Translational Studies in Sarcoidosis

临床方案(NHLBI06-H-0072)：阿托伐他汀作为第二期和第三期肺结节病的疾病改良剂：一项随机、双盲、安慰剂对照试验。 目标： 该方案的目的是在需要泼尼松治疗的肺结节病患者中进行一项随机、双盲、安慰剂对照试验，目的是确定在泼尼松剂量逐渐减少的情况下，阿托伐他汀是否能减少类固醇的使用和延长非类固醇的使用时间。 主要终点是类固醇节约期的持续时间。次级临床和生理终点旨在分析药物可能的抗炎和有益作用。由于结节病没有金标准的结果衡量标准，将使用四类次级终点来表征治疗剂对疾病临床病程的影响：成像(高分辨率胸部CT)；生活质量评估(SF-36和SGRQ)；抗炎效应(生物标记物和复发率)和功能效应(CPET、PFTs)。最后，我们将研究呼出的一氧化氮和一氧化碳在监测疾病活动中的作用。 取得的进展： 到目前为止，已联系了544多名受试者，并使用IRB批准的筛查问卷采访了约500人。大约有30名患者符合进一步筛查的条件。24名受试者已经登记，随机，并开始使用研究代理。 招聘数据： 招聘参数(n，受试者) 主题联系人(544) 电话筛选问卷(500份) 完成临床检查/筛查(91) 接受(27) 招生(24人) 符合进一步筛查条件的受试者(30) 人体组织(体液) 2008年样本总数 血液(血清/血浆)29 101 BAL 8 11

Clinical Protocol (NHLBI 06-H-0072): Atorvastatin as a disease-modifying agent in Stage II and Stage III Pulmonary Sarcoidosis: A randomized, double-blinded, placebo-controlled trial. Objectives: The objective of this protocol is to conduct, in patients with pulmonary sarcoidosis who require prednisone treatment, a randomized, double-blind placebo-controlled, trial which aims to determine, as prednisone dose is tapered, if atorvastatin administration results in less steroid use and longer steroid-free intervals. The primary endpoint is the duration of the steroid-sparing period. Secondary clinical and physiological endpoints are intended to analyze possible anti-inflammatory and beneficial effects of the drugs. Since there is no gold standard outcome measure in sarcoidosis, four categories of secondary endpoints will be used to characterize the effects of the therapeutic agent on the clinical course of the disease: imaging (high resolution chest CT); quality of life assessments (SF-36 and SGRQ), anti-inflammatory effects (biomarkers and relapse rates), and functional effects (CPET, PFTs). Finally, we will study the utility of exhaled nitric oxide and carbon monoxide in monitoring disease activity. Progress achieved: To date, over 544 subjects have been contacted, and approximately 500 have been interviewed using an IRB-approved screening questionnaire. Approximately 30 patients were eligible for further screening. Twenty four subjects have been enrolled, randomized, and started on study agent. Recruitment Data: Recruitment Parameters (n, subjects) Subject Contacts (544) Telephone Screening Questionnaires (500) Clinical Exams/Screens Completed (91) Accepted (27) Enrolled (24) Subjects Eligible for Further Screening (30) Human Tissues (Fluids) Samples 2008 Total Blood (serum/plasma) 29 101 BAL 8 11