基于肿瘤成分评估依维莫司对结节性硬化症相关肾血管平滑肌脂肪瘤的治疗作用

Evaluation of the therapeutic effect of everolimus on tuberous sclerosis related renal angiomyolipoma based on tumor components

目的基于肿瘤成分评估依维莫司对结节性硬化症相关肾血管平滑肌脂肪瘤(TSC-RAML)的治疗作用,明确依维莫司治疗后RAML中缩小的主要成分。方法回顾性分析2017年9月-2022年9月于空军军医大学第一附属医院泌尿外科就诊且符合国际结节性硬化症共识会议(ITSCCC)诊断标准的47例TSC-RAML患者的临床资料。依据CT组织特异性阈值的衰减范围,将患者分为富脂肪组(HU≤−10,n=26)与乏脂肪组(HU≥30,n=21)。收集患者基线资料及治疗6个月后的CT数据,记录治疗前后RAML的平均CT值,并采用Mimics软件对RAML进行三维重建,记录治疗前后RAML的体积。比较两组治疗前后RAML的体积及平均CT值。结果两组基线特征比较差异无统计学意义(P>0.05)。两组患者肿瘤体积缩小的中位数分别为4.94(3.12,27.23)cm^(3)和27.31(10.83,40.38)cm^(3),体积反应率分别为11.52%±0.96%和62.09%±12.60%,差异均有统计学意义(P<0.05)。治疗前后富脂肪组与乏脂肪组肿瘤平均CT差值分别为(4.23±3.01)HU、(14.52±3.61)HU,肿瘤平均CT值降低率分别为14.25%±11.94%、29.23%±0.53%,差异均有统计学意义(P<0.05)。治疗后乏脂肪组肿瘤平均CT值降低程度明显高于富脂肪组,差异有统计学意义(P<0.05)。依维莫司治疗6个月后,RAML肿瘤内高密度条索状血管组织成分明显减少并出现脂肪转化现象。结论依维莫司治疗乏脂肪性RAML时,缩小肿瘤体积及降低肿瘤平均CT值的效果更加显著,且缩小的是TSC-RAML中以血管成分为主的高密度成分。

Objective To evaluate the therapeutic effect of everolimus on tuberous sclerosis related renal angiomyolipoma(TSC-RAML)based on tumor components,and identify the types of main components in reducing RAML by everolimus.Methods To retrospectiely analyze the clinical data of 47 patients with TSC-RAML who were treated in the Urology Department of the First Affiliated Hospital of the Air Force Medical University and met the diagnostic criteria of ITSCCC from September 2017 to September 2022.According to the attenuation range of CT tissue specific threshold,patients were divided into fat rich group(HU≤−10,n=26)and fat deficient group(HU≥30,n=21).Collect patients'baseline CT data and 6 months after treatment,record the average CT value of RAML before and after treatment.Three-dimensional reconstruction of RAML was performed using Mimics software,and record the volume of RAML before and after treatment.The volume of RAML and mean CT value were compared between the two groups before and after treatment.Results No statistical difference existed in baseline characteristics between the two groups(P>0.05).The median reduction of tumor volume in fat rich group and fat deficient group of patients were 4.94(3.12,27.23)cm^(3) and 27.31(10.83,40.38)cm^(3),respectively,and the volume response rates were 11.52%±0.96%and 62.09%±12.60%respectively,the differences were statistically significant(P<0.05).The differences of average CT values between the fat rich group and fat deficient group were(4.23±3.01)HU and(14.52±3.61)HU respectively,and the reduction rates of CT values were 14.25%±11.94%and 29.23%±0.53%respectively,all were statistically significant(P<0.05).After treatment,the average CT value of tumors in fat deficient group decreased significantly compared to that in fat rich group with statistically significant difference(P<0.05).Six months after treatment with everolimus,the composition of high-density cord like vascular tissue in RAML tumors reduced significantly and fat conversion occurred.Conclusions The effect of everolimus on reducing tumor volume and average CT value of tumor in fat deficient RAML is better and significant,which confirmed that the reduction is the high-density component of TSC-RAML mainly composed of vascular components.