Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

描述（由申请人提供）：很大比例的患者可能不在SCD专科护理中，并且可能面临护理障碍。对于成年人来说，不接受专科治疗可能是致命的，因为专科医生可能会开出羟基脲（HU），这是FDA批准的第一个也是唯一一个治疗SCD的药物。HU的其他障碍包括医生对其使用的恐惧或误解，不一致的HU资格确定和不一致的剂量递增，以及由于对相对益处和危害的无知或不信任，与治疗相关的不便或难以获得药物而导致患者未能服用HU。为了克服这些障碍，弗吉尼亚州，包括弗吉尼亚州卫生部和两个学术医疗中心，计划进行两阶段的示范，首先是提高接受SCD专业护理的成人SCD患者的百分比（阶段i），然后是提高符合条件的SCD成人对HU的依从性（阶段i）。这两个阶段都将使用现有的学术SCD提供者，以及一种创新的多模式策略，以经过专门培训的SCD患者导航员（PNs）为特色，解决护理和HU使用的障碍。在第一阶段，我们将展示利用PNs克服患者获得SCD护理障碍的可行性。在第二阶段，我们将测试PNs在克服接受和坚持Hu治疗障碍方面的功效。患者将被随机分配到PN组或常规护理组。提供者将在两组中执行NIH关于HU资格和处方的指南。第一阶段的主要结果变量将是在随后的三个月内与专业SCD提供者进行访问的入组者的百分比。II期的主要结局将是随机分组后1年HbF水平的改善。次要结局将包括：四项对HU依从性的测量；达到最大耐受剂量、临床终点或最大剂量的患者百分比；急诊科和医院探访；平均红细胞体积；总血红蛋白;白细胞计数；网织红细胞计数;以及生活质量。ii期的Co变量将包括：患者人口统计学；基因型;社会支持;SCD相关疼痛状况；精神健康状况和酒精使用情况；以及应对措施。这个项目将是非常重要和有影响力的，因为它展示了一个全州范围内基于社区的战略的可行性，以帮助弱势的SCD成年人获得SCD专业护理，并可能延长生命，这是其他州可以采用的模式。

DESCRIPTION (provided by applicant): Large percentage of patients may not be in SCD specialty care, and may face barriers to care. For adults, not having specialty care may be fatal, because specialists may prescribe Hydroxyurea (HU), the first and only FDA approved remittive drug for SCD. Additional barriers to HU include physician fears or misconceptions about its use, inconsistent determination of HU eligibility and inconsistent dose escalation, and patient failure to take HU, due to ignorance or mistrust of relative benefit sand harms, inconvenience associated with therapy, or poor medication access. To overcome these barriers, the state of Virginia, including the Virginia Department of Health and two academic medical centers, plans a two phase demonstration, first of improvement in the percentage of adults with SCD who are in SCD specialty care (PhaseI), then of improvement in adherence to HU of eligible SCD adults (PhaseII). Both phases will use existing academic SCD providers, and an innovative, multimodal strategy, featuring specially trained SCD patient navigators (PNs), that addresses barriers to care and to HU use. In Phase I we will demonstrate the feasibility of utilizing PNs to overcome patient access barriers to SCD care. In Phase II we will test the efficacy of PNs for overcoming barriers to acceptance of and adherence with Hu therapy. Patients will be randomized to a PN or usual care arm. Providers will implement NIH guidelines for HU eligibility and prescribing in both arms. The primary outcome variable for Phase I will be the percentage of enrollees who in the subsequent three months achieve a visit with a specialty SCD provider. The primary outcome for Phase II will be improvement in the HbF level at 1 year post randomization. Secondary outcomes will include: four measures of adherence to HU; percent of patients achieving either maximum tolerated dose, a clinical endpoint, or maximum dose; ED and hospital visits; mean corpuscular volume; total hemoglobin; white blood cell count; reticulocyte count; and quality of life. Co variates in PhaseII will include: patient demographics; genotype; social support; SCD associated pain conditions; mental health status and alcohol use; and coping measures. This project will be critically important and impactful by demonstrating the feasibility of a statewide community based strategy to assist vulnerable SCD adults in obtaining SCD specialty care and likely prolonging life, a model that other states could adopt.