Back to Search
Start Over
Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study.
- Source :
-
Cancer control : journal of the Moffitt Cancer Center [Cancer Control] 2023 Jan-Dec; Vol. 30, pp. 10732748221144930. - Publication Year :
- 2023
-
Abstract
- Introduction: Plexiform neurofibromas (PN) represent the main cause of morbidity in patients affected by Neurofibromatosis Type 1 (NF1). Until recently, surgery has been the main treatment option in these patients, but it is burdened with a low efficacy rate and a high incidence of side effects as well as recurrence. In recent years, MEK inhibitors (MEKi) such as selumetinib and trametinib have shown great promise.<br />Methods: We retrospectively describe a single center cohort of NF1 patients affected by PN1 and treated with MEKi since 2019 to 2021. Patients recruited in the study were affected by PN that were not eligible to complete surgical excision, symptomatic or with major cosmetic deformation or functional neurological deficits.<br />Results: Most patients experienced improvement in clinical symptoms and quality of life, with reduction or stabilization of lesions. However, no complete response was achieved. The most common adverse effects involved the skin, affecting every patient. Importantly, no life-threatening adverse effects occurred.<br />Conclusions: In our experience, MEKi treatment has been shown to be both safe and effective in improving symptomatology and quality of life.
- Subjects :
- Humans
Retrospective Studies
Quality of Life
Protein Kinase Inhibitors adverse effects
Mitogen-Activated Protein Kinase Kinases therapeutic use
Neurofibroma, Plexiform drug therapy
Neurofibroma, Plexiform pathology
Neurofibroma, Plexiform surgery
Neurofibromatosis 1 drug therapy
Neurofibromatosis 1 chemically induced
Neurofibromatosis 1 pathology
Subjects
Details
- Language :
- English
- ISSN :
- 1526-2359
- Volume :
- 30
- Database :
- MEDLINE
- Journal :
- Cancer control : journal of the Moffitt Cancer Center
- Publication Type :
- Academic Journal
- Accession number :
- 36598023
- Full Text :
- https://doi.org/10.1177/10732748221144930