在中低收入国家实施的镰状细胞病管理循证干预措施：随机对照试验的系统评价。
Evidence-based interventions implemented in low-and middle-income countries for sickle cell disease management: A systematic review of randomized controlled trials.
机构信息
Global Health Program, New York University School of Global Public Health, New York, New York, United States of America.
Department of Social and Behavioral Sciences, New York University School of Global Public Health, New York, New York, United States of America.
出版信息
PLoS One. 2021 Feb 17;16(2):e0246700. doi: 10.1371/journal.pone.0246700. eCollection 2021.
BACKGROUND
Despite ~90% of sickle cell disease (SCD) occurring in low-and middle-income countries (LMICs), the vast majority of people are not receiving evidence-based interventions (EBIs) to reduce SCD-related adverse outcomes and mortality, and data on implementation research outcomes (IROs) and SCD is limited. This study aims to synthesize available data on EBIs for SCD and assess IROs.
METHODS
We conducted a systematic review of RCTs reporting on EBIs for SCD management implemented in LMICs. We identified articles from PubMed/Medline, Global Health, PubMed Central, Embase, Web of Science medical subject heading (MeSH and Emtree) and keywords, published from inception through February 23, 2020, and conducted an updated search through December 24, 2020. We provide intervention characteristics for each study, EBI impact on SCD, and evidence of reporting on IROs.
MAIN RESULTS
29 RCTs were analyzed. EBIs identified included disease modifying agents, supportive care agents/analgesics, anti-malarials, systemic treatments, patient/ provider education, and nutritional supplements. Studies using disease modifying agents, nutritional supplements, and anti-malarials reported improvements in pain crisis, hospitalization, children's growth and reduction in severity and prevalence of malaria. Two studies reported on the sustainability of supplementary arginine, citrulline, and daily chloroquine and hydroxyurea for SCD patients. Only 13 studies (44.8%) provided descriptions that captured at least three of the eight IROs. There was limited reporting of acceptability, feasibility, fidelity, cost and sustainability.
CONCLUSION
EBIs are effective for SCD management in LMICs; however, measurement of IROs is scarce. Future research should focus on penetration of EBIs to inform evidence-based practice and sustainability in the context of LMICs.
CLINICAL TRIAL REGISTRATION
This review is registered in PROSPERO #CRD42020167289.
背景
尽管约 90%的镰状细胞病（SCD）发生在中低收入国家（LMICs），但绝大多数人并未接受循证干预（EBIs）以减少与 SCD 相关的不良结局和死亡率，且有关实施研究结果（IROs）和 SCD 的数据有限。本研究旨在综合现有关于 SCD 管理中 EBIs 的数据，并评估 IROs。
方法
我们对在 LMICs 中实施的针对 SCD 管理的 EBIs 的 RCTs 进行了系统回顾。我们从 PubMed/Medline、全球健康、PubMed Central、Embase、Web of Science 医学主题词（MeSH 和 Emtree）和关键词中确定了从成立到 2020 年 2 月 23 日发表的文章，并通过 2020 年 12 月 24 日进行了更新搜索。我们为每项研究提供了干预特征、EBI 对 SCD 的影响以及报告 IRO 的证据。
主要结果
分析了 29 项 RCTs。确定的 EBIs 包括疾病修饰剂、支持性护理剂/镇痛药、抗疟药、全身治疗、患者/提供者教育和营养补充剂。使用疾病修饰剂、营养补充剂和抗疟药的研究报告称，疼痛危机、住院、儿童生长以及疟疾的严重程度和流行率有所改善。两项研究报告了补充精氨酸、瓜氨酸和每日氯喹和羟基脲对 SCD 患者的可持续性。只有 13 项研究（44.8%）提供了至少描述了八项 IROs 中的三项的描述。可接受性、可行性、保真度、成本和可持续性的报告有限。
结论
EBIs 对 LMICs 中的 SCD 管理有效；但是，IROs 的测量却很少。未来的研究应重点关注 EBIs 的渗透，以在 LMICs 背景下为循证实践和可持续性提供信息。
临床试验注册
本综述在 PROSPERO 中注册，编号为 CRD42020167289。
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