PDF(Pubmed)
Abstract：
BACKGROUND: Tuberous sclerosis complex (TSC) is a disorder of the mammalian target of the rapamycin (mTOR) pathway associated with the development of multisystem tumors, including renal angiomyolipoma (AML). These renal tumors are benign by nature but locally invasive and carry a risk for the progression of chronic kidney disease (CKD) to end stage kidney disease (ESKD). The frequency of subsequent renal transplantation in this population is largely uncharacterized, although single-center data suggests that 5%-15% of adult TSC patients are kidney transplant recipients.
METHODS: This retrospective cohort study utilized United Network for Organ Sharing (UNOS) data. We included candidates waitlisted between 1987 and 2020 for a first kidney transplant with TSC-associated kidney failure. We utilized descriptive statistics to characterize the frequency of first-time kidney transplant waitlisting and transplantation among persons with TSC and the Fine-Gray subdistribution hazard model to evaluate characteristics associated with progression from waitlist.
RESULTS: We identified 200 TSC-associated kidney failure patients within the waitlist cohort. Of these, 12 were pediatric patients. Two-thirds (N = 134) of waitlisted persons were female. One hundred forty patients received a transplant with a median waitlist time of 2 years. Younger age at waitlisting was associated with a greater probability of progressing to transplant (HR 0.98 [95% CI: 0.96-0.99]). 91.8% of kidney transplant recipients survived 1-year post-transplant with a functioning allograft.
CONCLUSIONS: The majority of patients with TSC who are waitlisted for a kidney transplant progress onto transplantation with excellent 1-year post transplant patient and allograft survival.
METHODS: This retrospective cohort study utilized United Network for Organ Sharing (UNOS) data. We included candidates waitlisted between 1987 and 2020 for a first kidney transplant with TSC-associated kidney failure. We utilized descriptive statistics to characterize the frequency of first-time kidney transplant waitlisting and transplantation among persons with TSC and the Fine-Gray subdistribution hazard model to evaluate characteristics associated with progression from waitlist.
RESULTS: We identified 200 TSC-associated kidney failure patients within the waitlist cohort. Of these, 12 were pediatric patients. Two-thirds (N = 134) of waitlisted persons were female. One hundred forty patients received a transplant with a median waitlist time of 2 years. Younger age at waitlisting was associated with a greater probability of progressing to transplant (HR 0.98 [95% CI: 0.96-0.99]). 91.8% of kidney transplant recipients survived 1-year post-transplant with a functioning allograft.
CONCLUSIONS: The majority of patients with TSC who are waitlisted for a kidney transplant progress onto transplantation with excellent 1-year post transplant patient and allograft survival.
摘要：
背景：结节性硬化症（TSC）是与多系统肿瘤发展相关的雷帕霉素（mTOR）途径的哺乳动物靶标疾病，包括肾血管平滑肌脂肪瘤（AML）。这些肾肿瘤本质上是良性的,但是局部侵入性的,并且具有使慢性肾病(CKD)进展为终末期肾病(ESKD)的风险。在这一人群中，随后的肾移植的频率在很大程度上没有被描述，尽管单中心数据显示5%-15%的成人TSC患者是肾移植受者.
方法：这项回顾性队列研究利用了联合器官共享网络(UNOS)数据。我们纳入了1987年至2020年之间等待上市的候选人，以进行首次TSC相关肾衰竭的肾脏移植。我们利用描述性统计来表征TSC患者中首次肾移植等待名单和移植的频率，并利用Fine-Gray子分布风险模型来评估与等待名单进展相关的特征。
结果：我们在等待队列中确定了200名TSC相关肾衰竭患者。其中,12名儿科患者。三分之二（N=134）的等候人员是女性。一百四十名患者接受了移植，等待名单的中位时间为2年。等待名单年龄越小，移植进展概率越大（HR0.98[95%CI：0.96-0.99]）。91.8％的肾移植受者在移植后1年存活，同种异体移植物正常。
结论：大多数等待肾移植的TSC患者在移植后1年和同种异体移植后存活率优异。
方法：这项回顾性队列研究利用了联合器官共享网络(UNOS)数据。我们纳入了1987年至2020年之间等待上市的候选人，以进行首次TSC相关肾衰竭的肾脏移植。我们利用描述性统计来表征TSC患者中首次肾移植等待名单和移植的频率，并利用Fine-Gray子分布风险模型来评估与等待名单进展相关的特征。
结果：我们在等待队列中确定了200名TSC相关肾衰竭患者。其中,12名儿科患者。三分之二（N=134）的等候人员是女性。一百四十名患者接受了移植，等待名单的中位时间为2年。等待名单年龄越小，移植进展概率越大（HR0.98[95%CI：0.96-0.99]）。91.8％的肾移植受者在移植后1年存活，同种异体移植物正常。
结论：大多数等待肾移植的TSC患者在移植后1年和同种异体移植后存活率优异。
