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September 9, 2026Journal of the American College of Cardiology163 citationsOpen Access

Multicenter, Randomized, Double-Blind, Placebo-Controlled Study on the Effect of Oral Tolvaptan on Left Ventricular Dilation and Function in Patients With Heart Failure and Systolic Dysfunction

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JUJames E. UdelsonFMFrank McGrewEFEnrique Flores

Key Result

Tolvaptan 30 mg/day for 1 year did not significantly reduce left ventricular end-diastolic volume compared with placebo in patients with heart failure and reduced systolic function (p=0.21).

Key Points

  • To evaluate whether vasopressin V2 receptor antagonism with tolvaptan prevents or reverses left ventricular dilation over one year in patients with heart failure and systolic dysfunction.
  • Multicenter, randomized, double-blind, placebo-controlled trial (NCT00043758) enrolling 240 patients with heart failure and reduced systolic function randomly assigned to oral tolvaptan (30 mg/day, n=120) or placebo (n=120).
  • Left ventricular end-diastolic volume (LVEDV) was evaluated using quantitative radionuclide ventriculography at baseline, after 1 year of therapy, and approximately 1 week after drug withdrawal.
  • At 1 year, LVEDV changed by -1.8 ± 10.7 ml/m2 with tolvaptan compared to 0.0 ± 10.0 ml/m2 with placebo, showing no significant difference between groups (p = 0.21).
  • Tolvaptan significantly reduced the composite endpoint of all-cause mortality or heart failure hospitalization compared with placebo (6 deaths [5%] and 21 hospitalizations [18%] vs 11 deaths [9%] and 34 hospitalizations [28%]; log-rank p < 0.03).

Study Design

Type

RCT (n=240)

Blinding

Double-blind

Randomization

Randomized

Multicenter

Yes

Structured PICO

Does oral tolvaptan reduce left ventricular end-diastolic volume in stable patients with heart failure and reduced systolic function?

P
Population
240 patients with heart failure and reduced systolic function followed for 1 year.
I
Intervention
Oral tolvaptan 30 mg/day for 1 year
C
Comparator
Placebo for 1 year
O
Outcome
Change in left ventricular end-diastolic volume (LVEDV) after 1 year of therapy, measured using quantitative radionuclide ventriculographysurrogate

Tolvaptan 30 mg/day for 1 year did not significantly reduce left ventricular end-diastolic volume in stable heart failure patients, though a nonprespecified analysis suggested a potential reduction in mortality or heart failure hospitalization.

Main Result

Absolute Event Rate: -1.8% vs 0%

p-value: p=0.21

Abstract

OBJECTIVES This study sought to examine the effects of vasopressin V2 receptor antagonism with tolvaptan on the changes in left ventricular (LV) volumes over time. BACKGROUND Vasopressin levels may be increased in patients with heart failure (HF) and may be a factor driving the progression of HF. METHODS This was a multicenter, randomized, double-blind, placebo-controlled trial conducted to evaluate the effect of long-term administration of the vasopressin V2-receptor antagonist tolvaptan (30 mg/day) on reducing left ventricular end-diastolic volume (LVEDV) compared with placebo in patients with HF and reduced systolic function, using quantitative radionuclide ventriculography at baseline, repeated after 1 year of therapy, and repeated again approximately 1 week after withdrawal of study drug. RESULTS A total of 120 patients were randomized to tolvaptan and 120 were randomized to placebo. In the placebo group, there was no change in LVEDV over the course of follow-up (change of 0.0 +/- 10.0 ml/m2). After 1 year of tolvaptan, there was a small reduction in LV volume (decrease of 1.8 +/- 10.7 ml/m2); the between-group difference was not significant (p = 0.21). During the course of the trial, there were 6 deaths (5%) and 21 HF hospitalizations (18%) in the tolvaptan group, compared with 11 deaths (9%) and 34 HF hospitalizations (28%) in the placebo group. In a time-to-event analysis, there was a significant favorable effect of tolvaptan on the composite of mortality or heart failure hospitalization (p < 0.03 by log-rank test). CONCLUSIONS In a well-treated population of stable HF patients, there was no significant effect of tolvaptan therapy on LV volumes observed during 1 year of therapy. Nonprespecified natural history data favored therapy with tolvaptan, with a reduction in the combined end point of mortality and heart failure hospitalization observed. (Multicenter, Randomized, Double-Blind, Placebo Controlled, Efficacy Study on the Effects of Tolvaptan on Left Ventricular Dilatation in Congestive Heart Failure Patients; http://clinicaltrials.gov/ct/show/NCT00043758?order=1; NCT00043758).

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Cite This Study

Udelson et al. (2007) conducted an RCT in Heart failure and systolic dysfunction (n=240). Tolvaptan vs. Placebo was evaluated on Change in left ventricular end-diastolic volume (LVEDV) (p=0.21). Tolvaptan 30 mg/day for 1 year did not significantly reduce left ventricular end-diastolic volume compared with placebo in patients with heart failure and reduced systolic function (p=0.21).

synapsesocial.com/papers/6aa178dc5f13e43af75dd25ahttps://doi.org/10.1016/j.jacc.2007.01.091
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