Aggressive therapy for neurosarcoidosis: long-term follow-up of 48 treated patients

Abstract

Background: Neurosarcoidosis (NS) is a relatively rare neurologic disorder for which no accepted treatment guidelines are available. Treatment with corticosteroids has been described as the primary means of controlling progressive symptoms. However, some physicians have recently advocated early intervention with alternative immunosuppressive therapies in patients who present with disabling symptoms.

Objective: To investigate our experience during the last decade regarding alternative immunosuppressive treatments, including corticosteroids and alternative therapies, in patients with NS.

Design: Observational, retrospective, consecutive case series with longitudinal follow-up.

Setting: Allegheny Neurological Clinic. Patients Seventy-eight patients with sarcoidosis were evaluated and classified as having possible, probable, or definite NS according to accepted criteria. Five cases of isolated NS were also included.

Main outcome measures: Patients with probable, definite, or isolated NS were scored before treatments and at final follow-up using estimated modified Rankin scores and the Disease Steps in Multiple Sclerosis scales.

Results: Forty-three patients were categorized as having either definite or probable NS according to accepted criteria and an additional 5 as having isolated NS. Thirty patients were categorized as having possible NS and were not included in the analysis of treatment response. Patients had a mean +/- SD number of visits of 7.2 +/- 6.4 and were followed up for a mean +/- SD of 44.1 +/- 43.6 months. Twenty patients were treated with pulse and/or maintenance corticosteroids alone. Twenty-six patients were treated with alternative immunosuppressive medications, with 23 of them receiving these medications at the time of diagnosis or within 6 months of the diagnosis of NS. Of the patients treated with alternative immunosuppressive therapies, 18 (69%) improved, 4 (15%) remained stable, and 4 (15%) worsened (including 1 death). Of the patients treated with corticosteroids alone, 7 (35%) improved, 11 (55%) remained stable, and 2 (10%) worsened. Two patients received no treatment.

Conclusions: Approximately half of all patients with NS seen at our clinic were believed to have disabling disease and to be at high risk for disease progression. These high-risk patients were treated with corticosteroids plus alternative immunosuppressive therapy, and favorable outcomes were obtained in almost all patients. Toxic effects related to treatments were minimal.