Multi-centre pilot study of 2-chlorodeoxyadenosine and cytosine arabinoside combined chemotherapy in refractory Langerhans cell histiocytosis with haematological dysfunction

• Published in: European journal of cancer (1990) Vol. 41; no. 17; pp. 2682 - 2689
• Main Authors: Bernard, F., Thomas, C., Bertrand, Y., Munzer, M., Landman Parker, J., Ouache, M., Colin, V. Minard, Perel, Y., Chastagner, P., Vermylen, C., Donadieu, J.
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.11.2005
• Subjects: 2-CdA; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Ara-C; Chronic Disease; Cladribine - administration & dosage; Cytarabine - administration & dosage; Drug Therapy, Combination; Female; Haematopoietic dysfunction; Hematologic Diseases - complications; Histiocytosis, Langerhans-Cell - drug therapy; Humans; Infant; Infusions, Intravenous; Langerhans cell histiocytosis; Male; Pilot Projects; Pilot study; Survival Analysis; Thrombocytopaenia; Pilot study; 2-CdA; Langerhans cell histiocytosis; Haematopoietic dysfunction; Thrombocytopaenia; Ara-C
• ISSN: 0959-8049; 1879-0852
• DOI: 10.1016/j.ejca.2005.02.007

The aim of this study was to assess the efficacy and adverse effects of 2-chlorodeoxyadenosine (2-CdA) and cytosine arabinoside (Ara-C) in children with refractory Langerhans cell histiocytosis (LCH) and haematopoietic dysfunction. Ten patients, with a median age at diagnosis of 0.5 years, were enrolled in this study. Treatment comprised at least two courses of Ara-C (1000 mg/m 2/d) and 2-CdA (9 mg/m 2/d) administered for 5 d every 4 weeks; subsequent median follow-up was 2.8 years (range 0.03–6.4 years). Among the 7 patients who received at least two courses of therapy, disease activity decreased in 6 patients, and control of disease was achieved in all patients after a median delay of 5.5 months. All patients suffered World Health Organisation (WHO) grade 4 haematological toxicity. Two septic deaths occurred shortly after administration of the first course of 2-CdA/Ara-C; a third patient was withdrawn from the trial after the first course and subsequently died following haematopoietic stem cell transplantation. This series is small, but we conclude that 2-CdA and Ara-C combined chemotherapy probably has major activity in childhood refractory Langerhans cell histiocytosis.