title: [Cladribine for 13 cases refractory high-risk children Langerhans cell histiocytosis].
abstract: OBJECTIVE
  To observe the efficacy and adverse reaction of the improvement program of cladribine combined with cytarabine (2-CdA+Ara-C) in treatment of children with refractory high-risk Langerhans cell histiocytosis (LCH).
  
  
  METHODS
  13 patients with refractory high-risk LCH or recurrent LCH were treated by combined 2-CdA+Ara-C chemotherapy. The treatment efficacy and the disease state in the process were evaluated according to the Histiocyte Society Evaluation and Treatment Guidelines (2009). The drug toxicity was evaluated according to the Common Terminology Criteria Adverse Events Version 4.0 (CTCAE v4.0, 2009).
  
  
  RESULTS
  Of 13 patients, 10 cases achieved non active disease (NAD); 2 patients with liver cirrhosis before the improvement program with CIP-LCH-2012 gave up the treatment after 1 course of therapy; 1 patient died of infectious shock after chemotherapy with severe pulmonary infection and intestinal infection. All 13 patients had grade 3 of blood and lymphatic system toxicity; 10 patients had grade 1 of hepatobiliary and gastrointestinal side effects; 3 patients with liver cirrhosis before the improvement program had grade 2 or grade 3 of hepatobiliary system and gastrointestinal system side effects, including 1 patient of death.
  
  
  CONCLUSIONS
  The improvement program of CIP-LCH-2012 had significant efficacy for children with refractory high-risk and relapsed LCH. The cladribine-associated toxicity was of significant myelosuppression, which may be tolerated in the most children patients. The program could be considered as a recommended salvage therapy for multi-system LCH (MS-LCH) after failure of first-line therapy, and as a first-line therapy for MS-LCH with risk organ injury. The program should be used with caution or dose-adjustment consideration for pre-treatment of severe organ damage exist, especially cirrhosis.
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Tao
authors.initials: T
authors.lastname: Hu
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Rong
authors.initials: R
authors.lastname: Liu
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Junhui
authors.initials: J
authors.lastname: Li
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Jing
authors.initials: J
authors.lastname: Cao
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Lei
authors.initials: L
authors.lastname: Zhang
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Juanjuan
authors.initials: J
authors.lastname: Li
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Wei
authors.initials: W
authors.lastname: Fan
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Dixiao
authors.initials: D
authors.lastname: Zhong
authors.affiliation: The Department of Hematology, The Capital Institute of Pediatrics, Beijing　100020, China.
authors.forename: Xiaodong
authors.initials: X
authors.lastname: Shi
mesh_terms: D002648:Child; D017338:Cladribine / Q000627:therapeutic use*; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D012307:Risk Factors; D016879:Salvage Therapy
chemical_list: D007166:Immunosuppressive Agents; D017338:Cladribine