Language
English
Publication Date
5-1-2024
Journal
British Journal of Haematology
DOI
10.1111/bjh.19380
PMID
38501390
PMCID
PMC12700493
PubMedCentral® Posted Date
12-13-2025
PubMedCentral® Full Text Version
Author MSS
Abstract
Optimal therapeutic approaches for advanced Langerhans cell histiocytosis (LCH) are not known. We assessed the safety and efficacy of combined chemotherapy with MAPK pathway inhibition in 10 patients with refractory systemic disease and/or LCH-associated neurodegeneration. Overall response rate was 9/10 (90%) for the entire cohort: 5/5 (100%) for patients with systemic disease and 6/7 (86%) for patients with central nervous system disease. BRAFV600E+ peripheral blood fraction decreased in 5/6 (83%). Toxicities included fever, skin rash, myalgias, neuropathy, cytopenias and hypocalcaemia. Prospective trials are required to optimize combination strategies, determine potential to achieve cure and compare outcomes to chemotherapy or MAPK inhibitor monotherapy.
Keywords
Female, Humans, Male, Antineoplastic Combined Chemotherapy Protocols, Histiocytosis, Langerhans-Cell, MAP Kinase Signaling System, Protein Kinase Inhibitors, Proto-Oncogene Proteins B-raf, Recurrence, Treatment Outcome
Published Open-Access
yes
Recommended Citation
Karri, Vivekanudeep; Lin, Howard; Velazquez, Jessica; et al., "Clinical, Radiological and Molecular Responses to Combination Chemotherapy With Mapk Pathway Inhibition in Relapsed and Refractory Langerhans Cell Histiocytosis" (2024). Faculty, Staff and Students Publications. 6424.
https://digitalcommons.library.tmc.edu/baylor_docs/6424