Efficacy of BRAF-Inhibitor Therapy in BRAF V600E-Mutated Adult Langerhans Cell Histiocytosis

Author:

Hazim Antonious Z.1,Ruan Gordon J.1,Ravindran Aishwarya2,Abeykoon Jithma P.3,Scheckel Caleb3,Vassallo Robert4,Ryu Jay H.4,Tobin W. Oliver5,Koster Matthew J.6,Bennani N. Nora3,Rech Karen L.2,Young Jason R.7,Shah Mithun V.3,Goyal Gaurav89,Go Ronald S.3

Affiliation:

1. Division of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA

2. Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA

3. Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA

4. Division of Pulmonary and Critical Care Medicine, Mayo Clinic, Rochester, Minnesota, USA

5. Division of Neurology, Mayo Clinic, Rochester, Minnesota, USA

6. Division of Rheumatology, Mayo Clinic, Rochester, Minnesota, USA

7. Division of Radiology, Mayo Clinic, Rochester, Minnesota, USA

8. Division of Hematology-Oncology, University of Alabama at Birmingham, Birmingham, Alabama, USA

9. Department of Research Collaborator (limited-tenure), on behalf of the Mayo Clinic Histiocytosis Working Group Mayo Clinic, Rochester, Minnesota, USA

Abstract

Abstract Langerhans cell histiocytosis (LCH) is a rare histiocytic neoplasm. To date, there is a lack of U.S. Food and Drug Administration–approved treatments in adult LCH to establish optimal first-line therapy. We conducted a retrospective, single-center case series evaluating the use of BRAF inhibitors in adult patients with BRAFV600E- LCH proven by biopsy. Our case series is the first to report the use of BRAF inhibitors as first-line therapy in adults with LCH. We also report the efficacy with single-agent dabrafenib in adult LCH. All but one of our patients had favorable response to targeted therapy.

Funder

University of Iowa/Mayo Clinic Lymphoma

Publisher

Oxford University Press (OUP)

Subject

Cancer Research,Oncology

Reference13 articles.

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2. Langerhans cell histiocytosis;Rodriguez-Galindo;Blood,2020

3. Recurrent BRAF mutations in Langerhans cell histiocytosis;Badalian-very;Blood,2010

4. Vemurafenib for BRAF V600-mutant Erdheim-Chester disease and langerhans cell histiocytosis analysis of data from the histology-independent, phase 2, open-label VE-BASKET study;Diamond;JAMA Oncol,2018

5. Major response to vemurafenib in patient with severe cutaneous Langerhans cell histiocytosis harboring BRAF V600E mutation;Charles;J Am Acad Dermatol,2014

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