PMID- 38094112 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20231214 IS - 2297-055X (Print) IS - 2297-055X (Electronic) IS - 2297-055X (Linking) VI - 10 DP - 2023 TI - Does sacubitril/valsartan work in children with heart failure?-a pilot study. PG - 1274990 LID - 10.3389/fcvm.2023.1274990 [doi] LID - 1274990 AB - BACKGROUND: Sacubitril/valsartan is an angiotensin receptor neprilysin antagonist (ARNI) approved for adult heart failure (HF). Its safety and efficacy in pediatric HF patients with cardiomyopathy or congenital heart disease are poorly understood. A pilot study was conducted to assess the clinical response, efficacy and safety of sacubitril/valsartan in this population at a tertiary care hospital in China. METHODS: Clinical parameters of patients who received sacubitril/valsartan from January 2019 to March 2023 were retrospectively collected and analyzed. Children over 1 month with a left ventricular ejection fraction (LVEF) <45% were included. Clinical efficacy was evaluated by echocardiographic LVEF, N-terminal pro-brain natriuretic peptide (NT-proBNP), New York Heart Association (NYHA) HF classification, HF re-admission, and death or transplantation. The initial dose was either 0.2 mg/kg bid or 0.4 mg/kg bid, with a target dose of 2.3 mg/kg bid or 3.1 mg/kg bid. RESULTS: Forty-five patients (60% male) with a median age of 7.86 years were enrolled. Among them, 23 had congenital heart disease and 22 had cardiomyopathies. The median maintenance dose was 0.76 mg/kg. The primary endpoint of LVEF up to 45% was reached by 24 patients (53.3%). The median NT-proBNP was significantly decreased from 5,501.5 pg/ml to 2,241.5 pg/ml (P < 0.001), more in congenital heart disease than in cardiomyopathies (P = 0.032). The NYHA HF class was improved or remained stable in 42 cases (93.3%). During a median follow-up of 1.23 years, 13 patients (28.9%) were re-hospitalized due to HF, and 9 patients (20%) died or underwent transplantation. Hypotension was the main adverse event, occurring in 8 patients. CONCLUSIONS: Sacubitril/valsartan may be effective in children with HF, but its safety and outcomes may differ depending on the etiology and anatomy of HF. Early post-operative congenital heart disease patients had less tolerance, more hypotension but better recovery and outcomes, while mid- and late- post-operative congenital heart disease patients and cardiomyopathy patients had less side effects but poorer clinical outcomes. CI - (c) 2023 Xu, Zhang, Chen, Chen, Song, Zhu, Liu, Gong, Zhang and Xu. FAU - Xu, Yahe AU - Xu Y AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Zhang, Mingjie AU - Zhang M AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Chen, Yijun AU - Chen Y AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Chen, Xi AU - Chen X AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Song, Wenting AU - Song W AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Zhu, Limin AU - Zhu L AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Liu, Liping AU - Liu L AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Gong, Xiaolei AU - Gong X AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Zhang, Yuqi AU - Zhang Y AD - Department of Pediatric Cardiology, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. FAU - Xu, Zhuoming AU - Xu Z AD - Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, China. LA - eng PT - Journal Article DEP - 20231129 PL - Switzerland TA - Front Cardiovasc Med JT - Frontiers in cardiovascular medicine JID - 101653388 PMC - PMC10716201 OTO - NOTNLM OT - HFrEF-heart failure with reduced ejection fraction OT - cardiomyopathy OT - congenital heart disease OT - heart failure OT - sacubitril/valsartan COIS- The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. EDAT- 2023/12/14 06:42 MHDA- 2023/12/14 06:43 PMCR- 2023/01/01 CRDT- 2023/12/14 04:10 PHST- 2023/08/11 00:00 [received] PHST- 2023/11/16 00:00 [accepted] PHST- 2023/12/14 06:43 [medline] PHST- 2023/12/14 06:42 [pubmed] PHST- 2023/12/14 04:10 [entrez] PHST- 2023/01/01 00:00 [pmc-release] AID - 10.3389/fcvm.2023.1274990 [doi] PST - epublish SO - Front Cardiovasc Med. 2023 Nov 29;10:1274990. doi: 10.3389/fcvm.2023.1274990. eCollection 2023.