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Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas
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Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas
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Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas
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Journal Article
Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas
2016
Overview
Plexiform neurofibroma is a complication of the
NF1
mutation in neurofibromatosis that results in overactivity of the RAS pathway. Selumetinib, a mitogen-activated protein kinase (MAPK) kinase inhibitor, induced tumor regressions in a majority of patients.
Neurofibromatosis type 1 is a common genetic disorder that is characterized by multiple manifestations including tumors of the nervous system.
1
,
2
Plexiform neurofibromas develop in 20 to 50% of persons with neurofibromatosis type 1 and can cause substantial complications including pain, functional impairment, disfigurement, and malignant transformation.
3
–
7
Most plexiform neurofibromas are diagnosed in early childhood and grow most rapidly during this period.
8
,
9
Complete surgical resection of these tumors is often not feasible, and regrowth of the tumor after incomplete surgical resection has been observed.
10
,
11
The
NF1
product neurofibromin functions as a negative regulator of RAS activity. Lack . . .
Publisher
Massachusetts Medical Society
Subject
/ Animals
/ Benzimidazoles - administration & dosage
/ Benzimidazoles - adverse effects
/ Benzimidazoles - pharmacokinetics
/ Child
/ Children
/ Creatine
/ Female
/ Humans
/ Kinases
/ Male
/ Mice
/ Mitogen-Activated Protein Kinase Kinases - antagonists & inhibitors
/ Neurofibroma, Plexiform - diagnostic imaging
/ Neurofibroma, Plexiform - drug therapy
/ Neurofibromatosis 1 - drug therapy
/ Pain
/ Protein Kinase Inhibitors - administration & dosage
/ Protein Kinase Inhibitors - adverse effects
/ Protein Kinase Inhibitors - pharmacokinetics
/ Tumors
