利妥昔单抗治疗韩国难治性炎性肌病患者的疗效和安全性。
Efficacy and Safety of Rituximab in Korean Patients with Refractory Inflammatory Myopathies.
机构信息
Department of Rheumatology, Hanyang University Hospital for Rheumatic Diseases, Seoul, Korea.
Department of Rheumatology, Ajou University School of Medicine, Suwon, Korea.
出版信息
J Korean Med Sci. 2020 Sep 28;35(38):e335. doi: 10.3346/jkms.2020.35.e335.
BACKGROUND
Rituximab (RTX), a monoclonal antibody that selectively binds to CD20+ B cells, showed favorable outcomes in patients with idiopathic inflammatory myopathies (IIM) in small case series, but the evidence is still not enough. Our goal was to determine the efficacy and safety of RTX for Korean patients with refractory IIM.
METHODS
We retrospectively analyzed the medical records of 16 patients with refractory IIM treated with RTX in seven tertiary rheumatology clinics in the Korea. The efficacy of RTX was evaluated with the improvement of serum creatine phosphokinase (CPK) level and physician's global assessment (PGA), and daily corticosteroid dose reduction. A > 25% decrease in CPK level, corticosteroid dose, or PGA was considered significant. A complete response (CR) was designated by meeting three efficacy criteria and a partial response (PR) by only two criteria.
RESULTS
Sixteen patients with IIM were evaluated (13 female; median age, 51.8 years). All patients had received at least one conventional immunosuppressive agent (median, 3.6 [2.0-5.0]) and concomitant corticosteroids. The median CPK level and median dose of prednisolone was 421.0 units/L and 20.0 mg/day respectively. Eleven patients were treated with intravenous immunoglobulin. Seven patients received 2,000 mg of RTX and the others received lower dose. Twenty-four weeks after RTX treatment, 11 patients achieved a > 25% reduction in corticosteroid dose and CPK levels, and nine showed improved PGA. The overall response rate was 68.8% (11 patients). At the end of follow-up (median 24 weeks), 12 (75.0%) patients responded overall: four (25.0%) and eight (50.0%) patients achieved CR and PR, respectively. Baseline muscle enzyme levels were higher in responders than non-responders, but disease duration, RTX dose, ESR and serum CRP were not significantly different between the two groups. The rate of adverse event was 25.4/1,000 person-years.
CONCLUSION
RTX could be an effective and relatively safe therapeutic option in patients with refractory IIM.
背景
利妥昔单抗（RTX）是一种单克隆抗体，可选择性结合 CD20+B 细胞，在小病例系列中显示出对特发性炎性肌病（IIM）患者的良好疗效，但证据仍不足。我们的目标是确定 RTX 对韩国难治性 IIM 患者的疗效和安全性。
方法
我们回顾性分析了在韩国 7 个三级风湿病诊所接受 RTX 治疗的 16 例难治性 IIM 患者的病历。通过血清肌酸磷酸激酶（CPK）水平和医生总体评估（PGA）的改善以及每日皮质类固醇剂量的减少来评估 RTX 的疗效。CPK 水平、皮质类固醇剂量或 PGA 降低>25%被认为有显著意义。满足三项疗效标准为完全缓解（CR），满足两项标准为部分缓解（PR）。
结果
共评估了 16 例 IIM 患者（13 例女性；中位年龄 51.8 岁）。所有患者均接受了至少一种常规免疫抑制剂（中位数，3.6[2.0-5.0]）和皮质类固醇的联合治疗。中位 CPK 水平和泼尼松龙的中位剂量分别为 421.0 U/L 和 20.0mg/天。11 例患者接受了静脉注射免疫球蛋白治疗。7 例患者接受了 2000mg 的 RTX 治疗，其余患者接受了较低剂量的 RTX 治疗。RTX 治疗 24 周后，11 例患者的皮质类固醇剂量和 CPK 水平降低>25%，9 例患者的 PGA 得到改善。总体缓解率为 68.8%（11 例）。在随访结束时（中位时间 24 周），12 例（75.0%）患者总体上有反应：4 例（25.0%）和 8 例（50.0%）患者分别达到 CR 和 PR。反应者的基线肌肉酶水平高于无反应者，但两组之间的疾病持续时间、RTX 剂量、ESR 和血清 CRP 无显著差异。不良事件发生率为 25.4/1000 人年。
结论
RTX 可能是治疗难治性 IIM 患者的一种有效且相对安全的治疗选择。
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