Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABIT

描述：在镰状细胞疗法中，羟基尿素坚持“个人最佳”：“习惯”总体目标是测试一种干预措施，通过提高长期自我管理的坚持羟基尿素(HU)疗法来改善镰状细胞病(SCD)儿童的健康。镰状细胞病(SCD)是一种遗传性血液疾病，影响着美国医疗服务不足的人群，其特征是贫血、痛苦的危机、生活质量(QOL)下降和医疗保健利用率高。HU是一种口服、每日一次的药物，也是唯一被批准用于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)，并导致胎儿血红蛋白(HBF)水平升高。HbF与HU具有稳定的剂量依赖关系，使其成为监测用药依从性的极佳生物标志物。由于没有统一的HBF靶点存在，个体化的HU诱导的HBF水平应该在治疗后达到稳定的“个人最佳”反应，但通常不是这样。许多年轻人没有从HU中充分受益，因为日常坚持没有充分融入他们的日常自我管理习惯中。遵守的障碍可能包括对毒品益处的不完全了解以及文化、后勤和发展方面的障碍。胡面临的具体挑战包括对好处的认识有限，以及对其安全性的担忧。年轻人还需要从父母那里进行适合发展的自我管理过渡。社区卫生工作者(CHW)是医疗之家的可靠合作伙伴，通过文化、行为和发展一致的干预措施来解决障碍。我们假设，两种既定干预措施的独特组合--基于社区的慢性儿科疾病支持和移动健康交流--将提高自我管理对HU的依从性。这项6个月的随机对照试验的具体目标是：1)评估父母、青少年和社区卫生工作者6个月干预CHW支持的可行性和可接受性，以提高对HU治疗的依从性；2)估计干预对HU治疗依从性的影响程度(主要结果)；3)6个月时合作的青少年-父母自我管理责任、生活质量和资源使用(次要结果)。这项可行性研究的目标是支持多点随机试验的设计，以通过加强对SCD儿童的疾病自我管理来改善HU依从性。这项研究还可能为服务不足社区中其他儿科慢性病的干预提供参考。

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.