First name
Jonathan
Middle name
H
Last name
Soslow

Title

Transforming Growth Factor-β Analysis of the VANISH Trial Cohort.

Year of Publication

2023

Number of Pages

e010314

Date Published

04/2023

ISSN Number

1941-3297

DOI

10.1161/CIRCHEARTFAILURE.122.010314

Alternate Title

Circ Heart Fail

PMID

36999957
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Featured Publication
No
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Title

Transforming Growth Factor-β Analysis of the VANISH Trial Cohort.

Year of Publication

2023

Number of Pages

e010314

Date Published

04/2023

ISSN Number

1941-3297

DOI

10.1161/CIRCHEARTFAILURE.122.010314

Alternate Title

Circ Heart Fail

PMID

36999957
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Featured Publication
No
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Publication Image

Title

Transforming Growth Factor-β Analysis of the VANISH Trial Cohort.

Year of Publication

2023

Number of Pages

e010314

Date Published

03/2023

ISSN Number

1941-3297

DOI

10.1161/CIRCHEARTFAILURE.122.010314

Alternate Title

Circ Heart Fail

PMID

36999957
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Featured Publication
No
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Title

Valsartan in early-stage hypertrophic cardiomyopathy: a randomized phase 2 trial.

Year of Publication

2021

Number of Pages

Date Published

2021 Sep 23

ISSN Number

1546-170X

Abstract

<p>Hypertrophic cardiomyopathy (HCM) is often caused by pathogenic variants in sarcomeric genes and characterized by left ventricular (LV) hypertrophy, myocardial fibrosis and increased risk of heart failure and arrhythmias. There are no existing therapies to modify disease progression. In this study, we conducted a multi-center, double-blind, placebo-controlled phase 2 clinical trial to assess the safety and efficacy of the angiotensin II receptor blocker valsartan in attenuating disease evolution in early HCM. In total, 178 participants with early-stage sarcomeric HCM were randomized (1:1) to receive valsartan (320 mg daily in adults; 80-160 mg daily in children) or placebo for 2 years ( NCT01912534 ). Standardized changes from baseline to year 2 in LV wall thickness, mass and volumes; left atrial volume; tissue Doppler diastolic and systolic velocities; and serum levels of high-sensitivity troponin T and N-terminal pro-B-type natriuretic protein were integrated into a single composite z-score as the primary outcome. Valsartan (n = 88) improved cardiac structure and function compared to placebo (n = 90), as reflected by an increase in the composite z-score (between-group difference +0.231, 95% confidence interval (+0.098, +0.364); P = 0.001), which met the primary endpoint of the study. Treatment was well-tolerated. These results indicate a key opportunity to attenuate disease progression in early-stage sarcomeric HCM with an accessible and safe medication.</p>

DOI

10.1038/s41591-021-01505-4

Alternate Title

Nat Med

PMID

34556856
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