Evidence map›Paper›PMID 41182427›Full record

SynthesisJournal of neuro-oncology2025

The current status of immune checkpoint inhibitors in pediatric CNS tumors: a systematic review with a representative case of CMMRD-associated glioma.

Beste Gulsuna, Rachel Lopez, Robert D W Kemp, Mehmet Denizhan Yurtluk, Beyza Erkan, Burak Özaydin, Rene Y McNall-Knapp, Karl Balsara

Abstract readCase ReportsSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Journal of neuro-oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. 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

8 authors.

Beste GulsunaDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Rachel LopezDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Robert D W KempDepartment of Biochemistry, University of Illinois at Urbana-Champaign, Champaign, IL, USA.
Mehmet Denizhan YurtlukDepartment of Neurosurgery, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
Beyza ErkanDepartment of Emergency Medicine, Fethiye State Hospital, Fethiye, Muğla, Türkiye.
Burak ÖzaydinDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA.
Rene Y McNall-KnappDepartment of Pediatrics, University of Oklahoma Health Sciences Center, Oklahoma, OK, USA.
Karl BalsaraDepartment of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N Lincoln Blvd, #4000, Oklahoma City, OK, 73104, USA. Karl-Balsara@ouhsc.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeImmune checkpoint inhibitors (ICIs) have shown promise in adult oncology, but their role in pediatric central nervous system (CNS) tumors remains unclear. This systematic review evaluates the efficacy, safety, and clinical outcomes associated with ICI use in children and adolescents with primary CNS tumors, with the addition of a representative illustrative case to contextualize findings.

methodsA systematic literature search was conducted across databases through September 2025 to identify studies reporting ICI use in pediatric CNS tumors. Eligible studies included clinical trials, retrospective cohorts, and case reports. Extracted data included patient demographics, tumor characteristics, treatment regimens, clinical outcomes, and adverse events. Disease progression was analyzed quantitatively; other endpoints were synthesized descriptively.

resultsTwelve studies with 309 pediatric CNS tumor patients (mean age 10.35 years, range 1-21; F/M ratio 0.8) using ICIs were analyzed. Eight studies (n = 198, 64%) predominantly included high-grade gliomas (HGGs); others (n = 111, 36%) included medulloblastoma, ependymoma, ATRT, and rare tumors. Pooled ORR was 4.1% (95% CI: 1.8-6.4), with 3 complete and 9 partial responses. Stable disease occurred in 30.7% (n = 67), often transient; progressive disease was common. HGG ORR was 4.5% (95% CI: 2.0-7.0), with PFS 1.5-6.2 months and OS 3.2-25.5 months. Non-HGG ORR was 3.6% (95% CI: 1.0-6.5), with PFS 2.1-4.5 months and OS 9.7-22.9 months. CMMRD/hypermutated HGGs had higher ORR (10%). Grade ≥3 irAEs occurred in up to 50% with dual ICI/combination therapy, mainly rash, colitis, and fatigue; no treatment-related deaths reported.

conclusionsIn pediatric CNS tumors, ICIs provide limited benefit outside select molecularly defined subgroups. Although toxicities are generally manageable, evidence remains insufficient to support routine clinical use. Future efforts should emphasize biomarker-driven selection and combination regimens.

Indexed as

Brain NeoplasmsCentral Nervous System NeoplasmsGliomaImmune Checkpoint InhibitorsAdolescentChildChild, PreschoolFemaleHumansInfantMaleYoung AdultImmune Checkpoint InhibitorsBrain tumorsImmune checkpoint inhibitorsImmunotherapyNeuro-oncologyPediatric

Identifiers

PMID41182427

What OpenQuestion holds

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Registered trials

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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.