Lebertransplantation bei metabolischen Lebererkrankungen im Erwachsenenalter

Zusammenfassung

Das klinische Erscheinungsbild der metabolischen Lebererkrankungen ist heterogen. Es reicht vom akuten Leberversagen über Leberzirrhose und Lebertumoren bis hin zur gesunden Leber mit extrahepatischen Komplikationen. Die extrahepatischen Manifestationen haben häufig einen entscheidenden Einfluss auf die Prognose im Spontanverlauf sowie nach Lebertransplantation. Viele metabolische Lebererkrankungen können bei frühzeitiger Diagnose erfolgreich konservativ behandelt werden. Kommt es zum Leberversagen oder drohen irreversible extrahepatische Schäden, ist eine Lebertransplantation indiziert. Die Indikationsstellung orientiert sich an den Standardkriterien zur Lebertransplantation, wobei abhängig von der Grunderkrankung jeweils spezifische Kriterien hinzutreten. Bei extrahepatischen Manifestationen kann sich auch die Notwendigkeit einer Mehrorgantransplantation ergeben. Im Allgemeinen ist die Prognose nach Lebertransplantation bei metabolischen Erkrankungen gut. Zusätzlich werden viele metabolische Defekte durch die Lebertransplantation phänotypisch geheilt. Alternative Ansätze wie die Hepatozytentransplantation oder die Gentherapie sind demgegenüber noch experimentell.

Abstract

The clinical presentation of metabolic liver disease is highly variable, covering acute liver failure, liver cirrhosis, hepatic cancer and various extrahepatic manifestations. Both natural course and prognosis after liver transplantation are substantially influenced by extrahepatic manifestations. In many types of metabolic liver disease, timely diagnosis allows for successful medical treatment. However, progressive liver failure and severe extrahepatic damage can be the indication for liver transplantation. In general, standard transplantation criteria also apply for metabolic liver disease. They have to be modified by disease-specific criteria, and extrahepatic damage may necessitate multiorgan transplantation. The overall prognosis after liver transplantation for metabolic liver disease is favorable. Furthermore, several metabolic defects are phenotypically cured by liver transplantation. Alternative treatments like hepatocyte transplantation or gene therapy are still in the experimental stage.

Schlüsselwörter

Metabolische Lebererkrankungen - Lebertransplantation - M. Wilson - hereditäre Hämochromatose - α₁-Antitrypsinmangel - zystische Fibrose - Glykogenspeichererkrankungen - Amyloidose - primäre Hyperoxalurie

Key words

Metabolic liver disease - liver transplantation - Wilson’s disease - hereditary hemochromatosis - α-1-antitrypsin deficiency - cystic fibrosis - glycogen storage disease - amyloidosis - primary hyperoxaluria

Literatur

• 1 Mailliard M E, Gollan J L. Metabolic liver disease in the young adult.  Best Pract Res Clin Gastroenterol. 2003;  17 307-322
• 2 Perlmutter D H. Metabolic liver disease.  J Pediatr Gastroenterol Nutr. 2002;  35 (Suppl 1) S24-28
• 3 Shneider B L. Pediatric liver transplantation in metabolic disease: clinical decision making.  Pediatr Transplant. 2002;  6 25-29
• 4 McDiarmid S V. The liver and metabolic diseases of childhood.  Liver Transpl Surg. 1998;  4 S34-S50
• 5 Kayler L K, Merion R M, Lee S. et al . Long-term survival after liver transplantation in children with metabolic disorders.  Pediatr Transplant. 2002;  6 295-300
• 6 Burdelski M, Rogiers X. Liver transplantation in metabolic disorders.  Acta Gastroenterol Belg. 1999;  62 300-305
• 7 Khanna A, Jain A, Eghtesad B. et al . Liver transplantation for metabolic liver diseases.  Surg Clin North Am. 1999;  79 153-162
• 8 Lucey M R, Brown K A, Everson G T. et al . Minimal criteria for placement of adults on the liver transplant waiting list: a report of a national conference organized by the American Society of Transplant Physicians and the American Association for the Study of Liver Diseases.  Liver Transpl Surg. 1997;  3 628-637
• 9 Pratschke J, Steinmüller T, Bechstein W O. et al . Orthotopic liver transplantation for hepatic associated metabolic disorders.  Clin Transplant. 1998;  12 228-232
• 10 Rela M, Muiesan P, Heaton N D. et al . Orthotopic liver transplantation for hepatic-based metabolic disorders.  Transpl Int. 1995;  8 41-44
• 11 Ishak K G. Inherited metabolic diseases of the liver.  Clin Liver Dis. 2002;  6 455-479
• 12 Pugh R N, Murray-Lyon I M, Dawson J L. et al . Transection of the oesophagus for bleeding oesophageal varices.  Br J Surg. 1973;  60 646-649
• 13 Freeman R B Jr, Wiesner R H, Harper A. et al . The new liver allocation system: moving toward evidence-based transplantation policy.  Liver Transpl. 2002;  8 851-858
• 14 Kamath P S, Wiesner R H, Malinchoc M. et al . A model to predict survival in patients with end-stage liver disease.  Hepatology. 2001;  33 464-470
