Vemurafenib combined with chemotherapy achieved sustained remission in pediatric LCH: a multi-center observational study.

ABSTRACT:

Background

Langerhans cell histiocytosis (LCH) is a myeloid neoplasia with potentially fatal consequences, and about 2/3 of cases involve the BRAF^V600E kinase-activated mutation. Vemurafenib, a BRAF inhibitor, has demonstrated significant clinical improvements in LCH. However, the high relapse rate of LCH following cessation of vemurafenib therapy remains a major challenge, and alternative treatment strategies require further investigation.

Methods

In this retrospective multi-center study, we evaluated the efficacy and safety of vemurafenib combined with conventional chemotherapy in patients with severe or refractory LCH.

Results

Seventeen patients were enrolled in the study, with eleven classified as risk organ involvement (RO +). Six received the combinat

SUBMITTER: Lei J 

PROVIDER: S-EPMC10794359 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature