Northwestern University Feinberg School of Medicine, McGaw Pavilion, Suite M300, 240 East Huron Street, Chicago, IL 60611, USA.
J Rheumatol. 2011 Oct;38(10):2172-9. doi: 10.3899/jrheum.101243. Epub 2011 Aug 15.
Pulmonary arterial hypertension (PAH) increases mortality in systemic sclerosis (SSc). The multicenter PHAROS registry (Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma) prospectively follows subjects with SSc at high risk for or with incident pulmonary hypertension (PH). We describe the registry design and baseline characteristics of subjects enrolled during the first 18 months since the start of the study.
High-risk subjects are enrolled and classified as Pre-PAH if they have (1) carbon monoxide diffusing capacity (DLCO) < 55% predicted; (2) percentage of predicted forced vital capacity/DLCO ratio ≥ 1.6; or (3) an estimated right ventricular systolic pressure > 35 mm Hg on echocardiography. Subjects with right heart catheterization (RHC)-confirmed incident PH (mean pulmonary artery pressure ≥ 25 mm Hg within previous 6 months) are subclassified into PAH, pulmonary venous hypertension secondary to left-side heart disease (PVH), and PH due to interstitial lung disease (PH-ILD). Baseline and biannual demographic, clinical, and laboratory data and patient-reported health questionnaires are collected.
There are 237 subjects enrolled in PHAROS. The majority are white (73%) and women (87%). There are 166 Pre-PAH and 71 Definite PH subjects (49 PAH, 7 PVH, and 15 PH-ILD).
PHAROS is the largest US and Canadian cohort of subjects with SSc at high risk for or with incident PAH. PAH-specific therapies are approved for 49/71 subjects with RHC-confirmed PAH. Analyses of PHAROS registry data will permit identification of risk factors for development of PAH among SSc patients at high risk for PAH and enhance understanding of the course of SSc-PAH.
肺动脉高压（PAH）会增加系统性硬化症（SSc）患者的死亡率。多中心 PHAROS 注册研究（肺动脉高压评估和认识硬皮病中的结局）前瞻性地随访 SSc 高危或新发肺动脉高压（PH）患者。我们描述了该注册研究的设计以及在研究启动后 18 个月内入组患者的基线特征。
高危患者被招募并分为 Pre-PAH，如果他们有：（1）一氧化碳弥散量（DLCO）<预测值的 55%；（2）预测用力肺活量/DLCO 比值的百分比≥1.6；或（3）超声心动图估计右心室收缩压>35mmHg。有右心导管（RHC）确诊的新发 PH（在过去 6 个月内平均肺动脉压≥25mmHg）的患者进一步分为 PAH、由左心疾病引起的肺静脉高压（PVH）和特发性肺间质纤维化相关 PH（PH-ILD）。收集基线和每半年的人口统计学、临床和实验室数据以及患者报告的健康问卷。
PHAROS 共入组 237 例患者。大多数患者为白人（73%）和女性（87%）。有 166 例 Pre-PAH 和 71 例确诊 PH 患者（49 例 PAH、7 例 PVH 和 15 例 PH-ILD）。
PHAROS 是美国和加拿大最大的 SSc 高危或新发 PAH 患者队列。有 49/71 例 RHC 确诊 PAH 患者接受了 PAH 特异性治疗。PHAROS 注册研究数据的分析将能够确定 SSc 高危患者发生 PAH 的危险因素，并加深对 SSc-PAH 病程的认识。
