Transplantation for inherited metabolic disorders of the liver

Transplantation Proceedings

Volumn 45, Issue 2, 2013, Pages 455-462

  Schilsky, M L ^a  

^a YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; HFE PROTEIN; LOW DENSITY LIPOPROTEIN RECEPTOR;

ACUTE LIVER FAILURE; ALPHA 1 ANTITRYPSIN DEFICIENCY; CHRONIC LIVER DISEASE; CYSTIC FIBROSIS; FAMILIAL AMYLOIDOSIS; FAMILIAL HYPERCHOLESTEROLEMIA; GENE THERAPY; GENETIC COUNSELING; GENETIC DISORDER; GLYCOGEN STORAGE DISEASE; GRAFT SURVIVAL; HEART TRANSPLANTATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOCHROMATOSIS; HEMOPHILIA A; HEMOPHILIA B; HETEROZYGOSITY; HOMOZYGOSITY; HUMAN; HYPEROXALURIA; INHERITED METABOLIC DISORDER; LIVER FUNCTION; LIVER TRANSPLANTATION; METABOLIC DISORDER; PATHOPHYSIOLOGY; PATIENT SELECTION; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN SECRETION; QUALITY OF LIFE; REVIEW; UREA CYCLE DISORDER; WILSON DISEASE;

EID: 84875201170     PISSN: 00411345     EISSN: 18732623     Source Type: Journal    
DOI: 10.1016/j.transproceed.2013.02.001     Document Type: Article

References (37)

