Bethesda, Maryland, USA.ORCID 0000-0002-5875-2606
Nada JabadoResearch Institute of the McGill University Health Center, Montreal, QuebecCANADA.
Maryam FouladiPediatric Hematology Oncology, Nationwide Children's Hospital, Columbus, OH, USA.ORCID 0000-0002-5693-035X
Jason FangusaroDepartment: Pediatric Hematology/Oncology and Stem Cell Transplantation, Atlanta, GA, USA.ORCID 0000-0003-3099-5259
Arzu Onar-ThomasDepartment of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Ira J DunkelDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, USA.
Katherine E WarrenDepartment of Pediatric Oncology, Dana Farber Cancer Institute/Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0003-2173-4129
National Cancer Institute · USSt. Jude Children's Research Hospital · USBoston Children's Hospital · USMcGill University Health Centre · CAMemorial Sloan Kettering Cancer Center · USChildren's Cancer Center · USChildren's Hospital of Los Angeles · USChildren's National · USLucile Packard Children's Hospital · USLurie Children's Hospital · USNationwide Children's Hospital · 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 UM1 CA081457
6 · The paper itself

Abstract

backgroundDiffuse intrinsic pontine glioma (DIPG) is a lethal childhood cancer with median survival of less than 1 year. Panobinostat is an oral multihistone deacetylase inhibitor with preclinical activity in DIPG models. Study objectives were to determine safety, tolerability, maximum tolerated dose (MTD), toxicity profile, and pharmacokinetics of panobinostat in children with DIPG. PATIENTS AND

methodsIn stratum 1, panobinostat was administered 3 days per week for 3 weeks on, 1 week off to children with progressive DIPG, with dose escalation following a two-stage continual reassessment method. After this MTD was determined, the study was amended to evaluate the MTD in children with nonprogressive DIPG/Diffuse midline glioma (DMG) (stratum 2) on an alternate schedule, 3 days a week every other week in an effort to escalate the dose.

resultsFor stratum 1, 19 subjects enrolled with 17/19 evaluable for dose-finding. The MTD was 10 mg/m2/dose. Dose-limiting toxicities included thrombocytopenia and neutropenia. Posterior reversible encephalopathy syndrome was reported in 1 patient. For stratum 2, 34 eligible subjects enrolled with 29/34 evaluable for dose finding. The MTD on this schedule was 22 mg/m2/dose. DLTs included thrombocytopenia, neutropenia, neutropenia with grade 4 thrombocytopenia, prolonged intolerable nausea, and increased ALT.

conclusionsThe MTD of panobinostat is 10 mg/m2/dose administered 3 times per week for 3 weeks on/1 week off in children with progressive DIPG/DMG and 22 mg/m2/dose administered 3 times per week for 1 week on/1 week off when administered in a similar population preprogression. The most common toxicity for both schedules was myelosuppression.

Indexed as

Brain Stem NeoplasmsDiffuse Intrinsic Pontine GliomaGliomaNeutropeniaPosterior Leukoencephalopathy SyndromeThrombocytopeniaChildHumansPanobinostatPanobinostatbrainstem gliomaDIPGepigeneticsHDAC inhibitorsmidline glioma

Identifiers

PMID37526549
PMCPMC10708931
OpenAlexW4385441608

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.