Erdheim-Chester disease (ECD) is a rare multisystem non-Langerhans histiocytosis. CNS involvement is a major complication,\nwhich is often rapidly progressive and confers a poor prognosis. However, treatment of CNS ECD is difficult due to poor CNS\npenetrance by the most effective chemotherapeutic drugs commonly used in this disorder (e.g., interferon and cladribine). We\ndescribe a case of a 60-year-old lady with a 5-year history of stable systemic ECD who presented with new brainstem lesions and\nrapid, steroid-refractory neurological deterioration which required immediate intervention. High-dose methotrexate was chosen\ndue to its rapid onset of action and excellent CNS penetration. Her neurological deterioration was quickly arrested with significant\nfunctional improvement, which was sustained for 4 months with consolidation doses of high-dose methotrexate. Subsequent\ntreatment with cladribine and interferon did not confer any appreciable clinical improvement. High-dose methotrexate is effective\nin controlling rapidly progressive CNS ECD and should be considered as a salvage agent prior to commencement ofmore definitive\ntreatment.
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