{"id":238175,"date":"2026-06-02T02:26:35","date_gmt":"2026-06-02T07:26:35","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2026\/06\/therapeutic-effects-of-selumetinib-on-diffuse-neurofibroma-and-optic-pathway-glioma-in-neurofibromatosis-type-1"},"modified":"2026-06-02T02:26:35","modified_gmt":"2026-06-02T07:26:35","slug":"therapeutic-effects-of-selumetinib-on-diffuse-neurofibroma-and-optic-pathway-glioma-in-neurofibromatosis-type-1","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2026\/06\/therapeutic-effects-of-selumetinib-on-diffuse-neurofibroma-and-optic-pathway-glioma-in-neurofibromatosis-type-1","title":{"rendered":"Therapeutic effects of selumetinib on diffuse neurofibroma and optic pathway glioma in neurofibromatosis type 1"},"content":{"rendered":"<p style=\"padding-right: 20px\"><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/therapeutic-effects-of-selumetinib-on-diffuse-neurofibroma-and-optic-pathway-glioma-in-neurofibromatosis-type-1.jpg\"><\/a><\/p>\n<p>Selumetinib is approved for the treatment of inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1). However, its efficacy in treating NF1-associated diffuse neurofibromas (NF1-DN) or optic pathway gliomas (NF1-OPG) remains unclear. We evaluated the efficacy and safety of selumetinib in these subgroups.<\/p>\n<p>This was a sub-analysis of a Korean phase II open-label trial focusing on non-target treatment effects on NF1-DN and NF1-OPG. A total of 88 pediatric and adult patients with NF1-PN (59 children and 29 adults) in this trial had been treated for at least 2 years (~ 26 cycles, 28-day cycle) with oral selumetinib (20 or 25 mg\/m\u00b2, or 50 mg\/dose every 12 h). Tumor volume, quality of life (QoL), and visual acuity were assessed.<\/p>\n<p>Among the 88 included patients, NF1-DN was diagnosed in 25 (28%), and NF1-OPG in 3 (3%). All NF1-DN patients exhibited disfigurement, two experienced pain, and a partial response (PR; \u226520% tumor reduction at a single time) was achieved in 9 of these cases (36%). The median time to PR was 6 cycles (range, 6\u201312), and the median time to best response was 18 cycles (range, 6\u201326), with a median volume change of \u2212 11.9% (range, \u2212 55.4% to + 36.3%). Confirmed PR (cPR; PR sustained for 6 cycles) was observed in 6 NF1-DN patients (24%), stable disease (SD) was observed in 9 of these patients (36%), and progressive disease (PD) in 10 cases (40%). In a paired comparison, cPR was significantly lower for NF1-DN than for NF1-PN (24% vs. 88%, P 0.001), and the median best volume reduction was also smaller (\u2212 11.9% vs. \u221242.1%, P 0.001). For the 3 NF1-OPG patients, visual impairment was present in all cases at baseline. One patient achieved PR at cycle 12 (\u2212 36.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Selumetinib is approved for the treatment of inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1). However, its efficacy in treating NF1-associated diffuse neurofibromas (NF1-DN) or optic pathway gliomas (NF1-OPG) remains unclear. We evaluated the efficacy and safety of selumetinib in these subgroups. This was a sub-analysis of a Korean phase II open-label [\u2026]<\/p>\n","protected":false},"author":662,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11],"tags":[],"class_list":["post-238175","post","type-post","status-publish","format-standard","hentry","category-biotech-medical"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/238175","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/662"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=238175"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/238175\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=238175"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=238175"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=238175"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}