Cyclosporine A therapy for multisystem Langerhans cell histiocytosis

BACKGROUND: Treatment of multisystem Langerhans cell histiocytosis (LCH) remains difficult. Various regimens of single and multiagent chemotherapy have been used, but a significant proportion of patients fail to respond to treatment. PROCEDURE: We have evaluated the use of cyclosporine A (CSA) in a controlled group of patients, who had received a systematic primary therapy (LCH-I). Patients received CSA either as a single agent (10 patients) or in combination with vinblastine, etoposide, prednisolone, and/or antithymocyte globulin (16 patients). RESULTS: Among the total of 26 patients treated, a single patient developed a complete response and three a partial response, whereas 85% (22 patients) had no response to CSA. CONCLUSIONS: CSA is at best of limited value in the treatment of patients with multisystem LCH, particularly those who had progressive disease while receiving chemotherapy.