期刊
JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION
卷 63, 期 5, 页码 E92-E97出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/MPG.0000000000001366
关键词
cystic fibrosis; endpoints for treatment trials; intestinal current measurement; nasal potential difference
资金
- North American CF Foundation
Objectives:Nasal potential difference (NPD) measurement is part of the diagnostic criteria for cystic fibrosis (CF) and now used routinely as an endpoint in clinical trials of correcting the basic defect in CF. Intestinal current measurement (ICM), measured ex vivo on a rectal biopsy, has been used to study cystic fibrosis transmembrane conductance regulator (CFTR) function but has not been compared to NPD in the same subject in adults and children. The aim of the study is to evaluate the potential usefulness of ICM as a marker of CFTR function for treatment studies compared NPD in patients with CF and in healthy control subjects.Methods:ICM and NPD were performed on healthy controls and patients with CF. The healthy adults were individuals undergoing routine screening colonoscopy at the Beth Israel Deaconess Medical Center. The healthy children were undergoing colonoscopy for suspicion of inflammation in Hadassah Hebrew University Medical Center. The CF adults were recruited from Boston Children's Hospital CF Center and CF Center Worcester Mass, the children with CF from Hadassah CF Center.Results:ICM measurements in healthy control subjects (n=16) demonstrated a mean (SE) carbachol response of 16.0 (2.2) A/cm(2), histamine response of 13.2 (2.1) A/cm(2) and a forskolin response of 6.3 (2.0) A/cm(2). Basal NPD of -15.9 (1.9) and response to Cl- free+isoproterenol of -13.8 (2.0). These responses were inverted in CF subjects (n=12) for ICM parameters with carbachol response of -3.0 (0.5) A/cm(2), histamine -1.0 (0.8) A/cm(2) and a forskolin response of 0.5 (0.3) and also for NPD parameters; basal NPD of -42.2 (4.3) and response to Cl- free+isoproterenol of 4.3 (0.7). Pearson correlation test showed the comparability of ICM and NPD in assessing CFTR function.Conclusions:ICM is equivalent to NPD in the ability to distinguish patients with CF from controls and could be used as surrogate markers of CFTR activity in treatment protocols.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据