OBJECTIVETo compare the clinical characteristics and survival on Chinese patients with idiopathic pulmonary arterial hypertension (IPAH) and familiar pulmonary arterial hypertension (FPAH) during conventional therapy era and targeted therapy era.

METHODSIPAH and FPAH patients who were referred between Jan 1999 and Oct 2004 in Fuwai Hospital were defined as conventional therapy era group (before 2005 no PAH-specific drug was available in China). All patients in this group were followed up till Jun 2005. IPAH and FPAH patients who were referred between Sep 2006 and Aug 2011 were defined as targeted therapy era group (new PAH-specific drugs were available in China since 2006) were analyzed. All patients in this group were followed up till Dec 2013. The primary endpoints were death and therapy medicine.

RESULTSSeventy-two patients were enrolled in conventional therapy era group, 375 were enrolled in targeted therapy era group. The mean age was (35.9 ± 12.2) years and (34.5 ± 17.4) years respectively (P = 0.67), and women was predominant in both groups. There was no difference in WHO functional class and hemodynamic data between the two groups. About 90.3% patients were treated by calcium-channel blockers (CCB) in conventional therapy era group. In targeted therapy era group, almost all patients were treated by at least one PAH-specific drug, only 3.2% patients who had a positive response to acute pulmonary vasodilator testing were treated by CCB. The median survival time was 30.4 months in conventional therapy era group and 66.2 months in targeted therapy era group. The 1-, 2-, 3- and 5- year survival rates of IPAH and FPAH patients were 68.0%, 56.9%, 38.9% and 20.8% in conventional therapy era group, and 89.3%, 78.1%, 68.2% and 53.7% in targeted therapy era group respectively (P < 0.000 1).

CONCLUSIONCompared with conventional therapy era, the survival rate of Chinese IPAH and FPAH patients is significantly improved in targeted therapy era.