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Kidney transplantation in patients with tuberous sclerosis complex
Amber M Goedken1, Wesam W Ismail1, Lucas D G Barrett2,3,4
1Department of Pharmacy Practice and Science, University of Iowa College of Pharmacy, Iowa City, Iowa, USA.
Pediatric Transplantation
|May 23, 2024
Summary
Patients with Tuberous Sclerosis Complex (TSC) kidney failure who are waitlisted for a kidney transplant have a high likelihood of receiving one. Post-transplant outcomes are favorable, with excellent patient and allograft survival rates at one year.
Area of Science:
- Nephrology
- Transplantation Immunology
- Genetics
Background:
- Tuberous Sclerosis Complex (TSC) is a genetic disorder affecting the mTOR pathway, leading to tumors, including renal angiomyolipomas.
- These tumors can cause chronic kidney disease (CKD) and end-stage kidney disease (ESKD), necessitating kidney transplantation.
- The frequency of kidney transplantation in TSC patients is not well-established.
Purpose of the Study:
- To characterize the frequency of kidney transplant waitlisting and transplantation in patients with TSC-associated kidney failure.
- To identify factors associated with progression from kidney transplant waitlist to transplantation.
Main Methods:
- Retrospective cohort study using United Network for Organ Sharing (UNOS) data from 1987-2020.
- Included patients waitlisted for a first kidney transplant due to TSC-associated kidney failure.
- Descriptive statistics and Fine-Gray subdistribution hazard models were used.
Main Results:
- 200 patients with TSC-associated kidney failure were identified on the waitlist; 140 received a transplant.
- Median waitlist time was 2 years; younger age at waitlisting correlated with higher transplant probability.
- One-year post-transplant survival was 91.8% for patients and their allografts.
Conclusions:
- Most TSC patients listed for kidney transplant successfully receive one.
- Excellent one-year patient and allograft survival rates are observed post-transplantation in this cohort.
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