2026 American Academy of Neurology Abstract Website

Objective:

To report a case of successful treatment of refractory neurosarcoidosis using tofacitinib.

Background:

Neurosarcoidosis develops in 5% of the sarcoidosis patients. Manifestations of neurosarcoid range from self-limiting cases such as facial nerve palsy to debilitating parenchymal and spinal cord presentations requiring aggressive treatment. Anti-TNF monoclonal antibodies such as infliximab and adalimumab are used when steroids and steroid sparing agents (methotrexate, mycophenolate) are not adequate, fail in up to one-third of patients. Options in these patients are limited with variable response reported to cyclophosphamide. We report the use of Tofacitinib, a JAK-STAT pathway inhibitor in a patient that worsened while on anti-TNF therapy.

Design/Methods:

A 47-year-old male presented with a six-month history of progressive gait instability, frequent falls, and asymmetric lower extremity weakness (right > left). Additional symptoms included distal hand numbness (in the fourth and fifth digits), reduced grip strength, urinary urgency, hesitancy, intermittent retention, and erectile dysfunction. MRI of the brain and spine showed diffuse leptomeningeal enhancement. CT chest/abdomen/pelvis revealed mediastinal and inguinal lymphadenopathy. Lymph node biopsy demonstrated non-necrotizing granulomas, consistent with sarcoidosis.

Results:

The patient started steroids and methotrexate followed by infliximab but lost hearing in one ear. He was found to have anti-infliximab antibodies and switched to adalimumab. Repeat MRI showed worsening of meningeal disease, prompting initiation of Tofacitinib. After six months of Tofacitinib therapy, the patient showed radiographic improvement in the posterior fossa and clinical improvement in gait and hearing. No major adverse effects were observed.

Conclusions:

This case supports the potential utility of Tofacitinib as an alternative treatment for refractory neurosarcoidosis. One published case report indicated the utility of tofacitinib in neurosarcoidosis but was complicated by the simultaneous use of cyclophosphamide. Our case shows that Tafacitinib by itself is efficacious in neurosarcoidosis. Prospective clinical studies may help define its place in therapy.