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霉酚酸酯成功长期治疗儿童复发性特发性视神经炎

Successful Long-Term Treatment of Pediatric Relapsing Idiopathic Optic Neuritis with Mycophenolate Mofetil.

作者信息

Fujino Shuhei, Akamine Keiji, Noda Eiichiro, Miyama Sahoko

机构信息

Department of Neurology, Tokyo Metropolitan Children's Medical Center, Tokyo 183-8561, Japan.

Department of Nephrology, Tokyo Metropolitan Children's Medical Center, Tokyo 183-8561, Japan.

出版信息

Neurol Int. 2025 Mar 18;17(3):44. doi: 10.3390/neurolint17030044.

Abstract

Pediatric optic neuritis (ON) is a rare but severe condition characterized by acute visual impairment, with 3-5% of relapsing cases lacking identifiable markers for associated conditions, such as neuromyelitis optica spectrum disorder (NMOSD) or multiple sclerosis (MS); these cases are thus classified as relapsing idiopathic optic neuritis (RION). Corticosteroids are typically used for acute management; however, their prolonged use in children poses significant risks, including central obesity, hypertension, and growth impairment, underscoring the need for nonsteroidal, long-term treatment options. Current strategies for preventing recurrence in pediatric RION are limited due to a lack of data on immunosuppressive efficacy and safety. Given its rarity and the challenges of long-term immunosuppression in children, identifying optimal therapeutic approaches remains critical. We report a case of a six-year-old girl with RION, who was initially treated with intravenous methylprednisolone (IVMP) and prednisolone (PSL) tapering, and who experienced recurrence eight months post-treatment. Additional corticosteroids and intravenous immunoglobulin (IVIg) were administered during relapse, but, due to adverse effects, treatment was transitioned to mycophenolate mofetil (MMF), enabling early PSL tapering. With MMF, the patient maintained stable vision and achieved a five-year recurrence-free period without notable side effects. In conclusion, this case suggests MMF's efficacy as a long-term management option for pediatric RION, potentially reducing corticosteroid-related risks.

摘要

儿童视神经炎(ON)是一种罕见但严重的疾病,其特征为急性视力损害,3%至5%的复发病例缺乏与相关疾病(如视神经脊髓炎谱系障碍(NMOSD)或多发性硬化症(MS))相关的可识别标志物;因此,这些病例被归类为复发性特发性视神经炎(RION)。皮质类固醇通常用于急性治疗;然而,在儿童中长期使用会带来重大风险,包括中心性肥胖、高血压和生长发育受损,这凸显了对非甾体类长期治疗方案的需求。由于缺乏关于免疫抑制疗效和安全性的数据,目前预防儿童RION复发的策略有限。鉴于其罕见性以及儿童长期免疫抑制的挑战,确定最佳治疗方法仍然至关重要。我们报告了一例六岁患RION的女孩,她最初接受静脉注射甲基泼尼松龙(IVMP)和逐渐减量的泼尼松龙(PSL)治疗,治疗后八个月复发。复发期间给予了额外的皮质类固醇和静脉注射免疫球蛋白(IVIg),但由于不良反应,治疗改为霉酚酸酯(MMF),从而能够提前逐渐减少PSL用量。使用MMF后,患者视力保持稳定,实现了五年无复发期,且无明显副作用。总之,该病例表明MMF作为儿童RION长期管理选择的有效性,可能降低与皮质类固醇相关的风险。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/86ed/11945395/a314f2e68c10/neurolint-17-00044-g001.jpg

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