Efficacy of BRAF-Inhibitor Therapy in BRAFV600E-Mutated Adult Langerhans Cell Histiocytosis

Antonious Z. Hazim; Gordon J. Ruan; Aishwarya Ravindran; Jithma P. Abeykoon; Caleb Scheckel; Robert Vassallo; Jay H. Ryu; W. Oliver Tobin; Matthew J. Koster; N. Nora Bennani; Karen L. Rech; Jason R. Young; Mithun V. Shah; Gaurav Goyal; Ronald S. Go

doi:10.1002/onco.13541

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

Antonious Z. Hazim, Gordon J. Ruan, Aishwarya Ravindran, Jithma P. Abeykoon, Caleb Scheckel, Robert Vassallo, Jay H. Ryu, W. Oliver Tobin, Matthew J. Koster, N. Nora Bennani, Karen L. Rech, Jason R. Young, Mithun V. Shah, Gaurav Goyal, Ronald S. Go

Research output: Contribution to journal › Comment/debate › peer-review

2 Scopus citations

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 BRAF^V600E- 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.

Original language	English (US)
Pages (from-to)	1001-1004
Number of pages	4
Journal	Oncologist
Volume	25
Issue number	12
DOIs	https://doi.org/10.1002/onco.13541
State	Published - Dec 2020

Keywords

Dabrafenib
Histiocytosis
Targeted therapy
Vemurafenib

ASJC Scopus subject areas

General Medicine

Access to Document

10.1002/onco.13541

Cite this

Hazim, A. Z., Ruan, G. J., Ravindran, A., Abeykoon, J. P., Scheckel, C., Vassallo, R., Ryu, J. H., Tobin, W. O., Koster, M. J., Bennani, N. N., Rech, K. L., Young, J. R., Shah, M. V., Goyal, G., & Go, R. S. (2020). Efficacy of BRAF-Inhibitor Therapy in BRAF^V600E-Mutated Adult Langerhans Cell Histiocytosis. Oncologist, 25(12), 1001-1004. https://doi.org/10.1002/onco.13541

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title = "Efficacy of BRAF-Inhibitor Therapy in BRAFV600E-Mutated Adult Langerhans Cell Histiocytosis",

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.",

keywords = "Dabrafenib, Histiocytosis, Targeted therapy, Vemurafenib",

author = "Hazim, {Antonious Z.} and Ruan, {Gordon J.} and Aishwarya Ravindran and Abeykoon, {Jithma P.} and Caleb Scheckel and Robert Vassallo and Ryu, {Jay H.} and Tobin, {W. Oliver} and Koster, {Matthew J.} and Bennani, {N. Nora} and Rech, {Karen L.} and Young, {Jason R.} and Shah, {Mithun V.} and Gaurav Goyal and Go, {Ronald S.}",

note = "Publisher Copyright: {\textcopyright} AlphaMed Press 2020",

year = "2020",

month = dec,

doi = "10.1002/onco.13541",

language = "English (US)",

volume = "25",

pages = "1001--1004",

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T1 - Efficacy of BRAF-Inhibitor Therapy in BRAFV600E-Mutated Adult Langerhans Cell Histiocytosis

AU - Hazim, Antonious Z.

AU - Ruan, Gordon J.

AU - Ravindran, Aishwarya

AU - Abeykoon, Jithma P.

AU - Scheckel, Caleb

AU - Vassallo, Robert

AU - Ryu, Jay H.

AU - Tobin, W. Oliver

AU - Koster, Matthew J.

AU - Bennani, N. Nora

AU - Rech, Karen L.

AU - Young, Jason R.

AU - Shah, Mithun V.

AU - Goyal, Gaurav

AU - Go, Ronald S.

PY - 2020/12

Y1 - 2020/12

N2 - 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.

AB - 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.

KW - Dabrafenib

KW - Histiocytosis

KW - Targeted therapy

KW - Vemurafenib

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