Clinical Development, Shionogi Inc., Florham Park, New Jersey, USA.
Division of Infectious Diseases and International Health, University of Virginia School of Medicine, Charlottesville, Virginia, USA.
J Pediatric Infect Dis Soc. 2021 Apr 30;10(4):477-484. doi: 10.1093/jpids/piaa145.
Baloxavir marboxil has demonstrated safety and efficacy in treating adult and adolescent outpatients with acute influenza (CAPSTONE-1 trial). Here, we report a subgroup analysis of outcomes in adolescents from the trial.
CAPSTONE-1 was a randomized, double-blind, placebo-controlled study. Eligible adolescent outpatients (aged 12-17 years of age) were randomized in a ratio of 2:1 to a single dose of baloxavir 40/80 mg if less than/greater than or equal to 80 kg or placebo. The main outcomes were the time to alleviation of symptoms (TTAS), duration of infectious virus detection, and incidence of adverse events (AEs).
Among 117 adolescent patients, 90 (77%) comprised the intent-to-treat infected population (63 baloxavir and 27 placebo; 88.9% A(H3N2)). The median TTAS was 38.6 hours shorter (95% confidence interval: -2.6, 68.4) in the baloxavir group compared with placebo (median TTAS, 54.1 hours vs 92.7 hours, P = .0055). The median time to sustained cessation of infectious virus detection was 72.0 hours for baloxavir compared with 120.0 hours for placebo recipients (P < .0001). Treatment-emergent PA/I38X-substituted viruses were detected in 5 of the 51 (9.8%) baloxavir recipients. In the safety population (76 baloxavir and 41 placebo), AEs were less common in baloxavir than placebo recipients (17.1% vs 34.1%; P = .0421). In the baloxavir group, no AEs except for diarrhea were reported in 2 or more patients.
Baloxavir demonstrated clinical and virologic efficacy in the otherwise healthy adolescents with acute influenza compared with placebo. There were no safety concerns identified. These results were similar to the adult population in CAPSTONE-1 and support baloxavir as a treatment option in adolescents.
巴洛沙韦治疗成人和青少年门诊急性流感（CAPSTONE-1 试验）具有安全性和疗效。在此，我们报告该试验中青少年亚组的结果分析。
CAPSTONE-1 是一项随机、双盲、安慰剂对照研究。合格的青少年门诊患者（年龄 12-17 岁）按 2:1 的比例随机分为单次剂量 40/80mg 巴洛沙韦或安慰剂治疗。主要终点为症状缓解时间（TTAS）、病毒检测持续时间和不良事件（AE）发生率。
在 117 例青少年患者中，90 例（77%）为意向治疗感染人群（63 例巴洛沙韦和 27 例安慰剂；88.9%为 A(H3N2)）。与安慰剂相比，巴洛沙韦组 TTAS 缩短了 38.6 小时（95%置信区间：-2.6，68.4）（中位数 TTAS：54.1 小时比 92.7 小时，P=0.0055）。巴洛沙韦组持续停止检测到传染性病毒的中位时间为 72.0 小时，而安慰剂组为 120.0 小时（P<0.0001）。在 51 例（9.8%）接受巴洛沙韦治疗的患者中检测到治疗后出现的 PA/I38X 取代病毒。在安全性人群（76 例巴洛沙韦和 41 例安慰剂）中，巴洛沙韦组的 AE 发生率低于安慰剂组（17.1%比 34.1%；P=0.0421）。在巴洛沙韦组中，除腹泻外，没有任何 AE 在 2 例或更多患者中出现。
与安慰剂相比，巴洛沙韦在健康的青少年急性流感患者中表现出临床和病毒学疗效。未发现安全性问题。这些结果与 CAPSTONE-1 中的成年人群相似，支持巴洛沙韦作为青少年的治疗选择。
