Epidemiol Rev. 2022 Dec 21;44(1):2-16. doi: 10.1093/epirev/mxac001.
The uveitides consist of >30 diseases characterized by intraocular inflammation. Noninfectious intermediate, posterior, and panuveitides typically are treated with oral corticosteroids and immunosuppression, with a similar treatment approach for most diseases. Because these uveitides collectively are considered a rare disease, single-disease trials are difficult to impractical to recruit for, and most trials have included several different diseases for a given protocol treatment. However, measures of uveitis activity are disease specific, resulting in challenges for trial outcome measures. Several trials of investigational immunosuppressive drugs or biologic drugs have not demonstrated efficacy, but design problems with the outcome measures have limited the ability to interpret the results. Successful trials have included diseases for which a single uveitis activity measure suffices or a composite measure of uveitis activity is used. One potential solution to this problem is the use of a single, clinically relevant outcome, successful corticosteroid sparing, defined as inactive uveitis with a prednisone dose ≤7.5 mg/day coupled with disease-specific guidelines for determining inactive disease. The clinical relevance of this outcome is that active uveitis is associated with increased risks of visual impairment and blindness, and that prednisone doses ≤7.5 mg/day have a minimal risk of corticosteroid side effects. The consequence of this approach is that trial visits require a core set of measures for all participants and a disease-specific set of measures, both clinical and imaging, to assess uveitis activity. This approach is being used in the Adalimumab Versus Conventional Immunosuppression (ADVISE) Trial.
葡萄膜炎由 >30 种疾病组成，其特征为眼内炎症。非传染性中间型、后葡萄膜炎和全葡萄膜炎通常采用口服皮质类固醇和免疫抑制治疗，大多数疾病的治疗方法相似。由于这些葡萄膜炎被认为是一种罕见病，单一疾病的试验难以招募，而且大多数试验为特定方案治疗纳入了几种不同的疾病。然而，葡萄膜炎活动的测量方法是疾病特异性的，这导致试验结果测量存在挑战。一些针对免疫抑制药物或生物药物的研究性药物试验并未显示出疗效，但结果测量的设计问题限制了对结果的解释能力。成功的试验包括那些只需要单一葡萄膜炎活动测量或使用葡萄膜炎活动综合测量的疾病。解决这个问题的一个潜在方法是使用单一的、临床上相关的结果，即成功的皮质类固醇保留，定义为泼尼松剂量≤7.5mg/天的无活动葡萄膜炎，同时结合确定无活动疾病的疾病特异性指南。该结果的临床相关性在于，活动性葡萄膜炎与视力损害和失明风险增加有关，泼尼松剂量≤7.5mg/天的皮质类固醇副作用风险最小。这种方法的结果是，试验访视需要为所有参与者提供一套核心测量指标，以及一套特定于疾病的临床和影像学测量指标，以评估葡萄膜炎的活动情况。这种方法正在 Adalimumab Versus Conventional Immunosuppression（ADVISE）试验中使用。