• 15 Wiesner R, Edwards E, Freeman R. et al . Model for end-stage liver disease (MELD) and allocation of donor livers.  Gastroenterology. 2003;  124 91-96
• 16 Muiesan P, Rela M, Kane P. et al . Liver transplantation for neonatal haemochromatosis.  Arch Dis Child Fetal Neonatal Ed. 1995;  73 F178-F180
• 17 Durand P, Debray D, Mandel R. et al . Acute liver failure in infancy: a 14-year experience of a pediatric liver transplantation center.  J Pediatr. 2001;  139 871-876
• 18 Corbally M T, Rela M, Heaton N D. et al . Orthotopic liver transplantation for acute hepatic failure in children.  Transpl Int. 1994;  7 (Suppl 1) S104-107
• 19 Bernuau J, Samuel D, Durand F. et al . Criteria for emergency liver transplantation in patients with acute viral hepatitis and factor V below 50 % of normal: a prospective study (Abstract).  Hepatology. 1991;  14 49A
• 20 O’Grady J G, Alexander G J, Hayllar K M. et al . Early indicators of prognosis in fulminant hepatic failure.  Gastroenterology. 1989;  97 439-445
• 21 Eng S C, Kowdley K V. Expansion of criteria for liver transplantation in HCC: a slippery slope?.  Gastroenterology. 2002;  122 579-582
• 22 Mela M, Mancuso A, Burroughs A K. Review article: hepatocellular carcinoma: indications for liver transplantation.  Aliment Pharmacol Ther. 2003;  17 (Suppl 2) 130-137
• 23 Steinmüller T, Jonas S, Neuhaus P. Review article: liver transplantation for hepatocellular carcinoma.  Aliment Pharmacol Ther. 2003;  17 (Suppl 2) 138-144
• 24 Wudel L J Jr, Chapman W C. Indications and limitations of liver transplantation for hepatocellular carcinoma.  Surg Oncol Clin N Am. 2003;  12 77-90
• 25 Starzl T E, Murase N, Demetris A. et al . The mystique of hepatic tolerogenicity.  Semin Liver Dis. 2000;  20 497-510
• 26 Loudianos G, Gitlin J D. Wilson’s disease.  Semin Liver Dis. 2000;  20 353-364
• 27 Schilsky M L, Tavill A S. Wilson disease. Schiff ER, Sorrell MF, Maddrey WC Schiff’s diseases of the liver Philadelphia; Lippincott, Williams & Wilkins 2003: 1169-1186
• 28 El-Youssef M. Wilson disease.  Mayo Clin Proc. 2003;  78 1126-1136
• 29 Schoenberger M, Ellis P P. Disappearance of Kayser-Fleischer rings after liver transplantation.  Arch Ophthalmol. 1979;  97 1914-1915
• 30 Lui C C, Chen C L, Weng H H. et al . Changes in neuroimaging in Wilson’s disease following orthotopic liver transplantation.  Transplant Proc. 1996;  28 1710-1713
• 31 Song H S, Ku W C, Chen C L. Disappearance of Kayser-Fleischer rings following liver transplantation.  Transplant Proc. 1992;  24 1483-1485
• 32 Bellary S, Hassanein T, Van Thiel D H. Liver transplantation for Wilson’s disease.  J Hepatol. 1995;  23 373-381
• 33 Eghtesad B, Nezakatgoo N, Geraci L C. et al . Liver transplantation for Wilson’s disease: a single-center experience.  Liver Transpl Surg. 1999;  5 467-474
• 34 Emre S, Atillasoy E O, Ozdemir S. et al . Orthotopic liver transplantation for Wilson’s disease: a single-center experience.  Transplantation. 2001;  72 1232-1236
• 35 Rela M, Heaton N D, Vougas V. et al . Orthotopic liver transplantation for hepatic complications of Wilson’s disease.  Br J Surg. 1993;  80 909-911
• 36 Schilsky M L, Scheinberg I H, Sternlieb I. Liver transplantation for Wilson’s disease: indications and outcome.  Hepatology. 1994;  19 583-587
• 37 Schumacher G, Platz K P, Mueller A R. et al . Liver transplantation in neurologic Wilson’s disease.  Transplant Proc. 2001;  33 1518-1519
• 38 Sutcliffe R P, Maguire D D, Muiesan P. et al . Liver transplantation for Wilson’s disease: long-term results and quality-of-life assessment.  Transplantation. 2003;  75 1003-1006
• 39 Bax R T, Hassler A, Luck W. et al . Cerebral manifestation of Wilson’s disease successfully treated with liver transplantation.  Neurology. 1998;  51 863-865
• 40 Mason A L, Marsh W, Alpers D H. Intractable neurological Wilson’s disease treated with orthotopic liver transplantation.  Dig Dis Sci. 1993;  38 1746-1750
• 41 Lui C C, Chen C L, Cheng Y F. et al . Recovery of neurological deficits in a case of Wilson’s disease after liver transplantation.  Transplant Proc. 1998;  30 3324-3325
• 42 Stracciari A, Tempestini A, Borghi A. et al . Effect of liver transplantation on neurological manifestations in Wilson disease.  Arch Neurol. 2000;  57 384-386
• 43 Polson R J, Rolles K, Calne R Y. et al . Reversal of severe neurological manifestations of Wilson’s disease following orthotopic liver transplantation.  Q J Med. 1987;  64 685-691
• 44 Guarino M, Stracciari A, D’Alessandro R. et al . No neurological improvement after liver transplantation for Wilson’s disease.  Acta Neurol Scand. 1995;  92 405-408
• 45 Suzuki S, Sato Y, Ichida T. et al . Recovery of severe neurologic manifestations of Wilson’s disease after living-related liver transplantation: a case report.  Transplant Proc. 2003;  35 385-386
• 46 Hermann W, Eggers B, Wagner A. The indication for liver transplant to improve neurological symptoms in a patient with Wilson’s disease.  J Neurol. 2002;  249 1733-1734
• 47 Wu J C, Huang C C, Jeng L B. et al . Correlation of neurological manifestations and MR images in a patient with Wilson’s disease after liver transplantation.  Acta Neurol Scand. 2000;  102 135-139
• 48 Tavill A S, Bacon B R. Hemochromatosis and the iron overload syndromes. Zakim D, Boyer TD Hepatology Philadelphia; Saunders 2003: 1289-1316
• 49 Brandhagen D J. Liver transplantation for hereditary hemochromatosis.  Liver Transpl. 2001;  7 663-672
• 50 Fracanzani A L, Conte D, Fraquelli M. et al . Increased cancer risk in a cohort of 230 patients with hereditary hemochromatosis in comparison to matched control patients with non-iron-related chronic liver disease.  Hepatology. 2001;  33 647-651
• 51 Westra W H, Hruban R H, Baughman K L. et al . Progressive hemochromatotic cardiomyopathy despite reversal of iron deposition after liver transplantation.  Am J Clin Pathol. 1993;  99 39-44
• 52 Mosimann F, Gardaz J P, Fontolliet C. et al . Fatal cardiomyopathy after liver transplantation for genetic hemochromatosis.  Transplant Proc. 1994;  26 3635-3636
• 53 Tallgren M, Hockerstedt K, Makinen J. et al . Cardiac evaluation of liver transplant recipients: QT dispersion in electrocardiogram.  Clin Transplant. 1996;  10 408-413
• 54 Aguirrezabalaga J, Gomez M, Novas S. et al . Combined liver-pancreas transplantation: contribution of five cases.  Transplant Proc. 2002;  34 211-212
• 55 Surakomol S, Olson L J, Rastogi A. et al . Combined orthotopic heart and liver transplantation for genetic hemochromatosis.  J Heart Lung Transplant. 1997;  16 573-575
• 56 Kowdley K V, Hassanein T, Kaur S. et al . Primary liver cancer and survival in patients undergoing liver transplantation for hemochromatosis.  Liver Transpl Surg. 1995;  1 237-241
• 57 Poulos J E, Bacon B R. Liver transplantation for hereditary hemochromatosis.  Dig Dis. 1996;  14 316-322
• 58 Brandhagen D J, Alvarez W, Therneau T M. et al . Iron overload in cirrhosis-HFE genotypes and outcome after liver transplantation.  Hepatology. 2000;  31 456-460
• 59 Tung B Y, Farrell F J, McCashland T M. et al . Long-term follow-up after liver transplantation in patients with hepatic iron overload.  Liver Transpl Surg. 1999;  5 369-374
• 60 Farrell F J, Nguyen M, Woodley S. et al . Outcome of liver transplantation in patients with hemochromatosis.  Hepatology. 1994;  20 404-410
• 61 Carrell R W, Lomas D A. Alpha1-antitrypsin deficiency - a model for conformational diseases.  N Engl J Med. 2002;  346 45-53
• 62 Perlmutter D H. Liver injury in alpha1-antitrypsin deficiency: an aggregated protein induces mitochondrial injury.  J Clin Invest. 2002;  110 1579-1583
• 63 de Serres F J. Worldwide racial and ethnic distribution of alpha1-antitrypsin deficiency: summary of an analysis of published genetic epidemiologic surveys.  Chest. 2002;  122 1818-1829
• 64 Anonym . American Thoracic Society/European Respiratory Society Statement: Standards for the Diagnosis and Management of Individuals with Alpha-1 Antitrypsin Deficiency.  Am J Respir Crit Care Med. 2003;  168 818-900
• 65 Graziadei I W, Joseph J J, Wiesner R H. et al . Increased risk of chronic liver failure in adults with heterozygous alpha1-antitrypsin deficiency.  Hepatology. 1998;  28 1058-1063
• 66 Loreno M, Boccagni P, Rigotti P. et al . Combined liver-kidney transplantation in a 15-year-old boy with alpha1-antitrypsin deficiency.  J Hepatol. 2002;  36 565-568
• 67 Hood J M, Koep L J, Peters R L. et al . Liver transplantation for advanced liver disease with alpha-1-antitrypsin deficiency.  N Engl J Med. 1980;  302 272-275
• 68 van Furth R, Kramps J A, van der Putten A B. et al . Change in alpha 1-antitrypsin phenotype after orthotopic liver transplant.  Clin Exp Immunol. 1986;  66 669-672
• 69 O’Riordan K, Blei A, Rao M S. et al . alpha 1-antitrypsin deficiency-associated panniculitis: resolution with intravenous alpha 1-antitrypsin administration and liver transplantation.  Transplantation. 1997;  63 480-482
• 70 Elzouki A N, Lindgren S, Nilsson S. et al . Severe alpha1-antitrypsin deficiency (PiZ homozygosity) with membranoproliferative glomerulonephritis and nephrotic syndrome, reversible after orthotopic liver transplantation.  J Hepatol. 1997;  26 1403-1407
• 71 Noble-Jamieson G, Barnes N D, Thiru S. et al . Severe hypertension after liver transplantation in alpha 1 antitrypsin deficiency.  Arch Dis Child. 1990;  65 1217-1221
• 72 Esquivel C O, Vicente E, Van Thiel D. et al . Orthotopic liver transplantation for alpha-1-antitrypsin deficiency: an experience in 29 children and ten adults.  Transplant Proc. 1987;  19 3798-3802
• 73 Filipponi F, Soubrane O, Labrousse F. et al . Liver transplantation for end-stage liver disease associated with alpha-1-antitrypsin deficiency in children: pretransplant natural history, timing and results of transplantation.  J Hepatol. 1994;  20 72-78
• 74 Vennarecci G, Gunson B K, Ismail T. et al . Transplantation for end stage liver disease related to alpha 1 antitrypsin.  Transplantation. 1996;  61 1488-1495
• 75 Francavilla R, Castellaneta S P, Hadzic N. et al . Prognosis of alpha-1-antitrypsin deficiency-related liver disease in the era of paediatric liver transplantation.  J Hepatol. 2000;  32 986-992
• 76 Prachalias A A, Kalife M, Francavilla R. et al . Liver transplantation for alpha-1-antitrypsin deficiency in children.  Transpl Int. 2000;  13 207-210
• 77 Ratjen F, Doring G. Cystic fibrosis.  Lancet. 2003;  361 681-689
• 78 Greger R, Schreiber R, Mall M. et al . Cystic fibrosis and CFTR.  Pflügers Arch. 2001;  443 (Suppl 1) S3-7
• 79 Colombo C, Crosignani A, Battezzati P M. Liver involvement in cystic fibrosis.  J Hepatol. 1999;  31 946-954
• 80 Diwakar V, Pearson L, Beath S. Liver disease in children with cystic fibrosis.  Paediatr Respir Rev. 2001;  2 340-349
• 81 Feranchak A P, Sokol R J. Cholangiocyte biology and cystic fibrosis liver disease.  Semin Liver Dis. 2001;  21 471-488
• 82 Boucher R C. An overview of the pathogenesis of cystic fibrosis lung disease.  Adv Drug Deliv Rev. 2002;  54 1359-1371
• 83 Bright-Thomas R J, Webb A K. The heart in cystic fibrosis.  J R Soc Med. 2002;  95 (Suppl 41) 2-10
• 84 Modolell I, Guarner L, Malagelada J R. Digestive system involvement in cystic fibrosis.  Pancreatology. 2002;  2 12-16
• 85 Moran A. Endocrine complications of cystic fibrosis.  Adolesc Med. 2002;  13 145-159
• 86 Hankard R, Munck A, Navarro J. Nutrition and growth in cystic fibrosis.  Horm Res. 2002;  58 (Suppl 1) 16-20
• 87 Colombo C, Battezzati P M, Crosignani A. et al . Liver disease in cystic fibrosis: A prospective study on incidence, risk factors, and outcome.  Hepatology. 2002;  36 1374-1382
• 88 Efrati O, Barak A, Modan-Moses D. et al . Liver cirrhosis and portal hypertension in cystic fibrosis.  Eur J Gastroenterol Hepatol. 2003;  15 1073-1078
• 89 Cheng K, Ashby D, Smyth R. Ursodeoxycholic acid for cystic fibrosis-related liver disease.  Cochrane Database Syst Rev. 2000;  CD000222
• 90 Genyk Y S, Quiros J A, Jabbour N. et al . Liver transplantation in cystic fibrosis.  Curr Opin Pulm Med. 2001;  7 441-447
• 91 Stern R C, Mayes J T, Weber F L Jr. et al . Restoration of exocrine pancreatic function following pancreas-liver-kidney transplantation in a cystic fibrosis patient.  Clin Transplant. 1994;  8 1-4
• 92 Fridell J A, Mazariegos G V, Orenstein D. et al . Liver and intestinal transplantation in a child with cystic fibrosis: a case report.  Pediatr Transplant. 2003;  7 240-242
• 93 Cox K L, Ward R E, Furgiuele T L. et al . Orthotopic liver transplantation in patients with cystic fibrosis.  Pediatrics. 1987;  80 571-574
• 94 Noble-Jamieson G, Valente J, Barnes N D. et al . Liver transplantation for hepatic cirrhosis in cystic fibrosis.  Arch Dis Child. 1994;  71 349-352
• 95 Mack D R, Traystman M D, Colombo J L. et al . Clinical denouement and mutation analysis of patients with cystic fibrosis undergoing liver transplantation for biliary cirrhosis.  J Pediatr. 1995;  127 881-887
• 96 Fridell J A, Bond G J, Mazariegos G V. et al . Liver transplantation in children with cystic fibrosis: a long-term longitudinal review of a single center’s experience.  J Pediatr Surg. 2003;  38 1152-1156
• 97 Molmenti E P, Squires R H, Nagata D. et al . Liver transplantation for cholestasis associated with cystic fibrosis in the pediatric population.  Pediatr Transplant. 2003;  7 93-97
• 98 Couetil J P, Houssin D P, Soubrane O. et al . Combined lung and liver transplantation in patients with cystic fibrosis. A 4 œ-year experience.  J Thorac Cardiovasc Surg. 1995;  110 1415-1423
• 99 Dennis C M, McNeil K D, Dunning J. et al . Heart-lung-liver transplantation.  J Heart Lung Transplant. 1996;  15 536-538
• 100 Couetil J P, Soubrane O, Houssin D P. et al . Combined heart-lung-liver, double lung-liver, and isolated liver transplantation for cystic fibrosis in children.  Transpl Int. 1997;  10 33-39
• 101 Wise P E, Wright J K, Chapman W C. et al . Heart-lung-liver transplant for cystic fibrosis.  Transplant Proc. 2001;  33 3568-3571
• 102 Milkiewicz P, Skiba G, Kelly D. et al . Transplantation for cystic fibrosis: outcome following early liver transplantation.  J Gastroenterol Hepatol. 2002;  17 208-213
• 103 Klima L D, Kowdley K V, Lewis S L. et al . Successful lung transplantation in spite of cystic fibrosis-associated liver disease: a case series.  J Heart Lung Transplant. 1997;  16 934-938
• 104 Ghishan F K. Inborn errors of metabolism that lead to permanent hepatic injury. Zakim D, Boyer TD Hepatology Philadelphia; Saunders 2003: 1397-1459
• 105 Vogt M, Gitzelmann R, Allemann J. Dekompensierte Leberzirrhose infolge Galaktosämie bei einem 52jährigen Mann.  Schweiz Med Wochenschr. 1980;  110 1781-1783
• 106 Mowat A P. Orthotopic liver transplantation in liver-based metabolic disorders.  Eur J Pediatr. 1992;  151 (Suppl 1) S32-38
• 107 Froesch E R, Wolf H P, Baitsch H. et al . Hereditary fructose intolerance. An inborn defect of hepatic fructose-1-phosphate splitting aldolase.  Am J Med. 1963;  34 151-167
• 108 Lameire N, Mussche M, Baele G. et al . Hereditary fructose intolerance: a difficult diagnosis in the adult.  Am J Med. 1978;  65 416-423
• 109 Locher S. Akutes Leber- und Nierenversagen nach Sorbitinfusion bei 28jährigem Patienten mit undiagnostizierter Fruktoseintoleranz.  Anästh Intensivther Notfallmed. 1987;  22 194-197
• 110 Rey M, Behrens R, Zeilinger G. Fatale Folgen einer Fructose-Infusion bei undiagnostizierter Fructose-Intoleranz.  Dtsch Med Wochenschr. 1988;  113 945-947
• 111 Sachs M, Asskali F, Förster H. et al . Wiederholte perioperative Applikation von Fructose und Sorbit bei einer Patientin mit hereditärer Fructoseintoleranz (HFI).  Z Ernährungswiss. 1993;  32 56-66
• 112 Schulte M J, Lenz W. Fatal sorbitol infusion in patient with fructose-sorbitol intolerance.  Lancet. 1977;  2 188
• 113 Bianchi L. Glycogen storage disease I and hepatocellular tumours.  Eur J Pediatr. 1993;  152 (Suppl 1) S63-70
• 114 Labrune P, Trioche P, Duvaltier I. et al . Hepatocellular adenomas in glycogen storage disease type I and III: a series of 43 patients and review of the literature.  J Pediatr Gastroenterol Nutr. 1997;  24 276-279
• 115 Malatack J J, Finegold D N, Iwatsuki S. et al . Liver transplantation for type I glycogen storage disease.  Lancet. 1983;  1 1073-1075
• 116 Koestinger A, Gillet M, Chiolero R. et al . Effect of liver transplantation on hepatic glucose metabolism in a patient with type I glycogen storage disease.  Transplantation. 2000;  69 2205-2207
• 117 Faivre L, Houssin D, Valayer J. et al . Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia.  J Inherit Metab Dis. 1999;  22 723-732
• 118 Martinez-Olmos M A, Lopez-Sanroman A, Martin-Vaquero P. et al . Liver transplantation for type Ib glycogenosis with reversal of cyclic neutropenia.  Clin Nutr. 2001;  20 375-377
• 119 Matern D, Starzl T E, Arnaout W. et al . Liver transplantation for glycogen storage disease types I, III, and IV.  Eur J Pediatr. 1999;  158 (Suppl 2) S43-48
• 120 Sokal E M, Lopez-Silvarrey A, Buts J P. et al . Orthotopic liver transplantation for type I glycogenosis unresponsive to medical therapy.  J Pediatr Gastroenterol Nutr. 1993;  16 465-467
• 121 Kirschner B S, Baker A L, Thorp F K. Growth in adulthood after liver transplantation for glycogen storage disease type I.  Gastroenterology. 1991;  101 238-241
• 122 Labrune P. Glycogen storage disease type I: indications for liver and/or kidney transplantation.  Eur J Pediatr. 2002;  161 (Suppl 1) S53-55
• 123 Haagsma E B, Smit G P, Niezen-Koning K E. et al . Type IIIb glycogen storage disease associated with end-stage cirrhosis and hepatocellular carcinoma. The Liver Transplant Group.  Hepatology. 1997;  25 537-540
• 124 Rosenthal P, Podesta L, Grier R. et al . Failure of liver transplantation to diminish cardiac deposits of amylopectin and leukocyte inclusions in type IV glycogen storage disease.  Liver Transpl Surg. 1995;  1 373-376
• 125 Selby R, Starzl T E, Yunis E. et al . Liver transplantation for type I and type IV glycogen storage disease.  Eur J Pediatr. 1993;  152 (Suppl 1) S71-76
• 126 Dhawan A, Tan K C, Portmann B. et al . Glycogenosis type IV: liver transplant at 12 years.  Arch Dis Child. 1994;  71 450-451
• 127 Sokal E M, Van Hoof F, Alberti D. et al . Progressive cardiac failure following orthotopic liver transplantation for type IV glycogenosis.  Eur J Pediatr. 1992;  151 200-203
• 128 Buxbaum J N, Tagoe C E. The genetics of the amyloidoses.  Annu Rev Med. 2000;  51 543-569
• 129 Lobato L. Portuguese-type amyloidosis (transthyretin amyloidosis, ATTR V30M).  J Nephrol. 2003;  16 438-442
• 130 Saraiva M J. Transthyretin amyloidosis: a tale of weak interactions.  FEBS Lett. 2001;  498 201-203
• 131 Suhr O B, Svendsen I H, Andersson R. et al . Hereditary transthyretin amyloidosis from a Scandinavian perspective.  J Intern Med. 2003;  254 225-235
• 132 Shaz B H, Gordon F, Lewis W D. et al . Orthotopic liver transplantation for familial amyloidotic polyneuropathy: a pathological study.  Hum Pathol. 2000;  31 40-44
