临床报道

1例青少年难治性视神经脊髓炎谱系疾病的诊治

  • 郑诺 ,
  • 陈曼 ,
  • 张冬丽 ,
  • 刘晓鸣
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  • 徐州市儿童医院(江苏徐州 221000)
刘晓鸣 电子信箱:xzlrlxm@163.com

收稿日期: 2024-07-17

  录用日期: 2024-11-01

  网络出版日期: 2025-03-31

Diagnosis and treatment of one case of refractory neuromyelitis optica spectrum disorder in adolescents

  • ZHENG Nuo ,
  • CHEN Man ,
  • ZHANG Dongli ,
  • LIU Xiaoming
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  • Xuzhou Children's Hospital, Xuzhou 221000, Jiangsu, China

Received date: 2024-07-17

  Accepted date: 2024-11-01

  Online published: 2025-03-31

摘要

探讨奥法妥木单抗(OFA)治疗难治性视神经脊髓炎谱系疾病(NMOSD)患者的可行性和有效性。患儿,女,15岁,血清水通道蛋白4抗体(AQP4-IgG)阳性多次复发的NMOSD,曾先后给予静脉注射甲基泼尼松龙琥珀酸钠、免疫球蛋白、利妥昔单抗(RTX),并口服醋酸泼尼松、吗替麦考酚酯治疗5年,9个月前开始间隔接受4剂OFA治疗(20 mg,皮下注射),观察患儿在OFA治疗的9个月期间NMOSD是否复发,血清AQP4-IgG抗体滴度,外周血B细胞水平以及头颅和脊髓MRI变化。患儿在OFA治疗的9个月期间症状稳定,无复发,血清AQP4-IgG滴度下降,复查头颅及脊髓MRI病灶减少,无新发病灶,耐受性好,无过敏反应或继发感染,除B细胞完全耗竭,未发现淋巴细胞减少或低丙种球蛋白血症。对于难治性NMOSD或其他免疫治疗不耐受的患儿,皮下注射OFA可能是一种安全有效的替代治疗。

本文引用格式

郑诺 , 陈曼 , 张冬丽 , 刘晓鸣 . 1例青少年难治性视神经脊髓炎谱系疾病的诊治[J]. 临床儿科杂志, 2025 , 43(4) : 301 -305 . DOI: 10.12372/jcp.2025.24e0688

Abstract

To preliminarily explore the feasibility and effectiveness of olfatomuzumab (OFA) in the treatment of patients with refractory neuromyelitis optica spectrum disorder (NMOSD). A 15-year-old adolescent female with seropositive AQP4-IgG and multiple relapses of refractory neuromyelitis optica spectrum disorder was given intravenous methylprednisolone, immunoglobulin, rituximab (RTX), and oral prednisone and mycophenolate mofetil for 5 years. She started to receive 4 doses of OFA (20 mg, subcutaneous injection) at intervals 9 months ago. The patient was observed for NMOSD recurrence, serum AQP4-IgG antibody titer, peripheral blood B cell level, and changes in cranial and spinal cord MRI during the 9-month period of OFA treatment. The patient's symptoms were stable without recurrence during the 9-month period of OFA treatment. The serum AQP4-IgG titer decreased, and the lesions in the cranial and spinal cord MRI decreased. There were no new lesions, and the tolerance was good without anaphylactic reactions or infectious adverse events. In addition to complete depletion of B cells, no lymphopenia or hypogammaglobulinemia was found. For patients with refractory NMOSD or other intolerant to immunotherapy, subcutaneous injection of OFA may be a safe and effective alternative treatment.

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