title: Activity and toxicity of 2-CDA in Langerhans cell histiocytosis: a single institutional experience.
abstract: BACKGROUND
  Langerhans cell histiocytosis (LCH) is a rare disorder characterized by clonal proliferation of immature and abnormal bone marrow derived langerhans cells. Treatment is usually multimodal. Potent anti-monocyte as well as immunomodulatory activity of 2-CDA and its proven efficacy in many lymphoproliferative disorders has made 2-CDA a rational choice in treatment of LCH.
  
  
  OBJECTIVE
  To evaluate the efficacy and toxicity profile of 2-CDA in children with relapsed or refractory LCH.
  
  
  METHODS
  This is a pilot study and we present the initial data of the first seven patients treated at our institution.
  
  
  METHODS
  Seven patients of relapsed and refractory LCH were enrolled from July 2000 to June 2004. The cohort of seven patients included six males and one female with a median age at initiation of cladribine was 2.25 years (range, 1.67 to 7.0 years). Three patients had received one prior chemotherapy regimen while the rest were heavily pretreated. Cladribine was administered over two hours IV daily for five days and repeated every four weeks.
  
  
  RESULTS
  After a median of six courses of cladribine (range, 2 to 9), two (33%) patients achieved PR and two (33%) patients have SD on imaging but are clinically better. None experienced grade 3 or 4 hematologic toxicity. At a median follow-up of 19 months (range, 8 to 52 months), five patients remain alive and one patient has died.
  
  
  CONCLUSIONS
  Our study shows that single agent 2-CDA is active and well-tolerated in children with relapsed or refractory LCH.
authors.affiliation: Department of Medical Oncology, Tata Memorial Centre, Parel, Mumbai, India.
authors.forename: G
authors.initials: G
authors.lastname: Biswas
authors.forename: A
authors.initials: A
authors.lastname: Khadwal
authors.forename: B
authors.initials: B
authors.lastname: Arora
authors.forename: R
authors.initials: R
authors.lastname: Bhagwat
authors.forename: S D
authors.initials: SD
authors.lastname: Banavali
authors.forename: C N
authors.initials: CN
authors.lastname: Nair
authors.forename: S K
authors.initials: SK
authors.lastname: Pai
authors.forename: P A
authors.initials: PA
authors.lastname: Kurkure
authors.forename: P M
authors.initials: PM
authors.lastname: Parikh
mesh_terms: D015762:2-Chloroadenosine / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000276:immunology / Q000627:therapeutic use; D000964:Antimetabolites, Antineoplastic / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use; D002675:Child, Preschool; D017338:Cladribine / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D003839:Deoxyadenosines / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D013997:Time Factors
chemical_list: D000964:Antimetabolites, Antineoplastic; D000970:Antineoplastic Agents; D003839:Deoxyadenosines; D015762:2-Chloroadenosine; D017338:Cladribine; C055994:2-chloro-3'-deoxyadenosine