A randomized trial of treatment for multisystem Langerhans' cell histiocytosis

Helmut Gadner, Nicole Grois, Maurizio Arico, Valerie Broadbent, Adriana Ceci, Ake Jakobson, Diane Komp, Jörg Michaelis, Stacy Nicholson, Ulrike Pötschger, Jon Pritchard, Stephan Ladisch

Research output: Contribution to journalArticlepeer-review

387 Scopus citations


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/m2, given intravenously every week) or etoposide (150 mg/m2/d, given intravenously for 3 days every 3 weeks), and a single initial close of corticosteroids. Results: Vinblastine and etoposide were equivalent (P ≥ .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 80%), 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 ≥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 close 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.

Original languageEnglish (US)
Pages (from-to)728-734
Number of pages7
JournalJournal of Pediatrics
Issue number5
StatePublished - 2001
Externally publishedYes

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health


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