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. 2024 May;204(5):1888-1893.
doi: 10.1111/bjh.19376. Epub 2024 Mar 19.

Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis

Deevyashali Parekh  1   2 , Howard Lin  1   2 , Akanksha Batajoo  1   2 , Erin Peckham-Gregory  1   2 , Vivekanudeep Karri  1   2 , Whitney Stanton  1   2 , Brooks Scull  1   2 , Ryan Fleishmann  1   2 , Nader El-Mallawany  1   2 , Olive S Eckstein  1   2 , Zachary D Prudowsky  1   2 , Nitya Gulati  1   2   3 , Jennifer E Agrusa  1   2   4 , Asra Z Ahmed  5 , Roland Chu  6 , Matthew S Dietz  7 , Stanton C Goldman  8 , Michael D Hogarty  9 , Hamayun Imran  10 , Stefanos Intzes  11 , Jenny M Kim  12 , Lisa M Kopp  13 , Carolyn Fein Levy  14 , Philip Neff  15 , Pallavi M Pillai  16 , Bryan A Sisk  17 , Deborah E Schiff  12 , Angela D Trobaugh-Lotrario  11 , Kelly Walkovich  4 , Kenneth L McClain  1   2 , Carl E Allen  1   2
Affiliations
Free article

Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis

Deevyashali Parekh et al. Br J Haematol. 2024 May.
Free article

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.

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References

REFERENCES

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