Improved outcome in the treatment of pediatric multifocal Langerhans cell histiocytosis

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Abstract
BACKGROUND.: The treatment outcome of multifocal childhood Langerhans cell histiocytosis (LCH) has not been satisfactory and has resulted in poor therapeutic responses with high mortality and a high incidence of reactivation with late sequelae. To overcome these issues, the Japan LCH Study Group‐96 (JLSG‐96) protocol was conducted prospectively from 1996 to 2001 in Japan.METHODS.: Newly diagnosed children with multifocal LCH were classified into 2 groups: a single‐system multisite (SS‐m) group and a multisystem (MS) group. All patients initially were treated on Protocol A, which consisted of 6 weeks of induction therapy with combined cytosine arabinoside, vincristine (VCR), and prednisolone (PSL) followed by 6 months of maintenance therapy. Patients who had a poor response to the induction of Protocol A were switched to a salvage regimen (Protocol B), which consisted of an intensive combination of doxorubicin, cyclophosphamide, VCR, and PSL.RESULTS.: In total, 91 patients were treated, including 32 patients in the SS‐m group and 59 patients in the MS group. At the median 5‐year follow‐up, 96.9% of patients in the SS‐m group and 78.0% of patients in the MS group had good response status. Diabetes insipidus developed in 3.1% of patients in the SS‐m group and in 8.9% of patients in the MS group. The overall survival rate at 5 years for the SS‐m and MS groups was 100% and 94.4% ± 3.2%, respectively.CONCLUSIONS.: The JLSG‐96 protocol attained very low mortality for pediatric patients with multifocal LCH. Cancer 2006. © 2006 American Cancer Society.