Open Access
Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study
Antonella Cacchione
1
,
Francesco Fabozzi
1, 2
,
Andrea Carai
3
,
Giovanna Stefania Colafati
4
,
Giada Del Baldo
1
,
Sabrina Rossi
5
,
Diana Martino
1
,
Giacomina Megaro
1
,
Giuseppe Maria Milano
1
,
Marina Macchiaiolo
6
,
Alessandro Crocoli
7
,
Maria Antonietta De Ioris
1
,
Luigi Boccuto
8
,
Domitilla Elena Secco
9
,
Mario Zama
7
,
Emanuele Agolini
10
,
Paolo Tomà
4
,
Angela Mastronuzzi
1, 11
1
Department of Hematology/Oncology, Cell Therapy, Gene Therapies and Hemopoietic Transplant, Bambino Gesù Children’s Hospital, Rome, Italy
|
3
Neurosurgery Unit, Department of Neuroscience, Bambino Gesù Children’s Hospital, Rome, Italy
|
5
Pathology Unit, Department of Laboratories, Bambino Gesù Children’s Hospital, Rome, Italy
|
6
Rare Diseases and Medical Genetics Unit, Academic Department of Pediatrics, Bambino Gesù Children’s Hospital, Rome, Italy
|
7
Surgery Department, Bambino Gesù Children’s Hospital, Rome, Italy
|
9
PsD of Department of Paediatric Haematology/Oncology, Bambino Gesù Children’s Hospital, Rome, Italy
|
10
Laboratory of Medical Genetics, Bambino Gesù Children’s Hospital, Rome, Italy
|
11
Unicamillus, Saint Camillus International University of Health Sciences, Rome, Italy
|
Publication type: Journal Article
Publication date: 2023-01-04
scimago Q2
wos Q3
SJR: 0.881
CiteScore: 4.0
Impact factor: 2.6
ISSN: 10732748, 15262359
PubMed ID:
36598023
Oncology
General Medicine
Hematology
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.
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.
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Metrics
11
Total citations:
11
Citations from 2024:
11
(100%)
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GOST
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Cacchione A. et al. Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study // Cancer Control. 2023. Vol. 30. p. 107327482211449.
GOST all authors (up to 50)
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Cacchione A., Fabozzi F., Carai A., Colafati G. S., Baldo G. D., Rossi S., Martino D., Megaro G., Milano G. M., Macchiaiolo M., Crocoli A., De Ioris M. A., Boccuto L., Secco D. E., Zama M., Agolini E., Tomà P., Mastronuzzi A. Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study // Cancer Control. 2023. Vol. 30. p. 107327482211449.
Cite this
RIS
Copy
TY - JOUR
DO - 10.1177/10732748221144930
UR - https://journals.sagepub.com/doi/10.1177/10732748221144930
TI - Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study
T2 - Cancer Control
AU - Cacchione, Antonella
AU - Fabozzi, Francesco
AU - Carai, Andrea
AU - Colafati, Giovanna Stefania
AU - Baldo, Giada Del
AU - Rossi, Sabrina
AU - Martino, Diana
AU - Megaro, Giacomina
AU - Milano, Giuseppe Maria
AU - Macchiaiolo, Marina
AU - Crocoli, Alessandro
AU - De Ioris, Maria Antonietta
AU - Boccuto, Luigi
AU - Secco, Domitilla Elena
AU - Zama, Mario
AU - Agolini, Emanuele
AU - Tomà, Paolo
AU - Mastronuzzi, Angela
PY - 2023
DA - 2023/01/04
PB - SAGE
SP - 107327482211449
VL - 30
PMID - 36598023
SN - 1073-2748
SN - 1526-2359
ER -
Cite this
BibTex (up to 50 authors)
Copy
@article{2023_Cacchione,
author = {Antonella Cacchione and Francesco Fabozzi and Andrea Carai and Giovanna Stefania Colafati and Giada Del Baldo and Sabrina Rossi and Diana Martino and Giacomina Megaro and Giuseppe Maria Milano and Marina Macchiaiolo and Alessandro Crocoli and Maria Antonietta De Ioris and Luigi Boccuto and Domitilla Elena Secco and Mario Zama and Emanuele Agolini and Paolo Tomà and Angela Mastronuzzi},
title = {Safety and Efficacy of Mek Inhibitors in the Treatment of Plexiform Neurofibromas: A Retrospective Study},
journal = {Cancer Control},
year = {2023},
volume = {30},
publisher = {SAGE},
month = {jan},
url = {https://journals.sagepub.com/doi/10.1177/10732748221144930},
pages = {107327482211449},
doi = {10.1177/10732748221144930}
}