Parameter [unit] | Placebo | Ambrisentan | 95% CI of difference | ||||
---|---|---|---|---|---|---|---|
Changes (n = 15) | Changes (n = 17) | ||||||
Baseline–6 months | Baseline–6 months | ||||||
Mean ± SD | 95% CI | n | Mean ± SD | 95% CI | n | ||
Hemodynamics at rest | |||||||
 CVP [mmHg] | − 0.20 ± 2.76 | (− 1.73 to 2.94) |  | 0.82 ± 4.11 | (− 1.29 to 2.94) |  | (− 1.54 to 3.59) |
 mPAP [mmHg] | − 0.73 ± 3.59 | (− 2.72 to 1.26) |  | − 1.00 ± 6.40 | (− 4.29 to 2.29) |  | (− 4.09 to 3.56) |
 PAWP [mmHg] | 0.13 ± 3.20 | (− 1.64 to 1.91) |  | 1.24 ± 5.31 | (− 1.50 to 3.97) |  | (− 2.12 to 4.32) |
 CO [l/min] | − 0.26 ± 1.11 | (−  0.88 to 0.36) |  | 0.58 ± 1.17 | (− 0.03 to 1.18) |  | (0.01 to 1.66) |
 CI [l/min/m2] | − 0.31 ± 0.71 | (− 0.71 to 0.08) |  | 0.36 ± 0.66 | (0.01 to 0.71) | (16) | (0.18 to 1.18) |
 SvO2 [%] | − 3.48 ± 12.26 | (− 10.89 to 3.93) | (13)# | − 2.79 ± 7.56 | (− 7.60 to 2.01) | (12)# | (− 7.83 to 9.20) |
 PVR [WU] | 0.02 ± 0.76 | (− 0.48 to 0.43) |  | − 0.59 ± 0.79 | (− 1.05 to − 0.14) |  | (− 1.25 to − 0.17) |
Hemodynamics at peak exercise | |||||||
 mPAP [mmHg] | 1.08 ± 7.39 | (− 3.39 to 5.54) | (13) | − 0.73 ± 6.23 | (− 4.18 to 2.71) | (15) | (− 7.10 to 3.48) |
 PAWP [mmHg] | 0.85 ± 6.3 | (− 2.96 to 4.65) | (13) | 4.93 ± 6.52 | (1.16 to 8.69) | (14) | (− 1.01 to 9.17) |
 CO [l/min] | − 0.05 ± 1.46 | (− 0.94 to 0.83) | (13) | 1.24 ± 1.47 | (0.43 to 2.05) | (15) | (0.15 to 2.44) |
 CI [l/min/m2] | − 0.45 ± 1.36 | (− 1.27 to 0.37) | (13) | 0.70 ± 0.81 | (0.25 to 1.15) | (15) | (0.29 to 2.00) |
 SvO2 [%] | − 28.35 ± 10.5 | (− 35.41 to − 21.30) | (11) | − 30.05 ± 17.73 | (− 42.73 to − 17.37) | (10) | (− 14.85 to 11.46) |
 Workload [Watt] | 1.92 ± 16.01 | (− 7.75 to 11.60) | (13) | 5.00 ± 16.9 | (− 4.36 to 14.36) | (15) | (− 9.77 to 15.93) |
 HR max [b/min] | 6.00 ± 18.74 | (− 5.32 to 17.32) | (13) | 7.47 ± 12.01 | (0.82 to 14.12) | (15) | (− 10.59 to 13.53) |
 PVR [WU] | − 0.003 ± 0.34 | (− 0.21 to 0.21) | (13) | − 0.84 ± 0.48 | (− 1.12 to − 0.57) | (14) | (− 1.17 to − 0.51) |
 TPR [mmHg min L− 1] | 0.16 ± 0.63 | (− 0.22 to 0.54) | (13) | − 0.56 ± 0.48 | (− 0.83 to − 0.29) | (15) | (− 1.16 to − 0.29) |
6MWD | |||||||
 6MWD [m] | − 16.53 ± 77.32 | (− 59.35 to 26.29) |  | 21.53 ± 34.6 | (3.74 to 39.31) |  | (− 4.30 to 80.43) |
 Borg dyspnea score | 0.08 ± 1.54 | (− 0.77 to 0.93) |  | 0.62 ± 1.7 | (− 0.25 to 1.50) |  | (− 0.63 to 1.72) |
