Abstract：
To describe the use of oral cyclophosphamide (PO CYC) in a single center longitudinal cohort of patients with idiopathic inflammatory myopathies (IIM). Patients using PO CYC were identified through a retrospective chart review of a myositis cohort at a single academic center. PO CYC dose, duration, adverse events, and disease activity measures before and after CYC were analyzed. Disease activity measures included muscle enzymes, manual muscle testing (MMT8), 100-mm visual analog scale (VAS), and 1-4 Likert scale for physician global assessment. Fourteen patients were treated with PO CYC within the cohort between 2008 and 2017; 9 dermatomyositis (DM), 3 polymyositis (PM), and 2 with immune-mediated necrotizing myopathy (IMNM). Age was 51.1 (40-72) years and the cumulative dose of PO CYC was 41 (2-131) grams over duration of 12.4 (0.5-43) months, mean (range) for all. All patients had severe refractory IIM, 10 (72%) with ILD, 3 (21%) with cardiac involvement and 4 (29%) were dependent in most activities of daily living. Median number of prior failed therapies was 4.5 (range 3-6) including intravenous CYC in 5 patients. Disease activity measures significantly improved following CYC use and concomitant daily prednisone dose decreased. The most common adverse events during CYC therapy were infections. We report the first cohort study of PO CYC use in IIM patients with severe, treatment refractory disease. Further trials are needed to verify these results as well as to evaluate long-term safety outcomes.
摘要：
描述口服环磷酰胺（POCYC）在特发性炎症性肌病（IIM）患者的单中心纵向队列中的使用。通过在单个学术中心对肌炎队列进行回顾性图表回顾，确定了使用POCYC的患者。POCYC剂量，持续时间,不良事件,并对CYC前后的疾病活动指标进行分析。疾病活动措施包括肌肉酶，手动肌肉测试（MMT8），100-mm可视模拟量表(VAS),和1-4个用于医生全球评估的李克特量表。在2008年至2017年的队列中，有14例患者接受了POCYC治疗；9例皮肌炎（DM），3多发性肌炎（PM），和2与免疫介导的坏死性肌病（IMNM）。年龄为51.1（40-72）岁，POCYC的累积剂量为41（2-131）克，持续时间为12.4（0.5-43）个月，所有的平均值（范围）。所有患者均有重度难治性IIM,10（72％）与ILD，3（21％）与心脏受累和4（29％）在大多数日常生活活动中具有依赖性。先前治疗失败的中位数为4.5（范围3-6），包括5例患者的静脉CYC。使用CYC后，疾病活动措施显着改善，并且伴随的每日泼尼松剂量减少。CYC治疗期间最常见的不良事件是感染。我们报告了在严重的IIM患者中使用POCYC的第一个队列研究，治疗难治性疾病。需要进一步的试验来验证这些结果以及评估长期安全性结果。
