Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study
| Autor: | Antonella Cacchione MD, Francesco Fabozzi MD, Andrea Carai MD, PhD, Giovanna Stefania Colafati MD, Giada del Baldo MD, Sabrina Rossi MD, Martino Diana MD, Giacomina Megaro MD, Giuseppe Maria Milano MD, PhD, Marina Macchiaiolo MD, Alessandro Crocoli MD, Maria Antonietta De Ioris MD, Luigi Boccuto MD, Domitilla Elena Secco MD, Mario Zama MD, Emanuele Agolini MD, Paolo Tomà, Angela Mastronuzzi MD, PhD |
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| Jazyk: | angličtina |
| Rok vydání: | 2023 |
| Předmět: | |
| Zdroj: | Cancer Control, Vol 30 (2023) |
| Druh dokumentu: | article |
| ISSN: | 1526-2359 10732748 |
| DOI: | 10.1177/10732748221144930 |
| Popis: | 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. 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. 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. Conclusions In our experience, MEKi treatment has been shown to be both safe and effective in improving symptomatology and quality of life. |
| Databáze: | Directory of Open Access Journals |
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