Kidney transplantation in patients with tuberous sclerosis complex

• Published in: Pediatric transplantation Vol. 28; no. 4; pp. e14765 - n/a
• Main Authors: Goedken, Amber M., Ismail, Wesam W., Barrett, Lucas D. G., Harshman, Lyndsay A.
• Format: Journal Article
• Language: English
• Published: Denmark Wiley Subscription Services, Inc 01-06-2024
• Subjects: Adolescent; Adult; Allografts; Angiomyolipoma; Child; Child, Preschool; Disease Progression; Female; Humans; Infant; kidney; Kidney diseases; Kidney Failure, Chronic - surgery; Kidney Transplantation; Kidney transplants; Male; Pediatrics; Rapamycin; Renal failure; Retrospective Studies; TOR protein; transplantation; Tuberous sclerosis; Tuberous Sclerosis - complications; Tuberous Sclerosis - surgery; Tumors; Waiting Lists; waitlist; Young Adult; kidney; waitlist; angiomyolipoma; tuberous sclerosis; transplantation
• ISSN: 1397-3142; 1399-3046
• DOI: 10.1111/petr.14765

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.
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 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.