Successful use of tacrolimus for treatment‐refractory neuroblastoma‐associated opsoclonus‐myoclonus‐ataxia syndrome: A case series

Abstract

AbstractOpsoclonus‐myoclonus‐ataxia syndrome (OMAS) is an autoimmune central nervous system disorder, primarily manifesting as a paraneoplastic sequalae to neuroblastoma, and characterized by motor disorders and behavioral disturbances. OMAS is typified by aberrant B‐cell and T‐cell activation. Current treatment involves immunosuppression using corticosteroids, intravenous immunoglobulin, and rituximab. However, these approaches often lead to treatment‐related toxicities and symptomatic recurrences with chronic neurocognitive impairment. We treated three children with refractory neuroblastoma‐associated OMAS with tacrolimus, a T‐cell‐targeting calcineurin inhibitor, effectively controlling symptoms within a month and enabling the discontinuation of immunosuppression with minimal side effects. Tacrolimus shows promise as a therapeutic option for refractory OMAS.