Abstract：
BACKGROUND: The first angiotensin receptor/neprilysin inhibitor on the market, sacubitril-valsartan, has shown marked improvements in death and hospitalization for heart failure among adults, and is now approved for use in pediatric heart failure. While the ongoing PANORAMA-HF trial is evaluating the effectiveness of sacubitril-valsartan for pediatric patients with a failing systemic left ventricle, the enrollment criteria do not include the majority of pediatric heart failure patients. Additional studies are needed.
METHODS: Using the TriNetX database, we performed a propensity score matched, retrospective cohort study to assess the incidence of a composite of all-cause mortality or heart transplant within 1 year. The 519 patients who received sacubitril-valsartan were compared to 519 matched controls who received an angiotensin converting enzyme inhibitor (ACE) or angiotensin II receptor blocker (ARB).
RESULTS: There was no significant difference in the incidence of the composite outcome with sacubitril-valsartan over an ACE/ARB (13.3% vs 13.2%, p = 0.95), or among the components of mortality (5.0% vs 5.8%, p = 0.58) or heart transplantation (8.7% vs 7.5%, p = 0.50). Patients who were receiving full goal-directed medical therapy (14.4% vs 16.0%, p = 0.55) also showed no difference in the composite outcome. We observed a significantly increased incidence of hypotension (10% vs 5.2%, p = 0.006) and a trend toward reduced number of hospitalizations per year (mean (SD) 1.3 (4.4) vs 2.0 (9.1), p = 0.09).
CONCLUSIONS: Sacubitril-valsartan is not associated with a decrease in the composite of all-cause mortality or heart transplantation within 1 year. Future studies should evaluate the possible reduction in hospitalizations and optimal dosing to minimize hypotension.
METHODS: Using the TriNetX database, we performed a propensity score matched, retrospective cohort study to assess the incidence of a composite of all-cause mortality or heart transplant within 1 year. The 519 patients who received sacubitril-valsartan were compared to 519 matched controls who received an angiotensin converting enzyme inhibitor (ACE) or angiotensin II receptor blocker (ARB).
RESULTS: There was no significant difference in the incidence of the composite outcome with sacubitril-valsartan over an ACE/ARB (13.3% vs 13.2%, p = 0.95), or among the components of mortality (5.0% vs 5.8%, p = 0.58) or heart transplantation (8.7% vs 7.5%, p = 0.50). Patients who were receiving full goal-directed medical therapy (14.4% vs 16.0%, p = 0.55) also showed no difference in the composite outcome. We observed a significantly increased incidence of hypotension (10% vs 5.2%, p = 0.006) and a trend toward reduced number of hospitalizations per year (mean (SD) 1.3 (4.4) vs 2.0 (9.1), p = 0.09).
CONCLUSIONS: Sacubitril-valsartan is not associated with a decrease in the composite of all-cause mortality or heart transplantation within 1 year. Future studies should evaluate the possible reduction in hospitalizations and optimal dosing to minimize hypotension.
摘要：
背景：市场上第一个血管紧张素受体/脑啡肽抑制剂，沙库巴曲-缬沙坦，已经显示出成年人死亡和心力衰竭住院的显着改善，现在被批准用于小儿心力衰竭。虽然正在进行的PANORAMA-HF试验正在评估沙库巴曲-缬沙坦对系统性左心室衰竭的儿科患者的有效性，入选标准不包括大多数小儿心力衰竭患者.还需要进一步的研究。
方法：使用TriNetX数据库，我们做了一个匹配的倾向评分，回顾性队列研究，以评估1年内全因死亡率或心脏移植的复合发病率。将接受沙库巴曲-缬沙坦的519例患者与接受血管紧张素转换酶抑制剂（ACE）或血管紧张素II受体阻滞剂（ARB）的519例匹配对照进行比较。
结果：与ACE/ARB相比，沙库巴曲-缬沙坦复合结局的发生率没有显着差异（13.3％vs13.2％，p=0.95),或死亡率的组成部分(5.0%对5.8%，p=0.58）或心脏移植（8.7%vs7.5%，p=0.50)。接受完全目标导向药物治疗的患者（14.4%vs16.0%，p=0.55）也显示综合结果没有差异。我们观察到低血压的发生率显着增加（10％vs5.2％，p=0.006）和每年住院人数减少的趋势（平均值（SD）1.3（4.4）对2.0（9.1），p=0.09)。
结论：沙库必曲-缬沙坦与1年内全因死亡率或心脏移植的复合降低无关。未来的研究应评估可能减少住院和最佳剂量以最大程度地减少低血压。
方法：使用TriNetX数据库，我们做了一个匹配的倾向评分，回顾性队列研究，以评估1年内全因死亡率或心脏移植的复合发病率。将接受沙库巴曲-缬沙坦的519例患者与接受血管紧张素转换酶抑制剂（ACE）或血管紧张素II受体阻滞剂（ARB）的519例匹配对照进行比较。
结果：与ACE/ARB相比，沙库巴曲-缬沙坦复合结局的发生率没有显着差异（13.3％vs13.2％，p=0.95),或死亡率的组成部分(5.0%对5.8%，p=0.58）或心脏移植（8.7%vs7.5%，p=0.50)。接受完全目标导向药物治疗的患者（14.4%vs16.0%，p=0.55）也显示综合结果没有差异。我们观察到低血压的发生率显着增加（10％vs5.2％，p=0.006）和每年住院人数减少的趋势（平均值（SD）1.3（4.4）对2.0（9.1），p=0.09)。
结论：沙库必曲-缬沙坦与1年内全因死亡率或心脏移植的复合降低无关。未来的研究应评估可能减少住院和最佳剂量以最大程度地减少低血压。
