| Sumario: | 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.
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