囊性纤维化患儿补充胆碱与结构化脂质：一项随机安慰剂对照试验
Choline Supplementation With a Structured Lipid in Children With Cystic Fibrosis: A Randomized Placebo-Controlled Trial.
作者信息
Schall Joan I, Mascarenhas Maria R, Maqbool Asim, Dougherty Kelly A, Elci Okan, Wang Dah-Jyuu, Altes Talissa A, Hommel Kevin A, Shaw Walter, Moore Jeff, Stallings Virginia A
机构信息
*Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA ‡The Richard Stockton College of New Jersey NJ §Biostatistics and Data Management Core ||Department of Radiology, Children's Hospital of Philadelphia, Philadelphia, PA ¶Department of Radiology and Medical Imaging, University of Virginia, Charlottesville, VA #Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH **Avanti Polar Lipids, Inc, Alabaster, AL.
出版信息
J Pediatr Gastroenterol Nutr. 2016 Apr;62(4):618-26. doi: 10.1097/MPG.0000000000001004.
BACKGROUND
Choline depletion is seen in cystic fibrosis (CF) and pancreatic insufficiency in spite of enzyme treatment and may result in liver, fatty acid, and muscle abnormalities. This study evaluated the efficacy and safety of an easily absorbed choline-rich structured lipid (LYM-X-SORB™ [LXS]) to improve choline status.
METHODS
Children with CF and pancreatic insufficiency were randomized to LXS or placebo in a 12-month double blind trial. Dietary choline intake, plasma cholines, plasma and fecal phospholipids, coefficient of fat absorption, pulmonary function, growth status, body composition, and safety measures were assessed. Magnetic resonance spectroscopy for calf muscle choline and liver fat were assessed in a subgroup and compared with a healthy comparison group matched for age, sex, and body size.
RESULTS
A total of 110 subjects were enrolled (age 10.4 ± 3.0 years). Baseline dietary choline, 88% recommended, increased 3-fold in the LXS group. Plasma choline, betaine, and dimethylglycine increased in the LXS but not placebo (P = 0.007). Plasma lysophosphatidylcholine and phosphatidylcholine increased, and fecal phosphatidylcholine/phosphatidylethanolamine ratio decreased (P ≤ 0.05) in LXS only, accompanied by a 6% coefficient of fat absorption increase (P = 0.001). Children with CF had higher liver fat than healthy children and depleted calf muscle choline at baseline. Muscle choline concentration increased in LXS and was associated with improvement in plasma choline status. No relevant changes in safety measures were evident.
CONCLUSIONS
LXS had improved choline intake, plasma choline status, and muscle choline stores compared with placebo group. The choline-rich supplement was safe, accepted by participants, and improved choline status in children with CF.
背景
尽管接受了酶治疗，但囊性纤维化（CF）和胰腺功能不全患者仍存在胆碱缺乏的情况，这可能导致肝脏、脂肪酸和肌肉异常。本研究评估了一种易于吸收的富含胆碱的结构化脂质（LYM-X-SORB™ [LXS]）改善胆碱状态的疗效和安全性。
方法
在一项为期12个月的双盲试验中，将患有CF和胰腺功能不全的儿童随机分为LXS组或安慰剂组。评估饮食中胆碱摄入量、血浆胆碱、血浆和粪便磷脂、脂肪吸收系数、肺功能、生长状况、身体成分和安全指标。对一个亚组进行小腿肌肉胆碱和肝脏脂肪的磁共振波谱分析，并与年龄、性别和体型相匹配的健康对照组进行比较。
结果
共纳入110名受试者（年龄10.4±3.0岁）。基线饮食胆碱摄入量为推荐量的88%，LXS组增加了3倍。LXS组血浆胆碱、甜菜碱和二甲基甘氨酸增加，而安慰剂组未增加（P = 0.007）。仅LXS组血浆溶血磷脂酰胆碱和磷脂酰胆碱增加，粪便磷脂酰胆碱/磷脂酰乙醇胺比值降低（P≤0.05），同时脂肪吸收系数增加6%（P = 0.001）。CF患儿在基线时肝脏脂肪含量高于健康儿童，小腿肌肉胆碱缺乏。LXS组肌肉胆碱浓度增加，且与血浆胆碱状态改善相关。安全指标未见相关变化。
结论
与安慰剂组相比，LXS改善了胆碱摄入、血浆胆碱状态和肌肉胆碱储备。这种富含胆碱的补充剂安全，受试者可接受，且改善了CF患儿的胆碱状态。
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