A circulating subset of BRAFV600E -positive cells in infants with high-risk Langerhans cell histiocytosis treated with BRAF inhibitors.
Br J Haematol
; 194(4): 745-749, 2021 08.
Article
em En
| MEDLINE
| ID: mdl-34312844
ABSTRACT
BRAF inhibitors are an effective treatment for BRAFV600E -mutated, risk-organ-positive Langerhans cell histiocytosis (RO+ LCH). However, cell-free BRAFV600E DNA often persists during therapy and recurrence frequently occurs after therapy discontinuation. To identify a pathological reservoir of BRAFV600E -mutated cells, we studied peripheral blood cells obtained from six infants with RO+ multisystem (MS) LCH that received targeted therapy. After cell sorting, the BRAFV600E mutation was detected in monocytes (n = 5), B lymphocytes (n = 3), T lymphocytes (n = 2), and myeloid and plasmacytoid dendritic cells (n = 2 each). This biomarker may offer an interesting tool for monitoring the effectiveness of new therapeutic approaches for weaning children with RO+ LCH from targeted therapy.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Histiocitose de Células de Langerhans
/
Mutação Puntual
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Proteínas Proto-Oncogênicas B-raf
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Inibidores de Proteínas Quinases
Tipo de estudo:
Etiology_studies
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Risk_factors_studies
Limite:
Child
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Child, preschool
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Humans
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Infant
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article