Langerhans cell histiocytosis: 40 years' experience.

Results: Overall survival (OS) for all patients was 93% at 10 years from diagnosis, event-free survival (EFS) 77%. OS for patients younger than 2 years and older than or equal to 2 years was 82% and 97% (P=0.003); EFS 48% and 87% (P=0.001). OS for patients diagnosed before and after April 1, 1991 was 84% and 98% (P=0.007), EFS 66% and 85% (P=0.03). OS for monosystemic and multisystemic disease was 100% and 71% (P<0.001); EFS 88% and 45% (P<0.001). OS for “risk” patients (involvement of bone marrow, spleen, liver, lungs) and “low-risk” patients was 50% and 94% (P=0.007), EFS 37% and 54% (P=0.06). Fourteen patients developed diabetes insipidus, 7 patients growth hormone deficiency, 2 hypothyroidism, and 1 neurodegeneration.