Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor.
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用ivacaftor治疗的囊性纤维化青春期前儿童的生长。
DOI：
10.1542/peds.2016-2522
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发表时间：
2017-02
期刊：
影响因子：
8
通讯作者：
Rowe SM
中科院分区：
文献类型：
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作者：
Stalvey MS;Pace J;Niknian M;Higgins MN;Tarn V;Davis J;Heltshe SL;Rowe SM
囊性纤维化（CF）以其对受影响个体的肺和胰腺的影响而闻名;然而，生长受损也是一种常见的并发症。我们假设，靶向CF跨膜传导调节因子（CFTR）蛋白的生物缺陷可能会影响生长结果。在这项事后分析中，我们评估了83名儿童（6-11岁）的线性生长和体重，这些儿童参加了2项临床试验，即观察性GOAL研究和安慰剂对照的Brachsion研究，以评估CFTR增效剂ivacaftor的作用。我们计算了身高和体重的z评分以及身高和体重的生长速度（GV）。在GOAL中接受依伐卡托治疗的儿童中，身高和体重z评分从基线到6个月显著增加（分别增加0.1 [P < .05]和0.26 [P < .0001]）;身高GV从3个月到6个月显著增加（2.10 cm/y增加[P < .01]）。在依伐卡托治疗的儿童中，身高和体重z评分从基线到48周显著增加（分别增加0.17 [P < .001]和0.35 [P < .001]）。从基线到48周，依伐卡托组的身高和体重GV也显著高于安慰剂组（差异分别为1.08 cm/y [P < .05]和3.11 kg/y [P < .001]）。依伐卡托治疗青春期前儿童可能有助于解决CF儿童的身材矮小和GV改变;这些分析的结果支持CF儿童生长存在内在缺陷，可以通过CFTR调节来改善。
Cystic fibrosis (CF) is known for its impact on the lung and pancreas of affected individuals; however, impaired growth is also a common complication. We hypothesized that targeting the biological defect in the CF transmembrane conductance regulator (CFTR) protein may impact growth outcomes. In this post hoc analysis, we assessed linear growth and weight in 83 children (aged 6–11) enrolled in 2 clinical trials, the longitudinal-observation GOAL study and the placebo-controlled ENVISION study, to evaluate the effects of ivacaftor, a CFTR potentiator. We calculated height and weight z scores and height and weight growth velocities (GVs). In ivacaftor-treated children in GOAL, height and weight z scores increased significantly from baseline to 6 months (increases of 0.1 [P < .05] and 0.26 [P < .0001], respectively); height GV increased significantly from 3 to 6 months (2.10 cm/y increase [P < .01]). In ivacaftor-treated children in ENVISION, height and weight z scores increased significantly from baseline to 48 weeks (increases of 0.17 [P < .001] and 0.35 [P < .001], respectively). Height and weight GVs from baseline to 48 weeks were also significantly higher with ivacaftor than with placebo (differences of 1.08 cm/y [P < .05] and 3.11 kg/y [P < .001], respectively). Ivacaftor treatment in prepubescent children may help to address short stature and altered GV in children with CF; results from these analyses support the existence of an intrinsic defect in the growth of children with CF that may be ameliorated by CFTR modulation.