Health & Technology Research Center (H&TRC), Escola Superior de Tecnologia da Saúde de Lisboa, Instituto Politécnico de Lisboa, Lisbon, Portugal.
Pharmaceutical Sciences Postgraduate Programme, Federal University of Paraná, Curitiba, Brazil.
BMJ Open. 2023 Feb 6;13(2):e064872. doi: 10.1136/bmjopen-2022-064872.
Sickle cell disease (SCD), an inherited haemoglobinopathy, has important impact on morbidity and mortality, especially in paediatrics. Previous systematic reviews are limited to adult patients or focused only on few therapies. We aim to synthesise the evidence on efficacy and safety of pharmacological interventions for managing SCD in children and adolescents.
This systematic review protocol is available at Open Science Framework (doi:10.17605/OSF.IO/CWAE9). We will follow international recommendations on conduction and report of systematic reviews and meta-analyses. Searches will be conducted in PubMed, Scopus and Web of Science (no language nor time restrictions) (first pilot searches performed in May 2022). We will include randomised controlled trials comparing the effects of disease-modifying agents in patients with SCD under 18 years old. Outcomes of interest will include: vaso-occlusive crisis, haemoglobin levels, chest syndrome, stroke, overall survival and adverse events. We will provide a narrative synthesis of the findings, and whenever possible, results will be pooled by means of pairwise or Bayesian network meta-analyses with surface under the cumulative ranking curve analyses. Different statistical methods and models will be tested. Dichotomous outcomes will be reported as OR, risk ratio or HR, while continuous data will be reported as standard mean differences, both with 95% CI/credibility interval. The methodological quality of the trials will be evaluated using the Risk of Bias 2.0 tool, and the certainty of the evidence will be assessed with the Grading of Recommendations Assessment, Development and Evaluation approach.
This study refers to a systematic review, so no ethics approval is necessary. We intent to publish our findings in international, peer-reviewed journal. Data will also be presented to peers in scientific events. Additionally, the results obtained in this study may contribute towards the update of therapeutic guidelines and for the development of health policies for SCD.
CRD42022328471.
镰状细胞病（SCD）是一种遗传性血红蛋白病，对发病率和死亡率有重要影响，尤其是在儿科。先前的系统评价仅限于成人患者或仅关注少数几种治疗方法。我们旨在综合评估药物干预治疗儿童和青少年镰状细胞病的疗效和安全性。
本系统评价方案可在 Open Science Framework（doi：10.17605/OSF.IO/CWAE9）上获取。我们将遵循关于系统评价和荟萃分析的国际建议进行研究。将在 PubMed、Scopus 和 Web of Science 中进行检索（无语言和时间限制）（2022 年 5 月首次进行试点检索）。我们将纳入比较 18 岁以下 SCD 患者疾病修饰药物效果的随机对照试验。感兴趣的结局将包括：血管阻塞性危象、血红蛋白水平、胸痛综合征、中风、总生存和不良事件。我们将对研究结果进行叙述性综合分析，只要有可能，将通过成对或贝叶斯网络荟萃分析以及累积排序曲线下面积分析来汇总结果。将测试不同的统计方法和模型。二分类结局将报告为 OR、风险比或 HR，而连续数据将报告为标准均数差，均带有 95%CI/可信度区间。试验的方法学质量将使用风险偏倚 2.0 工具进行评估，证据的确定性将使用推荐评估、制定和评估方法进行评估。
本研究为系统评价，因此无需伦理批准。我们打算在国际同行评审期刊上发表研究结果。还将在科学会议上向同行介绍研究结果。此外，本研究获得的结果可能有助于更新治疗指南和制定 SCD 卫生政策。
PROSPERO 注册号：CRD42022328471。
