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

BACKGROUND

Dapagliflozin (DAPA), a sodium-glucose cotransporter-2 inhibitor, has shown promise in adult patients with heart failure (HF), but its safety and efficacy in children remain underexplored.

METHODS

In this randomized controlled pilot study, 20 children (aged 1-12 years, median 2.2 years, and eight boys) with dilated cardiomyopathy and HF with reduced ejection fraction (HFrEF) were randomized to receive either DAPA plus standard therapy (n = 9) or standard therapy alone (n = 11). The primary outcome was a composite of worsening HF, HF hospitalization, and cardiovascular death over a 6-month follow-up period. Secondary outcomes included adverse events, such as changes in renal function, volume status, serum electrolyte levels, blood glucose levels, ketoacidosis, and urinary tract infections. The other secondary efficacy outcomes included N-terminal pro-brain natriuretic peptide (NT-proBNP) levels and changes in functional class from baseline to 6 months.

RESULTS

The primary composite outcome occurred in three patients: one (cardiovascular death) in the DAPA group and two (cardiovascular death and HF hospitalization) in the control group (11.1% vs. 18.25%, P = 0.58). Two patients in the DAPA group developed transient prerenal acute kidney injury. Mild hyponatremia was present in 45% of patients during follow-up, with no significant difference between the groups (P = 0.34). No cases of ketoacidosis or urinary infection were observed in DAPA group. Both the groups demonstrated improvements in NT-proBNP levels and functional class, with no statistically significant differences observed.

CONCLUSIONS

Dapagliflozin was generally well tolerated in children with HFrEF, with a safety profile similar to that of the control group. However, no significant differences were observed in efficacy outcomes between the groups in this pilot study.