REFERENCES
1. Hirbe AC, Gutmann DH. Neurofibromatosis type 1: a multidisciplinary approach to care. Lancet Neurol. 2014;13:834-43.
2. Fisher MJ, Blakeley JO, Weiss BD, et al. Management of neurofibromatosis type 1-associated plexiform neurofibromas. Neuro Oncol. 2022;24:1827-44.
3. Na B, Shah SR, Vasudevan HN. Past, present, and future therapeutic strategies for NF-1-associated tumors. Curr Oncol Rep. 2024;26:706-13.
4. Weiss B, Widemann BC, Wolters P, et al. Sirolimus for progressive neurofibromatosis type 1-associated plexiform neurofibromas: a neurofibromatosis Clinical Trials Consortium phase II study. Neuro Oncol. 2015;17:596-603.
5. Robertson KA, Nalepa G, Yang FC, et al. Pilot phase II trial of imatinib mesylate in neurofibromatosis type 1 patients with plexiform neurofibromas. Lancet Oncol. 2012;13:1218-24.
6. Widemann BC, Babovic-Vuksanovic D, Dombi E, et al. Phase II trial of pirfenidone in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas. Pediatr Blood Cancer. 2014;61:1598-602.
7. Widemann BC, Dombi E, Gillespie A, et al. Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas. Neuro Oncol. 2014;16:707-18.
8. Shih CS. Study of sutent®/sunitinib (SU11248) in subjects with NF-1 plexiform neurofibromas; 2018. Available from: https://clinicaltrials.gov/study/NCT01402817 [Last accessed on 11 Feb 2026].
9. Gorai S, Rathore G, Das K. Selumetinib-A comprehensive review of the new FDA-approved drug for neurofibromatosis. Indian Dermatol Online J. 2024;15:701-5.
10. Akbarzadeh M, Vaez-Gharamaleki Y, Hosseini M. Mirdametinib approval for neurofibromatosis type 1 with inoperable plexiform neurofibromas: the journey of MEK inhibitors. ESMO Rare Cancers. 2025;3:100015.
11. Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, Feliu J. Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review. ESMO Open. 2021;6:100223.
12. Gross AM, Wolters PL, Dombi E, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020;382:1430-42.
13. FDA. FDA approves selumetinib for adults with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selumetinib-adults-neurofibromatosis-type-1-symptomatic-inoperable-plexiform [Last accessed on 11 Feb 2026].
14. Chen AP, Coyne GO, Wolters PL, et al. Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind, phase 3 study. Lancet. 2025;405:2217-30.
15. Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: a pivotal, phase IIb trial of mirdametinib in adults and children with symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2025;43:716-29.
16. FDA. FDA approves mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mirdametinib-adult-and-pediatric-patients-neurofibromatosis-type-1-who-have-symptomatic [Last accessed on 11 Feb 2026].
17. Mol I, Bell T, Hu Y, Langseth A, Weber M, Moradian H. CO122 indirect treatment comparison (ITC) of mirdametinib and selumetinib for the treatment of children with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Value Health. 2025;28:S45.
18. Gross AM, Dombi E, Wolters PL, et al. Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas. Neuro Oncol. 2023;25:1883-94.
19. Healx Limited. A phase 2, open-label, single arm, non-controlled, single-stage study to evaluate the safety and effects of HLX-1502 in patients with plexiform neurofibroma and neurofibromatosis type 1 (INSPIRE-NF1). Available from: https://clinicaltrials.gov/study/NCT06541847 [Last accessed on 11 Feb 2026].
20. Korf B. Phase II study of binimetinib in children and adults with NF1 Plexiform neurofibromas (NF108-BINI). Available from: https://clinicaltrials.gov/study/NCT03231306 [Last accessed on 11 Feb 2026].
21. Fisher MJ, Shih CS, Rhodes SD, et al. Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial. Nat Med. 2021;27:165-73.
22. Dhall G. Study of cabozantinib with selumetinib for plexiform neurofibromas (NF113). Available from: https://clinicaltrials.gov/study/NCT06502171 [Last accessed on 11 Feb 2026].
23. Sundby RT, Szymanski JJ, Pan AC, et al. Early detection of malignant and premalignant peripheral nerve tumors using cell-free DNA fragmentomics. Clin Cancer Res. 2024;30:4363-76.
24. Bai RY, Shi J, Liu J, et al. Development of an adeno-associated virus vector for gene replacement therapy of NF1-related tumors. Nat Commun. 2025;16:8594.
25. Mueller S, Reddy AT, Dombi E, et al. NFB-17. MEK inhibitor binimetinib shows clinical activity in children with neurofibromatosis type 1- associated plexiform neurofibromas: a report from pnoc and the nf clinical trials consortium. Neuro Oncol. 2020;22:iii420-1.
