免疫介导性坏死性肌病中不进行干细胞救援的大剂量环磷酰胺治疗
High-dose cyclophosphamide without stem cell rescue in immune-mediated necrotizing myopathies.
作者信息
Mecoli Christopher A, Lahouti Arash H, Brodsky Robert A, Mammen Andrew L, Christopher-Stine Lisa
机构信息
Johns Hopkins University School of Medicine (C.A.M., A.H.L., R.A.B., A.L.M., L.C.-S.), Baltimore; and National Institute of Health (A.L.M.), Bethesda, MD.
出版信息
Neurol Neuroimmunol Neuroinflamm. 2017 Jul 7;4(5):e381. doi: 10.1212/NXI.0000000000000381. eCollection 2017 Sep.
OBJECTIVE
To describe the experience managing treatment-refractory immune-mediated necrotizing myopathies (IMNM) with high-dose cyclophosphamide (HiCy) therapy.
METHODS
Five patients with severe refractory IMNM who were treated with HiCy without stem cell rescue were identified. Their medical records were reviewed to assess demographic, clinical, and histologic characteristics as well as response to therapy.
RESULTS
Three patients with anti-signal recognition particle (SRP) and 2 patients with anti-HMG-CoA reductase autoantibodies were included. The mean follow-up time after HiCy therapy was 37 ± 28 months. Two patients demonstrated substantial response, evidenced by improved muscle strength and decreased muscle enzymes after HiCy therapy; both of these patients were anti-SRP positive. Four patients experienced febrile neutropenia after HiCy therapy, one of which required a prolonged intensive care unit stay for infectious complications, from which they eventually recovered.
CONCLUSIONS
These data suggest that HiCy therapy without stem cell rescue may be considered as an alternative for the treatment of refractory IMNM.
目的
描述采用大剂量环磷酰胺（HiCy）治疗难治性免疫介导坏死性肌病（IMNM）的经验。
方法
确定5例接受HiCy治疗且未进行干细胞救援的重度难治性IMNM患者。回顾他们的病历以评估人口统计学、临床和组织学特征以及对治疗的反应。
结果
包括3例抗信号识别颗粒（SRP）患者和2例抗HMG-CoA还原酶自身抗体患者。HiCy治疗后的平均随访时间为37±28个月。2例患者表现出显著反应，HiCy治疗后肌力改善和肌肉酶水平降低证明了这一点；这2例患者均为抗SRP阳性。4例患者在HiCy治疗后出现发热性中性粒细胞减少，其中1例因感染并发症需要在重症监护病房长期住院，最终康复。
结论
这些数据表明，不进行干细胞救援的HiCy治疗可被视为难治性IMNM治疗的一种替代方案。
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