Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT

产品说明：在镰状细胞病治疗中坚持“个人最佳”的羟基脲：“习惯”总体目标是测试一种干预措施，通过加强对羟基脲（HU）治疗的长期自我管理坚持来改善镰状细胞病（SCD）儿童的健康。镰状细胞病（SCD）是一种遗传性血液疾病，影响美国服务不足的人群，其特征是贫血、疼痛危象、生活质量下降和高卫生保健利用率。HU是一种每日一次的口服药物，也是唯一获批用于SCD治疗的药物。HU可显著降低症状、发病率和死亡率，改善生活质量（QOL），并诱导胎儿血红蛋白（HbF）水平升高。HbF与HU具有稳定的剂量依赖关系，使其成为监测药物依从性的极好生物标志物。由于不存在统一的HbF目标，个体化的HU诱导的HbF水平应该达到对治疗的稳定的“个人最佳”反应，但通常不会。许多青少年没有充分受益于胡由于日常坚持到他们的日常自我管理的习惯整合不足。坚持的障碍可能包括对药物益处的不完全了解以及文化、后勤和发展障碍。HU特有的挑战包括对其益处的认识有限以及对其安全性的担忧。青少年还需要从父母那里获得适当的自我管理过渡。社区卫生工作者（CHW）是医疗之家的长期合作伙伴，通过文化，行为和发展协调的干预措施来解决障碍。我们假设，两个既定的干预措施，以社区为基础的支持慢性儿科疾病和移动的健康传播的独特组合，将提高自我管理的坚持HU。这项为期6个月的随机对照试验的具体目的是：1）评估父母、青少年和CHW对CHW支持进行为期6个月的干预的可行性和可接受性，并通过定制的短信进行增强，以提高HU治疗的依从性; 2）估计干预对HU依从性的影响大小（主要结果）;和3）合作的青年-父母自我管理的责任，生活质量和资源利用（次要结果）在6个月。本可行性研究的目的是支持多中心随机试验的设计，以通过加强SCD儿童的疾病自我管理来改善HU依从性。这项研究也可以为服务不足社区的其他儿科慢性病的干预提供信息。

DESCRIPTION: Hydroxyurea Adherence for "Personal Best" in Sickle Cell Treatment: "HABIT" The overall goal is to test an intervention to improve the health of children with sickle cell disease (SCD) by enhancing long-term self-managed adherence to hydroxyurea (HU) therapy. Sickle cell disease (SCD), an inherited blood disorder affecting under-served populations in the U.S., is characterized by anemia, painful crises, reduced quality of life (QOL) and high health care utilization. HU is an oral, once-daily medication and sole approved drug for SCD therapy. HU dramatically reduces symptoms, morbidity and mortality, improves quality of life (QOL) and induces increased fetal hemoglobin (HbF) level. HbF has a stable dose- dependent relationship to HU, making it an excellent biomarker to monitor medication adherence. As no uniform HbF target exists, individualized HU-induced HbF levels should reach a stable "personal best" response to treatment, but often do not. Many youth do not fully benefit from HU due to inadequate integration of daily adherence into their routine self-managed habit. Barriers to adherence may include incomplete knowledge of drug benefit and cultural, logistical and developmental impediments. Challenges specific to HU include limited awareness about benefits and concerns about its safety. Youth also need a developmentally appropriate transition of self-management from their parents. Community-based health workers (CHW) are a well-established partner of the medical home to address barriers through culturally, behaviorally and developmentally aligned intervention. We hypothesize that a unique combination of two established interventions, community-based support for chronic pediatric illness and mobile health communication, will improve self-managed adherence to HU. The specific aims of this 6 month randomized controlled trial are to: 1) Assess the feasibility and acceptability by parents, youth and CHWs of a 6 month intervention of CHW support, augmented by tailored text messages, to improve adherence to HU therapy; 2) Estimate the effect size of the intervention on HU adherence (primary outcome); and on 3) Cooperative youth-parent self-management responsibility, QOL and resource use (secondary outcomes) at 6 months. The goal of this feasibility study is to support the design of a multi-site randomized tria to improve HU adherence through enhanced disease self-management in children with SCD. This research may also inform interventions for other pediatric chronic illnesses in underserved communities.