A randomized trial of treatment for multisystem Langerhans' cell histiocytosis

Objective: To compare 2 active agents, vinblastine and etoposide, in the tr eatment of multisystem Langerhans' cell histiocytosis (LCH) in an intel-nat ional randomized study. Study design: One hundred forty-three untreated patients were randomly assi gned to receive 24 weeks of vinblastine (6 mg/m(2), given intravenously eve ry 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 greater than or equal to 2.2) 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 del e loping permanent consequences (39% and 51%) including diabetes insipidus (2 2% and 23%). LCH reactivations were usually mild, as was toxicity. All chil dren greater than or equal to2 years old without risk organ involvement (li ver, lungs, hematopoietic system, or spleen) survived. With such involvemen t, lack of rapid (within 6 weeks) response was identified as a new prognost ic indicator, predicting a high (66%,) mortality rate. Conclusions: Vinblastine and etoposide, with one dose of corticosteroids, a re equally effective treatments for multisystem LCH, but patients who do no t respond within 6 weeks are at increased risk for treatment failure and ma y require different therapy.