Evidence map›Paper›PMID 41050069›Full record

ReviewOncology research2025

Pharmacological Phase I Clinical Trials in Pediatric Brain Tumors (1990-2024): A Historical Perspective.

Rosa Scarpitta, Emiliano Cappello, Alice Cangialosi, Veronica Gori, Giulia De Luca, Giovanni Gori, Guido Bocci

Abstract readReviewHistorical Article
In one paragraph

Review in Oncology research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

7 authors.

Rosa ScarpittaScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.
Emiliano CappelloScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.
Alice CangialosiScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.
Veronica GoriScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.
Giulia De LucaScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.
Giovanni GoriCentro di Farmacologia Clinica per la Sperimentazione dei Farmaci, Azienda Ospedaliera Universitaria Pisana, Pisa, 56126, Italy.
Guido BocciScuola di Specializzazione di Farmacologia e Tossicologia Clinica, Dipartimento di Ricerca Traslazionale e delle Nuove Tecnologie in Medicina e Chirurgia, University of Pisa, Pisa, 56126, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Central nervous system (CNS) tumors are the most common solid tumors in pediatric patients and the leading cause of childhood cancer-related mortality. Their rarity compared to adult cancers has made enrolling sufficient cases for clinical trials challenging. Consequently, pediatric CNS tumors were long treated with adult protocols despite distinct biological and clinical characteristics. This review examines key aspects of phase I pediatric oncology trials, including study design, primary outcomes, and pharmacological approaches, along with secondary considerations like clinical responses and ethical aspects. Firstly, we evaluated all phase I trial protocols focusing on pediatric CNS tumors with accessible results published in scientific databases (Pubmed, Scopus, Embase, Web of Science, and Google Scholar) from 1990 to November 2024. Secondly, we searched EudraCT and ClinicalTrials.gov on 30 November 2024 for ongoing trials. Our search yielded 60 completed phase I studies and 15 trials in progress. Dividing them by chronological order revealed that study designs and the response assessments evolved as the understanding of CNS tumor biology increased. Despite advancements improving diagnosis, management, and prognostication, mortality remains high, and morbidity persists. Notably, pediatric pharmacokinetics and pharmacodynamics differ from adults, complicating trial comparisons and dosage optimization. Future efforts should focus on large-scale clinical data collection to enhance trial efficiency.

Indexed as

Antineoplastic AgentsBrain NeoplasmsClinical Trials, Phase I as TopicChildHistory, 20th CenturyHistory, 21st CenturyHumansResearch DesignAntineoplastic Agentsclinical trialpediatric neuro-oncologyPhase I

Identifiers

PMID41050069
PMCPMC12494115

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.