Department of Medical Education, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Division of Hematology/Oncology, Comer Children's Hospital/University of Chicago, Chicago, IL, USA.
Biomed Res Int. 2022 Jul 18;2022:2122056. doi: 10.1155/2022/2122056. eCollection 2022.
Sickle cell disease (SCD) is a hemoglobinopathy with increasing global prevalence resulting in pain episodes and multiorgan complications. Complications of SCD have been shown to adversely impact health-related quality of life (HRQOL) comprised of physical, social, and emotional domains; hence, HRQOL measures can serve as an effective evaluator of disease burden. Hydroxyurea (HU) and other disease-modifying therapies have demonstrated to significantly improve clinical outcomes in patients with SCD. Medication adherence is an essential mediator of the clinical benefits of these therapies; low adherence has been shown to increase disease burden and healthcare utilization. This systematic literature review intends to determine the association between adherence to disease-modifying therapies and HRQOL in patients with SCD.
We found a total of 12 articles involving 788 participants, which included both patients with SCD and caregivers/parents. Adherence was measured using self-report instruments, laboratory markers, such as fetal hemoglobin and mean corpuscular volume, and mHealth medication trackers. HRQOL was measured using self-report instruments.
All studies demonstrated a correlation between higher HU adherence and better HRQOL scores. Higher HU adherence was associated with lower pain impact, less frequent pain episodes, less fatigue, and improved physical function and mobility, reflecting better physical HRQOL outcomes. Higher adherence was also associated with improved emotional response, decreased anxiety and depressive symptoms, and better social functioning and peer relationships. In addition, our findings indicated that having less frequent barriers to HU adherence was associated with better HRQOL scores. No studies evaluated HRQOL outcomes in relation to adherence to l-glutamine, voxelotor, or crizanlizumab.
Optimizing HU adherence has the potential to improve HRQOL in patients with SCD in addition to reducing healthcare utilization and improving treatment satisfaction. Addressing barriers to HU adherence can positively strengthen the relationship between adherence and HRQOL to potentially improve patient outcomes.
镰状细胞病（SCD）是一种血红蛋白病，其全球患病率不断增加，导致疼痛发作和多器官并发症。SCD 的并发症已被证明对健康相关生活质量（HRQOL）产生不利影响，包括身体、社会和情感领域；因此，HRQOL 测量可以作为疾病负担的有效评估者。羟基脲（HU）和其他疾病修正疗法已被证明可显著改善 SCD 患者的临床结局。药物依从性是这些疗法临床获益的重要中介；低依从性已被证明会增加疾病负担和医疗保健利用。本系统文献综述旨在确定 SCD 患者对疾病修正疗法的依从性与 HRQOL 之间的关联。
我们共发现了 12 篇涉及 788 名参与者的文章，其中包括 SCD 患者和照顾者/父母。依从性使用自我报告工具、胎儿血红蛋白和平均红细胞体积等实验室标志物以及移动医疗药物追踪器进行测量。HRQOL 使用自我报告工具进行测量。
所有研究均表明，HU 依从性越高，HRQOL 评分越高。较高的 HU 依从性与较低的疼痛影响、较少的疼痛发作、较少的疲劳、更好的身体功能和移动性相关，反映了更好的身体 HRQOL 结果。较高的依从性还与改善的情绪反应、减少焦虑和抑郁症状以及更好的社交功能和同伴关系相关。此外，我们的研究结果表明，HU 依从性的障碍较少与更好的 HRQOL 评分相关。没有研究评估 l-谷氨酰胺、沃洛特罗或克里扎尼鲁单抗的依从性与 HRQOL 之间的关系。
优化 HU 依从性除了减少医疗保健利用和提高治疗满意度外，还有潜力改善 SCD 患者的 HRQOL。解决 HU 依从性的障碍可以积极加强依从性与 HRQOL 之间的关系，从而有可能改善患者结局。
