Abstract:
Opsoclonus-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.
摘要:
视阵络-肌阵络-共济失调综合征(OMAS)是一种自身免疫性中枢神经系统疾病,主要表现为神经母细胞瘤的副肿瘤后遗症,以运动障碍和行为障碍为特征。OMAS以异常的B细胞和T细胞活化为代表。目前的治疗涉及使用皮质类固醇的免疫抑制,静脉注射免疫球蛋白,还有利妥昔单抗.然而,这些方法通常会导致治疗相关的毒性和症状性复发,并伴有慢性神经认知障碍.我们用他克莫司治疗3例难治性神经母细胞瘤相关OMAS患儿,一种T细胞靶向钙调磷酸酶抑制剂,在一个月内有效控制症状,并使免疫抑制停止,副作用最小。他克莫司有望作为难治性OMAS的治疗选择。