Achievements and Future Perspectives in the Treatment of Multisystem Langerhans Cell Histiocytosis

Clinical stratification and response evaluation The ignorance of the pathogenesis and the failure to establish generally accepted diagnostic criteria have inhibited the development of a rational treatment policy for multisystem LCH. The therapy for LCH, therefore, has varied over the past century according to what was believed to be the cause of the disease. Only with the introduction of new concepts of staging and diagnostic criteria it became possible to collect large enough numbers of patients to carry out prospective clinical trials.1 Empirically it has been shown that the treatment of LCH should depend on the extent of the disease, and patients were stratified as having “single system” disease (bone, skin, lymphnode, lung, or CNS) with single site and multi site involvement, and “multisystem” disease, often associated with dysfunction of the so called “risk organs”, i.e. liver, lungs, spleen or hematopoietic system.