Hydroxycarbamide therapy for pediatric relapsed Langerhans cell histiocytosis

Abstract

Despite significant improvements in the overall survival of pediatric patients with Langerhans cell histiocytosis (LCH), relapse is common, and a truly effective treatment for relapsed LCH has not yet been established. Hydroxycarbamide has been widely used for many years; however, thus far, few pediatric patients with LCH have received hydroxycarbamide therapy. Here, we present cases of two pediatric patients with relapsed LCH who received hydroxycarbamide with or without methotrexate therapy. In these two cases, the therapeutic response to hydroxycarbamide with or without methotrexate therapy was comparable to that to the conventional treatment for relapsed LCH in pediatric patients. No serious adverse events were observed, and dose reduction was unnecessary. Such an oral regimen reduces the physical and mental stress associated with painful injections in children, in addition to reducing the number of clinic visits and medical costs. It provides great benefits to pediatric patients with LCH who are likely to have repeat recurrences.