Therapeutic outcome of multifocal Langerhans cell histiocytosis in adults treated with the Special C regimen formulated by the Japan LCH Study Group

Little information is available regarding effective systemic therapies for adult Langerhans cell histiocytosis (LCH). The Japan LCH Study Group has formulated an ambulatory treatment regimen for adult patients with LCH. In total, 14 patients (median age 43 years, range 20–70 years) with multifocal LCH with biopsy-confirmed histology were enrolled. None had received cytoreductive agents for LCH previously. Four had single system (SS) and ten had multi system (MS) disease. All were treated with the Special C regimen, which consists of vinblastine/prednisolone and methotrexate with daily 6-mercaptopurine for 36 weeks. At the end of the therapeutic regimen, all SS patients achieved no active disease (NAD), and six of the ten MS patients showed a response (NAD in two, partial response in four). At the last follow-up (median 34 months), 11 patients were alive (NAD in eight and active disease in three). Of the three deceased, one died of hemorrhage during the Special C treatment, and two of infections during subsequent therapy. Although this study is limited by the small sample size, this ambulatory regimen shows signs of efficacy for adult LCH. This was particularly evident for patients with multifocal SS disease, but half of those with MS disease also benefited.