Diamond Ivan R, Grant Robert C, Feldman Brian M, Tomlinson George A, Pencharz Paul B, Ling Simon C, Moore Aideen M, Wales Paul W
Group for Improvement of Intestinal Function and Treatment (GIFT) Child Health Evaluative Sciences Program, Hospital for Sick Children, Toronto, Canada Department of Surgery Department of Health Policy Management and Evaluation.
Group for Improvement of Intestinal Function and Treatment (GIFT) Child Health Evaluative Sciences Program, Hospital for Sick Children, Toronto, Canada Department of Surgery.
JPEN J Parenter Enteral Nutr. 2014 Aug;38(6):702-10. doi: 10.1177/0148607113494213. Epub 2013 Aug 5.
To determine expert beliefs regarding the probability of intestinal failure-associated liver disease (IFALD) with novel lipid-based approaches (lipid minimization/ω-3 lipids) in managing IFALD to facilitate Bayesian analyses of clinical trials of these therapies.
Structured interviews were conducted using a validated approach to belief elicitation with 60 intestinal failure (IF) experts from across North America. Participants were asked to estimate, in an average population of infants referred for management of IF with early IFALD, the probability of advanced IFALD at 3 months following referral in each of 3 scenarios: (1) conventional lipid, (2) ω-3 lipids, and (3) lipid minimization. Probability distributions of the risk of advanced IFALD with each strategy were developed. Distributions of the elicited treatment effect for the novel approaches, relative to conventional lipid, were calculated.
Median duration of experience of participants managing patients with IF was 8.5 (range, 2-35) years. The median probability of advanced IFALD using conventional lipid was 32.5%; ω-3 lipids, 17.5%; and lipid minimization, 13%. The median of the elicited treatment effects relative to conventional lipid was a relative risk of 0.53 for the ω-3 lipid and 0.45 for lipid minimization.
There was consistent expert opinion that the novel lipid-based approaches are superior to conventional therapy, with similar estimates of treatment efficacy for the 2 approaches. The distributions of the elicited treatment effects can be used as prior distributions in Bayesian analyses of clinical trials of these novel strategies.
确定专家对于新型脂质疗法（脂质最小化/ω-3脂肪酸）治疗肠衰竭相关肝病（IFALD）的可能性的看法，以促进对这些疗法的临床试验进行贝叶斯分析。
采用经过验证的信念诱导方法，对来自北美各地的60名肠衰竭（IF）专家进行了结构化访谈。要求参与者估计，在因早期IFALD而转诊接受IF治疗的婴儿平均人群中，在以下三种情况中的每一种情况下，转诊后3个月出现晚期IFALD的概率：（1）传统脂质疗法；（2）ω-3脂肪酸疗法；（3）脂质最小化疗法。制定了每种策略下晚期IFALD风险的概率分布。计算了相对于传统脂质疗法，新型疗法所引发的治疗效果的分布情况。
参与者管理IF患者的中位经验时长为8.5年（范围为2至35年）。使用传统脂质疗法时晚期IFALD的中位概率为32.5%；ω-3脂肪酸疗法为17.5%；脂质最小化疗法为13%。相对于传统脂质疗法，所引发的治疗效果的中位数为，ω-3脂肪酸疗法的相对风险为0.53，脂质最小化疗法的相对风险为0.45。
专家们一致认为，新型脂质疗法优于传统疗法，且对这两种疗法的治疗效果估计相似。所引发的治疗效果分布可作为这些新策略临床试验贝叶斯分析中的先验分布。
