2-Chlorodeoxyadenosine therapy for disseminated Langerhans cell histiocytosis

Mayo Clin Proc. 2003 Mar;78(3):301-6. doi: 10.4065/78.3.301.


Objective: To evaluate the efficacy of 2-chlorodeoxyadenosine (2-CDA), a purine nucleoside analogue, in treating disseminated Langerhans cell histiocytosis (LCH).

Patients and methods: We retrospectively reviewed the clinical records of 5 patients who were seen at our institution for histologically confirmed disseminated LCH, including 1 patient with central nervous system parenchymal involvement. These patients were treated consecutively with 2-CDA chemotherapy between December 1994 and January 2001. The patients ranged in age from 19 to 81 years, and the median pretreatment duration of disease was 23 months. Median follow-up after initiation of 2-CDA treatment was 33 months. 2-Chlorodeoxyadenosine was used as frontline therapy for 1 patient and as salvage therapy for the other patients. Patients generally received 0.7 mg/kg over 5 or 7 days; the median number of courses was 4.

Results: Complete responses were achieved in 3 patients, including the patient with central nervous system disease, which, to our knowledge, has not been described previously. Two other patients achieved partial responses. The overall response rate was 100%. Toxic effects consisted mainly of myelosuppression; 1 patient developed dermatomal herpes zoster infection.

Conclusion: Our experience confirms the reported efficacy of 2-CDA in the treatment of LCH; however, the optimal timing and schedule of therapy remain to be determined.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Bone Marrow / drug effects
  • Brain Diseases / drug therapy
  • Brain Diseases / etiology
  • Cladribine / therapeutic use*
  • Female
  • Herpes Zoster / chemically induced
  • Histiocytosis, Langerhans-Cell / complications
  • Histiocytosis, Langerhans-Cell / drug therapy*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Middle Aged
  • Retrospective Studies


  • Immunosuppressive Agents
  • Cladribine