{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T02:11:02Z","timestamp":1787019062949,"version":"build-2736575974"},"reference-count":45,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Cancer Control"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Plexiform neurofibromas (PN) represent the main cause of morbidity in patients affected by Neurofibromatosis Type 1 (NF1). Until recently, surgery has been the main treatment option in these patients, but it is burdened with a low efficacy rate and a high incidence of side effects as well as recurrence. In recent years, MEK inhibitors (MEKi) such as selumetinib and trametinib have shown great promise.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We retrospectively describe a single center cohort of NF1 patients affected by PN1 and treated with MEKi since 2019 to 2021. Patients recruited in the study were affected by PN that were not eligible to complete surgical excision, symptomatic or with major cosmetic deformation or functional neurological deficits.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Most patients experienced improvement in clinical symptoms and quality of life, with reduction or stabilization of lesions. However, no complete response was achieved. The most common adverse effects involved the skin, affecting every patient. Importantly, no life-threatening adverse effects occurred.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>In our experience, MEKi treatment has been shown to be both safe and effective in improving symptomatology and quality of life.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/10732748221144930","type":"journal-article","created":{"date-parts":[[2023,1,4]],"date-time":"2023-01-04T06:22:21Z","timestamp":1672813341000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":16,"title":["Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study"],"prefix":"10.1177","volume":"30","author":[{"given":"Antonella","family":"Cacchione","sequence":"first","affiliation":[{"name":"Department of Hematology\/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3906-4410","authenticated-orcid":false,"given":"Francesco","family":"Fabozzi","sequence":"additional","affiliation":[{"name":"Department of Hematology\/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"},{"name":"Department of Pediatrics, University of Tor Vergata, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Carai","sequence":"additional","affiliation":[{"name":"Neurosurgery Unit, Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanna Stefania","family":"Colafati","sequence":"additional","affiliation":[{"name":"Department of Imaging, Bambino Ges\u00f9 Children\u2019s Hospital (IRCCS), Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giada del","family":"Baldo","sequence":"additional","affiliation":[{"name":"Department of Hematology\/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Rossi","sequence":"additional","affiliation":[{"name":"Pathology Unit, Department of Laboratories, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martino","family":"Diana","sequence":"additional","affiliation":[{"name":"Department of Hematology\/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giacomina","family":"Megaro","sequence":"additional","affiliation":[{"name":"Department of Hematology\/Oncology, 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Ioris","sequence":"additional","affiliation":[{"name":"Department of Hematology\/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luigi","family":"Boccuto","sequence":"additional","affiliation":[{"name":"Healthcare Genetics Program, School of Nursing, College of Behavioral, Social and Health Sciences, Clemson University, Clemson, SC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Domitilla Elena","family":"Secco","sequence":"additional","affiliation":[{"name":"PsD of Department of Paediatric Haematology\/Oncology, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Zama","sequence":"additional","affiliation":[{"name":"Surgery Department, Bambino Ges\u00f9 Children\u2019s Hospital, Rome, 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