Vemurafenib for Refractory Multisystem Langerhans Cell Histiocytosis in Children: An International Observational Study.

ABSTRACT:

Purpose

Off-label use of vemurafenib (VMF) to treat BRAF^V600E mutation-positive, refractory, childhood Langerhans cell histiocytosis (LCH) was evaluated.

Patients and methods

Fifty-four patients from 12 countries took VMF 20 mg/kg/d. They were classified according to risk organ involvement: liver, spleen, and/or blood cytopenia. The main evaluation criteria were adverse events (Common Terminology Criteria for Adverse Events [version 4.3]) and therapeutic responses according to Disease Activity Score.

Results

LCH extent was distributed as follows: 44 with positive and 10 with negative risk organ involvement. Median age at diagnosis was 0.9 years (range, 0.1 to 6.5 years). Median age at VMF initiation was 1.8 years (range, 0.18 to 14 years), with a med

SUBMITTER: Donadieu J 

PROVIDER: S-EPMC6823889 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature