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Table 1 Demographic and clinical characteristics of the study population

From: Early treatment with ambrisentan of mildly elevated mean pulmonary arterial pressure associated with systemic sclerosis: a randomized, controlled, double-blind, parallel group study (EDITA study)

Parameter [unit] Placebo
(N = 19)
Ambrisentan
(N = 19)
  Total
(N = 38)
Mean ± SD 95% CI n Mean ± SD 95% CI n 95% CI of difference Mean ± SD 95% CI n
Female sex no. [%] 14 (73.7)    16 (84.2)     30 (78.9)   
Age [years] 54.89 ± 11.23 (49.48 to 60.31)   58.79 ± 10.75 (53.61 to 63.97)   (− 3.33 to 11.12) 56.84 ± 11.02 (53.22 to 60.46)  
Height [cm] 166.21 ± 10.19 (161.30 to 171.12)   166.05 ± 5.59 (163.36 to 168.75)   (− 5.62 to 5.31) 166.13 ± 8.11 (163.47 to 168.80)  
Weight [kg] 74.81 ± 17.25 (66.48 to 83.10)   71.45 ± 15.83 (64.03 to 79.22)   (− 14.03 to 7.70) 73.13 ± 16.42 (67.83 to 78.59)  
Systolic blood pressure [mmHg] 121.32 ± 14.99 (114.09 to 128.54)   116.32 ± 11.53 (110.76 to 121.87)   (− 13.80 to 3.80) 118.82 ± 13.43 (114.40 to 123.23)  
Diastolic blood pressure [mmHg] 73.42 ± 9.14 (69.02 to 77.82)   73.68 ± 8.95 (69.37 to 78.00)   (− 5.69 to 6.21) 73.55 ± 8.92 (70.62 to 76.49)  
HR [beats/min] 77.58 ± 9.85 (72.83 to 82.33)   73.05 ± 11.12 (67.69 to 78.41)   (− 11.44 to 2.39) 75.32 ± 10.61 (72.83 to 78.81)  
WHO-FC no. [%]
 II 15 (78.9)    17 (89.5)     32 (84.2)   
 III 4 (21.1)    2 (10.5)     6 (15.8)   
Hemodynamic characteristic according to inclusion criteria
 mPAP rest 21–24 mmHg, mPAP exercise > 30 mmHg 12 (63.2)    10 (52.6)     22 (57.9)   
 mPAP rest 21–24 mmHg, mPAP exercise ≤ 30 mmHg 3 (15.8)    1 (5.3)     4 (10.5)   
 mPAP rest < 21 mmHg, mPAP exercise > 30 mmHg 4 (21.0)    8 (42.1)     2 (31.6)   
 mRSS [points] 11.47 ± 5.22 (8.72 to 14.23)   11.47 ± 5.71 (8.96 to 13.99)   (− 3.60 to 3.60) 11.47 ± 5.40 (9.70 to 13.25)  
SSc subgroup, no. (%)
 Diffuse 11 (57.9)    4 (21.1)     15 (39.5)   
 Limited 8 (42.1)    15 (78.9)     23 (60.5)   
 SSc disease duration [years] 13.28 ± 10.85 (8.05 to 18.51)   6.63 ± 4.80 (4.32 to 8.96)   (− 12.17 to 1.12) 9.96 ± 8.93 (7.02 to 12.90)  
Hemodynamics at rest
 CVP [mmHg] 5.63 ± 2.97 (4.20 to 7.06)   6.11 ± 2.94 (4.69 to 7.52)   (− 1.47 to 2.41) 5.87 ± 2.92 (4.90 to 6.82)  
 mPAP [mmHg] 21.32 ± 2.43 (20.15 to22.49)   19.84 ± 3.58 (18.12 to 21.57)   (− 3.49 to 0.54) 20.58 ± 3.11 (19.56 to 21.60)  
 PAWP [mmHg] 9.58 ± 2.97 (8.15 to 11.01)   9.42 ± 2.32 (8.30 to 10.54)   (− 1.91 to 1.59) 9.50 ± 2.63 (8.64 to 10.37)  
 CO [l/min] 5.66 ± 1.48 (4.95 to 6.37)   5.04 ± 1.26 (4.43 to 5.65)   (− 1.51 to 0.29) 5.35 ± 1.39 (4.89 to 5.81)  
 CI [l/min/m2] 3.20 ± 0.85 (2.79 to 3.61)   2.84 ± 0.64 (2.53 to 3.14)   (− 0.86 to 0.13) 3.02 ± 0.76 (2.77 to 3.27)  
 SvO2 [%] 73.13 ± 4.60 (70.67 to 75.58) (16) 73.91 ± 8.58 (69.50 to 78.32) (17) (− 4.15 to 5.71) 73.53 ± 6.85 (71.10 to 75.96) (33)
 PVR [WU] 2.09 ± 0.61 (1.80 to 2.39)   2.22 ± 0.93 (1.77 to 2.67)   (− 0.39 to 0.65) 2.16 ± 0.78 (1.90 to 2.41)  
Hemodynamics at peak exercise
 mPAP [mmHg] 36.94 ± 6.08 (33.82 to 40.07) (17) 37.78 ± 3.61 (35.98 to 39.57) (18) (− 2.58 to 2.25) 37.37 ± 4.91 (35.69 to 39.06) (35)
 PAWP [mmHg] 15.24 ± 5.43 (12.45 to 18.03) (17) 16.78 ± 6.42 (13.58 to 19.97) (18) (− 2.56 to 5.64) 16.03 ± 5.92 (13.99 to 18.06) (35)
 CO [l/min] 11.03 ± 3.62 (9.17 to 12.89) (17) 9.67 ± 2.67 (8.34 to 10.99) (18) (− 3.54 to 0.81) 10.33 ± 3.19 (9.23 to 11.43) (35)
 CI [l/min/m2] 6.04 ± 1.79 (5.12 to 6.96) (17) 5.41 ± 1.32 (4.75 to 6.06) (18) (− 1.71 to 0.45) 5.71 ± 1.58 (5.17 to 6.25) (35)
 SvO2 [%] 38.27 ± 6.10 (34.89 to 41.64) (15) 42.77 ± 11.56 (36.36 to 49.17) (15) (− 2.42 to 11.42) 40.52 ± 9.37 (37.02 to 44.02) (30)
 Workload [Watt] 75.00 ± 34.23 (57.40 to 92.60) (17) 75.00 ± 27.12 (61.52 to 88.48) (18) (− 21.18 to 21.17) 75.00 ± 30.32 (65.59 to 85.41) (35)
 HR max [/min] 117.65 ± 21.37 (106.66 to 128.63) (17) 111.39 ± 23.06 (99.92 to 122.86) (18) (− 21.57 to 9.06) 114.43 ± 22.15 (106.82 to 122.04) (35)
 PVR [WU] 2.07 ± 0.61 (1.76 to 2.38) (17) 2.29 ± 0.85 (1.87 to 2.71) (18) (− 0.29 to 0.73) 2.18 ± 0.74 (1.93 to 2.44) (35)
 TPR [mmHg min l− 1] 3.65 ± 1.18 (3.04 to 4.26) (17) 4.28 ± 1.65 (3.46 to 5.10) (18) (− 0.36 to 1.62) 3.97 ± 1.46 (3.47 to 4.47) (35)
6MWD
 6MWD [m] 448.11 ± 82.64 (408.28 to 487.93)   470.21 ± 77.03 (433.08 to 407.34)   (− 30.46 to 74.67) 459.16 ± 79.59 (433.00 to 485.32)  
 Borg dyspnea score 2.93 ± 1.97 (1.98 to 3.88)   2.41 ± 1.32 (1.77 to 3.04)   (− 1.63 to 0.58) 2.67 ± 1.68 (2.11 to 3.22)  
 SaO2 after 6MWD [%] 91.87 ± 4.63 (89.30 to 94.43) (15) 91.71 ± 4.79 (89.24 to 94.17) (17) (− 3.57 to 3.25) 91.78 ± 4.64 (90.10 to 93.45) (32)
 HR after 6MWD [/min] 99 ± 21.27 (88.07 to 109.93) (17) 107 ± 19.64 (96.90 to 117.10) (17) (− 6.30 to 22.30) 103 ± 20.56 (95.82 to 110.17) (34)
Echocardiography at rest
 Estimated sPAP [mmHg] 29.21 ± 5.16 (25.25 to 31.17)   28.58 ± 6.57 (26.04 to 31.43)   (− 4.52 to 3.26) 28.89 ± 5.83 (26.98 to 30.81)  
 RA area [cm2] 12.05 ± 4.24 (10.73 to 13.69)   11.68 ± 3.28 (10.45 to 13.66)   (− 2.86 to 2.13) 11.87 ± 3.74 (10.64 to 13.10)  
 RV area [cm2] 14.89 ± 4.56 (12.97 to 17.03)   14.13 ± 4.74 (12.65 to 16.82)   (− 3.82 to 2.30) 14.51 ± 4.60 (13.00–16.03)  
