Safety, pharmacokinetics and efficacy of selumetinib in Chinese adult and paediatric patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: The primary analysis of a phase 1 open-label study

Zhichao Wang; Xin Zhang; Chunyan Li; Yangbo Liu; Xiaoyun Ge; Jiajia Zhao; Xiaojun Yuan; Qingfeng Li

doi:10.1002/ctm2.1589

Clinical and Translational Medicine ›› 2024, Vol. 14 ›› Issue (3) : e1589 DOI: 10.1002/ctm2.1589

LETTER TO THE JOURNAL

Safety, pharmacokinetics and efficacy of selumetinib in Chinese adult and paediatric patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: The primary analysis of a phase 1 open-label study

Author information +

History +

PDF

Cite this article

Download citation ▾

Zhichao Wang,Xin Zhang,Chunyan Li,Yangbo Liu,Xiaoyun Ge,Jiajia Zhao,Xiaojun Yuan,Qingfeng Li. Safety, pharmacokinetics and efficacy of selumetinib in Chinese adult and paediatric patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: The primary analysis of a phase 1 open-label study. Clinical and Translational Medicine, 2024, 14(3): e1589 DOI:10.1002/ctm2.1589

登录浏览全文

4963

注册一个新账户忘记密码

References

Publishing order | Descend order by publishing year | Descend order by cited within

[1]	Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited—from bench to bedside. Oncotarget. 2014;5(15):5873-5892.

[2]	Bergqvist C, Servy A, Valeyrie-Allanore L, Ferkal S, Combemale P, Wolkenstein P. Neurofibromatosis 1 French national guidelines based on an extensive literature review since 1966. Orphanet J Rare Dis. 2020;15(1):37.

[3]	Blakeley JO, Plotkin SR. Therapeutic advances for the tumors associated with neurofibromatosis type 1, type 2, and schwannomatosis. Neuro Oncol. 2016;18(5):624-638.

[4]	Gross AM, Wolters PL, Dombi E, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020;382(15):1430-1442.

[5]	U.S. Food and Drug Administration. Selumetinib (Koselugo) Full Prescribing Information. Accessed January 5, 2024.

[6]	European Medicines Agency. Selumetinib (Koselugo) Summary of Product Characteristics. Accessed January 5, 2024.

[7]	AstraZeneca. Koselugo Approved in China for Paediatric Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas. Accessed January 5, 2024.

[8]	Dymond AW, Elks C, Martin P, et al. Pharmacokinetics and pharmacogenetics of the MEK1/2 inhibitor, selumetinib, in Asian and Western healthy subjects: a pooled analysis. Eur J Clin Pharmacol. 2017;73(6):717-726.

[9]	Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med. 2016;375(26):2550-2560.

[10]	Suenobu S, Terashima K, Akiyama M, et al. Selumetinib in Japanese pediatric patients with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas: an open-label, phase I study. Neurooncol Adv. 2023;5(1):vdad054.

RIGHTS & PERMISSIONS

2024 The Authors. Clinical and Translational Medicine published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.