“ Improving Self-Management in Adolescents with Sickle Cell Disease”

 描述(申请人提供)：镰状细胞病(SCD)是最常见的缩短寿命的遗传疾病，主要影响美国的非裔美国青年。SCD在婴儿期对健康造成第一次影响，但更严重的并发症(如器官损伤、慢性疼痛、早期死亡风险)出现在青春期。有效的自我管理技能对于减少青少年SCD的并发症和死亡率至关重要。先前关于SCD自我管理的研究集中在单一维度(例如，坚持)，并假设患者的积极性和参与度很高。然而，我们的试点工作表明，迫切需要制定量身定做的、以群体为基础的干预措施，以增加这一特别脆弱群体的积极性和参与度，以更广泛地提高他们的自我管理技能。行为激活(BA)或管理个人健康所需的知识、自我效能、准备和技能，可能是提高疾病自我管理和预防不良结局的重要机制。我们开发了一种新的、量身定做的、基于群体的自我管理干预工具SCThrive，以改善患有SCD的青少年和青年(AYA)的BA和自我管理。SCThrive使用面对面和在线混合的形式，并得到mHealth工具的支持，该工具由AYAS和SCD、iManage的输入设计。本申请的目的是确定SCThrive的可行性、可接受性和初步疗效，具体目的如下：1)确定SCThrive对患有SCD的AYA进行干预的可行性和可接受性；2)进行先导性随机试验(N=)，以确定在13-21岁SCD的AYA患者中，改进的SCThrive干预在增加BA方面是否优于注意控制(按年龄和疾病严重程度分层)。我们假设，与注意力控制相比，在6周内参与SCThrive将导致治疗后BA(主要结果)的增加。对于目标2，我们将继续评估可行性和可接受性，并检查SCThrive干预是否与治疗6周后BA的改善有关。我们还将探索在治疗6周后，与注意力控制相比，SCThrive是否与自我管理行为和生活质量(次要结果)的更大改善有关。该团队在行为医学、血液学和生物信息学方面拥有专业知识，并在儿科SCD干预研究方面进行了合作，这些研究为这一建议奠定了基础，包括开发iManage工具。这项研究具有重要意义，因为它解决了发展行为激活的SCD患者的需求，这些患者参与并合作管理他们的健康以减少症状。这项拟议的研究具有创新性，因为它挑战了主流的研究实践，即使用个人的、一维的和调查者设计的行为干预。这一开发/探索性项目的结果将为R01应用程序在全功率随机多点试验中检验SCThrive的疗效奠定基础。

 DESCRIPTION (provided by applicant): Sickle cell disease (SCD), the most common life-shortening genetic disorder, affects primarily African American youth in the U.S. SCD has its first health impact in infancy but more severe complications (e.g. organ damage, chronic pain, risk for early mortality) emerge in adolescence. Effective self-management skills are critical to decrease complications and mortality in adolescents with SCD. Prior studies in SCD self- management have focused on a single dimension (e.g. adherence) and assumed a high level of patient motivation and engagement. However, our pilot work indicates a compelling need to develop tailored, group- based interventions to increase motivation and engagement in this particularly vulnerable population to improve their self-management skills more broadly. Behavioral activation (BA) or the knowledge, self-efficacy, readiness and skills necessary to manage one's health, is potentially an important mechanism for improving disease self-management and preventing poor outcomes. We developed a novel, tailored, group-based self- management intervention, SCThrive, to improve BA and self-management in adolescents and young adults (AYAs) with SCD. SCThrive uses a mixed in-person and online format and is supported by a mHealth tool designed with input from AYAs with SCD, iManage. The objective of this application is to determine SCThrive's feasibility, acceptability and preliminary efficacy ia the following specific aims: 1) determine the feasibility and acceptability of the SCThrive intervention for AYAs with SCD, and 2) conduct a pilot randomized trial (N=64) to determine whether the refined SCThrive intervention is superior to an attention control in increasing BA in AYAs with SCD ages 13-21 (stratified by age and disease severity). We hypothesize that participation in SCThrive over a 6-week period as compared to attention control will lead to increased BA (primary outcome) at post-treatment. For Aim 2, we will continue to assess feasibility and acceptability and examine whether the SCThrive intervention is associated with improvements in BA 6 weeks after treatment. We will also explore whether SCThrive is associated with greater improvements in self-management behaviors and quality of life (secondary outcomes) compared to attention control 6-weeks after treatment. The team has expertise in behavioral medicine, hematology, and bioinformatics and has collaborated on pediatric SCD intervention studies that lay the foundation for this proposal including the development of the iManage tool. The study is significant because it addresses the need for developing behaviorally-activated patients with SCD who are engaged, collaborative partners in managing their health to decrease symptoms. The proposed research is innovative because it challenges the prevailing research practice of using individual, one-dimensional, and investigator-designed behavioral interventions. Results from this developmental/exploratory project will lay the foundation for an R01 application to examine SCThrive's efficacy in a fully powered randomized multisite trial.