Langerhans cell histiocytosis: a 16-year experience

Objectives: To describe the clinical course of Langerhans cell histiocytosis and to compare its outcome according to age, staging of the disease and treatment response. Methods: Retrospective analysis of data on 33 children with Langerhans cell histiocytosis followed at Hospital das Clinicas, Universidade Federal de Minas Gerais, Brazil, between 1988 and 2004. Results: Age at diagnosis ranged from 2 months to 16 years (median: 2.5 years). Seventeen children were male, The follow-up period varied from 21 days to 16.2 years (median: 3.4 years). The most common clinical manifestations at diagnosis were osteolytic lesions, enlarged lymph nodes and skin lesions. The overall survival rate for the whole group was 86.1% at 16 years (95%CI 66.6-94.6%). Deaths occurred in patients with multisystem disease and organ dysfunction at diagnosis. Those patients who had a "better" response to treatment in the sixth week were likely to have a significantly higher overall survival rate than those who showed disease progression. Overall survival rate was significantly higher for patients with single-system disease. The disease-free survival rate for the whole group was 30.9% at 16 years (95%CI 15.6-47.5%), and was significantly higher for those with single-system disease. Age groups were not associated with different disease-free survival rates. Diabetes insipidus was the most common sequela. No cases of secondary neoplasms were observed. Conclusion: The clinical manifestations of Langerhans cell histiocytosis vary widely, with a high relapse rate and low mortality rate.