To evaluate adalimumab treatment in CNS neurosarcoidosis in a retrospective observational analysis across multiple sites in the United States.

Inhibition of tumor necrosis factor alpha (TNF-α) via infliximab is a foundational treatment for neurosarcoidosis, especially in refractory or disabling cases. Limited evidence exists to guide management decisions beyond infliximab. Adalimumab, a self-injectable TNF-α inhibitor, has been reported as a successful treatment in single cases and small case series.

Adult patients with probable or definite neurosarcoidosis treated with adalimumab were included if they received at least 80 mg per month, underwent pre- and post-adalimumab MRIs, and were followed for ≥6 months following drug initiation. If on ≤10 mg prednisone by 6 months of treatment, responsiveness to adalimumab was defined as: 1) clinical improvement with stable or improved MRIs, or 2) clinical stability with improved MRIs.

Five US academic medical centers provided data for 21 patients: 11/21 male, average age 52.1 years at adalimumab initiation, average neurosarcoidosis disease duration 54.3 months, average 1.8 attacks prior to adalimumab, and average follow-up of 56.4 months since adalimumab initiation. Most common disease locations included: spinal cord parenchyma 9/21, leptomeninges 6/21, brain parenchyma 6/21, cauda equina 5/21, and spinal leptomeninges 5/21. Initial adalimumab dosing was 40 mg every other week in 18/21 and 40 mg weekly in 3/21. Line of treatment for adalimumab was: first 2/21, second 8/21, third 7/21, and fourth 4/21, including use after infliximab in 12/21. Concomitant steroid-sparing immunosuppressants were used in 9/21. Most (18/21, 85.7%) were responsive to adalimumab, except two patients experiencing breakthrough disease and one patient not improving clinically nor radiographically. All responders were on 10 mg daily of prednisone or less, including 14/18 on none at last follow-up.

Most patients, across a broad array of CNS phenotypes, responded favorably to adalimumab, including those who had previously been treatment refractory.