Plasma chitotriosidase activity versus CCL18 level for assessing type I Gaucher disease severity: protocol for a systematic review with meta-analysis of individual participant data.
Plasma chitotriosidase activity versus CCL18 level for assessing type I Gaucher disease severity: protocol for a systematic review with meta-analysis of individual participant data.
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DOI：
10.1186/s13643-017-0483-x
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发表时间：
2017-04-20
影响因子：
3.7
通讯作者：
Berger M
中科院分区：
文献类型：
--
作者：
Raskovalova T;Deegan PB;Yang R;Pavlova E;Stirnemann J;Labarère J;Zimran A;Mistry PK;Berger M
戈谢病（GD）是由酸性-葡萄糖苷酶缺乏引起的常染色体隐性溶酶体贮积症。GD表现出广泛的疾病严重程度的临床谱与不可预测的自然过程。血浆壳三醇苷酶活性和CC趋化因子配体18 （CCL18）可互换用于监测GD活性和对酶替代治疗的反应，并结合临床评估。然而，目前还缺乏对这两种生物标志物进行大规模的面对面比较。我们建议对个体参与者数据（IPD）进行meta分析，以比较血浆壳三醇苷酶活性和CCL18在评估I型（即非神经性）GD严重程度方面的准确性。符合条件的研究包括横断面、队列和随机对照研究，记录连续I型GD儿童或成人患者基线和/或随访时血浆壳三醇苷酶活性和CCL18水平。反映GD活性的预先指定的替代结果包括肝脏和脾脏体积、血红蛋白浓度、血小板计数和影像学证实的症状性骨事件。主要研究将通过检索Medline（1995年起）、EMBASE（1995年起）和Cochrane中央对照试验登记（Central）来确定。电子检索将通过联系研究小组来补充，以确定未发表的相关研究。在可能的情况下，IPD将从已发表的文章中提取。通讯作者将被邀请通过提供IPD进行合作。检索研究的方法学质量将根据每个研究结果进行评估，使用从诊断准确性研究质量评估-2工具改编的清单。主要终点将是肝体积（MN）的1.25倍，脾体积（MN）的5倍，血红蛋白浓度<11 g/dL，或血小板计数<100 × 109/L。预测结果的生物标志物比较准确性的效应大小估计将报告为受试者工作特征曲线下区域的差异以及95%置信区间。效应大小估计将根据结果报告为（加权）平均差异以及每个生物标志物的95%置信区间。IPD荟萃分析将采用一阶段和两阶段方法进行。根据GD的严重程度，将得到CCL18相对于血浆壳三醇苷酶活性的有效和精确的准确性估计。PROSPERO 2015 CRD42015027243本文的在线版本（doi:10.1186/s13643-017-0483-x）包含补充资料，仅供授权用户使用。
Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by deficiency in acid beta-glucosidase. GD exhibits a wide clinical spectrum of disease severity with an unpredictable natural course. Plasma chitotriosidase activity and CC chemokine ligand 18 (CCL18) have been exchangeably used for monitoring GD activity and response to enzyme replacement therapy in conjunction with clinical assessment. Yet, a large-scale head-to-head comparison of these two biomarkers is currently lacking. We propose a collaborative systematic review with meta-analysis of individual participant data (IPD) to compare the accuracy of plasma chitotriosidase activity and CCL18 in assessing type I (i.e., non-neuropathic) GD severity. Eligible studies include cross-sectional, cohort, and randomized controlled studies recording both plasma chitotriosidase activity and CCL18 level at baseline and/or at follow-up in consecutive children or adult patients with type I GD. Pre-specified surrogate outcomes reflecting GD activity include liver and spleen volume, hemoglobin concentration, platelet count, and symptomatic bone events with imaging confirmation. Primary studies will be identified by searching Medline (1995 onwards), EMBASE (1995 onwards), and Cochrane Central Register of Controlled Trials (CENTRAL). Electronic search will be complemented by contacting research groups in order to identify unpublished relevant studies. Where possible, IPD will be extracted from published articles. Corresponding authors will be invited to collaborate by supplying IPD. The methodological quality of retrieved studies will be appraised for each study outcome, using a checklist adapted from the Quality Assessment of Diagnostic Accuracy Studies-2 tool. The primary outcome will be a composite of liver volume >1.25 multiple of normal (MN), spleen volume >5 MN, hemoglobin concentration <11 g/dL, or platelet count <100 × 109/L. Effect size estimates for biomarker comparative accuracy in predicting outcomes will be reported as differences in areas under receiver operating characteristic curves along with 95% confidence intervals. Effect size estimates will be reported as (weighted) mean differences along with 95% confidence intervals for each biomarker according to outcomes. IPD meta-analysis will be conducted with both one- and two-stage approaches. Valid and precise accuracy estimates will be derived for CCL18 relative to plasma chitotriosidase activity in discriminating patients according to GD severity. PROSPERO 2015 CRD42015027243 The online version of this article (doi:10.1186/s13643-017-0483-x) contains supplementary material, which is available to authorized users.