@article{fe0907356b484c14b12a08bf68cdeff9,
title = "Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis",
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 a systemic objective response rate of 92.6%, higher in children (93.8%) than adults (83.3%). BRAFV600E+ variant allele frequency in peripheral blood is correlated with clinical responses. Prospective multicentre 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 = "Langerhans cell histiocytosis, clofarabine, myeloid neoplasia",
author = "Deevyashali Parekh and Howard Lin and Akanksha Batajoo and Erin Peckham-Gregory and Vivekanudeep Karri and Whitney Stanton and Brooks Scull and Ryan Fleishmann and Nader El-Mallawany and Eckstein, {Olive S.} and Prudowsky, {Zachary D.} and Nitya Gulati and Agrusa, {Jennifer E.} and Ahmed, {Asra Z.} and Roland Chu and Matthew S Dietz and Stanton C Goldman and Hogarty, {Michael D.} and Hamayun Imran and Stefanos Intzes and Jenny M Kim and Kopp, {Lisa M.} and Levy, {Carolyn Fein} and Philip Neff and Pillai, {Pallavi M.} and Sisk, {Bryan A.} and Schiff, {Deborah E.} and Angela D Trobaugh-Lotrario and Kelly Walkovich and McClain, {Kenneth L.} and Allen, {Carl E.}",
note = "Publisher Copyright: {\textcopyright} 2024 British Society for Haematology and John Wiley & Sons Ltd.",
year = "2024",
month = may,
doi = "10.1111/bjh.19376",
language = "English",
volume = "204",
pages = "1888--1893",
journal = "British Journal of Haematology",
issn = "0007-1048",
number = "5",
}