{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T18:27:05Z","timestamp":1772648825152,"version":"3.50.1"},"reference-count":22,"publisher":"American Academy of Pediatrics (AAP)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,3,1]]},"abstract":"<jats:p>Retinal astrocytic hamartoma (RAH) is a rare benign retinal tumor most commonly associated with tuberous sclerosis complex and is typically stable. RAH may also rarely occur in neurofibromatosis type 1 (NF1), an autosomal dominant disorder characterized by skin pigmentation abnormalities and multiple neurofibromas. We present a case of a child with NF1 and symptomatic, inoperable plexiform neurofibromas, for which selumetinib was initiated. Incidentally, the patient also had a progressive RAH involving the optic disc, leading to severe exudative retinal detachment. Following selumetinib therapy, the RAH showed measurable reduction in size (from 5.51\u2009\u00d7\u200910.69\u00a0mm to 5.46\u2009\u00d7\u20095.23\u00a0mm on ocular ultrasonography) and resolution of associated retinal detachment This case highlights a novel observation of selumetinib-associated RAH regression in an a patient with NF1. Although selumetinib was not prescribed for ocular disease, these findings suggest a potential off-target benefit and support further exploration of mitogen-activated protein kinase inhibitors in managing vision-threatening RAH in NF1.<\/jats:p>","DOI":"10.1542\/peds.2025-071507","type":"journal-article","created":{"date-parts":[[2026,2,19]],"date-time":"2026-02-19T00:02:11Z","timestamp":1771459331000},"source":"Crossref","is-referenced-by-count":0,"title":["Retinal Astrocytic Hamartoma in a Child With Neurofibromatosis Type 1 Treated With Selumetinib"],"prefix":"10.1542","volume":"157","author":[{"given":"Tsung-I","family":"Wang","sequence":"first","affiliation":[{"name":"1Department of Ophthalmology, Peking University People\u2019s Hospital, Beijing, China"},{"name":"2Beijing Key Laboratory of Ocular Disease and Optometry Science, Peking University People\u2019s Hospital, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuchen","family":"He","sequence":"additional","affiliation":[{"name":"1Department of Ophthalmology, Peking University People\u2019s Hospital, Beijing, China"},{"name":"2Beijing Key Laboratory of Ocular Disease and Optometry Science, Peking University People\u2019s Hospital, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xuemei","family":"Zhu","sequence":"additional","affiliation":[{"name":"1Department of Ophthalmology, Peking University People\u2019s Hospital, Beijing, China"},{"name":"2Beijing Key Laboratory of Ocular Disease and Optometry Science, Peking University People\u2019s Hospital, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jianhong","family":"Liang","sequence":"additional","affiliation":[{"name":"1Department of Ophthalmology, Peking University People\u2019s Hospital, Beijing, China"},{"name":"2Beijing Key Laboratory of Ocular Disease and Optometry Science, Peking University People\u2019s Hospital, Beijing, China."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"417","published-online":{"date-parts":[[2026,2,19]]},"reference":[{"issue":"6","key":"2026030411123037800_r1","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.1016\/j.mcna.2019.07.004","article-title":"The diagnosis and management of neurofibromatosis type 1","volume":"103","author":"Ly","year":"2019","journal-title":"Med Clin North Am."},{"key":"2026030411123037800_r2","doi-asserted-by":"crossref","first-page":"799","DOI":"10.1016\/B978-0-444-64076-5.00051-X","article-title":"Neurofibromatosis type 1","volume":"148","author":"Cimino","year":"2018","journal-title":"Handb Clin Neurol."},{"issue":"7","key":"2026030411123037800_r3","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1056\/NEJMoa1209288","article-title":"Selumetinib-enhanced radioiodine uptake in advanced thyroid cancer","volume":"368","author":"Ho","year":"2013","journal-title":"N Engl J Med."},{"issue":"1","key":"2026030411123037800_r4","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/S1470-2045(12)70489-8","article-title":"Selumetinib plus docetaxel for KRAS-mutant advanced non-small-cell lung cancer: a randomised, multicentre, placebo-controlled, phase 2 study","volume":"14","author":"J\u00e4nne","year":"2013","journal-title":"Lancet Oncol."},{"issue":"8","key":"2026030411123037800_r5","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1016\/S1470-2045(13)70237-7","article-title":"Selumetinib plus dacarbazine versus placebo plus dacarbazine as first-line treatment for BRAF-mutant metastatic melanoma: a phase 2 double-blind randomised study","volume":"14","author":"Robert","year":"2013","journal-title":"Lancet Oncol."},{"issue":"4","key":"2026030411123037800_r6","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1136\/bjo.85.4.420","article-title":"Ophthalmic manifestations of tuberous sclerosis: a population based study","volume":"85","author":"Rowley","year":"2001","journal-title":"Br J Ophthalmol."},{"issue":"6","key":"2026030411123037800_r7","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1159\/000088377","article-title":"Autofluorescence and angiographic findings of retinal astrocytic hamartomas in tuberous sclerosis","volume":"219","author":"Mennel","year":"2005","journal-title":"Ophthalmologica."},{"issue":"6","key":"2026030411123037800_r8","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1136\/bjo.56.6.487","article-title":"Glial hamartoma of the retina in generalized neurofibromatosis, Von Recklinghausen\u2019s disease","volume":"56","author":"Martyn","year":"1972","journal-title":"Br J Ophthalmol."},{"issue":"5","key":"2026030411123037800_r9","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1001\/archopht.1991.01080050076033","article-title":"Retinal manifestations of neurofibromatosis. 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