{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T20:07:16Z","timestamp":1789589236447,"version":"build-2803163510"},"reference-count":12,"publisher":"MDPI AG","issue":"5","license":[{"start":{"date-parts":[[2024,5,7]],"date-time":"2024-05-07T00:00:00Z","timestamp":1715040000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Current Oncology"],"abstract":"<jats:p>MEK inhibitors (MEKi) represent innovative and promising treatments for managing manifestations of neurofibromatosis type 1 (NF1). To mitigate potential ophthalmic side effects, such as MEKi-associated retinopathy (MEKAR), patients undergoing MEKi therapy routinely receive ophthalmology evaluations. Our study aims to assess the necessity of this regular screening within a predominantly pediatric NF1 population by examining the occurrence of ocular adverse events (OAE). A retrospective study evaluated 45 NF1 patients receiving MEKi. Inclusion criteria included baseline and follow-up examinations following the initiation of MEKi therapy. At each assessment, a comprehensive eye evaluation was performed, comprising a dilated fundus examination, ocular coherence tomography of the macula and nerve fiber layer, and Humphrey visual field testing. Twenty-six patients, with an average age of 13 years (range 2\u201323 years) and an average follow-up duration of 413 days were included in the analysis. Three different MEKi were used: selumetinib (77%), trametinib (23%), and mirdametinib (4%). None of the patients experienced retinopathy at any point during the study. Some patients had pre-existing optic neuropathies (27%), but no instances of nerve changes occurred after commencing MEKi therapy. Four patients (15%) exhibited symptoms of dry eye, all of which were effectively managed with topical lubrication.<\/jats:p>","DOI":"10.3390\/curroncol31050199","type":"journal-article","created":{"date-parts":[[2024,5,7]],"date-time":"2024-05-07T03:37:42Z","timestamp":1715053062000},"page":"2644-2649","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Incidence of Ophthalmological Complications in NF-1 Patients Treated with MEK Inhibitors"],"prefix":"10.3390","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5181-5010","authenticated-orcid":false,"given":"Lena","family":"Hummel","sequence":"first","affiliation":[{"name":"Department of Ophthalmology, University of Texas Medical Branch, Galveston, TX 77555, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"May","family":"Ameri","sequence":"additional","affiliation":[{"name":"McGovern School of Medicine, The University of Texas Health Science Center, Houston, TX 77021, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaikha","family":"Alqahtani","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Pediatric Hematology and Oncology, The University of Texas MD Anderson Cancer Center, Children\u2019s Cancer Hospital, Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zsila","family":"Sadighi","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Pediatric Hematology and Oncology, The University of Texas MD Anderson Cancer Center, Children\u2019s Cancer Hospital, Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nagham","family":"Al-Zubidi","sequence":"additional","affiliation":[{"name":"Department of Head and Neck, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,5,7]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1355","DOI":"10.1093\/brain\/111.6.1355","article-title":"Von Recklinghausen neurofibromatosis. A clinical and population study in south-east Wales","volume":"111","author":"Huson","year":"1988","journal-title":"Brain"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"100223","DOI":"10.1016\/j.esmoop.2021.100223","article-title":"Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: A comprehensive review","volume":"6","author":"Solares","year":"2021","journal-title":"ESMO Open"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"1845","DOI":"10.1093\/neuonc\/noac165","article-title":"MEK inhibitors for neurofibromatosis type 1 manifestations: Clinical evidence and consensus","volume":"24","author":"Gross","year":"2022","journal-title":"Neuro-Oncology"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1430","DOI":"10.1056\/NEJMoa1912735","article-title":"Selumetinib in Children with Inoperable Plexiform Neurofibromas","volume":"382","author":"Gross","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"ref_5","doi-asserted-by":"crossref","unstructured":"Wang, D., Ge, L., Guo, Z., Li, Y., Zhu, B., Wang, W., Wei, C., Li, Q., and Wang, Z. (2022). Efficacy and Safety of Trametinib in Neurofibromatosis Type 1-Associated Plexiform Neurofibroma and Low-Grade Glioma: A Systematic Review and Meta-Analysis. Pharmaceuticals, 15.","DOI":"10.3390\/ph15080956"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"1435","DOI":"10.1097\/IAE.0000000000002451","article-title":"Ocular adverse events associated with mek inhibitors","volume":"39","author":"Calvo","year":"2019","journal-title":"Retina"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1186\/s13023-023-02911-2","article-title":"Incidence and prevalence of neurofibromatosis type 1 and 2: A systematic review and meta-analysis","volume":"18","author":"Lee","year":"2023","journal-title":"Orphanet J. Rare Dis."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/S1470-2045(13)70024-X","article-title":"MEK162 for patients with advanced melanoma harbouring NRAS or Val600 BRAF mutations: A non-randomised, open-label phase 2 study","volume":"14","author":"Ascierto","year":"2013","journal-title":"Lancet Oncol."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1694","DOI":"10.1056\/NEJMoa1210093","article-title":"Combined BRAF and MEK inhibition in melanoma with BRAF V600 mutations","volume":"367","author":"Flaherty","year":"2012","journal-title":"N. Engl. J. Med."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.ejca.2016.06.018","article-title":"MEK inhibitor-associated retinopathy (MEKAR) in metastatic melanoma: Long-term ophthalmic effects","volume":"65","author":"Urner","year":"2016","journal-title":"Eur. J. Cancer"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"855","DOI":"10.1001\/jamaophthalmol.2016.0090","article-title":"Subretinal Fluid Associated with MEK Inhibitor Use in the Treatment of Systemic Cancer","volume":"134","author":"Weber","year":"2016","journal-title":"JAMA Ophthalmol."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"e1109","DOI":"10.1634\/theoncologist.2020-0069","article-title":"The Use of MEK Inhibitors in Neurofibromatosis Type 1-Associated Tumors and Management of Toxicities","volume":"25","author":"Klesse","year":"2020","journal-title":"Oncologist"}],"container-title":["Current Oncology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1718-7729\/31\/5\/199\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T14:40:33Z","timestamp":1760107233000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1718-7729\/31\/5\/199"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,5,7]]},"references-count":12,"journal-issue":{"issue":"5","published-online":{"date-parts":[[2024,5]]}},"alternative-id":["curroncol31050199"],"URL":"https:\/\/doi.org\/10.3390\/curroncol31050199","relation":{},"ISSN":["1718-7729"],"issn-type":[{"value":"1718-7729","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,5,7]]}}}