 TAPSE [cm] 2.50 ± 0.51 (2.23 to 2.71)   2.41 ± 0.33 (2.28 to 2.70)   (− 0.37 to 0.20) 16,834 ± 0.43 (2.31 to 2.60)  
Lung function
 FVC [%] 69.17 ± 13.72 (62.56 to 75.78)   70.51 ± 17.61 (62.02 to 79.00)   (− 6.37 to 1.84) 69.84 ± 15.58 (64.72 to 74.96)  
 FEV1 [L] 2.40 ± 0.77 (2.03 to 2.77)   2.26 ± 0.63 (1.96 to 2.57)   (− 0.60 to 0.33) 2.33 ± 0.70 (2.10 to 2.56)  
 FEV1% VC max [%] 79.31 ± 6.98 (75.95 to 82.68)   81.44 ± 15.38 (74.03 to 88.85)   (− 5.73 to 9.99) 80.38 ± 11.83 (76.49 to 84.27)  
 PEF [l/s] 5.46 ± 2.32 (4.34 to 6.58)   5.17 ± 1.72 (4.34 to 6.00)   (− 1.63 to 1.06) 5.31 ± 2.02 (4.65 to 5.98)  
 TLC [l] 4.96 ± 1.07 (4.44 to 5.48)   5.08 ± 1.25 (4.48 to 5.69)   (− 0.64 to 0.89) 5.02 ± 1.15 (4.64 to 5.40)  
 Residual volume [l] 1.94 ± 0.55 (1.68 to 2.21)   2.18 ± 0.70 (1.84 to 2.52)   (− 0.18 to 0.65) 2.06 ± 0.63 (1.85 to 2.27)  
 DLCO [mmol/min/kPa] 5.28 ± 1.52 (4.55 to 6.02)   5.03 ± 1.33 (4.38 to 5.67)   (− 1.20 to 0.68) 5.16 ± 1.42 (4.69 to 5.62)  
 DLCO % predicted 84.73 ± 2.61 (83.47 to 85.99)   84.16 ± 1.89 (83.25 to 85.08)   (− 2.07 to 0.93) 84.45 ± 2.27 (83.70 to 85.19)  
 SaO2 [%] 96.13 ± 1.80 (95.26 to 97.00)   96.82 ± 0.77 (96.45 to 97.17)   (− 0.23 to 1.60) 96.47 ± 1.41 (96.01 to 96.94)  
 PaO2 [mmHg] 78.78 ± 9.42 (74.24 to 83.32)   81.22 ± 5.91 (78.37 to 84.06)   (− 2.74 to 7.61) 80.00 ± 7.85 (77.42 to 82.58)  
 PaCO2 [mmHg] 39.01 ± 3.63 (37.25 to 40.76)   37.23 ± 2.57 (35.99 to 38.47)   (− 3.85 to 0.29) 38.12 ± 3.23 (37.05 to 39.18)  
Laboratory
 Hemoglobin [g/dl] 13.54 ± 1.26 (12.93 to 14.14)   13.62 ± 1.13 (13.07 to 14.16)   (− 0.71 to 0.87) 13.58 ± 1.18 (13.19 to 13.96)  
 Hematocrit [l/l] 0.42 ± 0.03 (0.40 to 0.43)   0.41 ± 0.03 (0.40 to 0.43)   (− 0.03 to 0.02) 0.41 ± 0.03 (0.40 to 0.42)  
 Platelet [100/nl] 2.53 ± 0.94 (2.08 to 2.98)   2.62 ± 0.63 (2.31 to 2.92)   (− 0.44 to 0.61) 2.58 ± 0.79 (2.32 to 2.83)  
 Creatinine [mg/dl] 0.83 ± 0.16 (0.75 to 0.91)   0.86 ± 0.12 (0.80 to 0.92)   (− 0.07 to 0.13) 0.85 ± 0.14 (0.80 to 0.89)  
 Potassium [mmol/l] 4.05 ± 0.38 (3.86 to 4.23)   4.20 ± 0.48 (3.97 to 4.43)   (− 0.13 to 0.44) 4.12 ± 0.43 (3.98 to 4.27)  
 AST [U/l] 24.11 ± 21.78 (13.61 to 34.60)   19.79 ± 7.06 (16.39 to 23.19)   (− 14.97 to 6.34) 21.95 ± 16.12 (16.65 to 27.25)  
 ALT [U/l] 29.68 ± 24.93 (17.67 to 41.70)   24.68 ± 9.85 (19.93 to 29.43)   (− 17.47 to 7.47) 27.18 ± 18.87 (20.98 to 33.39)  
 LDH [U/l] 197.63 ± 54.74 (171.25 to 224.01)   197.53 ± 35.59 (180.37 to 214.68)   (− 30.48 to 30.27) 197.58 ± 45.54 (182.61 to 212.55)  
