Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg-University, Mainz, Germany.
Department of Diagnostic and Interventional Radiology, Section of Pediatric Radiology, Medical Center of the Johannes Gutenberg-University, Mainz, Germany.
Mol Genet Metab. 2020 Jun;130(2):140-148. doi: 10.1016/j.ymgme.2020.03.006. Epub 2020 Mar 27.
To evaluate the feasibility of acoustic radiation force impulse point shear wave elastography (ARFI-pSWE) of the liver and spleen in patients with Gaucher disease type 1 (GD1), and to assess correlations between organ stiffness and clinico-radiologic data, particularly the GD1 Severity Scoring System (GD-DS3).
We retrospectively evaluated the results of ARFI-pSWE as measures of liver and spleen stiffness in 57 patients with GD1. The feasibility of the method was assessed. Correlations between elastography data and clinical data related to the metabolic syndrome, laboratory tests, and GD1-related clinico-radiologic data (bone marrow burden score, GD-DS3) were assessed.
ARFI-pSWE provided reliable results (i.e. standard deviation <30% of the mean value between the measurements) in 50/57 patients. Significant liver fibrosis was present in 35/50 patients (70%). Liver stiffness significantly correlated with GD-DS3 score (p = .03), and number of fulfilled criteria of metabolic syndrome (p = .03). Spleen stiffness significantly correlated with age (p = .021), body mass index (p = .002), number of fulfilled criteria of metabolic syndrome (p = .02), and several laboratory parameters (alanine aminotransferase, gamma glutamyltranspeptidase, triglycerides, cholesterol), and nearly significantly with GD-DS3 score (p = .059).
ARFI-pSWE is a useful tool for a more detailed assessment of disease severity in patients with GD1, which adds relevant information to the standard clinical scores. Thus, elastography might allow for extended therapy monitoring, especially in patients with significant liver fibrosis. Spleen elastography showed promising results; thus, its role should be further investigated.
评估声辐射力脉冲点剪切波弹性成像（ARFI-pSWE）在 1 型戈谢病（GD1）患者肝脏和脾脏中的可行性，并评估器官硬度与临床放射学数据之间的相关性，特别是 GD1 严重程度评分系统（GD-DS3）。
我们回顾性评估了 57 例 GD1 患者 ARFI-pSWE 作为肝脏和脾脏硬度测量值的结果。评估了该方法的可行性。评估了弹性成像数据与与代谢综合征、实验室检查和 GD1 相关临床放射学数据（骨髓负担评分、GD-DS3）相关的临床数据之间的相关性。
ARFI-pSWE 在 50/57 例患者中提供了可靠的结果（即测量值之间的平均值的标准偏差<30%）。35/50 例患者存在显著的肝纤维化（70%）。肝脏硬度与 GD-DS3 评分显著相关（p=0.03），与代谢综合征符合标准的数量显著相关（p=0.03）。脾脏硬度与年龄（p=0.021）、体重指数（p=0.002）、代谢综合征符合标准的数量（p=0.02）、几项实验室参数（丙氨酸氨基转移酶、γ-谷氨酰转肽酶、甘油三酯、胆固醇）显著相关，与 GD-DS3 评分接近显著相关（p=0.059）。
ARFI-pSWE 是评估 GD1 患者疾病严重程度的有用工具，它为标准临床评分提供了相关信息。因此，弹性成像可能允许进行更广泛的治疗监测，尤其是在有明显肝纤维化的患者中。脾脏弹性成像显示出有前途的结果；因此，应进一步研究其作用。
