{"doi":"10.1007/s00381-021-05138-3","title":"Targeted therapy for pediatric low-grade glioma","abstract":null,"journal":"Child's Nervous System","year":2021,"id":634662,"datarank":0.4335557636844247,"base_score":2.8903717578961645,"endowment":2.8903717578961645,"self_citation_contribution":0.4335557636844247,"citation_network_contribution":0.0,"self_endowment_contribution":0.4335557636844247,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":17,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":456960,"name":"Jordi Muchart","orcid":"0000-0003-4702-5072","position":1,"is_corresponding":false},{"id":1646131,"name":"Vicente Santa-María López","orcid":null,"position":2,"is_corresponding":false},{"id":1646133,"name":"Mariona Suñol Capella","orcid":null,"position":3,"is_corresponding":false},{"id":1646135,"name":"Noelia Salvador","orcid":null,"position":4,"is_corresponding":false},{"id":1646138,"name":"Sara Pérez Jaume","orcid":null,"position":5,"is_corresponding":false},{"id":1646140,"name":"Ofelia Cruz Martínez","orcid":null,"position":6,"is_corresponding":false},{"id":630459,"name":"Andrés Morales La Madrid","orcid":"0000-0002-0592-673X","position":7,"is_corresponding":false},{"id":1646127,"name":"Juan Pablo Muñoz Pérez","orcid":"0000-0002-4975-2436","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Targeted therapy for pediatric low-grade glioma","abstract":"<h4>Purpose</h4>Pediatric low-grade gliomas are the most frequent brain tumors in children. The standard approach for symptomatic unresectable tumors is chemotherapy. Recently, key molecular alterations/pathways have been identified and targeted drugs developed and tested in clinical trials. We describe our institutional experience with MAPK pathway targeted therapy.<h4>Methods</h4>We retrospectively reviewed the medical reports of 23 patients diagnosed with PLGG and treated with either trametinib or dabrafenib at Hospital Sant Joan de Dèu (Barcelona, Spain). Patients with neurofibromatosis were excluded. Objective response rate (ORR) and disease control rate (DCR) were determined using the Response Assessment in Pediatric Neuro-Oncology criteria in low-grade glioma. ORR was defined as the proportion of patients with the best overall response including complete remission (CR) or partial remission (PR). DCR was the sum of the CR, PR, and stable disease (SD) rates.<h4>Results</h4>ORR with trametinib was 0% (95% CI, 0%-23.2%) and DCR was 78.6% (95% CI, 49.2%-95.3%). Eleven patients had SD and three patients presented PD. ORR with dabrafenib was 41.7% (95% CI, 16.5%-71.4%), including four CR and one patient with PR. DCR with dabrafenib was 100% (95% CI, 73.5%-100%); there were seven SD and none PD. Treatment was well tolerated. Only three patients, on trametinib, presented grade 3 adverse effects: leukocytoclastic vasculitis, cheilitis, and bone infection.<h4>Conclusions</h4>Our experience adds to the growing data about the efficacy and tolerability of targeted therapy in patients with PLGG. When present, toxicity is mainly mild-moderate and transient. Ongoing prospective clinical trials are trying to address if its use should be advanced to first-line therapy.","is_dataset_classified":null,"base_score":2.8903717578961645,"endowment":2.8903717578961645,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"33864514","pmcid":null,"openalex_id":"https://openalex.org/W3155421121","authors":[],"funders":[],"total_grants":0,"fwci":1.7458,"citation_percentile":0.85169492,"influential_citations":0,"citation_trend":[{"year":2022,"count":8},{"year":2023,"count":2},{"year":2024,"count":5},{"year":2025,"count":1},{"year":2026,"count":1}],"oa_status":"closed","license":"https://www.springer.com/tdm","oa_locations":[{"url":"https://link.springer.com/content/pdf/10.1007/s00381-021-05138-3.pdf","host_type":"publisher"},{"url":"https://link.springer.com/article/10.1007/s00381-021-05138-3/fulltext.html","host_type":"publisher"},{"url":"https://doi.org/10.1007/s00381-021-05138-3","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/33864514","host_type":"repository"}],"fields_of_study":["Glioma Diagnosis and Treatment","Neurofibromatosis and Schwannoma Cases","Neuroblastoma Research and Treatments"],"mesh_terms":["Antineoplastic Combined Chemotherapy Protocols","Brain Neoplasms","Child","Glioma","Humans","Prospective Studies","Retrospective Studies","Spain"],"keywords":["Medicine","Dabrafenib","Trametinib","Tolerability","Internal medicine","Adverse effect","Glioma","Oncology","Clinical trial","Neurofibromatosis","Surgery","Pathology","Vemurafenib","MAPK/ERK pathway","Cancer","Targeted Therapy","Mapk Pathway","Pediatric Low-grade Gliomas"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Good health and well-being"}],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-06T13:58:32.225335Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}