 SaO2 after 6MWD [%] | 1.30 ± 5.1 | (− 2.35 to 4.95) | (10)# | 2.07 ± 5.7 | (− 1.22 to 5.37) | (14) | (− 3.92 to 5.46) |
 HR after 6MWD [/min] | 6.83 ± 24.97 | (− 9.04 to 22.70) | (12)# | − 1.53 ± 18.43 | (− 11.74 to 8.68) | (15) | (− 25.56 to 8.83) |
Quality of life SF-36 | |||||||
 Physical functioning | − 2.00 ± 25.20 | (− 15.96 to 11.96) |  | − 7.65 ± 21.66 | (− 18.78 to 3.49) |  | (− 22.56 to 11.27) |
 Physical role functioning | 13.33 ± 38.81 | (− 8.12 to 34.82) |  | − 10.29 ± 42.44 | (− 32.12–11.53) |  | (− 53.14 to 5.88) |
 Bodily pain | − 2.47 ± 28.19 | (− 18.08 to 13.15) |  | − 9.29 ± 23.87 | (− 21.57 to 2.98) |  | (− 25.62 to 11.97) |
 General health perceptions | 0.20 ± 19.39 | (− 10.54 to 10.94) |  | − 2.71 ± 10.62 | (− 8.17 to 2.75) |  | (− 14.01 to 8.20) |
 Vitality | − 5.00 ± 16.37 | (− 14.06 to 4.06) |  | − 3.53 ± 12.34 | (− 9.88 to 2.82) |  | (− 8.92 to 11.86) |
 Social role functioning | 0.87 ± 27.69 | (− 14.47 to 16.20) |  | − 2.18 ± 19.21 | (− 12.05 to 7.70) |  | (− 20.08 to 13.99) |
 Emotional role functioning | 8.80 ± 49.68 | (− 18.71 to 36.31) |  | − 9.82 ± 36.76 | (− 28.73 to 9.08) |  | (− 49.93 to 12.68) |
 Mental health | − 3.73 ± 11.85 | (− 10.30 to 2.83) |  | − 4.47 ± 10.94 | (− 10.10 to 1.16) |  | (− 8.97 to 7.49) |
 Physical health score | 0.87 ± 16.01 | (− 8.00 to 9.73) |  | − 6.71 ± 12.17 | (− 12.97 to − 0.45) |  | (− 17.77 to 2.62) |
 Mental health score | 0.27 ± 19.77 | (− 10.68 to 11.22) |  | − 4.65 ± 9.47 | (− 9.51 to 0.22) |  | (− 15.89 to 6.06) |
 mRSS | 0.73 ± 2.15 | (− 0.46 to 1.93) |  | 0.24 ± 0.97 | (− 0.26 to 0.73) |  | (− 1.68 to 0.68) |
Lung function | |||||||
 FVC [%] | − 1.04 ± 5.60 | (− 4.15 to − 2.06) |  | − 3. 31 ± 5.56 | (− 6.27 to − 0.34) | (16) | (− 6.37 to 1.84) |
 FEV1 [L] | − 0.06 ± 0.20 | (− 0.17 to 0.05) |  | − 0.11 ± 0.21 | (− 0.22 to 0.01) | (16) | (− 0.20 to 0.11) |
 FEV1% VC max [%] | − 0.67 ± 4.50 | (− 3.16 to 1.82) |  | − 4.17 ± 7.12 | (− 7.97 to − 0.38) | (16) | (− 7.91 to 0.91) |
 PEF [l/s] | −0.22 ± 1.76 | (−1.12 to 0.76) |  | 0.01 ± 1.15 | (−0.61 to 0.62) | (16) | (−0.86 to 1.31) |
 TLC [l] | − 0.03 ± 0.36 | (− 0.23 to 0.17) |  | − 0.06 ± 0.37 | (− 0.26 to 0.14) | (16) | (− 0.30 to 0.24) |
 Residual volume [l] | 0.05 ± 0.37 | (− 0.16 to 0.25) |  | − 0.03 ± 0.33 | (− 0.20 to 0.15) | (16) | (− 0.33 to 1.83) |
