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).

确认青少年神经性厌食症适应性治疗的功效/机制 在青少年神经性厌食症 (AN) 中，30-40% 通过家庭治疗 (FBT) 完全康复并保持健康状态 因此，治疗后 3-4 年，但那些未缓解的患者发生持久性 AN 的风险很高 伴随的合并症、生活质量大大降低、过早死亡，而目前没有任何治疗方法 基于证据的治疗研究发现，第 4 节的体重增加可以预测 85-90% 的结果。 因此，根据精准医疗改善结果的一种方法是将治疗与 患者根据第 4 次疗程的体重增加情况提供替代治疗以满足特定需求 早期反应不佳的人（~44%）。这项针对 RFA-MH-16-425 的研究的目的是确定是否存在 与 FBT 兼容的适应性门诊治疗可以通过解决贫困问题来显着改善结果 早期反应并增加我们对 FBT 治疗机制的理解我们开发了 3-。 课程（强化家长辅导-IPC）干预，以提高家长对这个贫困儿童的早期重新喂养技能 响应小组展示了采用 IPC 的随机自适应设计的可行性。 与早期反应不佳者的预期体重恢复率相比，达到目标的参与者多了 50% 体重恢复超出预期，EOT 平均体重达 96.7%（初步疗效信号为 Cohen's d= 0.82）之前的研究表明，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，数据来自 目前的试点随机对照试验表明，孩子在第 4 次会议前体重未增加 2.4 公斤的父母报告称，体重水平较低 早在第 2 节中，自我效能感就与重新喂养相关；然而，在接受 FBT+IPC 后，父母的自我效能感 该组的疗效分数有所提高，并且与早期反应者父母的疗效分数没有区别。 虽然这些初步数据很有希望，但使用自适应方法进行足够动力和受控的 RCT 需要采用 FBT+IPC 作为早期反应不佳者的适应性干预的随机设计 在推荐适应性疗法之前，了解父母效能和再喂养行为在 FBT 中的作用 进行一项充分有力的研究（0.87（α = 0.05，双尾）），150 名青少年（每人 75 名）。 站点）将被随机分配到 2 个站点（斯坦福大学和加州大学旧金山分校） 标准 FBT 或适应性治疗组 (FBT+IPC)，其中第 4 阶段体重未增加 2.4 公斤的患者将 接受额外治疗 (FBT+IPC) 将在以下时间进行评估（对治疗情况不知情）。 基线、第 3 个月、第 9 个月（EOT）以及第 6 个月和第 12 个月的随访时，我们的主要结果是达到体重。 缓解（根据年龄、身高和性别调整后的预期平均 BMI 百分比>94%）。