1. D. Permutter Alpha one antitrypsin deficiency In Schiffs Diseases of the Liver E.R. Schiff, W.C. Maddrey, M.F. Sorrell, 2011 Wiley-Blackwell Oxford, UK
2. J.H. Teckman, J. Burrows, T. Hidvegi, B. Schmidt, P.D. Hale, D.H. Perlmutter The proteasome participates in degradation of mutant alpha 1-antitrypsin Z in the endoplasmic reticulum of hepatoma-derived hepatocytes J Biol Chem 276 2001 44865 44872
3. J.M. Hunt, R. Tuder Alpha 1 anti-trypsin: one protein, many functions Curr Mol Med 12 7 2012 Aug 827 835 Clin Transplant 25 2011 731 736
4. A. Topic, M. Ljujic, D. Radojkovic Alpha-1-antitrypsin in pathogenesis of hepatocellular carcinoma Hepat Mon 12 2012 e7042
5. M.G. Hughes Jr, K.M. Khan, A.C. Gruessner, H. Sharp, M. Hill, T. Jie, R. Kandaswamy, A. Humar, W.D. Payne, R.W. Gruessner Long-term outcome in 42 pediatric liver transplant patients with alpha 1-antitrypsin deficiency: a single-center experience Clin Transplant 25 2011 731 736
6. E.A. Roberts, M.L. Schilsky Diagnosis and treatment of Wilson disease: an update.; American Association for Study of Liver Diseases (AASLD) Hepatology 47 2008 2089 2111
7. R. Arnon, R. Annunziato, M. Schilsky, T. Miloh, A. Willis, M. Sturdevant, A. Sakworawich, F. Suchy, N. Kerkar Liver transplantation for children with Wilson disease: comparison of outcomes between children and adults Clin Transplant 25 2011 E52 E60
8. S. Koppikar, A. Dhawan Evaluation of the scoring system for the diagnosis of Wilson's disease in children Liver Int 25 2005 680 681
9. G. Schumacher, K.P. Platz, A.R. Mueller, R. Neuhaus, W. Luck, J.M. Langrehr, U. Settmacher, T. Steinmueller, M. Becker, P. Neuhaus Liver transplantation in neurologic Wilson's disease Transplant Proc 33 2001 1518 1519
10. V. Medici, V.G. Mirante, L.R. Fassati, M. Pompili, D. Forti, M. Del Gaudio, C.P. Trevisan, U. Cillo, G.C. Sturniolo, S. Fagiuoli Monotematica AISF 2000 OLT Study Group Liver transplantation for Wilson's disease: The burden of neurological and psychiatric disorders Liver Transpl 11 2005 1056 1063
11. A. Pietrangelo Hereditary hemochromatosis: pathogenesis, diagnosis, and treatment Gastroenterology 139 2010 393 408
12. G. Bolondi, C. Garuti, E. Corradini, H. Zoller, W. Vogel, A. Finkenstedt, J.L. Babitt, H.Y. Lin, A. Pietrangelo Altered hepatic BMP signaling pathway in human HFE hemochromatosis Blood Cells Mol Dis 45 2010 308 312
13. K.V. Kowdley, D.J. Brandhagen, R.G. Gish, N.M. Bass, J. Weinstein, M.L. Schilsky, R.J. Fontana, T. McCashland, S.J. Cotler, B.R. Bacon, E.B. Keeffe, F. Gordon, N. Polissar National Hemochromatosis Transplant Registry Survival after liver transplantation in patients with hepatic iron overload: the national hemochromatosis transplant registry Gastroenterology 129 2005 494 503
14. F.S. Dar, W. Faraj, M.B. Zaman, A. Bartlett, A. Bomford, A. O'Sullivan, J. O'Grady, M. Heneghan, M. Rela, N.D. Heaton Outcome of liver transplantation in hereditary hemochromatosis Transpl Int 22 2009 717 724
15. A. Giakoustidis, T.P. Cherian, N. Antoniadis, D. Giakoustidis Combined cardiac surgery and liver transplantation: three decades of worldwide results J Gastrointestin Liver Dis 20 2011 415 421
16. D. Debray, D. Kelly, R. Houwen, B. Strandvik, C. Colombo Best practice guidance for the diagnosis and management of cystic fibrosis-associated liver disease J Cyst Fibros 10 Suppl 2 2011 Jun S29 S36
17. C.S. Desai, A. Gruessner, S. Habib, R. Gruessner, K.M. Khan Survival of Cystic Fibrosis Patients Undergoing Liver and Liver-Lung Transplantations Transplant Proc 345 2012 688
18. M.R. Miller, R.J. Sokol, M.R. Narkewicz, M.K. Sontag Pulmonary function in individuals who underwent liver transplantation: from the US cystic fibrosis foundation registry Liver Transpl 18 2012 585 593
19. G. Grannas, M. Neipp, M.M. Hoeper, J. Gottlieb, R. Lück, T. Becker, A. Simon, C.P. Strassburg, M.P. Manns, T. Welte, A. Haverich, J. Klempnauer, B. Nashan, M. Strueber Indications for and outcomes after combined lung and liver transplantation: a single-center experience on 13 consecutive cases Transplantation 85 2008 524 531
