DISEASE COURSE AND LATE SEQUELAE OF LANGERHANS CELL HISTIOCYTOSIS - 25-YEAR EXPERIENCE AT THE UNIVERSITY-OF-CALIFORNIA, SAN-FRANCISCO

Purpose: The purpose of our investigation was to correlate the extent and degree of organ involvement at presentation of Langerhans' cell hi stiocytosis (LCH) with subsequent disease course, survival, and late s equelae. Materials and Methods: The medical records of 71 patients wit h a pathologic diagnosis of LCH, age 0 to 21 years, who presented betw een January 1, 1969 and June 30, 1994, were reviewed for organ involve ment at diagnosis, treatment, disease course, and late sequelae. Suppl ementary data were obtained by mailed questionnaire. Results: The medi an follow-up time from diagnosis for all patients was 8.1 years. Invol vement at diagnosis included nine patients with skin-only disease, 22 with monostotic disease, 12 with polyostotic disease, and 28 with mult isystem presentation. Treatment was surgery only in 17 and chemotherap y and/or radiotherapy in 54 patients. Recurrences were seen in 35 pati ents, with the highest rate in the polyostotic group. Ten patients die d: seven with the multisystem presentation, two with monostotic diseas e, and one with skin-only disease. Causes included progressive LCH (n = 6) and late sequelae of either treatment (n = 3) or disease (n = 1). Late sequelae were seen in 64% of 51 patients with more than 3 years of follow-up data. The most common were skeletal defects in 42%, denta l problems in 30%, diabetes insipidus in 25%, growth failure in 20%, s ex hormone deficiency in 16%, hypothyroidism in 14%, hearing loss in 1 6%, and other CNS dysfunction in 14%. The overall estimated survival r ates at 5, 15, and 20 years are 88%, 88%, and 77%, with on estimated e vent-free survival rate of only 30% at 15 years. Conclusion: Despite t he favorable survival, more than half of LCH patients will have furthe r dissemination of disease or late sequelae, including even some patie nts with single-system disease at diagnosis. Future treatment needs to be designed to prevent disease progression and late sequelae. (C) 199 6 by American Society of Clinical Oncology.