Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis
Deevyashali Parekh, Howard Lin, Akanksha Batajoo, Erin C. Peckham‐Gregory, Vivekanudeep Karri, Whitney Stanton, Brooks Scull, Ryan Fleishmann, Nader Kim El‐Mallawany, Olive S. Eckstein, Zachary D. Prudowsky, Nitya Gulati, Jennifer E. Agrusa, Asra Ahmed, Roland Chu, Matthew S. Dietz, Stanton Goldman, Michael D. Hogarty, Hamayun Imran, Stefanos Intzes, Jenny M. Kim, Lisa M. Kopp, Carolyn Fein Levy, Philip Neff, Pallavi Pillai, Bryan A. Sisk, Deborah Schiff, Angela D. Trobaugh‐Lotrario, Kelly Walkovich, Kenneth L. McClain, Carl E. Allen
Abstract
Summary 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%). BRAF V600E+ 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.