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LCH-I (1991-1995)

Patients with histologically confirmed multisystem LCH were randomly assigned to receive 24 weeks treatment of vinblastine (Arm A) or etoposide (Arm B).1 An initial pulse of high-dose methyl-prednisolone was given in both arms to treat constitutional symptoms. Five hundred and twenty-three patients were registered on LCH-I, of whom 210 had multisystem disease and 143 were randomized.


  • Vinblastine and Etoposide, used as monotherapy, are equally effective in MS-LCH
  • Used as single drugs vinblastine and etoposide failed to provide adequate disease control, as could be judged by the high rate (47%) of switch to alternative therapy before trial completion
  • Poor prognosis is clearly associated with involvement of at least one “risk organ” (liver, lungs, hematological system, and/or spleen) at diagnosis
  • Response at 6 weeks is a very powerful and independent predictor of outcome


1. Gadner H, Grois N, Arico M, Broadbent V, Ceci A, Jakobson A, et al. A randomized trial of treatment for multisystem Langerhans' cell histiocytosis. J Pediatr, 2001, 138(5):728-34.

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