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


METHODS
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.
Gadner
H
H
St Anna Children's Hospital, Vienna, Austria.
Grois
N
N
Arico
M
M
Broadbent
V
V
Ceci
A
A
Jakobson
A
A
Komp
D
D
Michaelis
J
J
Nicholson
S
S
Pötschger
U
U
Pritchard
J
J
Ladisch
S
S
D000293:Adolescent; D002648:Child; D004359:Drug Therapy, Combination; D005047:Etoposide / Q000009:adverse effects / Q000627:therapeutic use*; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy*; D006801:Humans; D008775:Methylprednisolone / Q000627:therapeutic use*; D018570:Risk Assessment; D016019:Survival Analysis; D016896:Treatment Outcome; D014747:Vinblastine / Q000009:adverse effects / Q000627:therapeutic use*
D014747:Vinblastine; D005047:Etoposide; D008775:Methylprednisolone