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: 8659513
• 负责人: Nancy S. Green
• 金额: $ 17.75万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-05-08 至 2016-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8659513
• 关键词: Accident and Emergency department; Address; Adherence; Affect; African Caribbean; Anemia; Appointment; Awareness; Biological Markers; Child; Child health care; Childhood; Childhood Asthma; Chronic; Chronic Disease; Clinic; Communication; Communities; Community Health; Control Groups; Cues; Development; Disease; Dose; Effectiveness; Erythrocytes; Family; Family health status; Feasibility Studies; Fetal Hemoglobin; Goals; Habits; Health; Health Communication; Healthcare; Hematological Disease; Hemoglobin; Hispanics; Home environment; Hospitalization; Inherited; Intervention; Knowledge; Linguistics; Logistics; Measures; Medical; Minor; Monitor; Morbidity - disease rate; Oral; Organ; Outcome; Outpatients; Pain; Parents; Patient Self-Report; Pharmaceutical Preparations; Pharmacy facility; Productivity; Quality of life; Randomized; Randomized Controlled Trials; Research; Resources; Risk; Safety; Schools; Self Management; Sickle Cell; Sickle Cell Anemia; Site; Structure; Surveys; Symptoms; Testing; Text; Travel; Underserved Population; Visit; Youth; base; community intervention; design; family management; health care service utilization; hydroxyurea; improved; mHealth; medication compliance; mortality; multi-site trial; polymerization; primary outcome; programs; public health relevance; randomized trial; reinforced behavior; response; secondary outcome; sickling; social; standard care; successful intervention

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.

描述：镰状细胞病治疗中“个人最佳”羟基脲依从性：“习惯”总体目标是测试一种干预措施，通过增强对羟基脲（HU）治疗的长期自我管理依从性来改善镰状细胞病（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，其特点是贫血、痛苦危机、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，也是唯一批准用于 SCD 治疗的药物。 HU 显着降低症状、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性关系，使其成为监测药物依从性的极佳生物标志物。由于不存在统一的 HbF 目标，个体化 HU 诱导的 HbF 水平应该达到稳定的“个人最佳”治疗反应，但往往达不到。由于日常坚持没有充分融入他们的日常自我管理习惯，许多青少年没有从 HU 中充分受益。依从性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 面临的具体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进行适合发展的自我管理转变。社区卫生工作者 (CHW) 是医疗之家的成熟合作伙伴，通过文化、行为和发展方面的协调干预来消除障碍。我们假设，两种既定干预措施（基于社区的慢性儿科疾病支持和移动健康沟通）的独特组合将提高自我管理对 HU 的依从性。这项为期 6 个月的随机对照试验的具体目的是： 1) 评估家长、青少年和社区卫生工作者对社区卫生工作者支持进行为期 6 个月干预的可行性和可接受性，并通过定制短信进行增强，以提高对 HU 治疗的依从性； 2) 估计干预措施对 HU 依从性的影响大小（主要结果）； 3) 6 个月时青少年与父母合作的自我管理责任、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中心随机试验的设计，通过加强 SCD 儿童的疾病自我管理来提高 HU 依从性。这项研究还可以为服务欠缺社区中其他儿科慢性疾病的干预措施提供信息。

项目成果

Do difficulties in swallowing medication impede the use of hydroxyurea in children?

• DOI: 10.1002/pbc.25073
• 发表时间: 2014-09
• 期刊: PEDIATRIC BLOOD & CANCER
• 影响因子: 3.2
• 作者: Bekele, ElShadey;Thornburg, Courtney D.;Brandow, Amanda M.;Sharma, Mukta;Smaldone, Arlene M.;Jin, Zhezhen;Green, Nancy S.
• 通讯作者: Green, Nancy S.