Massachusetts Eye Research and Surgery Institution, Cambridge, Massachusetts, USA.
Ophthalmology. 2013 Jun;120(6):1201-9. doi: 10.1016/j.ophtha.2013.01.031. Epub 2013 Apr 16.
To analyze the success rate of pulsed intravenous (IV) cyclophosphamide (CyP) for noninfectious ocular inflammatory disease and to identify risk factors for failure of therapy.
Retrospective, interventional, noncomparative cohort study.
One hundred ten eyes of 65 patients.
Through a computer search of the Massachusetts Eye Research and Surgery Institution's database, we identified patients who were treated with IV CyP between May 2005 and April 2012. We obtained demographic and clinical information through review of the electronic health record of each patient.
Clinical response, corticosteroid-sparing effect, recurrence rate, calculated "risk factors" for failure, visual acuity, and adverse reactions.
Pulsed IV CyP achieved complete remission of inflammation (for ≥ 2 visits) in 54 patients (84.4%). Sustained remission of inflammation occurred in 70% of patients within 3 months, 86.6% of patients within 6 months, and 91.7% within 9 months. The mean time to achieving quiescence was 3.5 months. The success rate in reducing corticosteroid to prednisone ≤ 10 mg/d within 6 months, while maintaining control of ocular inflammation, was 89.7% (95% confidence interval [CI], 81.1-93.5%). The mean duration of clinical remission for those patients who had a positive response to CyP was 32.67 months (95% CI, 25.91-39.43). Relapse of vasculitis was observed in 1 patient (1.5%) after completing the course of therapy. Early initiation of therapy during the course of the disease was correlated with a lesser rate of recurrence (P = 0.028). The most common adverse effects were nausea (29%) and transient lymphopenia (26%). The mean best-corrected visual acuity (BCVA) improved from 0.59 ± 0.66 at baseline to 0.30 ± 0.54 at 6 months of follow-up (P<0.001). The mean follow-up period was 31.61 ± 20.47 months.
Pulsed IV CyP employing our protocol results in an extremely high rate of sustained complete remission in patients with recalcitrant and fulminant, vision-threatening ocular inflammatory disorders, with an excellent safety profile in the hands of physicians trained and skilled in the art of this therapy. It also allows tapering and discontinuing corticosteroids in most patients. Early initiation of therapy may decrease the risk of relapses.
FINANCIAL DISCLOSURE(S): The authors have no proprietary or commercial interest in any materials discussed in this article.
分析脉冲式静脉内（IV）环磷酰胺（CyP）治疗非传染性眼部炎症性疾病的成功率，并确定治疗失败的风险因素。
回顾性、干预性、非对照队列研究。
65 例患者的 110 只眼。
通过对马萨诸塞州眼研究与手术机构数据库的计算机检索，我们确定了 2005 年 5 月至 2012 年 4 月期间接受 IV CyP 治疗的患者。通过对每位患者电子病历的回顾，我们获得了人口统计学和临床信息。
临床反应、皮质类固醇节省效应、复发率、计算的“失败风险因素”、视力和不良反应。
脉冲式 IV CyP 使 54 例患者（84.4%）的炎症完全缓解（≥2 次就诊）。70%的患者在 3 个月内、86.6%的患者在 6 个月内、91.7%的患者在 9 个月内持续缓解炎症。达到静止状态的平均时间为 3.5 个月。在 6 个月内将皮质类固醇泼尼松降至≤10mg/d，同时保持眼部炎症控制的成功率为 89.7%（95%置信区间[CI]，81.1%-93.5%）。对 CyP 有阳性反应的患者的临床缓解平均持续时间为 32.67 个月（95%CI，25.91-39.43）。1 例患者（1.5%）在完成治疗疗程后出现血管炎复发。在疾病过程中早期开始治疗与较低的复发率相关（P=0.028）。最常见的不良反应是恶心（29%）和短暂性淋巴细胞减少症（26%）。治疗后平均最佳矫正视力（BCVA）从基线时的 0.59±0.66 提高到 6 个月随访时的 0.30±0.54（P<0.001）。平均随访时间为 31.61±20.47 个月。
采用我们方案的脉冲式 IV CyP 治疗难治性和暴发性、威胁视力的眼部炎症性疾病，可实现极高的持续完全缓解率，且在接受过这种治疗技术培训和熟练掌握的医生手中，具有极佳的安全性。它还允许大多数患者逐渐减少和停止使用皮质类固醇。早期开始治疗可能会降低复发的风险。
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