There are several reports of the use of this drug for this rare condition. Erdheim-Chester disease (ECD) is a rare xanthomatous non-Langerhans cell histiocytosis which involves the marrow space of the long bones. Extraosseous sites most commonly affected include the eyes, lungs, pituitary glands, and kidneys.
There are several reports of the use of cladribine. Unfortunately, there is little basis to consider that it is beneficail and some reports suggest that it may be harmful. In 1999, Saven and Burian described encouraging responses to cladribine in 13 patients with adult Langerhans cell histiocytosis. This information, together with our new evidence of increased monocyte activation in this patient, made clabribine, an agent toxic to monocytes, a rational choice. There has been one previous report of treatment of Erdheim-Chester disease with cladribine. That patient had orbital involvement and unfortunately developed bilateral blindness. It was postulated that cladribine might have caused toxic injury to the optic nerves which predisposed them to ischaemic injury. However, the clinical signs suggested progression of the Erdheim-Chester disease as the cause of the blindness. There has also been a case of transient blindness occurring during therapy with clabribine on a solid tumor population. That was, however, with orbital involvement.
Myra, Powell, R.Treatment of Erdheim-Chester disease with cladribine: a rational approach Br J Ophthalmol. 2004 June; 88(6): 844–847.
Saven A, Burian C. Cladribine activity in adult Langerhans-cell histiocytosis. Blood 1999;93:4125–30.
13.Sheidow TG, Nicolle DA, Heathcote JG. Erdheim-Chester disease: two cases of orbital involvement. Eye 2000;14:606–12.