Efficacy and safety of sacubitril valsartan in treatment of dilated cardiomyopathy in children

Objective To observe the efficacy and safety of sacubitril valsartan in the treatment of dilated cardiomyopathy (DCM) in children. Methods A total of 19 hospitalized children with DCM were retrospectively selected as study subjects. All patients received captopril combined with conventional anti-heart failure therapy for 1 to 4 weeks, but due to ineffective treatment, they were switched to treatment of sacubitril valsartan. Clinical data, biochemical indicators, and echocardiographic findings were collected to assess changes in clinical symptoms and related indicators after 1-, 3-, 6-, and 12-month sacubitril valsartan treatment. Information on the dosage and administration of sacubitril valsartan, as well as the occurrence of adverse reactions was collected. Results Among 19 patients, there were 8 males and 11 females, with a median age of 3 years. Heart function was classified as grade Ⅱ in 2 patients, grade Ⅲ in 10 patients, and grade Ⅳ in 7 patients. At the final follow-up, the efficacy assessment showed significant improvement in 8 patients, effective improvement in 6 patients, and no improvement in 5 patients, with a total effective rate of 73.7%. After 1-, 3-, 6-, and 12-month sacubitril valsartan treatment, the levels of N-terminal pro-B-type natriuretic peptide (NT-proBNP) were lower, left ventricular ejection fraction (LVEF) was higher, and left ventricular diastolic diameter (LVDd)-Z value was smaller compared with those before treatment (P<0.05). The initial single dose of sacubitril valsartan was 0.8 mg/kg, and the average maintenance dose was 1.7 mg/kg, administered twice daily. After treatment, 1 patient developed hypotension, and 5 patients exhibited mild abnormalities in renal function indicators (of which 4 recovered to normal levels after tolerance). Conclusion Sacubitril valsartan can effectively improve heart failure symptoms and cardiac function indicators in children with DCM, but close monitoring of blood pressure and renal function changes is required during treatment.