Pilot Study Of Leflunomide For The Treatment Of Uveitis

本试验的目的是研究来氟米特治疗16例葡萄膜炎患者的安全性和有效性，这些患者随机分为安慰剂和来氟米特治疗。这项为期一年的研究是一项双盲、随机、安慰剂对照的干预研究。患者在三天内每天服用100毫克的研究药物或安慰剂，然后在接下来的12个月里每天服用20毫克的研究药物或安慰剂。患者和诊所工作人员将被戴上面具执行治疗任务。所有患者，无论治疗分配如何，均将给予泼尼松1毫克/公斤/天，不超过80毫克，每日一次。随机化将根据当前使用(随机后两周内)泼尼松10毫克每日一次与当前泼尼松10毫克每日一次(包括不使用泼尼松)进行分层。在随机化后，泼尼松将根据标准时间表在16周内逐渐减少到10 mg，po，qd。患者将在基线以及开始研究药物治疗后的1、4、8、12、16、24周、9和12个月进行检查。主要研究结果包括最佳矫正视力较基线下降10个字母或以上；无法在16周前将泼尼松减少到10毫克，每日1次；无法在16周至12个月期间维持泼尼松10毫克，每日1次；增加治疗眼部炎症所需的系统治疗。主要的安全结果包括至少以下一种情况的发生：腹痛、腹泻、皮疹、高血压或脱发，此外还将描述体重减轻和肝功能测试的变化。次要疗效终点包括是否有玻璃体混浊和黄斑囊样水肿。

The purpose of this trial is to investigate the safety and efficacy of leflunomide for the treatment of 16 patients with uveitis, randomly assigned to treatment with placebo or leflunomide. This one year study is a double-masked, randomized, placebo-controlled intervention study. Patients receive 100 mg of study drug or placebo each day for three days, then 20 mg of study drug or placebo for the next 12 months. Patients and clinic staff will be masked to treatment assignments. All patients, regardless of treatment assignment, will placed on prednisone at a dose of 1 mg/kg/day, not to exceed 80 mg qd. Randomization will be stratified by current use (within two weeks of randomization) of prednisone at a dose > 10 mg qd versus current use (within two weeks of randomization) of prednisone at a dose < 10 mg qd (including no prednisone use). Subsequent to randomization, prednisone will be tapered to 10 mg po qd over 16 weeks according to a standard schedule. Patients will be examined at baseline and at 1, 4, 8, 12, 16, 24 weeks, 9 and 12 months after beginning study medication. Primary study outcomes include a best corrected visual acuity decrease of 10 letters or more from baseline; inability to taper prednisone to 10 mg qd by Week 16; inability to maintain prednisone dose of < 10 mg qd from Week 16 through Month 12; addition of systemic therapy required to treat ocular inflammatory disease. Primary safety outcomes include the occurrence of at least one of the following: abdominal pain, diarrhea, skin rash, hypertension, or alopecia and additionally weight loss and changes in liver function tests will be described. Secondary efficacy endpoints will include the presence or absence of vitreous haze and cystoid macular edema.