Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor
Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor
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DOI：
10.1007/s10620-015-3834-2
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发表时间：
2016-01-01
影响因子：
3.1
通讯作者：
Schwarzenberg, Sarah Jane
中科院分区：
文献类型：
--
作者：
Borowitz, Drucy;Lubarsky, Barry;Schwarzenberg, Sarah Jane
囊性纤维化（CF）跨膜电导调节因子（CFTR）门控突变G551D由于通道打开概率降低而阻止足够的离子运输。Ivacaftor是一种口服CFTR增强器，可增加通道打开概率。进一步分析两项研究中ivacaftor对年龄为千分之一的CF和G551D突变患者体重和体重指数（BMI）的改善。患者按1:1随机分组，每12小时服用150mg伊伐他汀或安慰剂，持续48周。主要终点（肺功能）之前有报道。其他结果包括体重和身高测量和CF问卷-修订（CFQ-R）。研究纳入213例患者（年龄为千分之一货币符号20岁，n = 105；年龄为千分之一货币符号20岁，n = 108）。在每千分之一货币符号为20年的患者中，从基线到第48周体重的调整平均变化为4.9和2.2 kg （ivvacaftor vs.安慰剂，p = 0.0008）。在第48周，平均年龄体重z评分与基线的变化为0.29对-0.06 (p < 0.0001)；平均年龄bmi z-score变化为0.26 vs -0.13 （p < 0.0001）。在bbb20岁的患者中，从基线到第48周体重的调整平均变化为2.7 vs -0.2 kg （p = 0.0003）。第48周的平均BMI变化为0.9 vs -0.1 kg/m(2) （p = 0.0003）。体重变化与肺功能或汗液氯化物的改善之间没有明显的线性相关性。在饮食感知、身体形象和增重能力方面，CFQ-R显著改善。ivacaftor治疗48周后营养状况改善。
The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability.To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged a parts per thousand yen6 years with CF and the G551D mutation.Patients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R).Studies included 213 patients (aged a parts per thousand currency sign 20 years, n = 105; aged > 20 years, n = 108). In patients a parts per thousand currency sign20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p < 0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p < 0.0001). In patients > 20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight.Nutritional status improved following treatment with ivacaftor for 48 weeks.