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combined liver-kidney transplantation for rare diseases

World Journal of Hepatology 2020年第10期12卷 722-737页

作者： Mladen Knotek Rafaela Novak alemka Jaklin-Kekez Anna Mrzljak Department of Medicine Tree Top HospitalHulhumale 23000Maldives Department of Medicine Merkur University HospitalZagreb 10000Croatia School of Medicine University of ZagrebZagreb 10000Croatia clinic for Pediatric Diseases Helena Zagreb 10000Croatia

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 *** aim of the present review is to provide the most up-to-date overview of the rare conditions as indications for *** are major indications for cLKT in ***,in some of them(e.g.,atypical hemolytic uremic syndrome or primary hyperoxaluria),cLKT may be required in adults as *** hyperoxaluria is divided into three types,of which type 1 and 2 lead to *** has been proven effective in renal function replacement,at the same time preventing recurrence of the *** is associated with liver fibrosis in 5%of cases and these patients are candidates for *** alpha 1-antitrypsin deficiency,hereditary c3 deficiency,lecithin cholesterol acyltransferase deficiency and glycogen storage diseases,glomerular or tubulointerstitial disease can lead to chronic kidney *** transplantation as a part of cLKT corrects underlying genetic and consequent metabolic *** atypical hemolytic uremic syndrome caused by mutations in the genes for factor H,successful cLKT has been reported in a small number of ***,for this indication,cLKT has been largely replaced by eculizumab,an anti-c5 *** 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.

关键词： combined liver-kidney transplantation Methylmalonic aciduria Hereditary complement c3 deficiency Glycogen storage diseases Homozygous protein c deficiency Primary hyperoxaluria Atypical hemolytic uremic syndrome Sensitization Donorspecific antibodies

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