Efficacy ofBRAF-InhibitorTherapy in BRAFV600E-MutatedAdult Langerhans Cell Histiocytosis

被引：28

作者：

Hazim, Antonious Z. ^{[4
]}

Ruan, Gordon J. ^{[4
]}

Ravindran, Aishwarya ^{[5
]}

Abeykoon, Jithma P. ^{[1
]}

Scheckel, Caleb ^{[1
]}

Vassallo, Robert ^{[2
]}

Ryu, Jay H. ^{[2
]}

Tobin, W. Oliver ^{[7
]}

Koster, Matthew J. ^{[3
]}

Bennani, N. Nora ^{[1
]}

Rech, Karen L. ^{[5
]}

Young, Jason R. ^{[6
]}

Shah, Mithun V. ^{[1
]}

Goyal, Gaurav ^{[8
,9
]}

Go, Ronald S. ^{[1
]}

机构：

[1] Mayo Clin, Div Hematol, 200 First St SW, Rochester, MN 55905 USA

[2] Mayo Clin, Div Pulm & Crit Care Med, Rochester, MN USA

[3] Mayo Clin, Div Rheumatol, Rochester, MN USA

[4] Mayo Clin, Div Internal Med, Rochester, MN USA

[5] Mayo Clin, Div Lab Med & Pathol, Rochester, MN USA

[6] Mayo Clin, Div Radiol, Rochester, MN USA

[7] Mayo Clin, Div Neurol, Rochester, MN USA

[8] Mayo Clin, Mayo Clin Histiocytosis Working Grp, Dept Res, Rochester, MN USA

[9] Univ Alabama Birmingham, Div Hematol Oncol, 2000 6th Ave S, Birmingham, AL 35233 USA

来源：

ONCOLOGIST | 2020年 / 25卷 / 12期

关键词：

Vemurafenib; Dabrafenib; Targeted therapy; Histiocytosis; OPEN-LABEL; BRAF; VEMURAFENIB; MELANOMA; PHASE-3; DISEASE;

D O I：

10.1002/onco.13541

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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 withBRAF(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.

引用

页码：1001 / 1004

页数：4

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