Combined liver-kidney transplantation for rare diseases

Area of Science:

• Nephrology
• Hepatology
• Transplantation Immunology

Background:

• Combined liver and kidney transplantation (CLKT) is an option for patients with combined liver and kidney failure or genetic liver defects causing end-stage chronic kidney disease (ESKD).
• Rare genetic conditions are significant indications for CLKT, particularly in pediatric patients, but also in adults for specific diseases.

Purpose of the Study:

• To provide an updated overview of rare conditions that serve as indications for combined liver and kidney transplantation (CLKT).

Main Methods:

• Literature review of rare indications for combined liver and kidney transplantation (CLKT).

Main Results:

• CLKT is effective for renal function replacement and preventing disease recurrence in conditions like primary hyperoxaluria.
• Nephronophthisis with liver fibrosis, alpha-1-antitrypsin deficiency, hereditary C3 deficiency, lecithin cholesterol acyltransferase deficiency, and glycogen storage diseases are candidates for CLKT.
• While CLKT has shown success in atypical hemolytic uremic syndrome, eculizumab is now the preferred treatment; however, CLKT remains vital for immune protection in highly sensitized recipients.

Conclusions:

• CLKT is a critical treatment for specific rare genetic disorders leading to combined organ failure or ESKD.
• Liver transplantation within CLKT addresses underlying genetic and metabolic abnormalities, crucial for conditions like primary hyperoxaluria and glycogen storage diseases.
• CLKT provides essential immune protection for transplanted kidneys, particularly in highly sensitized patients, though alternative treatments have emerged for certain indications like atypical hemolytic uremic syndrome.