High-dose methotrexate for the treatment of relapsed central nervous system erdheim-chester disease

Area of Science:

• Neurology
• Oncology
• Rare Diseases

Background:

• Erdheim-Chester disease (ECD) is a rare multisystem non-Langerhans histiocytosis.
• Central nervous system (CNS) involvement in ECD is a severe complication with poor prognosis.
• Standard treatments like interferon and cladribine have limited CNS penetration, complicating CNS ECD management.

Purpose of the Study:

• To evaluate the efficacy of high-dose methotrexate in a patient with rapidly progressive, steroid-refractory CNS Erdheim-Chester disease.
• To assess high-dose methotrexate as a potential salvage therapy for CNS ECD.

Main Methods:

• Case report of a 60-year-old female with a 5-year history of systemic ECD.
• Presentation of new brainstem lesions and rapid neurological deterioration.
• Treatment with high-dose methotrexate for CNS ECD, followed by cladribine and interferon.

Main Results:

• High-dose methotrexate rapidly arrested neurological deterioration and improved function.
• Clinical improvement was sustained for 4 months with methotrexate consolidation doses.
• Subsequent treatment with cladribine and interferon showed no significant clinical benefit.

Conclusions:

• High-dose methotrexate demonstrates effectiveness in controlling rapidly progressive CNS ECD.
• It serves as a valuable salvage agent for CNS ECD prior to more definitive therapies.
• Methotrexate's excellent CNS penetration makes it a suitable option for managing CNS ECD.