Language

English

Publication Date

5-1-2024

Journal

British Journal of Haematology

DOI

10.1111/bjh.19376

PMID

38501389

PMCID

PMC12690567

PubMedCentral® Posted Date

12-11-2025

PubMedCentral® Full Text Version

Author MSS

Abstract

Over 50% of patients with systemic LCH are not cured with front-line therapies and data to guide salvage options are limited. We describe 58 patients with LCH who were treated with clofarabine. Clofarabine monotherapy was active against LCH in this cohort including heavily pretreated patients with systemic objective response rate of 92.6%, higher in children (93.8%) than adults (83.3%). BRAFV600E+ variant allele frequency in peripheral blood correlated with positive clinical responses. Prospective multi-center trials are warranted to determine optimal dosing, long-term efficacy, late toxicities, relative cost and patient reported outcomes of clofarabine compared to alternative LCH salvage therapy strategies.

Keywords

Humans, Clofarabine, Histiocytosis, Langerhans-Cell, Male, Female, Adult, Adolescent, Child, Middle Aged, Child, Preschool, Young Adult, Aged, Recurrence, Proto-Oncogene Proteins B-raf, Infant, Treatment Outcome, Salvage Therapy, Adenine Nucleotides, Arabinonucleosides

Published Open-Access

yes

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