 DLCO [mmol/min/kPa] | − 0.45 ± 1.70 | (− 1.48 to 0.57) | (13) | − 0.32 ± 1.44 | (− 1.06 to 0.42) |  | (− 1.04 to 1.30) |
 DLCO % predicted | − 0.44 ± 1.84 | (− 1.55 to 0.67) | (13) | 1.19 ± 1.81 | (0.26 to 2.12) | (17) | (25.10 to 30.04) |
 SaO2 [%] | 0.15 ± 1.86 | (− 0.88 to 1.18) |  | − 0.62 ± 1.50 | (− 1.39 to 0.16) |  | (− 1.99 to 0.44) |
 PaO2 [mmHg] | 1.69 ± 9.95 | (− 3.82 to 7.21) |  | − 4.88 ± 7.60 | (− 8.78 to − 0.97) |  | (− 12.92 to − 0.22) |
 PaCO2 [mmHg] | − 0.03 ± 2.63 | (− 1.48 to 1.43) |  | − 0.65 ± 2.77 | (− 2.08 to 0.77) |  | (− 2.58 to 1.33) |
Echocardiography | |||||||
 Estimated sPAP [mmHg] | − 0.93 ± 6.08 | (− 4.30 to 2.43) |  | − 0.82 ± 4.46 | (− 3.11 to 1.47) |  | (− 3.71 to 3.93) |
 RA area [cm2] | − 0.47 ± 4.07 | (− 2.72 to 1.79) |  | 1.65 ± 2.67 | (0.28 to 3.01) |  | (− 0.34 to 4.57) |
 RV area [cm2] | − 0.80 ± 3.05 | (− 2.49 to 0.89) |  | − 0.15 ± 3.46 | (− 1.93 to 1.63) |  | (− 1.72 to 3.02) |
 TAPSE [cm] | − 0.19 ± 0.54 | (− 0.49 to 0.11) |  | 0.12 ± 0.41 | (− 0.09 to 0.33) |  | (− 0.04 to 0.65) |
Laboratory | |||||||
 Hemoglobin [g/dl] | 0.19 ± 0.68 | (− 0.19 to 0.56) |  | − 0.59 ± 0.86 | (− 1.03 to − 0.15) |  | (− 1.34 to − 0.21) |
 Hematocrit [l/l] | 0.00 ± 0.02 | (− 0.009 to 0.02) |  | − 0.01 ± 0.02 | (− 0.03 to − 0.0001) |  | (− 0.03 to 0.00) |
 Platelets [100/nl] | − 0.08 ± 0.39 | (− 0.29 to 0.14) |  | − 0.15 ± 0.37 | (− 0.34 to 0.04) |  | (− 0.35 to 0.20) |
 Creatinine [mg/dl] | − 0.03 ± 0.09 | (− 0.08 to 0.02) |  | − 0.04 ± 0.11 | (− 0.09 to 0.02) |  | (− 0.08 to 0.07) |
 Potassium [mmol/l] | 0.09 ± 0.40 | (− 0.13 to 0.31) |  | − 0.06 ± 0.62 | (− 0.38 to 0.26) |  | (− 0.53 to 0.24) |
 AST [U/l] | − 4.40 ± 13.94 | (− 12.12 to 3.32) |  | 3.59 ± 7.96 | (− 0.51 to 7.68) |  | (− 0.08 to 16.06) |
 ALT [U/l] | − 4.93 ± 15.01 | (− 13.25 to 3.38) |  | 5.12 ± 7.83 | (1.09 to 9.15) |  | (1.56 to 18.55) |
 LDH [U/l] | − 7.00 ± 27.36 | (− 22.15 to 8.15) |  | 2.82 ± 29.57 | (− 12.38 to 18.03) |  | (− 10.84 to 30.49) |
 CK [U/l] | 9.21 ± 40.50 | (− 14.17 to 32.60) | (14) | 5.53 ± 36.80 | (− 13.39 to 24.45) |  | (− 32.10 to 24.73) |
 CRP [mg/l] | − 1.08 ± 3.52 | (− 3.03 to 0.87) |  | − 2.71 ± 12.17 | (− 8.97 to 3.54) |  | (− 8.29 to 5.03) |
 NTproBNP [pg/ml] | 31.00 ± 85.83 | (− 20.87 to 82.87) | (13) | − 15.63 ± 207.48 | (− 126.18 to 94.93) | (16) | (−  172.95 to 79.70) |