Steroid-Sparing Therapy for Neurosarcoidosis

Steroid-Sparing Therapy for Neurosarcoidosis

Information sourced from NEJM Journal Watch:
Steroid-Sparing Therapy for Neurosarcoidosis

Relatively high rates of relapse occurred with either methotrexate or mycophenolate mofetil in a retrospective analysis.

Glucocorticoids are widely considered to be a first-line therapy for neurosarcoidosis, but steroid monotherapy is sometimes insufficient or the toxicity is prohibitive. Evidence to inform selection of steroid-sparing therapy for neurosarcoidosis is sparse, and practice patterns vary. Now, in an uncontrolled, retrospective study from four university hospitals in France, investigators compared relapse rates in neurosarcoidosis patients treated with two steroid-sparing regimens for at least 3 months.

Of 40 neurosarcoidosis patients, 32 were treated with methotrexate (MTX; at a median dose of 20 mg per week), and 14 were treated with mycophenolate mofetil (MMF; at a median dose of 2 grams per day); 6 patients had successive treatment with both agents. Median steroid dosage at initiation of treatment was 40 mg/day in the MTX group versus 20 mg/day in the MMF group. The median age was 43.5 years (range, 13–77 years), and median disease duration at time of MMF or MTX initiation was 7.5 years. Both central nervous system and peripheral nervous system manifestations were included under the “neurosarcoidosis” umbrella, with most patients having CNS manifestations. In the MTX group, 15 of 32 patients (46.8%) relapsed compared with 11 of 14 (78.6%) in the MMF group (P=0.06). Median survival time without relapse was 28 months in the MTX group versus 11 months in the MMF group (P=0.049). Treatment was discontinued because of serious infection in 2 of 32 (6.3%) methotrexate plus steroids recipients and in no MMF plus steroids recipients.


The higher glucocorticoid dose in the MTX group could have confounded interpretation of efficacy and risk for infection in this uncontrolled analysis. In any case, relapse rates were remarkably high in both the MTX and MMF groups, and more effective steroid-sparing regimens for neurosarcoidosis are clearly needed.

Jeffrey M. Gelfand, MD, MAS reviewing Bitoun S et al. Neurology 2016 Dec 13.

Dr. Gelfand is Assistant Professor of Clinical Neurology, MS Center, University of California, San Francisco.


Bitoun S et al. Treatment of neurosarcoidosis: A comparative study of methotrexate and mycophenolate mofetil. Neurology 2016 Dec 13; 87:2517.
[PubMed® abstract]

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