口服还原型谷胱甘肽可改善小儿囊性纤维化患者的生长情况。

Oral reduced L-glutathione improves growth in pediatric cystic fibrosis patients.

作者信息

Visca Alfredo, Bishop Clark T, Hilton Sterling, Hudson Valerie M

机构信息

*Federazione Italiana Medici Pediatri, Verbano Cusio Ossola, Turin, Italy †Intermountain Healthcare ‡Brigham Young University, Provo, UT §Texas A&M University, College Station.

出版信息

J Pediatr Gastroenterol Nutr. 2015 Jun;60(6):802-10. doi: 10.1097/MPG.0000000000000738.

DOI:10.1097/MPG.0000000000000738

PMID:25633497

Abstract

BACKGROUND AND OBJECTIVE

Consensus nutritional guidelines for patients with cystic fibrosis (CF) recommend aggressive treatment of growth failure. Oral reduced glutathione (GSH) has been shown to improve cachexia and case reports have demonstrated improved growth in pediatric patients with CF. No controlled studies using oral GSH in CF have, however, been reported. The aim of the study was to determine whether oral GSH could improve growth in CF. Secondarily, to determine whether oral GSH could improve other systemic clinical markers.

METHODS

We performed a placebo-controlled, randomized, double-blind, repeated-measures clinical trial in 44 pediatric patients with CF ages 18 months to 10 years. Primary outcomes were change in weight percentile, body mass index (BMI) percentile, height percentile, and fecal calprotectin. Secondary outcomes included liver function tests and measures of systemic inflammation. Each participant was studied for 6 months, with data obtained at baseline, 3 months, and 6 months. Blood samples were obtained on the baseline and 6-month visits. Subjects were treated with oral GSH or placebo (calcium citrate), each 65 mg · kg(-1) · day(-1) divided into 3 doses per day at mealtimes, and administered daily for 6 months.

RESULTS

The GSH treatment group gained an average of 0.67 standard deviation (SD) in weight-for-age-and sex z score (wfaszs), (19.1 weight percentile points) during the course of 6 months, with no adverse effects (vs placebo with an increase of 0.1 SD in wfaszs [2.1 weight percentile points], P < 0.0001). Fecal calprotectin improved, GSH -52.0 vs placebo 0.5), also BMI for GSH improved 0.69 SD BMI-adjusted-for-age-and-sex z score versus placebo 0.22 SD (BMI percentile 21.7 GSH vs 5.2 placebo), and height 0.2 SD in height-for-age-and-sex z score (hfaszs) GSH versus -0.06 SD hfaszs placebo [height percentile 7.0 GSH vs -2.6 placebo], all P < 0.0001). Secondary outcomes improved significantly, as well.

CONCLUSIONS

Oral reduced L-GSH significantly improves measures of growth status and gut inflammation in CF.

摘要

背景与目的

囊性纤维化（CF）患者的营养共识指南建议积极治疗生长发育迟缓。口服还原型谷胱甘肽（GSH）已被证明可改善恶病质，且病例报告显示CF患儿的生长情况有所改善。然而，尚未有关于CF患者口服GSH的对照研究报道。本研究的目的是确定口服GSH是否能改善CF患者的生长情况。其次，确定口服GSH是否能改善其他全身临床指标。

方法

我们对44名年龄在18个月至10岁的CF患儿进行了一项安慰剂对照、随机、双盲、重复测量的临床试验。主要结局指标为体重百分位数、体重指数（BMI）百分位数、身高百分位数和粪便钙卫蛋白的变化。次要结局指标包括肝功能检查和全身炎症指标。每位参与者接受6个月的研究，在基线、3个月和6个月时获取数据。在基线和6个月随访时采集血样。受试者接受口服GSH或安慰剂（柠檬酸钙）治疗，剂量均为65 mg·kg⁻¹·d⁻¹，分3次在进餐时服用，持续给药6个月。

结果

GSH治疗组在6个月期间年龄和性别校正体重Z评分（wfaszs）平均增加0.67标准差（SD）（体重百分位数增加19.1个百分点），且无不良反应（相比之下，安慰剂组wfaszs增加0.1 SD[体重百分位数增加2.1个百分点]，P<0.0001）。粪便钙卫蛋白有所改善（GSH组为-52.0，安慰剂组为0.5），GSH组的BMI年龄和性别校正Z评分也有所改善，为0.69 SD，而安慰剂组为0.22 SD（BMI百分位数GSH组为21.7，安慰剂组为5.2），身高年龄和性别校正Z评分（hfaszs）GSH组为0.2 SD，安慰剂组为-0.06 SD[身高百分位数GSH组为7.0，安慰剂组为-2.6]，所有P值均<0.0001）。次要结局指标也有显著改善。