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Sacubitril/Valsartan in Pediatric Heart Failure (PANORAMA-HF): A Randomized, Multicenter, Double-Blind Trial
Robert Shaddy1, Michael Burch2, Paul F Kantor1
1Children's Hospital Los Angeles and the Keck School of Medicine of the University of Southern California, Los Angeles (R.S., P.F.K., J.M.).
Circulation
|September 25, 2024
Summary
Sacubitril/valsartan did not prove superior to enalapril in treating pediatric heart failure (HF) with reduced left ventricular systolic dysfunction (LVSD). Both treatments demonstrated clinically meaningful improvements over 52 weeks in children.
Area of Science:
- Pediatric Cardiology
- Pharmacology
- Clinical Trials
Background:
- Sacubitril/valsartan, an angiotensin receptor-neprilysin inhibitor (ARNI), is approved for heart failure (HF) with reduced left ventricular ejection fraction.
- Limited direct comparative data exists between ARNIs and angiotensin-converting enzyme inhibitors (ACEIs) in pediatric HF populations.
- The PANORAMA-HF trial addresses this gap by comparing sacubitril/valsartan with enalapril in children with HF due to left ventricular systolic dysfunction (LVSD).
Purpose of the Study:
- To evaluate the pharmacokinetics, pharmacodynamics, safety, and efficacy of sacubitril/valsartan compared to enalapril in pediatric patients with HF.
- To determine if sacubitril/valsartan is superior to enalapril in treating children with HF attributable to systemic LVSD.
- To assess clinical outcomes and safety profiles of both treatment regimens over a 52-week follow-up period.
Main Methods:
- A randomized, double-blind trial involving 375 children (1 month to <18 years) with HF and LVSD.
- Participants were randomized to receive either sacubitril/valsartan or enalapril for 52 weeks.
- The primary efficacy endpoint was a global rank composite, considering clinical events, functional class (NYHA/Ross), and quality of life.
Main Results:
- No significant difference was found between sacubitril/valsartan and enalapril regarding the primary global rank endpoint (P=0.42).
- Both treatment groups showed clinically meaningful reductions in NYHA/Ross class, Patient Global Impression of Severity (PGIS), and NT-proBNP levels.
- Adverse event rates were similar between the groups (88.8% for sacubitril/valsartan vs. 87.8% for enalapril), with an acceptable safety profile for sacubitril/valsartan.
Conclusions:
- Sacubitril/valsartan did not demonstrate superiority over enalapril in the treatment of pediatric HF with LVSD based on the primary composite endpoint.
- Both sacubitril/valsartan and enalapril provided clinically meaningful benefits for pediatric patients with HF over 52 weeks.
- The safety profile of sacubitril/valsartan was deemed acceptable in this pediatric population.
