Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT

接受药物治疗的障碍在患有慢性病的年轻人中很常见，也是 未得到充分服务的社区的种族/族裔差距。一种遗传性血液疾病--镰状细胞病 慢性阻塞性肺疾病(SCD)的特点是慢性和急性疾病以及生活质量(QOL)下降。它影响着非洲人 美国人和其他服务不足的社区。羟基脲(HU)是FDA唯一批准的药物 对SCD的治疗非常有效，并提高了生活质量。忠诚度差在年轻人中很常见， 患有SCD的年轻人。这项应用，“羟基尿素坚持个人最好的镰刀细胞 治疗：习惯，《对PA-14-029的反应》，儿童的慢性疾病自我管理和 青春期。“这项拟议的研究是一项为期4年的5点随机对照试验(RCT)，直接源于 我们最近由R21资助的同名可行性研究。减少HU使用障碍的总体目标和 通过养成每天服药的习惯，提高10-18岁青少年的服药依从性。习惯 可行性研究通过文化匹配的方式提供给多民族的亲子-青年二人组样本 社区卫生工作者(CHWS)，通过定制的文本消息进行增强。两个站点的研究 在多民族样本中证明了干预的可行性和可接受性，以及充分的 对主要结果的影响，以推动一项多点疗效试验。拟议的多地点的目标是 这项研究是在6个月时测试习惯干预的有效性，并在12个月时测试效果的可持续性 月份。服药依从性差问题的重要性，利用CHW弥合差距 在健康服务和受SCD影响的服务不足的亲子二人组之间， 科学，我们多种族可行性研究的成功，以及研究结果可能应用于 患有其他严重慢性病的年轻人说明了这项试验的重要性。具体目标：研究 目的是测试习惯干预的效果：目标1：提高日常的胡适依从性， 主要结果以两种方式运作：生物标记物(接近或超过历史个人最好成绩 目标2：提高青年的生活质量和自我管理 通过三种次要结果衡量责任一致性：一般生活质量和疾病特定生活质量 和父母/青年在委派自我管理责任方面的一致性(次要 目标3：改善健康状况，以两个次要结果衡量：急性住院使用(12 月住院时间、入院时间和急诊室就诊)和自我报告的疾病 症状(疲劳、疼痛干扰和强度)；目标4：定性评估影响和可持续性 从社区的角度对养成日常用药习惯的干预 卫生工作者(CHW)和青年亲子二人组采用焦点小组和个人访谈。如果经过验证 这种方法是有效的，可以适用于受其他严重慢性病影响的青年。

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.