20. J. Hicks, E. Wartchow, G. Mierau Glycogen storage diseases: a brief review and update on clinical features, genetic abnormalities, pathologic features, and treatment Ultrastruct Pathol 35 5 2011 183 196
21. G. Carreiro, C.A. Villela-Nogueira, Hu Coelho, S. Basto, V.L. Pannain, A. Caroli-Bottino, J. Ribeiro Filho Orthotopic liver transplantation in glucose-6-phosphatase deficiency - Von Gierke disease - with multiple hepatic adenomas and concomitant focal nodular hyperplasia J Pediatr Endocrinol Metab 20 2007 545 549
22. J. Häberle, N. Boddaert, A. Burlina, A. Chakrapani, M. Dixon, M. Huemer, D. Karall, D. Martinelli, P.S. Crespo, R. Santer, A. Servais, V. Valayannopoulos, M. Lindner, V. Rubio, C. Dionisi-Vici Suggested guidelines for the diagnosis and management of urea cycle disorders Orphanet J Rare Dis 29 2012 7 32
23. P.M. Campeau, P.J. Pivalizza, G. Miller, K. McBride, S. Karpen, J. Goss, B.H. Lee Early orthotopic liver transplantation in urea cycle defects: follow up of a developmental outcome study Mol Genet Metab 100 Suppl 1 2010 S84 S87
24. S.P. Horslen, T.C. McCowan, T.C. Goertzen, P.I. Warkentin, H.B. Cai, S.C. Strom, I.J. Fox Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder Pediatrics 111 6 Pt 1 2003 Jun 1262 1267
25. G. Celik, S. Sen, S. Sipahi, C. Akkin, S. Tamsel, H. Töz, C. Hoscoskun Regressive course of oxalate deposition in primary hyperoxaluria after kidney transplantation Renal Fail 32 9 2010 1131 1136
26. J.I. Scheinman Liver transplantation in oxalosis prior to advanced chronic kidney disease Pediatr Nephrol 25 2010 2217 2222
27. S. Sato, S. Fuchinoue, M. Kimikawa, T. Tolimbara, I. Nakajima, S. Teraoka, H. Shiraga, K. Ito Sequential liver-kidney transplantation from a living-related donor in primary hyperoxaluria type 1 (oxalosis) Transplant Proc 35 2003 373 374
28. A.D. Marais, J.C. Firth, D.J. Blom Homozygous familial hypercholesterolemia and its management Semin Vasc Med 4 2004 43 50
29. N. Kawagishi, K. Satoh, Y. Akamatsu, S. Sekiguchi, Y. Ishigaki, S. Oikawa, S. Satomi Long-term outcome after living donor liver transplantation for two cases of homozygous familial hypercholesterolemia from a heterozygous donor J Atheroscler Thromb 14 2007 94 98
30. Y. Shirahata, N. Ohkohchi, N. Kawagishi, M. Syouji, S. Tsukamoto, S. Sekiguchi, N. Koyamada, S. Oikawa, S. Satomi Living-donor liver transplantation for homozygous familial hypercholesterolemia from a donor with heterozygous hypercholesterolemia Transpl Int 16 2003 276 279
31. X. Hou, M.I. Aguilar, D.H. Small Transthyretin and familial amyloidotic polyneuropathy Recent progress in understanding the molecular mechanism of neurodegeneration FEBS J 274 2007 1637 1650
32. S.H. Bradshaw, J.P. Veinot Cardiac amyloidosis: what are the indications for transplant? Curr Opin Cardiol 27 2012 143 147
33. E.Y. Yoshitoshi, Y. Takada, F. Oike, S. Sakamoto, K. Ogawa, H. Kanazawa, Y. Ogura, S. Okamoto, H. Haga, M. Ueda, H. Egawa, M. Kasahara, K. Tanaka, S. Uemoto Long-term outcomes for 32 cases of Wilson's disease after living-donor liver transplantation Transplantation 87 2009 261 267
34. F. Cheng, G.Q. Li, F. Zhang, X.C. Li, B.C. Sun, L.B. Kong, L.Y. Pu, K. Wang, X.F. Qian, W. You, X.H. Wang Outcomes of living-related liver transplantation for Wilson's disease: a single-center experience in China Transplantation 87 2009 751 757
35. D. Azoulay, C. Salloum, D. Samuel, V. Planté-Bordeneuve Operative risks of domino liver transplantation for the FAP liver donor and the FAP liver recipient Amyloid 19 2012 73 74
36. C. Liu, D.M. Niu, C.C. Loong, C.Y. Hsia, M.Y. Tsou, H.L. Tsai, C. Wei Domino liver graft from a patient with homozygous familial hypercholesterolemia Pediatr Transplant 14 2010 E30 E33
37. C. Ribes-Koninckx, E.P. Ibars, MÁ Calzado Agrasot, A. Bonora-Centelles, B.P. Miquel, J.J. Vila Carbó, E.D. Aliaga, J.M. Pallardó, M.J. Gómez-Lechón, J.V. Castell Clinical outcome of hepatocyte transplantation in four pediatric patients with inherited metabolic diseases Cell Transplant 21 2012 2267 2282