目的进行系统评价和荟萃分析,以评估酶替代疗法对Fabry病患者心脏MRI参数的影响。材料与方法从2000年1月1日至2024年1月1日在PubMed进行了系统的文献检索,ClinicalTrials.gov,Embase,和Cochrane图书馆数据库。研究结果是以下参数的变化:(a)左心室壁质量(LVM),以克为单位;(B)LVM以体重指数为索引,以克/平方米为单位;(c)最大左心室壁厚(MLVWT),以毫米为单位测量;(D)钆后期增强(LGE)程度,以LVM的百分比衡量;和(E)原生T1映射,以毫秒为单位测量。对基线和随访参数之间的合并均值差异进行了随机效应荟萃分析。研究方案在PROSPERO(CRD4202236223)中注册。结果最终分析包括11项研究,共445例Fabry病患者(平均年龄±SD,41岁±11岁;277名男性,168女)。在基线和随访心脏MRI之间,以下情况没有改变:T1映射(平均差,6毫秒[95%CI:-2,15];两项研究,70名患者,I2=88%)和LVM指数(平均差,-1g/m2[95%CI:-6,3];四项研究,290名患者,I2=81%)。以下措施最低限度地降低:LVM(平均差,-18克[95%CI:-33,-3];七项研究,107名患者,I2=96%)和MLVWT(平均差,-1毫米[95%CI:-2,-0.02];六项研究,151名患者,I2=90%)。LGE程度增加(平均差,1%[95%CI:1,1];三项研究,114名患者,I2=85%)。结论在Fabry病患者中,酶替代疗法与LVM的稳定有关,MLVWT,和T1映射值,而LGE程度轻度增加。关键词:法布里病,酶替代疗法(ERT),心脏MRI,晚钆增强(LGE)补充材料可用于本文。©RSNA,2024.
Purpose To perform a systematic review and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in patients with Fabry disease. Materials and Methods A systematic literature search was conducted from January 1, 2000, through January 1, 2024, in PubMed, ClinicalTrials.gov, Embase, and Cochrane Library databases. Study outcomes were changes in the following parameters: (a) left ventricular wall mass (LVM), measured in grams; (b) LVM indexed to body mass index, measured in grams per meters squared; (c) maximum left ventricular wall thickness (MLVWT), measured in millimeters; (d) late gadolinium enhancement (LGE) extent, measured in percentage of LVM; and (e) native T1 mapping, measured in milliseconds. A random-effects meta-analysis of the pooled mean differences between baseline and follow-up parameters was conducted. The study protocol was registered in PROSPERO (CRD42022336223). Results The final analysis included 11 studies of a total of 445 patients with Fabry disease (mean age ± SD, 41 years ± 11; 277 male, 168 female). Between baseline and follow-up cardiac MRI, the following did not change: T1 mapping (mean difference, 6 msec [95% CI: -2, 15]; two studies, 70 patients, I2 = 88%) and LVM indexed (mean difference, -1 g/m2 [95% CI: -6, 3]; four studies, 290 patients, I2 = 81%). The following measures minimally decreased: LVM (mean difference, -18 g [95% CI: -33, -3]; seven studies, 107 patients, I2 = 96%) and MLVWT (mean difference, -1 mm [95% CI: -2, -0.02]; six studies, 151 patients, I2 = 90%). LGE extent increased (mean difference, 1% [95% CI: 1, 1]; three studies, 114 patients, I2 = 85%). Conclusion In patients with Fabry disease, enzyme replacement therapy was associated with stabilization of LVM, MLVWT, and T1 mapping values, whereas LGE extent mildly increased. Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gadolinium Enhancement (LGE) Supplemental material is available for this article. © RSNA, 2024.