Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis.
Deevyashali ParekhHoward LinAkanksha BatajooErin Peckham-GregoryVivekanudeep KarriWhitney StantonBrooks ScullRyan FleishmannNader El-MallawanyOlive S EcksteinZachary D PrudowskyNitya GulatiJennifer E AgrusaAsra Z AhmedRoland ChuMatthew S DietzStanton C GoldmanMichael D HogartyHamayun ImranStefanos IntzesJenny M KimLisa M KoppCarolyn Fein LevyPhilip NeffPallavi M PillaiBryan A SiskDeborah E SchiffAngela D Trobaugh-LotrarioKelly WalkovichKenneth L McClainCarl E AllenPublished in: British journal of haematology (2024)
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.
Keyphrases
- patient reported outcomes
- peripheral blood
- combination therapy
- open label
- acute lymphoblastic leukemia
- acute myeloid leukemia
- young adults
- cell therapy
- single cell
- clinical trial
- multiple myeloma
- study protocol
- hodgkin lymphoma
- stem cells
- big data
- machine learning
- newly diagnosed
- wild type
- data analysis
- chemotherapy induced