Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis

Deevyashali Parekh, Howard Lin, Akanksha Batajoo, Erin Peckham-Gregory, Vivekanudeep Karri, Whitney Stanton, Brooks Scull, Ryan Fleishmann, Nader El-Mallawany, Olive S. Eckstein, Zachary D. Prudowsky, Nitya Gulati, Jennifer E. Agrusa, Asra Z. Ahmed, Roland Chu, Matthew S Dietz, Stanton C Goldman, Michael D. Hogarty, Hamayun Imran, Stefanos IntzesJenny M Kim, Lisa M. Kopp, Carolyn Fein Levy, Philip Neff, Pallavi M. Pillai, Bryan A. Sisk, Deborah E. Schiff, Angela D Trobaugh-Lotrario, Kelly Walkovich, Kenneth L. McClain, Carl E. Allen

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)1888-1893
Number of pages6
JournalBritish Journal of Haematology
Volume204
Issue number5
DOIs
StatePublished - May 2024

Keywords

  • Langerhans cell histiocytosis
  • clofarabine
  • myeloid neoplasia

Fingerprint

Dive into the research topics of 'Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis'. Together they form a unique fingerprint.

Cite this