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，随机分配到安慰剂组或来氟米特组。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。患者每天服用100毫克的研究药物或安慰剂，持续三天，然后在接下来的12个月里服用20毫克的研究药物或安慰剂。病人和诊所工作人员将被分配治疗任务。所有患者，无论治疗任务如何，泼尼松的剂量为1mg /kg/天，不超过80mg qd。随机分组将根据目前使用（随机分组后两周内）的强的松剂量为10mg / d与目前使用（随机分组后两周内）的强的松剂量< 10mg / d（包括不使用强的松）进行分层。在随机化之后，强的松将根据标准计划逐渐减少到每天10毫克，持续16周。患者将在基线以及开始研究用药后的1、4、8、12、16、24周、9和12个月接受检查。主要研究结果包括：最佳矫正视力较基线下降10个字母或更多；无法在第16周前将强的松减少到每天10毫克；从第16周到第12个月，不能维持强的松剂量< 10mg qd；眼部炎症性疾病需要增加全身治疗。主要安全结局包括以下至少一项的发生：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检查的变化。次要疗效终点包括是否存在玻璃体浑浊和囊样黄斑水肿。

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.