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Assessments included the clinical benefit rate (CBR) after 24 months and at the last observation. Major response (MR) was defined as a\u2009\u2265\u200950% reduction from baseline in tumor volume. Adverse events (AEs), patient-reported pain and quality of life (QoL), and Eastern Cooperative Oncology Group performance status (ECOG PS), were also evaluated.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of 45\/70 patients with available natural history data at C0, 33\/45 (73.3%) had progressive disease (PD). Radiological evaluation at C6 in 17\/33 patients showed 16 (94.1%) had stable disease (SD) or partial response (PR). 52\/58 patients (91.5%) had SD or PR\/MR at C12; final response at last radiological follow-up was PD (7.7%), SD (42.3%), PR (30.8%) and MR (19.2%). CBR was 83.3% (24\/70) at C24 and 91.5% (43\/47) at last radiological follow-up. Selumetinib significantly reduced pain perception and improved QoL and ECOG PS. The type of response at C6 seems to predict response at C12 and at last observation. Adverse events were generally mild (78% grade\u2009\u2264\u20092).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Our findings suggest that the response after 6 and 12 selumetinib cycles may predict long-term outcomes, providing clinicians with an early indicator for therapeutic decision-making.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration number<\/jats:title>\n                    <jats:p>Not applicable.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s11060-025-05197-5","type":"journal-article","created":{"date-parts":[[2025,8,13]],"date-time":"2025-08-13T06:52:04Z","timestamp":1755067924000},"page":"1027-1037","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study"],"prefix":"10.1007","volume":"175","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4547-0500","authenticated-orcid":false,"given":"Claudia","family":"Santoro","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariachiara","family":"Servedio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Cristina","family":"Diana","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irene","family":"Russo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Arkhangelskaya","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gianluca","family":"Piccolo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Santangelo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Mastronuzzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonella","family":"Cacchione","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"May","family":"El Hachem","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmela","family":"Russo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Cirillo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Cecconi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Grasso","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariateresa","family":"Loiotine","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Santoro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariachiara","family":"Resta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmela","family":"De Meco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Consolata","family":"Soddu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenia","family":"Spreafico","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bartolomeo","family":"Rossi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Fossati","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Leoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silverio","family":"Perrotta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teresa","family":"Perillo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,8,13]]},"reference":[{"key":"5197_CR1","doi-asserted-by":"publisher","first-page":"229","DOI":"10.1093\/nop\/npac014","volume":"9","author":"TA Gregory","year":"2022","unstructured":"Gregory TA, Molina PSB, Phillips GD, Henson JW (2022) Impact of neurofibromatosis type 1 in an adult community population. 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T.P. received payment for scientific consultation and speaker honoraria from Alexion, AstraZeneca Rare Disease; and participated on an advisory board for Alexion, AstraZeneca Rare Disease. A.M. received speaker honoraria from Alexion, AstraZeneca Rare Disease and participated on an advisory board for Ipsen. C.L. participated in the SeluPASS steering committee. All the other authors declare no conflict of interest.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"This study was approved by the territorial Ethics Committee of the Polyclinic of Bari, Italy, on April 24, 2024, and conducted in accordance with the Helsinki Declaration, Good Clinical Practice guidelines, and all applicable laws and regulations.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Written informed consent to participate was obtained from patients aged 18 years, and from parents\/legal guardians of patients aged\u2009<\u200918 years.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"Not applicable.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to publish"}}]}}