Background Renal angiomyolipoma(RAML)is the most common kidney lesion in patients with tuberous sclerosis complex(TSC),affecting about 80%of patients.It is a benign tumor that grows over time,usually bilaterally,and can easily lead to kidney complications such as acute hemorrhage.Herein,we investigated the efficacy and safety of sirolimus in children with TSC-associated RAML and explored the factors affecting tumor disappearance under sirolimus treatment through subgroup analysis.Methods A prospective cohort study was conducted.Sirolimus was initiated at 1 mg/(m^(2)×day),and dose adjustments were made by a 2-week titration period to attain a trough blood concentration of 5-10 ng/mL.The disappearance of RAML in chil-dren after sirolimus treatment was observed,and Cox regression was used to screen the factors affecting tumor disappearance.Results One hundred and twenty-six patients who met the criteria were analyzed.After 3 months,6 months,12 months,and 24 months of follow-up,tumors disappeared in 18(14.3%),30(23.8%),39(31.0%),and 42(33.3%)children,respec-tively.Tumors disappeared in 50(39.7%)children by the last visit of each individual,and 30(60%)of them occurred within 6 months.The multivariate Cox regression analysis showed that patients with a smaller maximum tumor diameter at baseline had a higher tumor disappearance rate.Thirty-six(29%)patients had stomatitis during the entire treatment period,and no serious adversereactionswereobserved.Conclusions Sirolimus could promote the disappearance of TSC-related RAML.The disappearance rate was correlated with the maximum diameter at baseline,and the smaller the tumor was,the higher the disappearance rate.It is well tolerated in the treatment of RAML associated with TSC.
目的探讨肝上皮样血管平滑肌脂肪瘤(Hepatic epithelioid angiomyolipoma,HEMAL)和无肝硬化背景的肝细胞肝癌(Hepatocellular carcinoma,HCC)的MRI征象特点。方法选取32例HEAML(HEAML组)和51例无肝硬化背景的HCC(HCC组)患者的临床及MRI影像资料。图像分析包括病灶的位置、大小、形状、有无出血及脂肪,延迟期信号、供血动脉、早期引流静脉、中心血管征、包膜及周围血管关系,对比MRI特征有无差异。结果两组病灶的位置:HEAML组左叶(15/32),右叶(16/32),尾状叶(1/32),HCC组左叶(13/51),右叶(36/51),尾状叶(2/51)。最大径为(39.28±22.68)mm vs(39.22±25.97)mm。病灶有脂肪(13/32 vs 5/51)、出血(2/32 vs 15/51),ADC值与肝脏比值(1.07±0.38)vs(0.93±0.21),差异有统计学意义(P<0.05)。动脉期明显强化(31/32 vs 42/51)、延迟期低信号(10/32 vs 45/51),强化方式:快进快出(10/32 vs 44/51),有供血动脉(1/32 vs 13/51),早期引流静脉(20/32 vs 2/51),中心血管征(14/32 vs 1/51),有包膜(7/32 vs 36/51),差异均有统计学意义(P<0.05)。而与周围血管关系:受压(11/32 vs 8/51)、肝胆期低信号(4/5 vs 19/23)差异无统计学意义(P>0.05)。结论HEAML与HCC的MRI表现有一定差异。特别是病灶在有无脂肪及出血、早期引流静脉、中心血管征和强化信号及方式有助于鉴别诊断。