St Vincent's Hospital, Sydney, John Wlodarczyk Consulting Services, Newcastle, New South Wales, Australia.
Intern Med J. 2011 Mar;41(3):227-34. doi: 10.1111/j.1445-5994.2009.02139.x.
BACKGROUND/AIMS: The Bosentan Patient Registry (BPR) was a prospective, multicentre, Australian registry funded by Actelion Pharmaceuticals. The primary aim of the registry was to collect survival data in patients with pulmonary arterial hypertension (PAH) treated with bosentan.
The BPR was initiated in 15 specialized PAH centres. All patients on or starting bosentan were invited to enrol. Treating physicians notified the registry if patients discontinued bosentan, because of either a change in therapy, transplantation, intervention or death. Survival data were validated against the Australian Institute of Health and Welfare National Death Index.
Between 2004 and 2007, a total of 528 patients (mean age 59 ± 17 years) were enrolled representing 69% of patients either previously taking or initiated on bosentan during that time. The BPR population was generally older with more advanced functional deficit than patients enrolled in randomized, placebo-controlled trials. Aetiology was idiopathic (iPAH) in 58% and connective tissue disease related (scleroderma (SSc)-PAH) in 42%. For iPAH patients, World Health Organisation functional classes II, III and IV at enrolment was 8.2%, 66.4% and 20.5%, and for the SSc-PAH cohort, 3.2%, 75.8% and 17.9% respectively. The observed annual mortality was 11.8% in patients with iPAH and 16.6% in patients SSc-PAH.
This large Australian registry provides 'real life' information on the characteristics and management of PAH in clinical practice. Treatment with bosentan improved survival outcomes in both iPAH and SSc-PAH compared with historical controls. Age, disease severity and aetiology were critical factors in determining clinical outcomes.
背景/目的：博森坦患者注册（BPR）是一项由 Actelion 制药公司资助的前瞻性、多中心、澳大利亚注册研究。该注册研究的主要目的是收集接受博森坦治疗的肺动脉高压（PAH）患者的生存数据。
该注册研究在 15 个专门的 PAH 中心启动。所有正在接受或开始接受博森坦治疗的患者均被邀请入组。如果患者因治疗改变、移植、介入或死亡而停止博森坦治疗，主治医生将向该注册研究报告。生存数据与澳大利亚卫生和福利研究所国家死亡指数进行了验证。
2004 年至 2007 年，共有 528 名患者（平均年龄 59 ± 17 岁）入组，占同期接受或开始接受博森坦治疗的患者的 69%。BPR 人群的年龄普遍较大，功能缺陷程度较随机、安慰剂对照试验中的患者更为严重。病因学方面，特发性肺动脉高压（iPAH）占 58%，结缔组织疾病相关肺动脉高压（硬皮病相关肺动脉高压（SSc-PAH））占 42%。在 iPAH 患者中，入组时的世界卫生组织功能分类 II、III 和 IV 分别为 8.2%、66.4%和 20.5%，而在 SSc-PAH 队列中，分别为 3.2%、75.8%和 17.9%。iPAH 患者的观察年死亡率为 11.8%，SSc-PAH 患者为 16.6%。
这项大型澳大利亚注册研究提供了 PAH 在临床实践中的特征和管理的“真实生活”信息。与历史对照相比，博森坦治疗改善了 iPAH 和 SSc-PAH 患者的生存结局。年龄、疾病严重程度和病因是决定临床结局的关键因素。
