调整Utrecht分级对TSC-AML患者接受mTOR抑制剂治疗效果的预测作用  

The predictive effect of adjusted Utrecht staging criteria on efficacy of mTOR inhibitors in TSCAML patients

作　　者：王文达 邱东旭 廖章诚 李汉忠 张玉石 Wang Wenda;Qiu Dongxu;Liao Zhangcheng;Li Hanzhong;Zhang Yushi(Department of Urology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院、北京协和医学院、北京协和医院泌尿外科,北京100730

出　　处：《中华泌尿外科杂志》2023年第10期731-735,共5页Chinese Journal of Urology

基　　金：北京协和医院中央高水平医院临床科研专项(2022-PUMCH-A-151)。

摘　　要：目的探讨调整的乌特勒支(Utrecht)分级对结节性硬化症相关肾血管平滑肌脂肪瘤(TSC-AML)患者接受哺乳动物雷帕霉素靶蛋白(mTOR)抑制剂治疗效果的预测作用。方法回顾性分析2014年12月至2020年12月北京协和医院收治的39例TSC-AML患者的病例资料,基于调整的Utrecht分级将患者分为4组,1组6例,Utrecht分级≤5级,AML直径均<5cm;2组14例,Utrecht分级6级,至少1个AML直径≥5cm,肾解剖结构正常;3组10例,Utrecht分级6级,至少1个AML直径≥5cm,肾解剖结构可辨认;4组9例,Utrecht分级6级,至少1个AML直径≥5cm,肾解剖结构不可辨认。1~4组的男/女性别比例分别为3/3例、7/7例、3/7例、1/8例(P=0.233),年龄分别为(27.33±7.84)、(28.64±12.36)、(31.10±5.88)、(29.11±7.15)岁(P=0.869),差异均无统计学意义。1~4组治疗前血管平滑肌脂肪瘤(AML)最大径分别为(3.54±0.52)、(8.11±2.08)、(11.58±4.60)、(17.08±3.61)cm,差异有统计学意义(P<0.01);治疗前肌酐水平分别为(80.17±16.01)、(76.36±18.72)、(76.10±27.61)、(71.89±18.66)μmol/L(P=0.900),差异无统计学意义;治疗前各组尿蛋白阳性患者分别为2、3、8、2例(P=0.023),差异有统计学意义。4组均接受依维莫司10mg每日1次治疗≥3个月。比较4组治疗后AML最大缩瘤率、肌酐水平、尿蛋白严重程度、镜下血尿严重程度的变化差异。结果1~4组治疗后最大缩瘤率分别为(76.06±13.16)%、(64.92±16.33)%、(55.30±20.49)%、(43.73±20.61)%,差异有统计学意义(P=0.009);肌酐升高值分别为(8.50±7.61)、(5.71±8.54)、(7.70±7.18)、(6.11±7.04)μmol/L,差异无统计学意义(P=0.856);尿蛋白增加患者的例数分别为3、4.6、9例,差异有统计学意义(P=0.014)。结论调整的Utrecht分级对mTOR抑制剂治疗TSC-AML的最大缩瘤率、尿蛋白增加等有一定预测作用。Objective To investigate the predictive effect of adjusted Utrecht staging criteria on efficacy of mammalian target of rapamycin(mTOR)inhibitors in patients with tuberous sclerosis-associated renal angiomyolipoma(TSC-AML).Methods In this study,39 adult patients with TSC-AML who attended the Peking Union Medical College Hospital from December,2014 to December,2020 were retrospectively analyzed,and were divided into 4 groups based on Utrecht staging criteria:Group 1,Utrecht staging≤stage 5,all AMLs<5 cm(n=6);Group 2,stage 6 with at least one AML≥5 cm and normal renal anatomy(n=14);Group 3,stage 6 with at least one AML≥5 cm and recognizable renal anatomy(n=10);Group 4,stage with at least one AML≥5 cm and unrecognizable renal anatomy(n=9).The mean age of patients in the 4 groups were 27.33±7.84,28.64±12.36,31.10±5.88 and 29.11±7.15(P=0.869).No statistic difference in gender was found among the dfferent groups(P=0.233).The mean maximum diameters of AML in the four groups were(3.54±0.52)cm,(8.11±2.08)cm,(11.58±4.60)cm and(17.08±3.61)cm,respectively(P<0.01).The mean levels of creatinine were(80.17±16.01)μmol/L,(76.36±18.72)μmol/L,(76.10±27.61)μmol/L and(71.89±18.66)μmol/L in the four groups(P=0.900).The numbers of patients with positive urine protein were 2,3,8 and 2(P=0.023).All the patients took Everolimus 10mg orally per day for at least 3 months.Differences in maximum AML shrinkage rate,creatinine level,severity of urine protein and microscopic hematuria were compared between the groups.Results As AML severity increased,the shrinkage rates decreased significantly in tun[Group 1:(76.06±13.16)%,Group 2:(64.92±16.33)%,Group 3:(55.30±20.49)%,Group 4:(43.73±20.61)%,P=0.009].After treatment,creatinine levels increased in all groups[Group 1:(8.50±7.61)μmol/L,Group 2:(5.71±8.54)μmol/L,Group 3:(7.70±7.18)μmol/L,Group 4:(6.11±7.04)μmol/L],but there was no significant diference among groups(P=0.856).Moreover the increase in urine protein worsened with the degree of severity(Group I:3,Group 2

关 键 词：结节性硬化症 血管平滑肌脂肪瘤 治疗效果 预测 

分 类 号：R751[医药卫生—皮肤病学与性病学]