Treatment of corticosteroid-resistant neurosarcoidosis with a short-course cyclophosphamide regimen.

Background/objectives: Many patients with neurosarcoidosis have disease that is refractory to corticosteroids or they are unable to tolerate high-dose corticosteroids because of detrimental side effects. We examined a short-course, pulse-dose regimen using cyclophosphamide to treat such patients.

Methods: We identified a population of patients with neurosarcoidosis refractory to standard therapy with corticosteroids. Patients who were unable to tolerate corticosteroid therapy due to side effects were also included. Alternative therapy for these patients was initiated using i.v. cyclophosphamide.

Results: Seven patients were identified for treatment with our cyclophosphamide regimen. The mean duration of therapy was 5.4 months. Four of the seven patients reported symptomatic improvement on therapy, and all seven patients demonstrated objective improvement in either MRI or cerebrospinal fluid abnormalities. Mean corticosteroid dose of the group was reduced from 42 mg/d before therapy to 18 mg/d after therapy. Relapse of neurologic symptoms was noted in one patient after the completion of therapy. One patient acquired an opportunistic infection, and a second patient required hospitalization for a central venous catheter infection.

Conclusion: Short-course cyclophosphamide appears to be a reasonable, steroid-sparing treatment option for patients with corticosteroid-refractory neurosarcoidosis.