Long-Term Therapeutic Outcomes with Cladribine in Patients with Xanthoma Disseminatum

Cladribine (2-chlorodeoxyadenosine or 2CdA) is a purine analogue that has been used in various non-Langerhans cell histiocytosis. We present a case series of six patients with xanthoma disseminatum having systemic involvement, treated with monthly cladribine for three to 12 cycles (median six cycles), leading to 63.3% improvement at the end of treatment, increasing to 75% at the end of 2.1 ± 1.6 years of follow-up. Three patients maintained their response, two continued to improve without further intervention, and one patient relapsed with partial response to reinstitution of therapy. Intracranial lesions improved significantly in all three patients with neurological involvement. One patient with severe laryngeal involvement causing stenosis was able to avoid tracheostomy. Cladribine was largely well tolerated. Half of the patients developed transient leucopenia requiring prophylactic granulocyte colony-stimulating factor, but none of the patients developed any serious infections.