• 133 Jonsen E, Suhr O B, Tashima K. et al . Early liver transplantation is essential for familial amyloidotic polyneuropathy patients’ quality of life.  Amyloid. 2001;  8 52-57
• 134 Suhr O B, Wiklund U, Eleborg L. et al . Impact of autonomic neuropathy on circulatory instability during liver transplantation for familial amyloidotic polyneuropathy.  Transplantation. 1997;  63 675-679
• 135 Viana J da S, Bento C, Vieira H. et al . Haemodynamics during liver transplantation in familial amyloidotic polyneuropathy: study of the intraoperative cardiocirculatory data of 50 patients.  Rev Port Cardiol. 1999;  18 689-697
• 136 Lobato L, Ventura A, Beirao I. et al . End-stage renal disease in familial amyloidosis ATTR Val30Met: a definitive indication to combined liver-kidney transplantation.  Transplant Proc. 2003;  35 1116-1120
• 137 Ruygrok P N, Gane E J, McCall J L. et al . Combined heart and liver transplantation for familial amyloidosis.  Intern Med J. 2001;  31 66-67
• 138 Grazi G L, Cescon M, Salvi F. et al . Combined heart and liver transplantation for familial amyloidotic neuropathy: considerations from the hepatic point of view.  Liver Transpl. 2003;  9 986-992
• 139 Herlenius G, Larsson M, Ericzon B G. FAP World Transplant Register and domino/sequential register update.  Transplant Proc. 2001;  33 1367
• 140 Herlenius G, Larsson M, Ericzon B G. Results from the familial amyloidotic polyneuropathy world transplant registry.  Transplant Proc. 2001;  33 2454
• 141 Suhr O B, Ericzon B G, Friman S. Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type).  Liver Transpl. 2002;  8 787-794
• 142 Adams D, Samuel D, Goulon-Goeau C. et al . The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation.  Brain. 2000;  123 1495-1504
• 143 Holmgren G, Steen L, Ekstedt J. et al . Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30).  Clin Genet. 1991;  40 242-246
• 144 Schmidt H H, Nashan B, Pröpsting M J. et al . Familial Amyloidotic Polyneuropathy: domino liver transplantation.  J Hepatol. 1999;  30 293-298
• 145 Rydh A, Suhr O, Hietala S O. et al . Serum amyloid P component scintigraphy in familial amyloid polyneuropathy: regression of visceral amyloid following liver transplantation.  Eur J Nucl Med. 1998;  25 709-713
• 146 Holmgren G, Ericzon B G, Groth C G. et al . Clinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosis.  Lancet. 1993;  341 1113-1116
• 147 de Carvalho M, Conceicao I, Bentes C. et al . Long-term quantitative evaluation of liver transplantation in familial amyloid polyneuropathy (Portuguese V30M).  Amyloid. 2002;  9 126-133
• 148 Bergethon P R, Sabin T D, Lewis D. et al . Improvement in the polyneuropathy associated with familial amyloid polyneuropathy after liver transplantation.  Neurology. 1996;  47 944-951
• 149 Suhr O B, Wiklund U, Ando Y. et al . Impact of liver transplantation on autonomic neuropathy in familial amyloidotic polyneuropathy: an evaluation by spectral analysis of heart rate variability.  J Intern Med. 1997;  242 225-229
• 150 Lang K, Wikstrom L, Danielsson A. et al . Outcome of gastrointestinal complications after liver transplantation for familial amyloidotic polyneuropathy.  Scand J Gastroenterol. 2000;  35 985-989
• 151 Olofsson B O, Backman C, Karp K. et al . Progression of cardiomyopathy after liver transplantation in patients with familial amyloidotic polyneuropathy, Portuguese type.  Transplantation. 2002;  73 745-751
• 152 Pomfret E A, Lewis W D, Jenkins R L. et al . Effect of orthotopic liver transplantation on the progression of familial amyloidotic polyneuropathy.  Transplantation. 1998;  65 918-925
• 153 Stangou A J, Hawkins P N, Heaton N D. et al . Progressive cardiac amyloidosis following liver transplantation for familial amyloid polyneuropathy: implications for amyloid fibrillogenesis.  Transplantation. 1998;  66 229-233
• 154 Bittencourt P L, Couto C A, Leitao R M. et al . No evidence of de novo amyloidosis in recipients of domino liver transplantation: 12 to 40 (mean 24) month follow-up.  Amyloid. 2002;  9 194-196
• 155 Furtado A J. Domino liver transplantation using FAP grafts. HUC experience - hopes and realities.  Amyloid. 2003;  10 Suppl 1 84-87
• 156 Casas-Melley A T, Thomas P G, Krueger L J. et al . Domino as a bridge to definitive liver transplantation in a neonate.  Pediatr Transplant. 2002;  6 249-254
• 157 Donckier V, El N akadi I, Closset J. et al . Domino hepatic transplantation using the liver from a patient with primary hyperoxaluria.  Transplantation. 2001;  71 1346-1348
• 158 Popescu I, Simionescu M, Tulbure D. et al . Homozygous familial hypercholesterolemia: specific indication for domino liver transplantation.  Transplantation. 2003;  76 1345-1350
• 159 Merlini G, Bellotti V. Molecular mechanisms of amyloidosis.  N Engl J Med. 2003;  349 583-596