 CRP [mg/l] 5.18 ± 5.17 (2.69 to 7.68)   5.28 ± 11.60 (− 0.48 to 11.05) (18) (− 5.84 to 6.04) 5.23 ± 8.77 (2.30 to 8.16) (37)
 NTproBNP [pg/ml] 123.42 ± 142.96 (54.52 to 192.32)   267.83 ± 303.11 (117.10 to 418.57) (18) (− 12.38 to 301.20) 193.68 ± 242.81 (112.71 to 274.63) (37)
Quality of life SF-36
 Physical functioning 50.26 ± 25.95 (37.75 to 62.77)   64.21 ± 25.83 (51.67 to 76.66)   (− 3.09 to 30.99) 57.24 ± 26.50 (48.52 to 65.95)  
 Physical role functioning 35.53 ± 40.24 (16.13 to 54.92)   51.32 ± 41.23 (31.45 to 79.19)   (− 11.01 to 42.59) 43.42 ± 40.97 (29.96 to 56.89)  
 Bodily pain 49.79 ± 28.96 (35.83 to 63.75)   62.00 ± 29.16 (47.95 to 76.05)   (− 6.91 to 31.33) 55.89 ± 29.33 (46.26 to 65.53)  
 General health perceptions 41.74 ± 13.07 (35.44 to 48.04)   54.42 ± 19.47 (45.03 to 63.81)   (1.72 to 23.65) 48.08 ± 17.58 (42.30 to 53.86)  
 Vitality 42.89 ± 19.32 (33.58 to 52.20)   50.53 ± 20.94 (40.03 to 60.62)   (− 5.62 to 20.89) 46.71 ± 20.24 (40.06 to 53.37)  
 Social role functioning 58.05 ± 25.99 (45.53 to 70.58)   74.47 ± 24.45 (62.69 to 86.26)   (− 0.18 to 33.03) 66.26 ± 26.24 (57.64 to 74.89)  
 Emotional role functioning 49.16 ± 46.33 (26.83 to 71.49)   63.16 ± 47.02 (40.50 to 85.82)   (− 16.71 to 44.71) 56.16 ± 46.58 (40.85 to 71.47)  
 Mental health 59.58 ± 18.85 (50.49 to 68.66)   64.42 ± 18.03 (55.73 to 73.11)   (− 7.30 to 16.98) 62.00 ± 18.36 (55.97 to 68.04)  
 Physical health score 43.89 ± 21.26 (33.65 to 54.14)   56.47 ± 23.87 (44.97 to 67.98)   (− 2.29 to 27.45) 50.18 ± 23.19 (42.56 to 57.81)  
 Mental health score 50.26 ± 20.70 (40.29 to 60.24)   61.42 ± 21.45 (51.08 to 71.76)   (− 2.71 to 25.03) 55.84 ± 21.55 (48.76 to 62.92)  
  1. SD standard deviation, HR heart rate, mRSS modified Rodnan Skin Score, WHO-FC World Health Organization functional class, CVP central venous pressure, mPAP mean pulmonary arterial pressure, PAWP pulmonary capillary wedge pressure, CO cardiac output, CI cardiac index, SvO2 venous oxygen saturation, PVR pulmonary vascular resistance, WU Wood Units, sPAP systolic pulmonary arterial pressure, RA right atrial, RV right ventricular, TAPSE tricuspid annular plane systolic excursion, FVC forced vital capacity, FEV1 forced expiratory volume in first second, VC vital capacity, PEF peak expiratory flow, TLC total lung capacity, DLCO diffusing capacity of the lung for carbon monoxide, SaO2 oxygen saturation, PaO2 partial pressure of oxygen, PaCO2 partial pressure of carbon dioxide, AST aspartate-aminotransferase, ALT alanine-aminotransferase, LDH lactate dehydrogenase, CRP C-reactive protein
  2. In case of missing data, sample sizes are given in brackets