Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium.

ABSTRACT: Histiocytic disorders are rare hematologic neoplasms characterized by a notable dependence on mitogen-activated protein kinase signaling. Targeted therapy is an emerging treatment option, yet the number of reported patients remains limited. Here, we describe 40 patients with histiocytic neoplasms who were treated with targeted therapy in 7 tertiary referral hospitals from the Netherlands and Belgium. The cohort comprised of 6 (15%) children and 34 (85%) adults with diverse histiocytoses, including Langerhans cell histiocytosis (LCH; n = 12), Erdheim-Chester disease (n = 14), central nervous system xanthogranuloma (n = 2), Rosai-Dorfman disease (n = 3), histiocytic sarcoma (n = 2), ALK-positive histiocytosis (n = 1), and mixed/unclassifiable histiocytosis (n = 6). Five patients were include

SUBMITTER: Kemps PG 

PROVIDER: S-EPMC12096411 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature