Evaluation of efficacy, safety and tolerability of Ambrisentan in Chinese adults with pulmonary arterial hypertension: a prospective open label cohort study

摘要: Although several new drugs have been approved in recent years, pulmonary arterial hypertension (PAH) remains a rapidly progressive disease with poor prognosis. Ambrisentan, selective endothelin type A antagonist, has for treatment of PAH. This open label study assessed the efficacy and safety ambrisentan Chinese subjects Eligible patients PAH (World Health Organisation [WHO] functional class [FC] II orIII) were enrolled received Ambrisentan (5 mg) once daily 12-week preliminary evaluation period, dose-adjustment period (dose titration to 10 mgallowed). Endpoints included: change from baseline 6-Minute Walk Distance (6-MWD), N-Terminal Pro B-Type Natriuretic Peptide (NT-pro-BNP), WHO FC, Borg Dyspnoea Index (BDI), clinical worsening incidences adverse events (AE). One hundred thirty-three (85 % women, mean age: 36 years) (WHOFC or III) (5 mg) period. Mean (SD) duration drug exposure was 161.7 (27.13) days. (average dose 6.27 mg) significantly improved exercise capacity (6MWD) (mean: 377.1 m [m]) at week 12 (+53.6 m, p < 0.001) (primary endpoint). Improvement noted as early 4, sustained up 24 (+ 64.4 m, p < 0.001). NT-pro-BNP plasma levels decreased (p < 0.001) (−861.4 ng/L) (−806 ng/L) 1600.7 ng/L). The FC showed improvements 44 51 24. BDI scores (−0.3, (−0.2, p = 0.003) 2.5). Four died during (sudden cardiac death [n = 2], cerebral haemorrhage [n = 1], failure [n = 1]). Drug related occurred 34.3 % subjects; peripheral oedema (11.2 %) flushing (8.2 %) most frequently. 10 mg, orally) profile similar that observed global studies. NCT No. (ClinicalTrials.gov): NCT01808313 ; Registration date (first time): February 28, 2013.