Does sacubitril/valsartan work in children with heart failure?—a pilot study
BackgroundSacubitril/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 res...
| Main Authors: | , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2023-11-01
|
| Series: | Frontiers in Cardiovascular Medicine |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fcvm.2023.1274990/full |
| _version_ | 1797448733417275392 |
|---|---|
| author | Yahe Xu Mingjie Zhang Yijun Chen Xi Chen Wenting Song Limin Zhu Liping Liu Xiaolei Gong Yuqi Zhang Zhuoming Xu |
| author_facet | Yahe Xu Mingjie Zhang Yijun Chen Xi Chen Wenting Song Limin Zhu Liping Liu Xiaolei Gong Yuqi Zhang Zhuoming Xu |
| author_sort | Yahe Xu |
| collection | DOAJ |
| description | BackgroundSacubitril/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.MethodsClinical 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.ResultsForty-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.ConclusionsSacubitril/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. |
| first_indexed | 2024-03-09T14:14:41Z |
| format | Article |
| id | doaj.art-5fcb75a1815b4306890c2efefe5c70e1 |
| institution | Directory Open Access Journal |
| issn | 2297-055X |
| language | English |
| last_indexed | 2024-03-09T14:14:41Z |
| publishDate | 2023-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Cardiovascular Medicine |
| spelling | doaj.art-5fcb75a1815b4306890c2efefe5c70e12023-11-29T04:49:17ZengFrontiers Media S.A.Frontiers in Cardiovascular Medicine2297-055X2023-11-011010.3389/fcvm.2023.12749901274990Does sacubitril/valsartan work in children with heart failure?—a pilot studyYahe Xu0Mingjie Zhang1Yijun Chen2Xi Chen3Wenting Song4Limin Zhu5Liping Liu6Xiaolei Gong7Yuqi Zhang8Zhuoming Xu9Department of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Cardiology, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaDepartment of Pediatric Thoracic and Cardiovascular Surgery, Shanghai Children’s Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, ChinaBackgroundSacubitril/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.MethodsClinical 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.ResultsForty-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.ConclusionsSacubitril/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.https://www.frontiersin.org/articles/10.3389/fcvm.2023.1274990/fullsacubitril/valsartanheart failurecongenital heart diseasecardiomyopathyHFrEF—heart failure with reduced ejection fraction |
| spellingShingle | Yahe Xu Mingjie Zhang Yijun Chen Xi Chen Wenting Song Limin Zhu Liping Liu Xiaolei Gong Yuqi Zhang Zhuoming Xu Does sacubitril/valsartan work in children with heart failure?—a pilot study Frontiers in Cardiovascular Medicine sacubitril/valsartan heart failure congenital heart disease cardiomyopathy HFrEF—heart failure with reduced ejection fraction |
| title | Does sacubitril/valsartan work in children with heart failure?—a pilot study |
| title_full | Does sacubitril/valsartan work in children with heart failure?—a pilot study |
| title_fullStr | Does sacubitril/valsartan work in children with heart failure?—a pilot study |
| title_full_unstemmed | Does sacubitril/valsartan work in children with heart failure?—a pilot study |
| title_short | Does sacubitril/valsartan work in children with heart failure?—a pilot study |
| title_sort | does sacubitril valsartan work in children with heart failure a pilot study |
| topic | sacubitril/valsartan heart failure congenital heart disease cardiomyopathy HFrEF—heart failure with reduced ejection fraction |
| url | https://www.frontiersin.org/articles/10.3389/fcvm.2023.1274990/full |
| work_keys_str_mv | AT yahexu doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT mingjiezhang doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT yijunchen doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT xichen doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT wentingsong doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT liminzhu doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT lipingliu doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT xiaoleigong doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT yuqizhang doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy AT zhuomingxu doessacubitrilvalsartanworkinchildrenwithheartfailureapilotstudy |