Abstract
Objective : To clarify the clinical characteristics, treatment and outcome of Langerhans cell histiocytosis (LCH) with multisystem organ dysfunction. Design : Retrospective chart review of children with multisystem LCH between 1983 and 1994. Setting : Collaborative study with 6 affiliated hospitals of Childhood Cancer Conference of Osaka. Results : Eleven patients had evidence of dysfunction of the liver, lungs and/or bone marrow. Their median age at diagnosis was 10months (range : 0.8-14 months). Treatment consisted initially of a combination of prednisolone and vinblastine or etoposide, which eventually failed to yield a good response. Subsequently, several chemotherapeutic drugs including cyclophosphamide, doxorubicin, vincristine, cytarabine, 6-mercaptopurine, and methotrexate were given. Cyclosporin, interferon-α and interleukin-2 as immunomodulatory agents were attempted. A fatal outcome occurred in 4 patients. Three of them died of respiratory failure due to pulmonary infiltration or pneumonia, and one died of mycotic infection. Of the 7survivors, 5 have been treated for disease-related late consequences. They have suffered from recurrent bone tumors (n=3), diabetes insipidus (n=2), leukoencephalopathy (n=1), and persistent splenomegaly (n=2). No secondary malignancy was noted. onclusions : Patients with LCH with organ dysfunction, particularly when accompanied by pulmonary involvement, have the poorest prognosis. At present, the long-term disease-free survival rate is low.
