{"doi":"10.3390/genes16060645","title":"Clinical Efficacy of Selumetinib in Alleviating Neuropathic Pain Associated with Plexiform Neurofibroma: A Case Series","abstract":"<jats:p>Background: Selumetinib, an MEK inhibitor, was approved by the U.S. Food and Drug Administration in April 2020 and became reimbursable in Italy in January 2020, for treating patients aged ≥3 years with neurofibromatosis type 1 (NF1) complicated by symptomatic, inoperable plexiform neurofibromas (PNs). Selumetinib has been shown to effectively reduce the volume of target PNs and alleviate neuropathic pain even in long-term-treated patients. We report the impact of Selumetinib on pain in three NF1 cases with inoperable symptomatic plexiform neurofibromas. Case Series: Three patients with NF1 (aged 13–27 years) presented with symptomatic, inoperable plexiform neurofibromas (PNs) associated with severe neuropathic pain. Following selumetinib administration, a marked reduction in or disappearance of neuropathic pain was observed within a few weeks, allowing the complete discontinuation of pain therapy. Interestingly, pain recurred whenever selumetinib was temporarily suspended, requiring patients to resume analgesic therapy. Conclusions: Selumetinib treatment has been shown to effectively reduce neuropathic pain in patients with NF1. These findings represent a significant advancement in managing pain related to PNs and support its potential application in treating other forms of neuropathic pain.</jats:p>","journal":"Genes","year":2025,"id":623004,"datarank":0.26876392038420827,"base_score":1.791759469228055,"endowment":1.791759469228055,"self_citation_contribution":0.26876392038420827,"citation_network_contribution":0.0,"self_endowment_contribution":0.26876392038420827,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":5,"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":1506841,"name":"Irene Russo","orcid":null,"position":1,"is_corresponding":false},{"id":1609965,"name":"Lorenzo Filipponi","orcid":null,"position":2,"is_corresponding":false},{"id":647913,"name":"Silverio Perrotta","orcid":"0000-0001-6149-6845","position":3,"is_corresponding":false},{"id":1609966,"name":"Elisabetta Maida","orcid":null,"position":4,"is_corresponding":false},{"id":1609967,"name":"Giulio Piluso","orcid":"0000-0001-9418-125X","position":5,"is_corresponding":false},{"id":1609968,"name":"Mariarosa Anna Beatrice Melone","orcid":"0000-0002-7213-9277","position":6,"is_corresponding":false},{"id":1609969,"name":"Claudia Santoro","orcid":"0000-0003-4547-0500","position":7,"is_corresponding":false},{"id":1609964,"name":"Giuseppina Miele","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Clinical Efficacy of Selumetinib in Alleviating Neuropathic Pain Associated with Plexiform Neurofibroma: A Case Series","abstract":"<jats:p>Background: Selumetinib, an MEK inhibitor, was approved by the U.S. Food and Drug Administration in April 2020 and became reimbursable in Italy in January 2020, for treating patients aged ≥3 years with neurofibromatosis type 1 (NF1) complicated by symptomatic, inoperable plexiform neurofibromas (PNs). Selumetinib has been shown to effectively reduce the volume of target PNs and alleviate neuropathic pain even in long-term-treated patients. We report the impact of Selumetinib on pain in three NF1 cases with inoperable symptomatic plexiform neurofibromas. Case Series: Three patients with NF1 (aged 13–27 years) presented with symptomatic, inoperable plexiform neurofibromas (PNs) associated with severe neuropathic pain. Following selumetinib administration, a marked reduction in or disappearance of neuropathic pain was observed within a few weeks, allowing the complete discontinuation of pain therapy. Interestingly, pain recurred whenever selumetinib was temporarily suspended, requiring patients to resume analgesic therapy. Conclusions: Selumetinib treatment has been shown to effectively reduce neuropathic pain in patients with NF1. These findings represent a significant advancement in managing pain related to PNs and support its potential application in treating other forms of neuropathic pain.</jats:p>","is_dataset_classified":null,"base_score":1.791759469228055,"endowment":1.791759469228055,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"40565537","pmcid":"PMC12192340","openalex_id":"https://openalex.org/W4410813590","authors":[],"funders":[],"total_grants":0,"fwci":3.8731,"citation_percentile":0.93636983,"influential_citations":0,"citation_trend":[{"year":2025,"count":2},{"year":2026,"count":3}],"oa_status":"gold","license":"cc-by","oa_locations":[{"url":"https://www.mdpi.com/2073-4425/16/6/645/pdf?version=1748409852","host_type":"journal"},{"url":"https://www.mdpi.com/2073-4425/16/6/645/pdf?version=1748409852","host_type":"publisher"},{"url":"https://www.mdpi.com/2073-4425/16/6/645/pdf","host_type":"publisher"},{"url":"https://doi.org/10.3390/genes16060645","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/40565537","host_type":"repository"},{"url":"https://www.ncbi.nlm.nih.gov/pmc/articles/12192340","host_type":"repository"},{"url":"https://europepmc.org/articles/PMC12192340","host_type":"Europe_PMC"},{"url":"https://europepmc.org/articles/PMC12192340?pdf=render","host_type":"Europe_PMC"}],"fields_of_study":["Neurofibromatosis and Schwannoma Cases","Neuroblastoma Research and Treatments","Management of metastatic bone disease","Humans","Neurofibroma, Plexiform","Adult","Benzimidazoles","Adolescent","Female","Male","Neuralgia","Young Adult","Neurofibromatosis 1","Treatment Outcome","Protein Kinase Inhibitors"],"mesh_terms":["Adolescent","Adult","Benzimidazoles","Female","Humans","Male","Neuralgia","Neurofibromatosis 1","Treatment Outcome","Neurofibroma, Plexiform","Protein Kinase Inhibitors","Young Adult"],"keywords":["Selumetinib","Neuropathic pain","Medicine","Plexiform neurofibroma","Dermatology","Neurofibroma","Neurofibromatosis","Anesthesia","Internal medicine","Pathology","Cancer","Neurofibromatosis Type 1","Mek Inhibitor"],"sdg_mappings":[],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-03T21:55:38.286104Z","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":[]}