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Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study

Abstract : An international phase 2 study combining cladribine and cytarabine (Ara-C) was initiated for patients with refractory, risk-organ–positive Langerhans cell histiocytosis (LCH) in 2005. The protocol, comprising at least two 5-day courses of Ara-C (1 g/m2 per day) plus cladribine (9 mg/m2 per day) followed by maintenance therapy, was administered to 27 patients (median age at diagnosis, 0.7 years; median follow-up, 5.3 years). At inclusion, all patients were refractory after at least 1 course of vinblastine (VBL) plus corticosteroid, all had liver and spleen involvement, and 25 patients had hematologic cytopenia. After 2 courses, disease status was nonactive (n = 2), better (n = 23), or stable (n = 2), with an overall response rate of 92%. Median disease activity scores decreased from 12 at the start of therapy to 3 after 2 courses (P \textless .0001). During maintenance therapy, 4 patients experienced reactivation in risk organs. There were 4 deaths; 2 were related to therapy toxicity and 2 were related to reactivation. All patients experienced severe toxicity, with World Health Organization grade 4 hematologic toxicity and 6 documented severe infections. The overall 5-year survival rate was 85% (95% confidence interval, 65.2%-94.2%). Thus, the combination of cladribine/Ara-C is effective therapy for refractory multisystem LCH but is associated with high toxicity
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https://hal-univ-rennes1.archives-ouvertes.fr/hal-01231424
Contributor : Laurent Jonchère <>
Submitted on : Friday, November 20, 2015 - 11:29:02 AM
Last modification on : Friday, May 15, 2020 - 12:22:03 PM

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Jean Donadieu, Frédéric Bernard, Max Van Noesel, Mohamed Barkaoui, Odile Bardet, et al.. Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study. Blood, American Society of Hematology, 2015, 126 (12), pp.1415--1423. ⟨10.1182/blood-2015-03-635151⟩. ⟨hal-01231424⟩

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