Cytosine-arabinoside, vincristine, and prednisolone in the treatment of children with disseminated Langerhans cell histiocytosis with organ dysfunction: experience at a single institution.

Fifty-two pediatric patients with Langerhans cell histiocytosis (LCH) were diagnosed at the Emma Kinderziekenhuis (EKZ) in Amsterdam over a 20-year period. Eight patients with multiorgan involvement with organ dysfunction and ten patients with multi-organ involvement without organ dysfunction received chemotherapy containing cytosine-arabinoside, vincristine, and prednisolone as part of their treatment. Five of the 8 patients (63%) with organ dysfunction and eight of the 10 (80%) without the organ dysfunction who needed chemotherapy because of the deteriorating of symptoms despite conventional therapy are presently in complete clinical remission. Two of those with organ dysfunction have died. Four of the total 18 patients developed diabetes insipidus either as an initial symptom or during the course of the disease. These results in both groups compare satisfactorily with other chemotherapeutic regimens, and since this combination is only mildly toxic, it has been well tolerated. Therefore we would recommend wider experience using this regimen in patients with widespread LCH with organ dysfunction as well as in patients with disseminated LCH in which chemotherapy proved to be necessary.