Confirming the Efficacy/Mechanism of an Adaptive Treatment for Adolescent Anorexia Nervosa

确认青少年神经性厌食症适应性治疗的功效/机制

• 批准号: 9884814
• 负责人: JAMES D LOCK
• 金额: $ 73.88万
• 依托单位: STANFORD UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-04-24 至 2022-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9884814
• 关键词: Achievement; Address; Adolescent; Aftercare; Age; Ambulatory Care; Anorexia Nervosa; Behavior; Behavioral; Blinded; Body Weight; Body Weight Changes; California; Cessation of life; Child; Code; DSM-V; Data; Development; Disease remission; Eating; Evidence based treatment; Failure; Family; Family Study; Feeding behaviors; Gender; Height; Intervention; Mediating; Mediator of activation protein; Obsession; Outcome; Parents; Participant; Patients; Pilot Projects; Psychotherapy; Quality of life; Randomized; Recovery; Reporting; Risk; San Francisco; Self Efficacy; Signal Transduction; Site; Tail; Treatment outcome; Universities; Videotape; Weight; Weight Gain; adaptive intervention; alternative treatment; arm; base; clinically significant; comorbidity; design; evidence base; experience; family structure; feeding; follow-up; high risk; improved; improved outcome; outcome prediction; parental role; precision medicine; premature; primary outcome; response; skills; treatment arm; treatment effect; weight restoration

Confirming the efficacy/mechanism of an adaptive treatment for adolescent anorexia nervosa In adolescent anorexia nervosa (AN) 30-40% fully recover with Family Based Treatment (FBT) and remain so 3-4 years after treatment, but those who do not remit have a high risk of developing enduring AN with accompanying co-morbidities, much reduced quality of life, premature death, and for which there is no evidence-based treatment. Studies of FBT find that weight gain by session 4 predicts outcome in 85-90% of cases;  thus, one way to improve outcomes in line with precision medicine is to match treatments to patients depending on weight gain at session 4 by offering an alternative treatment to meet the specific needs of poor early responders (~44%). The aim of this study in response to RFA-MH-16-425 is to determine if an adaptive outpatient treatment compatible with FBT could significantly improve outcomes by addressing poor early response and add to our understanding of the mechanisms of treatment in FBT. We developed a 3- session (Intensive Parent Coaching-IPC) intervention to increase parental skills at re-feeding for this poor early responding group demonstrating the feasibility of using a randomized adaptive design employing IPC. Compared to expected weight restoration rates in poor early responders, 50% more participants achieved weight restoration than expected with an average mean weight of 96.7% by EOT (preliminary efficacy signal of Cohen's d= 0.82). Previous studies have shown that FBT specifically targets parental self-efficacy as the mechanism to promote weight restoration in their children with AN. Consistent with these studies, data from the current pilot RCT suggest that parents whose children do not gain 2.4 kg by session 4 report lower levels of self-efficacy related to re-feeding as early as session 2; however, after receiving FBT+IPC, parental self- efficacy scores in this group improve and become indistinguishable from those of parents of early responders. While these preliminary data are promising, a sufficiently powered and controlled RCT using an adaptive randomized design employing FBT+IPC as the adaptive intervention for poor early responders is needed to understand the role of parental efficacy and re-feeding behaviors in FBT before recommending an adaptive approach. To conduct an adequately powered study (0.87 (alpha=.05, two-tailed)), 150 adolescents (75 per site) will be randomized at 2 sites (Stanford University and the University of California, San Francisco) to either standard FBT or an adaptive treatment arm (FBT+IPC) where those who do not gain 2.4 kg by session 4 will receive additional treatment (FBT+IPC). Assessments (blinded to treatment condition) will be conducted at baseline, 3, 9 (EOT) and at 6 and 12-month follow-up. Our primary outcome will be achievement of weight remission (>94% expected mean percent BMI adjusted for age, height and gender).

阐明青少年神经性厌食症适应性治疗的疗效/机制 在青少年神经性厌食症（AN）中，30-40%的人通过家庭治疗（FBT）完全康复， 因此，治疗后3-4年，但那些没有缓解的人有很高的风险发展为持久的AN， 伴随的合并症，生活质量大大降低，过早死亡，并且没有 循证治疗。FBT的研究发现，第4次治疗的体重增加可预测85-90%的患者的结局。 病例;因此，根据精准医学改善结果的一种方法是将治疗与 通过提供替代治疗来满足特定需求， 早期反应较差（约44%）。响应RFA-MH-16-425的本研究旨在确定 与FBT兼容的适应性门诊治疗可以通过解决不良反应， 早期反应，并增加我们对FBT治疗机制的理解。我们开发了一个3- 会议（密集的父母教练-IPC）干预，以提高父母的技能，重新喂养这个贫穷的早期 响应组证明了使用随机化自适应设计采用IPC的可行性。 与早期反应差的预期体重恢复率相比， 体重恢复高于预期，EOT时的平均体重为96.7%（初步疗效信号为 Cohen的d= 0.82）。先前的研究表明，FBT特别针对父母的自我效能感， 促进AN儿童体重恢复的机制。与这些研究一致，来自 目前的试点随机对照试验表明，在第4阶段，孩子体重没有增加2.4公斤的父母报告说， 早在第2阶段，自我效能就与重新喂养有关;然而，在接受FBT+IPC后，父母自我效能 该组中的疗效评分提高并且变得与早期应答者的父母的疗效评分难以区分。 虽然这些初步数据是有希望的，但使用自适应 需要采用FBT+IPC作为早期应答较差者的适应性干预的随机设计， 在推荐适应性的FBT之前， approach.为了进行一项具有充分把握度的研究（0.87（α = 0.05，双尾）），150名青少年（75名/ 研究中心）将在2个研究中心（斯坦福大学和加州大学旧金山分校弗朗西斯科）随机分配至 标准FBT或适应性治疗组（FBT+IPC），其中第4阶段未增加2.4 kg的患者将 接受额外治疗（FBT+IPC）。评估（对治疗条件设盲）将在 基线、3、9（EOT）以及6和12个月随访时。我们的主要成果将是实现体重 缓解（根据年龄、身高和性别调整的BMI预期平均百分比>94%）。