Efficacy And Safety Of Short-term Low-dose Everolimus In The Treatment Of Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex

ObjectiveTo evaluate the efficacy and safety of short-term low-dose everolimus in patients with tuberous sclerosis associated renal angiomyolipoma in Northwest China.MethodsFrom October 2015 to June 2020,48 patients with TSC-AML diagnosed by Department of Urology,Xijing Hospital,Air Force Medical University and treated with everolimus according to ITSCCC diagnostic criteria were selected,and they were divided into oral everolimus standard dose group and low dose group according to their willingness and financial situation.The standard dose group was given everolimus10.0mg/d.Low dose group was given everolimus 5.0 mg/d.The plasma concentration of everolimus was detected 2 weeks and 3 months after treatment.The volume of TSC-AML before treatment was defined as "baseline".The follow-up time was 3 months and 6months after treatment.The therapeutic effect was defined as the volume reduction rate of TSC-AML compared with baseline.The therapeutic effect and adverse reactions were compared between two groups.ResultThe average tumor volume reduction rate of 18 cases of low-dose group was 45.05±18.38%,and that of 30 cases of standard dose group was 52.16±21.68%.There was no significant difference between two groups(P = 0.251).The average tumor volume reduction rate was 53.26 ± 20.26% in the low dose group and 58.34 ± 21.75% in the standard dose group after six months treatment.There was no significant difference in the average tumor volume reduction rate between the two groups(P=0.426).The incidence of stomatitis in the low-dose group was significantly lower than that in the standard dose group(P < 0.001),and the severity of menstrual disorders was significantly lower than that in the standard dose group.ConclusionThe therapeutic effect of short-term low-dose everolimus in the treatment of TSC-AML was similar to that of short-term standard dose,but the adverse reactions and medical costs were significantly reduced.Therefore,we believe that low-dose everolimus can also be used as an option for the treatment of TSC-AML as the result of its advantages in safety and price,which may help patients to adhere to long-term treatment.