• 160 Gertz M A, Rajkumar S V. Primary systemic amyloidosis.  Curr Treat Options Oncol. 2002;  3 261-271
• 161 Perfetti V, Vignarelli M C, Casarini S. et al . Biological features of the clone involved in primary amyloidosis (AL).  Leukemia. 2001;  15 195-202
• 162 Nowak G, Westermark P, Wernerson A. et al . Liver transplantation as rescue treatment in a patient with primary AL kappa amyloidosis.  Transpl Int. 2000;  13 92-97
• 163 Sandberg-Gertzen H, Ericzon B G, Blomberg B. Primary amyloidosis with spontaneous splenic rupture, cholestasis, and liver failure treated with emergency liver transplantation.  Am J Gastroenterol. 1998;  93 2254-2256
• 164 Kumar K S, Lefkowitch J, Russo M W. et al . Successful sequential liver and stem cell transplantation for hepatic failure due to primary AL amyloidosis.  Gastroenterology. 2002;  122 2026-2031
• 165 Kobayashi T, Sato Y, Ichida T. et al . Primary amyloidosis with liver failure and acute renal failure treated with emergency living-related liver transplantation: a case report.  Transplant Proc. 2003;  35 356-357
• 166 Sharma P, Perri R E, Sirven J E. et al . Outcome of liver transplantation for familial amyloidotic polyneuropathy.  Liver Transpl. 2003;  9 1273-1280
• 167 Hawkins P N. Hereditary systemic amyloidosis with renal involvement.  J Nephrol. 2003;  16 443-448
• 168 Gillmore J D, Stangou A J, Tennent G A. et al . Clinical and biochemical outcome of hepatorenal transplantation for hereditary systemic amyloidosis associated with apolipoprotein AI Gly26Arg.  Transplantation. 2001;  71 986-992
• 169 Hamidi A sl L, Liepnieks J J, Uemichi T. et al . Renal amyloidosis with a frame shift mutation in fibrinogen A alpha-chain gene producing a novel amyloid protein.  Blood. 1997;  90 4799-4805
• 170 Zeldenrust S, Gertz M, Uemichi T. et al . Orthotopic liver transplantation for hereditary fibrinogen amyloidosis.  Transplantation. 2003;  75 560-561
• 171 Gillmore J D, Booth D R, Rela M. et al . Curative hepatorenal transplantation in systemic amyloidosis caused by the Glu526Val fibrinogen alpha-chain variant in an English family.  Q J Med. 2000;  93 269-275
• 172 Cochat P, Basmaison O. Current approaches to the management of primary hyperoxaluria.  Arch Dis Child. 2000;  82 470-473
• 173 Leumann E, Hoppe B. The primary hyperoxalurias.  J Am Soc Nephrol. 2001;  12 1986-1993
• 174 Pirulli D, Marangella M, Amoroso A. Primary hyperoxaluria: genotype-phenotype correlation.  J Nephrol. 2003;  16 297-309
• 175 Leumann E, Hoppe B. Pre-emptive liver transplantation in primary hyperoxaluria type 1: a controversial issue.  Pediatr Transplant. 2000;  4 161-164
• 176 Broyer M, Brunner F P, Brynger H. et al . Kidney transplantation in primary oxalosis: data from the EDTA Registry.  Nephrol Dial Transplant. 1990;  5 332-336
• 177 Jamieson N V. The results of combined liver/kidney transplantation for primary hyperoxaluria (PH1) 1984 - 1997. The European PH1 transplant registry report. European PH1 Transplantation Study Group.  J Nephrol. 1998;  11 (Suppl 1) 36-41
• 178 Nolkemper D, Kemper M J, Burdelski M. et al . Long-term results of pre-emptive liver transplantation in primary hyperoxaluria type 1.  Pediatr Transplant. 2000;  4 177-181
• 179 Shapiro R, Weismann I, Mandel H. et al . Primary hyperoxaluria type 1: improved outcome with timely liver transplantation: a single-center report of 36 children.  Transplantation. 2001;  72 428-432
• 180 Cochat P, Gaulier J M, Koch-Nogueira P C. et al . Combined liver-kidney transplantation in primary hyperoxaluria type 1.  Eur J Pediatr. 1999;  158 (Suppl 2) S75-80
• 181 Watts R W, Danpure C J, De Pauw L. et al . Combined liver-kidney and isolated liver transplantations for primary hyperoxaluria type 1: the European experience. The European Study Group on Transplantation in Hyperoxaluria Type 1.  Nephrol Dial Transplant. 1991;  6 502-511
• 182 Jamieson N V. The European Primary Hyperoxaluria Type 1 Transplant Registry report on the results of combined liver/kidney transplantation for primary hyperoxaluria 1984 - 1994. European PH1 Transplantation Study Group.  Nephrol Dial Transplant. 1995;  10 (Suppl 8) 33-37
• 183 Detry O, Honore P, DeRoover A. et al . Reversal of oxalosis cardiomyopathy after combined liver and kidney transplantation.  Transpl Int. 2002;  15 50-52
• 184 Fyfe B S, Israel D H, Quish A. et al . Reversal of primary hyperoxaluria cardiomyopathy after combined liver and renal transplantation.  Am J Cardiol. 1995;  75 210-212
• 185 Rodby R A, Tyszka T S, Williams J W. Reversal of cardiac dysfunction secondary to type 1 primary hyperoxaluria after combined liver-kidney transplantation.  Am J Med. 1991;  90 498-504
• 186 Toussaint C, De Pauw L, Vienne A. et al . Radiological and histological improvement of oxalate osteopathy after combined liver-kidney transplantation in primary hyperoxaluria type 1.  Am J Kidney Dis. 1993;  21 54-63
