Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT

坚持羟基脲可实现镰状细胞治疗的个人最佳效果​​：习惯

• 批准号: 9367887
• 负责人: Nancy S. Green
• 金额: $ 75.84万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-27 至 2021-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9367887
• 关键词: Acute; Adherence; Admission activity; Adolescent; Adolescent and Young Adult; Affect; African American; Age; Biological Markers; Caribbean region; Child; Chronic; Chronic Disease; Communities; Community Health Aides; Control Groups; Custom; Development; Disease; Dose; Eligibility Determination; Emergency department visit; Ethnic Origin; FDA approved; Fatigue; Feasibility Studies; Fetal Hemoglobin; Focus Groups; Funding; Generic Drugs; Goals; Habits; Health; Health Care Costs; Health Services; Health Status; Healthy People 2020; Hematological Disease; Hospitals; Individual; Inherited; Intervention; Interview; Knowledge; Latino; Length of Stay; Link; Logistics; Longevity; Measures; Medical Staff; Morbidity - disease rate; Organ; Outcome; Pain; Pain intensity; Pain interference; Parents; Patient Self-Report; Pharmaceutical Preparations; Pharmacotherapy; Pharmacy facility; Quality of life; Randomized; Randomized Controlled Trials; Reporting; Research; Sampling; Science; Self Management; Sickle Cell; Sickle Cell Anemia; Site; Source; Symptoms; Text; Underserved Population; Youth; base; burden of illness; clinical research site; design; efficacy testing; efficacy trial; experience; group intervention; hospital utilization; hydroxyurea; improved; medication compliance; mortality; primary outcome; racial and ethnic disparities; reduce symptoms; secondary outcome; stem; success; young adult

Barriers to medication adherence are common in youth with chronic illness and are a source of racial/ethnic disparities in underserved communities. An inherited blood disease, Sickle Cell Disease (SCD) is characterized by chronic and acute illness and reduced quality of life (QOL). It affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves QOL. Poor adherence is common among youth and young adults with SCD. This application, “Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT,” responds to PA-14-029, “Chronic Condition Self-Management in Children and Adolescents.” The proposed study, a 5-site four-year randomized control trial (RCT), stems directly from our recent R21-funded feasibility study of the same title. Overall goals of reducing barriers to HU use and improving adherence for youth 10-18 years through creation of a daily medication habit. The HABIT feasibility study was delivered to a multi-ethnic sample of parent-youth dyads by culturally aligned community health workers (CHWs), augmented by customized text messaging. The 2-site study demonstrated intervention feasibility and acceptability in a multi-ethnic sample, as well as a sufficient effect on the primary outcomes to power a multi-site efficacy trial. The goal of the proposed multi-site study is to test the efficacy of the HABIT intervention at 6 months and sustainability of the effect at 12 months. The importance of the problem of poor medication adherence, use of CHWs to bridge the gap between health services and underserved parent-youth dyads affected by SCD, the strength of the science, the success of our multi-ethnic feasibility study and the potential application of study findings to youth with other serious chronic illnesses speak to the importance of this trial. Specific Aims: Study aims are to test the efficacy of the HABIT intervention to: Aim 1: Improve daily HU adherence, the primary outcome operationalized two ways: biomarker (approach or exceed a historical Personal best HbF) and pharmacy refill (primary outcome); Aim 2: Improve youth quality of life and self-management responsibility concordance measured by three secondary outcomes: generic and disease-specific QOL and parent/youth concordance regarding delegation of self-management responsibility (secondary outcome); Aim 3: Improve health status measured by two secondary outcomes: acute hospital use (12 month hospital length of stay, admissions and emergency room visits) and self-reported disease symptoms (fatigue, pain interference and intensity); Aim 4: Qualitatively assess impact and sustainability of the intervention on developing a daily medication HU habit from the perspectives of the community health workers (CHWs) and youth-parent-dyads using focus group and individual interviews. If proven efficacious, this approach could be adapted to youth affected by other serious chronic conditions.

坚持服药的障碍在患有慢性病的年轻人中很常见， 服务不足社区的种族/族裔差异。一种遗传性血液病，镰状细胞病 (SCD)以慢性和急性疾病以及生活质量（QOL）降低为特征。影响非洲 美国人和其他弱势群体。羟基脲（HU）是FDA批准的唯一药物 治疗SCD，并且非常有效并改善QOL。年轻人中普遍存在依从性差的情况， 患有SCD的年轻人。这项申请，“羟基脲坚持个人最好的镰状细胞 治疗：HABIT，”对PA-14-029的回应，“儿童慢性疾病自我管理， 青少年。”这项拟议的研究是一项为期4年的5个地点的随机对照试验（RCT），直接源于 我们最近的R21资助的同一标题的可行性研究。减少使用HU的障碍的总体目标， 通过建立每日用药习惯，提高10-18岁青少年的依从性。的习惯 可行性研究是交付给一个多种族的样本的父母-青年的二对夫妇， 社区卫生工作者（CHW），并通过定制短信进行增强。双中心研究 在多种族样本中证明了干预的可行性和可接受性， 对多中心疗效试验的主要结局的影响。建议的多站点的目标 这项研究是为了测试6个月时HABIT干预的有效性和12个月时效果的可持续性。 个月药物依从性差问题的重要性，使用CHW弥合差距 在卫生服务和受SCD影响的服务不足的父母-青少年二人组之间， 科学，我们的多民族可行性研究的成功和研究结果的潜在应用， 患有其他严重慢性病的青年人说明了这项试验的重要性。具体目标：研究 目的是测试HABIT干预的有效性：目的1：改善每日HU依从性， 主要结局可操作性有两种方式：生物标志物（接近或超过历史个人最佳 HbF）和药物补充（主要结局）;目的2：改善青年生活质量和自我管理 责任一致性通过三个次要结局测量：一般和疾病特异性QOL 以及家长/青少年在自我管理责任下放方面的一致性（中学） 目的3：通过两个次要结局来衡量改善健康状况：急性住院使用（12 月住院时间、入院和急诊室就诊）和自我报告的疾病 症状（疲劳、疼痛干扰和强度）;目标4：质量评估影响和可持续性 从社区的角度出发，对养成每日服药的习惯进行了哪些干预 卫生工作者（CHWs）和青年父母二人组使用焦点小组和个人访谈。如果证明 这一方法有效，可适用于患有其他严重慢性病的青年。