Results of Treatment of 127 Patients with Systemic Histiocytosis (Letterer-Siwe Syndrome, Schuller-Christian Syndrome and Multifocal Eosinophilic Granuloma)

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Abstract
Systemic histiocytosis is a rare disease, the treatment of which has undergone significant changes over the past 50 yr. The results of therapy of 127 patients treated between 1941 and 1975 were reviewed. A staging system was developed to distinguish patients at presentation. Fourteen patients presented with single monostotic or multiple bone lesions (stage I); 28 patients presented at age > 24 mo. at diagnosis with 1 or more systems involved including diabetes insipidus, gingiva, lymph node, skin, pulmonary infiltrate or focal bone marrow positivity (stage II); 63 patients presented with diagnosis at < 24 mo. of age with multiple systems involved as in stage II or were > 24 mo. of age at diagnosis with involvement of liver and/or spleen, massive nodal involvement or massive lung involvement or packed bone marrow (stage III). Fifteen patients presented with splenomegaly > 6 cm palpable and fever of > 1 mo. duration (stage IV); a special group of 7 patients was noted to have peripheral blood monocytosis representing > 20% of the peripheral blood count in addition to stage III or stage IV findings (stage V). Median follow-up was 8 yr with 20 patients followed for 22 yr. Patients diagnosed prior to 24 mo. of age evidently had a significantly worse prognosis (42% 10-yr actuarial survival) compared to older patients (85%). The Lahey staging system was confirmed for assessing prognosis of patients. There was no significant effect of available single agent or combination chemotherapy on improving the survival of patients with stage II, III or IV histiocytosis. Radiation therapy was of significant benefit in producing local control of painful or fracture-prone bone lesions and in reversing diabetes insipidus. There was an increased associated incidence of developmental abnormalities and mental retardation. Five patients developed 2nd malignancies. The superior prognosis of patients with stages I-III compared to stage IV disease and the excellent survival of early-stage patients indicates that experimental and aggressive approaches might be properly directed at patients with stage IV disease.