title: Multi-centre pilot study of 2-chlorodeoxyadenosine and cytosine arabinoside combined chemotherapy in refractory Langerhans cell histiocytosis with haematological dysfunction.
abstract: 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 5d 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 Two septic deaths occurred shortly after administration of the first course of 2-CdA/Ara-C; a third patient was withdrawn from the trial after the first course and subsequently died following haematopoietic stem cell transplantation.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.
authors.affiliation: Service d'Hémato-Oncologie Pédiatrique, Hôpital Arnaud de Villeneuve, CHU de Montpellier, 34000 Montpellier, France. f-bernard@chu-montpellier.fr
authors.forename: F
authors.initials: F
authors.lastname: Bernard
authors.forename: C
authors.initials: C
authors.lastname: Thomas
authors.forename: Y
authors.initials: Y
authors.lastname: Bertrand
authors.forename: M
authors.initials: M
authors.lastname: Munzer
authors.forename: J
authors.initials: J
authors.lastname: Landman Parker
authors.forename: M
authors.initials: M
authors.lastname: Ouache
authors.forename: V Minard
authors.initials: VM
authors.lastname: Colin
authors.forename: Y
authors.initials: Y
authors.lastname: Perel
authors.forename: P
authors.initials: P
authors.lastname: Chastagner
authors.forename: C
authors.initials: C
authors.lastname: Vermylen
authors.forename: J
authors.initials: J
authors.lastname: Donadieu
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002908:Chronic Disease; D017338:Cladribine / Q000008:administration & dosage; D003561:Cytarabine / Q000008:administration & dosage; D004359:Drug Therapy, Combination; D005260:Female; D006402:Hematologic Diseases / Q000150:complications*; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D007262:Infusions, Intravenous; D008297:Male; D010865:Pilot Projects; D016019:Survival Analysis
chemical_list: D003561:Cytarabine; D017338:Cladribine