Cutaneous adverse events in children treated with vemurafenib for refractory BRAF
BRAF inhibitor
Langerhans cell histiocytosis
cutaneous adverse event
melanoma
neutrophilic panniculitis
pediatric
photosensitivity
rash
skin toxicity
squamous cell carcinoma
vemurafenib
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
09 2021
09 2021
Historique:
revised:
16
04
2021
received:
28
12
2020
accepted:
29
04
2021
pubmed:
11
6
2021
medline:
17
3
2022
entrez:
10
6
2021
Statut:
ppublish
Résumé
The somatic BRAF Multicentric retrospective observational study including patients <18 years treated with VMF alone for refractory BRAF Fifty-seven patients: 56% female, median age 2.1 years (0.2-14.6), median treatment duration 4.1 months (1.4-29.7). Forty-one patients (72%) had at least one CAE: photosensitivity (40%), keratosis pilaris (32%), rash (26%), xerosis (21%), and neutrophilic panniculitis (16%). No skin tumor was observed. Five percent of CAEs were grade 3. None were grade 4 or led to permanent VMF discontinuation. Dose reduction was necessary for 12% of patients, temporary treatment discontinuation for 16%, none leading to loss of efficacy. VMF dose, median RPL, and efficacy were not correlated with CAE occurrence. At doses used for pediatric LCH, CAEs are frequent but rarely severe and have little impact on the continuation of treatment when managed appropriately. Regular dermatological follow-up is essential to manage CAEs and screen for possible induced skin tumors.
Sections du résumé
BACKGROUND
The somatic BRAF
PROCEDURE
Multicentric retrospective observational study including patients <18 years treated with VMF alone for refractory BRAF
RESULTS
Fifty-seven patients: 56% female, median age 2.1 years (0.2-14.6), median treatment duration 4.1 months (1.4-29.7). Forty-one patients (72%) had at least one CAE: photosensitivity (40%), keratosis pilaris (32%), rash (26%), xerosis (21%), and neutrophilic panniculitis (16%). No skin tumor was observed. Five percent of CAEs were grade 3. None were grade 4 or led to permanent VMF discontinuation. Dose reduction was necessary for 12% of patients, temporary treatment discontinuation for 16%, none leading to loss of efficacy. VMF dose, median RPL, and efficacy were not correlated with CAE occurrence.
CONCLUSIONS
At doses used for pediatric LCH, CAEs are frequent but rarely severe and have little impact on the continuation of treatment when managed appropriately. Regular dermatological follow-up is essential to manage CAEs and screen for possible induced skin tumors.
Substances chimiques
Protein Kinase Inhibitors
0
Vemurafenib
207SMY3FQT
BRAF protein, human
EC 2.7.11.1
Proto-Oncogene Proteins B-raf
EC 2.7.11.1
Types de publication
Journal Article
Observational Study
Langues
eng
Sous-ensembles de citation
IM
Pagination
e29140Informations de copyright
© 2021 Wiley Periodicals LLC.
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