Combined treatment with vemurafenib and cobimetinib in Langerhans cell histiocytosis and Erdheim-Chester disease overlap syndrome: A case Report

Hidalgo-Soto Marta*, Poza-Santaella María, Pita-Suárez Daniel, Calbacho-Robles María, Pina-Sánchez José, González-Medina José and Baumann Tycho Stephan

Histiocytoses are clonal disorders diseases derived from the monocyte-macrophage lineage. The Erdheim–Chester Disease (ECD) and Langerhans Cell Histiocytosis (LCH) may occur in association with overlapping clinical, histopathological and molecular features, harboring somatic MAP2K1 mutations in more than 50% of patients. BRAF and MEK inhibitors have shown to be efficacious in ECD and LCH, including responses in patients with CNS involvement.

This case report describes a 59-year-old woman who presented with vemurafenib-refractory ECD/LCH overlap syndrome treated with vemurafenib/cobimetinib dual therapy, with rapidly progressing neurological involvement after its initiation.

Although targeted therapy plays a crucial role in the treatment of histiocytosis, only anecdotal clinical cases treated with dual therapy have been reported in ECD or LCH and collaborative trials are needed to improve outcomes.

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Published on: Jan 20, 2022 Pages: 1-5

Full Text PDF Full Text HTML DOI: 10.17352/ahcrr.000035
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