Details

Autor(en) / Beteiligte

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

Titel

A randomized trial of treatment for multisystem Langerhans’ cell histiocytosis

Ist Teil von

• The Journal of pediatrics, 2001-05, Vol.138 (5), p.728-734

Ort / Verlag

New York, NY: Mosby, Inc

Erscheinungsjahr

2001

Link zum Volltext

Quelle

Elsevier Journal Backfiles on ScienceDirect (DFG Nationallizenzen)

Beschreibungen/Notizen

• 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 dose 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 83%), 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 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. (J Pediatr 2001;138:728-34)