A circulating subset of BRAFV600E‐positive cells in infants with high‐risk Langerhans cell histiocytosis treated with BRAF inhibitors

Summary 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 o...

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Bibliographic Details
Published in:British journal of haematology 2021-08, Vol.194 (4), p.745-749
Main Authors: Poch, Rita, Le Louet, Solenne, Hélias‐Rodzewicz, Zofia, Hachem, Nawa, Plat, Geneviève, Barkaoui, Mohamed‐Aziz, Lapillonne, Hélène, Delhommeau, François, Emile, Jean‐François, Donadieu, Jean, Héritier, Sébastien
Format: Article
Language:English
Subjects:
Biomarker
Blood cells
BRAF
circulating tumour cells
Dendritic cells
Hematology
Histiocytosis
Infants
Langerhans cell histiocytosis
Life Sciences
Lymphocytes B
Lymphocytes T
Monocytes
Peripheral blood
targeted therapy
Weaning
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Summary:Summary 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.
ISSN:0007-1048
1365-2141
DOI:10.1111/bjh.17721