Successful Tocilizumab Treatment in a Child With Refractory Takayasu Arteritis

Takayasu arteritis (TA) in the child remains a therapeutic challenge because corticosteroids and conventional immunosuppressive agents are not always safe or efficacious. The complex formed by interleukin-6 (IL-6) and soluble IL-6 receptor appears to play a pivotal role in the pathogenesis of TA. We describe a favorable response to the anti-IL-6 receptor antibody tocilizumab (TCZ) in a child with aggressive and refractory TA including an assessment of the proinflammatory cytokine profile. A 3-year-old girl with TA consisting of thickening of the aortic arch wall, severe obstruction of the supra-aortic branches, and complete occlusion of both common carotid arteries failed to respond to corticosteroids, methotrexate, tumor necrosis factor alpha blockade, cyclophosphamide, and mycophenolate mofetil, and 3 years later, the disease remained active with severe manifestations (brain ischemia). The patient underwent percutaneous angioplasty, although significant restenosis was soon documented. After a severe relapse, the patient started TCZ infusions (8 mg/kg for 2 weeks), and a rapid clinical remission was observed, associated with a drastic reduction of inflammatory markers and IL-6 levels. Corticosteroids were withdrawn, the patient's weight and height improved, and bone mineral density values returned to normal. Two years later, TCZ infusions were extended, with no significant side effects. Cerebral ischemia resolved, and recanalization of the previously occluded supra-aortic branches was performed. Pediatrics 2012;130:e1720-e1724