英文标题:Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial 中文标题:Selumetinib在成人NF1患者中治疗不可手术的丛状神经纤维瘤:一项2期试验 发表日期:06 January 2025 文章类型:Article 所属期刊:Nature Medicine 文章作者:Andrea M. Gross | Brigitte C. Widemann 文章链接:...
Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trialdoi:10.1038/s41591-024-03361-4The MEK inhibitor selumetinib induces objective responses and provides clinical benefit in children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs). To ...
Cutaneous toxicities of mitogen-activated protein kinase inhibitors in children and young adults with neurofibromatosis-1 Brianna C. Peacock Sanjna Tripathy Anisha B. Patel Journal of Neuro-Oncology (2024) Mucocutaneous toxicities from MEK inhibitors: a scoping review of the literature Christopher Iri...
Phase I study of the MEK1/2 inhibitor selumetinib (AZD6244) hydrogen sulfate in children and young adults with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs) 10018 Background: Lack of functional neurofibromin in NF1 leads to dysregulated Ras and tumorigenesis. Selumet...
Phase II trial of the MEK 1/2 inhibitor selumetinib (AZD6244, ARRY-142886 Hydrogen Sulfate) in adults with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN). 3612Background: NF1-related PN are locally invasive tumors characterized by increased activation of the RAS pathwa...
neurofibromatosis 1neurofibromaplexiformBACKGROUND. The MEK inhibitor, selumetinib, reduces plexiform neurofibroma (PN) in pediatric patients with neurofibromatosis type 1 (NF1). Its safety and efficacy in adults with PN and effectiveness in other NF1 manifestations (eg, neurocognitive function, growth ...
Effects of selumetinib treatment for neurofibromatosis type 1 on pulmonary function in children and young adultsdoi:10.1183/13993003.congress-2023.PA4475TreatmentsSpirometryGeneticsBackground: Neurofibromatosis type 1 is an autosomal dominant disease caused by the defect of the antitumoral gene NF1 that ...
Here we report the initial results of an ongoing phase II study of selumetinib in adults with NF1 and inoperable PN, which includes pharmacodynamic (PD) evaluation of serial PN and cutaneous neurofibroma (cNF) biopsies, as well as patient-reported outcomes (PROs) (NCT02407405). Materials and ...
Widemann BC, Marcus LJ, Fisher MJ et al (2014) Phase I study of the MEK1/2 inhibitor selumetinib (AZD6244) hydrogen sulfate in children and young adults with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs). J Clin Oncol 32(suppl 15):abstract 10018–10018 10. ...
Conclusions: This study highlights patient characteristics that may be related to increased risk for nonadherence, as well as challenges with electronic pill caps that should be considered in future clinical trials for NF1-related PN. Results can inform future adherence interventions for adults with ...