Department of Neurology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
Department of Pathology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
Front Immunol. 2023 Dec 19;14:1301109. doi: 10.3389/fimmu.2023.1301109. eCollection 2023.
Immune-mediated necrotizing myopathies (IMNM) is a rare disease that was first described in 2004. Due to the lack of large case series, there are no formal treatment recommendations for IMNM.
We presented a case of a 47-year-old woman who experienced progressive limb weakness, starting from the lower limbs and gradually affecting the upper limbs. She also reported experiencing dyspnea after engaging in daily activities. When she was admitted to the hospital, her upper limbs were almost unable to move and she could not stand even with support. Her Creatine kinase (CK) level significantly increased (> 3500 u/l). Electromyography showed myogenic damage, anti-Signal recognition particle (anti-SRP) and anti-Ro52 antibodies were highly positive. Pathological biopsy of the right biceps muscle showed necrotizing myopathy in the skeletal muscle. She was ultimately diagnosed with anti-SRP IMNN, and was given monotherapy with methylprednisolone and combination therapy with immunoglobulin, but her symptoms continued to worsen. The patient refused to bear the possible further liver dysfunction and blood system damage caused by Cyclophosphamide and Rituximab, and she chose to try to use Ofatumumab (OFA).
After receiving three doses of OFA treatment without any adverse reactions, she reported that her muscle strength had basically recovered and she was able to walk independently. The B cells in the circulatory system have been depleted, and blood markers such as liver function have consistently remained within normal range. During the follow up, her activity tolerance continued to improve.
We have presented a severe case of SRP-IMNM in which the patient showed poor response to conventional immunotherapy. However, rapid symptom relief was achieved with early sequential use of OFA treatment. This provides a new option for the treatment of SRP-IMNM, and more large-scale studies will be needed in the future to verify our results.
免疫介导的坏死性肌病（IMNM）是一种罕见疾病，于 2004 年首次描述。由于缺乏大型病例系列，目前尚无针对 IMNM 的正式治疗建议。
我们报告了一例 47 岁女性病例，她出现进行性四肢无力，从下肢开始逐渐影响上肢。她还报告在日常活动后出现呼吸困难。当她入院时，她的上肢几乎无法移动，即使有支撑也无法站立。她的肌酸激酶（CK）水平显著升高（>3500u/l）。肌电图显示肌源性损害，抗信号识别颗粒（anti-SRP）和抗 Ro52 抗体高度阳性。右侧肱二头肌的病理活检显示骨骼肌坏死性肌病。最终诊断为抗 SRP IMNN，给予甲基强的松龙单药治疗和免疫球蛋白联合治疗，但症状持续恶化。患者拒绝承受环磷酰胺和利妥昔单抗可能导致的进一步肝功能和血液系统损害，她选择尝试使用奥法木单抗（OFA）。
在接受三剂 OFA 治疗且无不良反应后，她报告说肌肉力量基本恢复，能够独立行走。循环系统中的 B 细胞已被耗尽，肝功能等血液标志物一直保持在正常范围内。在随访期间，她的活动耐量持续改善。
我们报告了一例严重的 SRP-IMNM 病例，该患者对常规免疫治疗反应不佳。然而，早期序贯使用 OFA 治疗迅速缓解症状。这为 SRP-IMNM 的治疗提供了新的选择，未来需要进行更多大规模研究来验证我们的结果。
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