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Table 1 Patient characteristics

From: Tranilast for advanced heart failure in patients with muscular dystrophy: a single-arm, open-label, multicenter study

Items

 

FAS (n = 17)

Age (years)

Mean ± SD

33.6 ± 8.0

Sex

Male/female

16 (94.1%)/1 (5.9%)

Diseases

Duchenne muscular dystrophy

13 (76.5%)

 

Becker muscular dystrophy

3 (17.6%)

 

Limb-Girdle muscular dystrophy

1 (5.9%)

Previous illness

No/Yes

11 (64.7%)/6 (35.3%)

 

Severe heart failure

3 (17.6%)

 

Lethal arrhythmia

2 (11.8%)

 

acute nephritis/severe renal dysfunction

1 (5.9%)

 

Thrombosis/embolism

1 (5.9%)

Complications

No/yes

12 (70.6%)/5 (29.4%)

 

Biliary disorders (gall stone, cholecystolithiasis)

1 (5.9%)

 

Rash

2 (11.8%)

 

Others

2 (11.8%)

Allergy

No/yes

9 (52.9%)/8 (47.1%)

 

Rhinitis

3 (17.6%)

 

Hay fever

2 (11.8%)

 

Atopic dermatitis

3 (17.6%)

 

Drug hypersensitivity (Not for tranilast)

2 (11.8%)

Mitral regurgitation

Absent or trivial/mild/moderate

7 (41.2%)/7 (41.2%)/3 (17.6%)

BNP (pg/mL)

GM ± GSD IQR

192.9 ± 1.72 (132–260)

hANP (pg/mL)

GM ± GSD IQR

189.7 ± 1.74 (152–284)

cTnT (ng/mL)

GM ± GSD IQR

0.026 ± 1.76 (0.02–0.03)

FS (%)

Mean ± SD IQR

9.0 ± 3.8 (6–13)

LVDD (mm)

Mean ± SD IQR

58.1 ± 6.3 (55–61)

Motor function

Ambulant/non-ambulant

1 (5.9%)/ 16 (94.1%)

Mechanical ventilation

No/intermittent/full-time

2 (11.8%)/13 (76.5%)/ 2 (11.8%)

Oxygen inhalation

No/yes

16 (94.1%)/1 (5.9%)

Nutrition

Oral/tube feeding

8 (47.1%)/9 (52.9%)

ACEI/ARB

Yes/no

15 (88.2%)/2 (11.8%)

Beta-blocker

Yes/no

15 (88.2%)/2 (11.8%)

Digitalis

Yes/no

2 (11.8%)/15 (88.2%)

Diuretics (except aldosterone receptor antagonist)

Yes/no

6 (35.3%)/11 (64.7%)

Aldosterone receptor antagonist

Yes/no

8 (47.1%)/9 (52.9%)

Cardiotonic agents

Yes/no

3 (17.6%)/14 (82.4%)

Anti-arrhythmic agents

Yes/no

10 (58.8%)/7 (41.2%)

  1. *The diagnosis was based on genetic analysis in 15 patients and muscle biopsy in one DMD and one LGMD patient
  2. FAS, full analysis set; GM, geometric mean; GS, geometric standard deviation; IQR, inter quartile range; BNP, brain natriuretic peptide; hANP, human atrial; cTnT, cardiac troponin T; FS, fractional shortening; LVDD; Left Ventricular Diastolic Dysfunction; ACEI/ARB, angiotensin-converting enzyme inhibitors/ angiotensin-receptor blockers