% 0期刊文章%一个罗宾Deterding %丽莎·R·年轻%艾米丽·m . DeBoer %大卫沃伯顿%史蒂文·坎宁安%一个凯文·R·费拉Nicolaus Schwerk % %一个凯文·k·布朗%米卡尔Dumistracel %埃尔韦拉Erhardt %茱莉亚Bertulis %玛蒂娜Gahlemann %一个Matthias Griese Susanne Stowasser % %, % T Nintedanib在儿童和青少年fibrosing间质性肺疾病的2023% % D J R 10.1183/13993003.01512 -2022%欧洲呼吸杂志% P 2201512 % V 61% N 2% X背景儿童间质性肺病(ILD)由一系列的罕见ILDs影响婴儿、儿童和青少年。Nintedanib许可成人治疗肺纤维化。InPedILD试验的主要目标是确定剂量的和安全的儿童和青少年nintedanib fibrosing ILD。方法6岁到17岁的病人fibrosing ILD高分辨率计算机断层扫描和临床重大疾病随机2:1获得nintedanib 24周或安慰剂,然后开放nintedanib。剂量是根据weight-dependent异速生长的伸缩。Co-primary端点是在稳态血浆浓度时间曲线下的面积(AUCτ,ss)在2周和26和患者的比例在24周的治疗诱发的不良事件。结果26例患者接受nintedanib和13个病人则接受安慰剂治疗。几何均值(几何变异系数)AUCτnintedanib党卫军是175µg·h·L−1例(85.1%)患者11岁和160年µg·h·L−1(82.7%) 12 - 17岁的病人。在双盲阶段,不良事件被报道在每个治疗组84.6%的患者。两个病人停止nintedanib由于不良事件。据报道在38.5%和15.4%的腹泻nintedanib和安慰剂组,分别。 Adjusted mean±se changes in percentage predicted forced vital capacity at week 24 were 0.3±1.3% in the nintedanib group and −0.9±1.8% in the placebo group.Conclusions In children and adolescents with fibrosing ILD, a weight-based dosing regimen resulted in exposure to nintedanib similar to adults and an acceptable safety profile. These data provide a scientific basis for the use of nintedanib in this patient population.The results of the randomised placebo-controlled InPedILD trial support a positive benefit–risk assessment for use of nintedanib in children and adolescents with fibrosing ILD https://bit.ly/3qnAhUM %U //www.qdcxjkg.com/content/erj/61/2/2201512.full.pdf