Dapagliflozin in pediatric heart failure with reduced ejection fraction - A randomized pilot study

Area of Science:

• Cardiology
• Pediatrics
• Pharmacology

Background:

• Sodium-glucose cotransporter-2 inhibitors, like dapagliflozin, are effective in adults with heart failure (HF).
• The safety and efficacy of dapagliflozin in pediatric HF populations are not well-established.
• Dilated cardiomyopathy is a common cause of heart failure in children.

Purpose of the Study:

• To evaluate the safety and efficacy of dapagliflozin in children with heart failure with reduced ejection fraction (HFrEF).
• To assess adverse events and clinical outcomes in pediatric patients receiving dapagliflozin.

Main Methods:

• A randomized controlled pilot study involving 20 children (aged 1-12 years) with dilated cardiomyopathy and HFrEF.
• Patients were randomized to receive either dapagliflozin plus standard therapy or standard therapy alone for 6 months.
• Primary outcome: composite of worsening HF, HF hospitalization, and cardiovascular death. Secondary outcomes: adverse events, NT-proBNP levels, and functional class.

Main Results:

• The primary composite outcome occurred in 11.1% of the dapagliflozin group and 18.25% of the control group (P=0.58).
• Two patients in the dapagliflozin group experienced transient prerenal acute kidney injury; mild hyponatremia was observed in 45% without significant group difference.
• No ketoacidosis or urinary infections occurred in the dapagliflozin group. Both groups showed improvements in NT-proBNP and functional class.

Conclusions:

• Dapagliflozin was generally well-tolerated in children with HFrEF, exhibiting a safety profile comparable to the control group.
• This pilot study did not find statistically significant differences in efficacy outcomes between dapagliflozin and standard therapy in pediatric HFrEF patients.
• Further research is warranted to confirm the long-term safety and efficacy of dapagliflozin in pediatric heart failure.