Dramatic efficacy of vemurafenib in both multisystemic and refractory Erdheim-Chester disease and Langerhans cell histiocytosis harboring the BRAF V600E mutation

Author:

Haroche Julien12,Cohen-Aubart Fleur12,Emile Jean-François3,Arnaud Laurent12,Maksud Philippe4,Charlotte Frédéric5,Cluzel Philippe6,Drier Aurélie7,Hervier Baptiste12,Benameur Neïla8,Besnard Sophie9,Donadieu Jean10,Amoura Zahir12

Affiliation:

1. Department of Internal Medicine and French reference Center for Rare Auto-immune and Systemic Diseases, Assistance Publique–Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, Paris, France;

2. Université Pierre et Marie Curie, UPMC University Paris 6, Paris, France;

3. Research Unit (EA) EA4340 and Pathology Laboratory, Versailles University and AP-HP, Boulogne, France;

4. Departments of Nuclear Medicine,

5. Pathology,

6. Radiology,

7. Neuroradiology, and

8. Pharmacy, Hôpital Pitié-Salpêtrière, University Paris 6, AP-HP, Paris, France,

9. Department of Internal Medicine, Pontchaillou University Hospital, Rennes Cedex, France, and

10. Department of Pediatry, AP-HP, Centre de Référence des Histiocytoses, Hôpital Trousseau, Paris, France

Abstract

Key Points Treatment with vemurafenib induced a dramatic response in 3 patients with histiocytosis harboring BRAF V600E mutations. Tumor response was observed in both Erdheim-Chester disease and Langerhans cell histiocytosis.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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