Table 4.
VANISH (n=212) | INHERIT21 (n=133) | Shimada et al20(n=20) | CHANCE19 (n=24) | Yamazaki et al17 (n=19) | Kawano et al16 (n=23) | Araujo et al18 (n=30) | |
---|---|---|---|---|---|---|---|
Age (yr) | 22.2 (9.7) | 52 (13) | 51 (13) | 53 (13) | 55 (6)/58 (9) | 64 (range 31–79) | 34(12)/32(13) |
Female gender (%) | 40.6 | 35.3 | 15.0 | 54.0 | 0 | 21.7 | 50.0 |
Race (%) | |||||||
White | 95.8 | 94.0 | -- | -- | -- | -- | -- |
Black | 1.4 | 0 | -- | -- | -- | -- | -- |
Other | 2.8 | 6.0 | -- | -- | -- | -- | -- |
NYHA class (%) | |||||||
I | 93.4 | 63.9 | 90.0 | 35.0 | -- | -- | 33.3 |
II | 6.6 | 30.0 | 10.0 | 35.0 | -- | -- | 46.7 |
III and IV | 0 | 6.0 | 0 | 30.0 | -- | -- | 20.0 |
Pathogenic or likely pathogenic variant (%)* | 100 | 42.9# | -- | 81.8† | -- | -- | -- |
MYBPC3 | 51.4 | 50.9 | -- | 33.3 | -- | -- | -- |
MYH7 | 34.4 | 28.1 | -- | 50.0 | -- | -- | -- |
TNNI3 | 3.3 | 10.5 | -- | 16.0 | -- | -- | -- |
TNNT2 | 3.8 | 5.3 | -- | - | -- | -- | -- |
Cardiac medications (%) | |||||||
Beta-blocker | 17.0 | 57.1 | 35.0 | 33.0 | 10.5 | 17.4 | -- |
Calcium channel blocker | 2.4 | 14.2 | 20.0 | 33.0 | 15.7 | 43.5 | -- |
Heart rate (bpm) | 70 (14) | 65 (11) | 63 (9)/65 (10) | 65/66 | -- | 64 (7)/65 (7) | -- |
Blood pressure (mmHg) | -- | ||||||
Systolic | 117 (12) | 128 (12) | 118 (13)/123 (8) | 113/119 | 126 (9)/128 (11) | 133 (16)/123 (13) | |
Diastolic | 68 (10) | 79 (9) | -- | -- | 79 (8)/79 (6) | 76 (14)/76 (6) | |
Imaging findings | |||||||
Max. LV wall thickness (mm) | 15 (4) | 23 (6) | 16 (4)/15 (3)‡ | 20 (4)/20 (3) | 22 (6)/20(3) | 18 (4)/17 (4) ‡ | 26 (6)/24 (8) ‡ |
LVEF (%) | 66 (8) | 69 (8) | 73 (7)/71 (8) | 69 (5)/70 (6) | -- | 72 (10)/70 (17) | -- |
LA diameter (mm) | 34 (6) | 41 (7) | -- | -- | -- | 41 (5)/44 (7) | 44 (6)/44 (5) |
LV mass (g) | 116 (42) | -- | 155 (135–222)/139 (112–205) | 407 (139)/451 (228) | 203 (47)/177 (48) | -- | -- |
LV mass (g/m2) | 63 (17) | 103 (36) | -- | -- | -- | -- |
Results are presented as % or as mean (SD) or median (IQR). Where parameters appear twice they were only reported separately for the two treatment arms.
indicates Not reported. CMR indicates cardiac magnetic resonance imaging; LA, left atrial; LV, left ventricular; LVEF, left ventricular ejection fraction; NYHA, New York Heart Association Class.
5 participants with MYBPC3 also have MYH7 (n=4) or TNNT2 (n=1).
108 (81%) patients had genetic testing performed prior to inclusion in the INHERIT trial, four patients were compound heterozygous.
22 (92%) patients had genetic testing performed in the CHANCE trial.
Diameter of interventricular septum (maximum wall thickness not reported).