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Record W7135019574 · doi:10.1093/neuped/wuaf001.184

LGG-01. Managing side effects of MAPK inhibitor therapies in pediatric populations: a Delphi consensus initiative

2025· article· en· W7135019574 on OpenAlex
E. Bouffet, Darren Hargrave, Daniel C. Bowers, Stewart Goldman, Grant T Liu, Ashley S. Plant-Fox, Nathan Robison, Michal Zápotocký, Jennifer T. Huang, Hanneke M. van Santen

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

affAt least one author lists a Canadian institution in the pinned OpenAlex snapshot.

Bibliographic record

VenueNeuro-Oncology Pediatrics · 2025
Typearticle
Languageen
FieldMedicine
TopicNeuroblastoma Research and Treatments
Canadian institutionsSickKids Foundation
Fundersnot available
KeywordsAdverse effectDelphi methodMultidisciplinary approachClinical trialMEDLINETrametinibDelphi

Abstract

fetched live from OpenAlex

Abstract Pediatric low-grade gliomas (pLGGs), among other pediatric brain/CNS tumors, are often driven by activating mutations in the MAPK pathway; MAPK inhibitors (MAPKis) have emerged as key treatments. However, challenges have arisen regarding their toxicity and potential late effects, creating a need for additional guidance in diagnosing, monitoring, and managing adverse events (AEs). Here, we report preliminary findings from a literature search supporting a Delphi consensus initiative, aiming to provide recommendations for clinicians to improve management of AEs with MAPKis. PubMed, Web of Science, and Cochrane Trials databases were searched for articles in English published between 2010–2024. Congress abstracts published between 2018–2024 were also considered. Up to 100 multidisciplinary global experts will aim to reach consensus on statements conceived by an international steering committee, supported by the literature search. In total, 638 publications were identified; 208 were considered relevant for data extraction. Most publications were case reports/series (64), retrospective studies (59), or phase 1/2 trials (46). The most common cancer types reported were pLGG and plexiform neurofibroma. In total, 152 publications reported data for MEK inhibitors (most commonly trametinib and selumetinib) and 116 reported data for BRAF/RAF inhibitors (most commonly dabrafenib and vemurafenib). Publications most frequently reported AE incidence, severity, and dose modifications, and less frequently reported AE management strategies, risk factors, and patient experiences. Several classes of AE, including cutaneous, gastrointestinal, hematological, cardiac, laboratory abnormalities, and general constitutional symptoms, were reported with all MAPKi types. Cutaneous toxicities were consistently common and were the AEs for which management strategies were most frequently reported. There is a paucity of information regarding AE prophylaxis, management, risk factors, and patient experiences in pediatric patients receiving MAPKis. Our findings will form the basis of a series of consensus statements and recommendations to guide and improve management of AEs with MAPKis.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.002
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.014
Threshold uncertainty score0.987

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.002
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0010.002
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.029
GPT teacher head0.336
Teacher spread0.307 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it