Combined liver-kidney transplantation for rare diseases

• Published in: World journal of hepatology Vol. 12; no. 10; pp. 722 - 737
• Main Authors: Knotek, Mladen, Novak, Rafaela, Jaklin-Kekez, Alemka, Mrzljak, Anna
• Format: Journal Article
• Language: English
• Published: United States Baishideng Publishing Group Inc 27-10-2020
• Subjects: Review; Methylmalonic aciduria; Combined liver-kidney transplantation; Glycogen storage diseases; Homozygous protein C deficiency; Atypical hemolytic uremic syndrome; Primary hyperoxaluria; Donor-specific antibodies; Sensitization; Hereditary complement C3 deficiency
• ISSN: 1948-5182; 1948-5182
• DOI: 10.4254/wjh.v12.i10.722

Combined liver and kidney transplantation (CLKT) is indicated in patients with failure of both organs, or for the treatment of end-stage chronic kidney disease (ESKD) caused by a genetic defect in the liver. The aim of the present review is to provide the most up-to-date overview of the rare conditions as indications for CLKT. They are major indications for CLKT in children. However, in some of them (
atypical hemolytic uremic syndrome or primary hyperoxaluria), CLKT may be required in adults as well. Primary hyperoxaluria is divided into three types, of which type 1 and 2 lead to ESKD. CLKT has been proven effective in renal function replacement, at the same time preventing recurrence of the disease. Nephronophthisis is associated with liver fibrosis in 5% of cases and these patients are candidates for CLKT. In alpha 1-antitrypsin deficiency, hereditary C3 deficiency, lecithin cholesterol acyltransferase deficiency and glycogen storage diseases, glomerular or tubulointerstitial disease can lead to chronic kidney disease. Liver transplantation as a part of CLKT corrects underlying genetic and consequent metabolic abnormality. In atypical hemolytic uremic syndrome caused by mutations in the genes for factor H, successful CLKT has been reported in a small number of patients. However, for this indication, CLKT has been largely replaced by eculizumab, an anti-C5 antibody. CLKT has been well established to provide immune protection of the transplanted kidney against donor-specific antibodies against class I HLA, facilitating transplantation in a highly sensitized recipient.