Transforming pediatric heart failure: Efficacy of low-dose sacubitril/valsartan

IF 3 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Current Problems in Cardiology Pub Date : 2024-11-06 DOI:10.1016/j.cpcardiol.2024.102926

Hui Zha , Xuejia Zhai , Lin Wang , Quancheng Yang , Qing Guo , Yanyu Chen , Jie Liu , Hua Peng

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Abstract

Aims

Pediatric heart failure is a significant cause of illness and death in children. We aimed to assess sacubitril/valsartan's effectiveness and find the proper dosage for pediatric patients.

Methods

Patients unresponsive to traditional medicines for at least 12 months prescribed sacubitril/valsartan from January 2020 to March 2023 were reviewed. The initial dose was 0.2 mg/kg bid, gradually increasing in 0.1 mg/kg increments. The target dose was determined based on blood pressure fluctuations at 70/50 mmHg. Clinical efficacy and quantity-effect relationship were evaluated using echocardiography, NT-proBNP, and the concentration of valsartan, sacubitril, sacubitrilat.

Results

A total of 23 pediatric patients with dilated cardiomyopathy and advanced heart failure were enrolled. Mean sacubitril/valsartan dose was 1.84 mg/kg/day. After 6 months, LVEF increased significantly (38.09% to 45% at 3 months, 52% at 6 months; p < 0.001). LV size reduced to 4.4 cm (IQR, 4.1–5.2) and 4.5 cm (IQR, 4–5.1) at 3 and 6 months, respectively, from 4.6 cm (IQR, 4.2–5.6) at baseline (both P < 0.05). NT-proBNP levels reduced by 5.7 at 3 months (p < 0.05) and 5.38 at 6 months (p < 0.001). Sacubitrilat is the active form of Sacubitril. The highest concentration of sacubitrilat was observed at approximately 1.6 mg/kg. The maintenance dose correlated positively with time (p < 0.001) and valsartan/sacubitril concentration (p < 0.05).

Conclusion

Low dose sacubitril/valsartan is effective in children with heart failure, with dosage adjustments to avoid hypotension. Adjusted dosing can increase EF and reduce heart size, offering new possibilities for pediatric heart failure treatment.

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改变小儿心力衰竭：小剂量沙库比妥/缬沙坦的疗效。

目的：小儿心力衰竭是导致儿童患病和死亡的重要原因。我们旨在评估沙库比特利/缬沙坦的疗效，并找到适合小儿患者的剂量：方法：我们对 2020 年 1 月至 2023 年 3 月期间至少 12 个月对传统药物无反应的患者处方沙库比妥/缬沙坦的情况进行了审查。初始剂量为 0.2 mg/kg bid，以 0.1 mg/kg 为增量逐渐增加。目标剂量根据 70/50 mmHg 的血压波动确定。使用超声心动图、NT-proBNP以及缬沙坦、沙库比特利、沙库比特利钠的浓度评估临床疗效和量效关系：共有23名患有扩张型心肌病和晚期心力衰竭的儿童患者入选。萨库比特利/缬沙坦的平均剂量为 1.84 毫克/千克/天。6个月后，LVEF显著增加（3个月时为38.09%，6个月时为45%，6个月时为52%；P < 0.001）。左心室大小从基线时的 4.6 厘米（IQR，4.2-5.6）分别缩小到 3 个月时的 4.4 厘米（IQR，4.1-5.2）和 6 个月时的 4.5 厘米（IQR，4-5.1）（P 均<0.05）。3 个月时，NT-proBNP 水平降低了 5.7（P < 0.05），6 个月时降低了 5.38（P < 0.001）。Sacubitrilat 是 Sacubitril 的活性形式。在约 1.6 毫克/千克的剂量下，sacubitrilat 的浓度最高。维持剂量与时间（p < 0.001）和缬沙坦/沙库比特利浓度（p < 0.05）呈正相关：结论：小剂量沙库比特利/缬沙坦对心力衰竭儿童有效，但需调整剂量以避免低血压。调整剂量可增加EF，缩小心脏体积，为小儿心衰治疗提供了新的可能性。

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来源期刊

Current Problems in Cardiology 医学-心血管系统

CiteScore

4.80

自引率

2.40%

发文量

392

审稿时长

6 days

期刊介绍： Under the editorial leadership of noted cardiologist Dr. Hector O. Ventura, Current Problems in Cardiology provides focused, comprehensive coverage of important clinical topics in cardiology. Each monthly issues, addresses a selected clinical problem or condition, including pathophysiology, invasive and noninvasive diagnosis, drug therapy, surgical management, and rehabilitation; or explores the clinical applications of a diagnostic modality or a particular category of drugs. Critical commentary from the distinguished editorial board accompanies each monograph, providing readers with additional insights. An extensive bibliography in each issue saves hours of library research.