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

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.

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.

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.