Successful treatment of multisystem Langerhans cell histiocytosis (histiocytosis X) with etoposide.

Purpose: Langerhans cell histiocytosis (LCH) in its disseminated form usually occurs in the very young, and has a fulminant, rapidly progressive, and fatal course despite different forms of therapy.

Patients and methods: We treated two patients, who had failed on vinblastine treatment, with i.v. etoposide (VP-16) at a dose of 150 mg/kg/day for 3 days. Patient I, 8 months of age, presented with failure to thrive and huge bilateral granulomatous lesions of the external auditory canal with erosion and extensive destruction of the petrous pyramids and mastoid area. Patient II, 20 months of age, presented with widespread purpuric skin rash, hepatosplenomegaly, and bone marrow involvement.

Results: Both patients sustained complete remission (CR) following three to six courses of VP-16 and continued to be in unmaintained CR for > 48 months from diagnosis. No major toxicity was noted.

Conclusions: Etoposide (VP-16), an epipodophyllotoxin known for its usefulness in the treatment of malignancies of the monocyte/macrophage lineage, appears to be an effective treatment for the severe multisystem (disseminated) LCH of childhood and should be strongly considered as front-line therapy for this subgroup of patients with poor prognostic factors.