Luvometinib in patients with Langerhans cell histiocytosis, Erdheim-Chester disease, and other histiocytic neoplasms: a single-arm, multicentre, phase 2 study.

ABSTRACT:

Background

Histiocytic neoplasms are a heterogeneous group of haematologic disorders marked by a high frequency of mutations in the somatic mitogen-activated protein kinase pathway. This single-arm, multicentre, phase 2 study evaluated the efficacy and safety of the selective MEK1/2 inhibitor luvometinib in adult patients with histiocytic neoplasms.

Methods

Patients (aged >16 years), regardless of tumour genotype and who were either treatment-naïve or relapse/refractory, were enrolled and received oral luvometinib, 8 mg, once daily in 28-day cycles until disease progression, death, unacceptable toxicity, withdrawal of consent, or end of the study. The primary end point was overall response rate (ORR) assessed by an independent review committee according to positron emission

SUBMITTER: Cao XX 

PROVIDER: S-EPMC12481021 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature