Histiocytosis X: Natural History And Management In Childhood

Abstract

Multi-system histiocytosis X is still a potentially fatal disorder. Death occurs from pulmonary, hepatic, small bowel or bone marrow failure often with severe intercurrent infection as the terminal illness. Chemotherapy has had some impact on mortality but morbidity in survivors is still a real problem. Around 50% of survivors will have a significant handicap. Complete response can be achieved with a variety of chemotherapeutic agents but the introduction of combination therapy has led to unacceptable toxicity especially in the very young. There is preliminary evidence that treatment with calf ‘thymic humoral factor’ may benefit a proportion of patients. This recent work supports the notion that histiocytosis X is not a true malignancy, but rather a subtle form of immune deficiency possibly involving a soluble thymic or T-lymphocyte product.