Selumetinib Use as Targeted Therapy for Inoperable Plexiform Neurofibroma: Review of the Literature
Author: Eman Alibrahim
Base Hospital / Institution: Dhahran Eye Specialist Hospital, Dammam , KSA
ePoster presentation
Abstract ID: 24-519
Purpose
In this article, we comprehensively reviewed the recently published literature on the use of selumetinib to treat inoperable plexiform neurofibroma in neurofibromatosis type 1 patients.
Methods
We reviewed all English articles published from 1999 to 2024. A systemic medline search was performed on PubMed using search terms (Selumetinib OR Plexiform Neurofibroma OR Neurofibromatosis OR Targeted therapy OR mitogen-activated protein kinase).
Results
Gross et al., 2020 found that the median time to initial response was eight cycles, and the median time to best response was 16 cycles. Initially developed for melanoma and lung cancer, Selumetinib showed favorable results in the treatment of plexiform neurofibroma in children at a dose of 20 to 30 mg per square meter, with 71-74% of the treated children showing a partial response, which was defined as a decrease in tumor volume of 20% or more when compared to baseline on vMRI, furthermore, none of them showed any tumor progression.
Gross et al., 2020 found that progression-free survival was favorable, being 84% as of 3 years after the start of treatment. They also found that pain scores significantly decreased, and quality of life scores significantly increased when assessing children with PN who underwent Selumetinib treatment.
The most commonly reported side effects with Gross et al., 2023 were skin manifestations (58 patients out of 74), vomiting (58 patients), and increased Creatine Phosphokinase (56 patients).
Conclusion
In essence, all studies reporting on the use of Selumetinib in different neurofibromatosis lesions have demonstrated a high success rate with a low-risk side effects profile, favoring the use of the drug in the treatment of different neurofibromatosis lesions.
Additional Authors
| First name | Last name | Base Hospital / Institution |
|---|---|---|
| Hammam | A. Alotaibi | Dhahran Eye Specialist Hospital, Dammam , KSA |
| , Askar | K. Alshaibani | Dhahran Eye Specialist Hospital, Dammam , KSA |
| Osama | AlSheikh | King Khaled Eye Specialist Hospital, Riyadh Saudi Arabia |
| Hunter K | Yuen | Hong Kong eye hospital , Hong Kong |
