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

 描述（由申请人提供）：镰状细胞病（SCD）是最常见的缩短寿命的遗传性疾病，主要影响美国的非洲裔美国青年。SCD在婴儿期对健康产生首次影响，但在青春期出现更严重的并发症（例如器官损伤、慢性疼痛、早期死亡风险）。有效的自我管理技能对于减少SCD青少年的并发症和死亡率至关重要。SCD自我管理的先前研究集中于单个维度（例如，依从性），并假设患者的动机和参与度高。然而，我们的试点工作表明，迫切需要制定有针对性的、基于群体的干预措施，以提高这一特别脆弱人群的积极性和参与度，从而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，可能是改善疾病自我管理和预防不良结果的重要机制。我们开发了一种新颖的、量身定制的、基于小组的自我管理干预措施SCThrive，以改善患有SCD的青少年和年轻人（AYA）的BA和自我管理。SCThrive使用混合的面对面和在线格式，并由mHealth工具提供支持，该工具是根据具有SCD的AYAs的输入设计的，iManage。本申请旨在确定SCThrive的可行性、可接受性和初步有效性，具体目标如下：1）确定对患有SCD的AYA进行SCThrive干预的可行性和可接受性，以及2）进行初步随机试验（N=64），以确定在13-21岁患有SCD的AYA中，在增加BA方面，改进的SCThrive干预是否优于注意力控制上级（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（主要结局）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干预是否与治疗后6周BA的改善相关。我们还将探讨与治疗后6周的注意力控制相比，SCThrive是否与自我管理行为和生活质量（次要结局）的更大改善相关。该团队拥有行为医学、血液学和生物信息学方面的专业知识，并在儿科SCD干预研究方面进行了合作，为该提案奠定了基础，包括开发iManage工具。这项研究意义重大，因为它解决了发展行为激活的SCD患者的需求，这些患者参与了管理其健康以减少症状的合作伙伴。拟议的研究是创新的，因为它挑战了流行的研究实践，使用个人，一维，和干扰器设计的行为干预。该开发/探索性项目的结果将为R 01应用奠定基础，以在一项完全有效的随机多中心试验中检查SCThrive的疗效。

 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.