A randomized trial of treatment for multisystem Langerhans' cell histiocytosis

J Pediatr. 2001 May;138(5):728-34. doi: 10.1067/mpd.2001.111331.


Objective: To compare 2 active agents, vinblastine and etoposide, in the treatment of multisystem Langerhans' cell histiocytosis (LCH) in an international randomized study.

Study design: One hundred forty-three untreated patients were randomly assigned to receive 24 weeks of vinblastine (6 mg/m(2), given intravenously every week) or etoposide (150 mg/m(2)/d, given intravenously for 3 days every 3 weeks), and a single initial dose of corticosteroids.

Results: Vinblastine and etoposide were equivalent (P > or = .2) in all respects: response at week 6 (57% and 49%); response at the last evaluation (58% and 69%); toxicity (47% and 58%); and probability of survival (76% and 83%) [corrected], of disease reactivation (61% and 55%), and of developing permanent consequences (39% and 51%) including diabetes insipidus (22% and 23%). LCH reactivations were usually mild, as was toxicity. All children > or = 2 years old without risk organ involvement (liver, lungs, hematopoietic system, or spleen) survived. With such involvement, lack of rapid (within 6 weeks) response was identified as a new prognostic indicator, predicting a high (66%) mortality rate.

Conclusions: Vinblastine and etoposide, with one dose of corticosteroids, are equally effective treatments for multisystem LCH, but patients who do not respond within 6 weeks are at increased risk for treatment failure and may require different therapy.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Child
  • Drug Therapy, Combination
  • Etoposide / adverse effects
  • Etoposide / therapeutic use*
  • Histiocytosis, Langerhans-Cell / drug therapy*
  • Humans
  • Methylprednisolone / therapeutic use*
  • Risk Assessment
  • Survival Analysis
  • Treatment Outcome
  • Vinblastine / adverse effects
  • Vinblastine / therapeutic use*


  • Vinblastine
  • Etoposide
  • Methylprednisolone