Abstract：
Sacubitril/valsartan has been approved for the management of heart failure (HF) with reduced ejection fraction in adults. PANORAMA-HF trial (Prospective Trial to Assess the Angiotensin Receptor Blocker Neprilysin Inhibitor LCZ696 Versus Angiotensin-Converting Enzyme Inhibitor for the Medical Treatment of Pediatric HF) investigated its effects on clinical outcomes in pediatric patients with HF.
PANORAMA-HF is a multicenter, Phase II/III study using an adaptive, seamless, 2-part design. The study aimed to evaluate the pharmacokinetics and pharmacodynamics of single doses of sacubitril/valsartan (Part 1), and the efficacy and safety of sacubitril/valsartan versus enalapril administered twice daily for 52 weeks (Part 2) in pediatric patients with HF due to left ventricular systolic dysfunction with biventricular heart physiology. An innovative trial design using a novel global rank assessment of severity was employed. For analysis, eligible patients were stratified into 3 age groups (Group 1, 6 to <18 years; Group 2a, 2 to <6 years; and Group 3a, 1 month to <2 years) and functional classification (New York Heart Association/Ross class I/II and III/IV).
We report the key demographic, baseline, and clinical characteristics of 375 pediatric patients randomized to receive the study medication. The mean age for patients in Groups 1, 2a, and 3a was 12.2, 3.2, and 1.3 years, respectively. About 70% of patients had a prior HF hospitalization, 85% had New York Heart Association/Ross class I/II HF, and ≈8% were angiotensin-converting enzyme inhibitor/angiotensin receptor blocker naïve.
Compared to other pediatric HF studies, PANORAMA-HF recruited a relatively homogeneous pediatric HF population across 3 age groups, enabling a more robust evaluation of pharmacokinetics/pharmacodynamics and efficacy/safety of sacubitril/valsartan. Most patients had mildly symptomatic HF at baseline.
URL: https://www.
gov; Unique identifier: NCT02678312.
PANORAMA-HF is a multicenter, Phase II/III study using an adaptive, seamless, 2-part design. The study aimed to evaluate the pharmacokinetics and pharmacodynamics of single doses of sacubitril/valsartan (Part 1), and the efficacy and safety of sacubitril/valsartan versus enalapril administered twice daily for 52 weeks (Part 2) in pediatric patients with HF due to left ventricular systolic dysfunction with biventricular heart physiology. An innovative trial design using a novel global rank assessment of severity was employed. For analysis, eligible patients were stratified into 3 age groups (Group 1, 6 to <18 years; Group 2a, 2 to <6 years; and Group 3a, 1 month to <2 years) and functional classification (New York Heart Association/Ross class I/II and III/IV).
We report the key demographic, baseline, and clinical characteristics of 375 pediatric patients randomized to receive the study medication. The mean age for patients in Groups 1, 2a, and 3a was 12.2, 3.2, and 1.3 years, respectively. About 70% of patients had a prior HF hospitalization, 85% had New York Heart Association/Ross class I/II HF, and ≈8% were angiotensin-converting enzyme inhibitor/angiotensin receptor blocker naïve.
Compared to other pediatric HF studies, PANORAMA-HF recruited a relatively homogeneous pediatric HF population across 3 age groups, enabling a more robust evaluation of pharmacokinetics/pharmacodynamics and efficacy/safety of sacubitril/valsartan. Most patients had mildly symptomatic HF at baseline.
URL: https://www.
gov; Unique identifier: NCT02678312.
摘要：
背景：萨库必曲/缬沙坦已被批准用于治疗成人射血分数降低的心力衰竭（HF）。PANORAMA-HF试验（评估血管紧张素受体阻滞剂Neprilysin抑制剂LCZ696与血管紧张素转换酶抑制剂治疗小儿HF的前瞻性试验）研究了其对小儿HF患者临床结局的影响。
方法：PANORAMA-HF是一个多中心，II/III期研究使用适应性，无缝,2部分设计。该研究旨在评估单剂量沙库巴曲/缬沙坦的药代动力学和药效学（第1部分），以及沙库巴曲/缬沙坦与依那普利每天两次给药52周的疗效和安全性（第2部分），用于因左心室收缩功能障碍而导致的双室心脏生理学的HF患儿。采用了一种创新的试验设计，使用了一种新型的严重程度全球等级评估。为了进行分析，符合条件的患者被分为3个年龄组(第1组，6至<18岁；第2a组，2至<6年；第3a组，1个月至<2年）和功能分类（纽约心脏协会/罗斯I/II和III/IV级）。
结果：我们报告了关键的人口统计，基线,和随机接受研究药物的375名儿科患者的临床特征。第1、2a、3a分别为12.2、3.2和1.3年，分别。大约70%的患者先前曾因HF住院，85%的人患有纽约心脏协会/罗斯I/II级HF，血管紧张素转换酶抑制剂/血管紧张素受体阻滞剂初始治疗约8%。
结论：与其他儿科HF研究相比，PANORAMA-HF招募了3个年龄组的相对同质的小儿HF人群，能够更可靠地评估沙库巴曲/缬沙坦的药代动力学/药效学和疗效/安全性。大多数患者在基线时具有轻度症状性HF。
背景：URL：https://www。
结果：gov；唯一标识符：NCT02678312。
方法：PANORAMA-HF是一个多中心，II/III期研究使用适应性，无缝,2部分设计。该研究旨在评估单剂量沙库巴曲/缬沙坦的药代动力学和药效学（第1部分），以及沙库巴曲/缬沙坦与依那普利每天两次给药52周的疗效和安全性（第2部分），用于因左心室收缩功能障碍而导致的双室心脏生理学的HF患儿。采用了一种创新的试验设计，使用了一种新型的严重程度全球等级评估。为了进行分析，符合条件的患者被分为3个年龄组(第1组，6至<18岁；第2a组，2至<6年；第3a组，1个月至<2年）和功能分类（纽约心脏协会/罗斯I/II和III/IV级）。
结果：我们报告了关键的人口统计，基线,和随机接受研究药物的375名儿科患者的临床特征。第1、2a、3a分别为12.2、3.2和1.3年，分别。大约70%的患者先前曾因HF住院，85%的人患有纽约心脏协会/罗斯I/II级HF，血管紧张素转换酶抑制剂/血管紧张素受体阻滞剂初始治疗约8%。
结论：与其他儿科HF研究相比，PANORAMA-HF招募了3个年龄组的相对同质的小儿HF人群，能够更可靠地评估沙库巴曲/缬沙坦的药代动力学/药效学和疗效/安全性。大多数患者在基线时具有轻度症状性HF。
背景：URL：https://www。
结果：gov；唯一标识符：NCT02678312。