• 187 Toussaint C, Vienne A, De Pauw L. et al . Combined liver-kidney transplantation in primary hyperoxaluria type 1. Bone histopathology and oxalate body content.  Transplantation. 1995;  59 1700-1704
• 188 Bastani B, Mistry B M, Nahass G T. et al . Oxalate kinetics and reversal of the complications after orthotopic liver transplantation in a patient with primary hyperoxalosis type 1 awaiting renal transplantation.  Am J Nephrol. 1999;  19 64-69
• 189 Russo P A, Mitchell G A, Tanguay R M. Tyrosinemia: a review.  Pediatr Dev Pathol. 2001;  4 212-221
• 190 Holme E, Lindstedt S. Nontransplant treatment of tyrosinemia.  Clin Liver Dis. 2000;  4 805-814
• 191 Perez-Cerda C, Merinero B, Sanz P. et al . Liver transplantation in nine Spanish patients with tyrosinaemia type I.  J Inherit Metab Dis. 1995;  18 119-122
• 192 Wijburg F A, Reitsma W C, Slooff M J. et al . Liver transplantation in tyrosinaemia type I: the Groningen experience.  J Inherit Metab Dis. 1995;  18 115-118
• 193 Mohan N, McKiernan P, Preece M A. et al . Indications and outcome of liver transplantation in tyrosinaemia type 1.  Eur J Pediatr. 1999;  158 (Suppl 2) S49-54
• 194 Laine J, Salo M K, Krogerus L. et al . The nephropathy of type I tyrosinemia after liver transplantation.  Pediatr Res. 1995;  37 640-645
• 195 Jansen P L. Diagnosis and management of Crigler-Najjar syndrome.  Eur J Pediatr. 1999;  158 (Suppl 2) S89-94
• 196 van der Veere C N, Sinaasappel M, McDonagh A F. et al . Current therapy for Crigler-Najjar syndrome type 1: report of a world registry.  Hepatology. 1996;  24 311-315
• 197 Kaneko K, Takei Y, Aoki T. et al . Bilirubin adsorption therapy and subsequent liver transplantation cured severe bilirubin encephalopathy in a long-term survival patient with Crigler-Najjar disease type I.  Intern Med. 2000;  39 961-965
• 198 Whitington P F, Emond J C, Heffron T. et al . Orthotopic auxiliary liver transplantation for Crigler-Najjar syndrome type 1.  Lancet. 1993;  342 779-780
• 199 Rela M, Muiesan P, Vilca-Melendez H. et al . Auxiliary partial orthotopic liver transplantation for Crigler-Najjar syndrome type I.  Ann Surg. 1999;  229 565-569
• 200 Onaca N, Goldstein R M, Klintmalm G B. Xanthomas disappear rapidly after liver transplantation for familial hypercholesterolemia.  Liver Transpl. 2003;  9 1334-1335
• 201 Shrotri M, Fernando B S, Sudhindran S. et al . Long-term outcome of liver transplantation for familial hypercholesterolemia.  Transplant Proc. 2003;  35 381-382
• 202 Starzl T E, Bilheimer D W, Bahnson H T. et al . Heart-liver transplantation in a patient with familial hypercholesterolaemia.  Lancet. 1984;  1 1382-1383
• 203 Offstad J, Schrumpf E, Geiran O. et al . Plasma exchange and heart-liver transplantation in a patient with homozygous familial hypercholesterolemia.  Clin Transplant. 2001;  15 432-436
• 204 Levi D, Pefkarou A, Fort J A. et al . Liver transplantation for factor VII deficiency.  Transplantation. 2001;  72 1836-1837
• 205 Gordon F H, Mistry P K, Sabin C A. et al . Outcome of orthotopic liver transplantation in patients with haemophilia.  Gut. 1998;  42 744-749
• 206 Wilde J, Teixeira P, Bramhall S R. et al . Liver transplantation in haemophilia.  Br J Haematol. 2002;  117 952-956
• 207 Samuel D, Boboc B, Bernuau J. et al . Liver transplantation for protoporphyria. Evidence for the predominant role of the erythropoietic tissue in protoporphyrin overproduction.  Gastroenterology. 1988;  95 816-819
• 208 Bloomer J R, Rank J M, Payne W D. et al . Follow-up after liver transplantation for protoporphyric liver disease.  Liver Transpl Surg. 1996;  2 269-275
• 209 Meerman L, Haagsma E B, Gouw A S. et al . Long-term follow-up after liver transplantation for erythropoietic protoporphyria.  Eur J Gastroenterol Hepatol. 1999;  11 431-438
• 210 Dellon E S, Szczepiorkowski Z M, Dzik W H. et al . Treatment of recurrent allograft dysfunction with intravenous hematin after liver transplantation for erythropoietic protoporphyria.  Transplantation. 2002;  73 911-915
• 211 Do K D, Banner B F, Katz E. et al . Benefits of chronic plasmapheresis and intravenous heme-albumin in erythropoietic protoporphyria after orthotopic liver transplantation.  Transplantation. 2002;  73 469-472
• 212 Thunell S, Henrichson A, Floderus Y. et al . Liver transplantation in a boy with acute porphyria due to aminolaevulinate dehydratase deficiency.  Eur J Clin Chem Clin Biochem. 1992;  30 599-606
• 213 Burton B K. Urea cycle disorders.  Clin Liver Dis. 2000;  4 815-830
• 214 Heringlake S, Böker K, Manns M. Fatal clinical course of ornithine transcarbamylase deficiency in an adult heterozygous female patient.  Digestion. 1997;  58 83-86
• 215 Todo S, Starzl T E, Tzakis A. et al . Orthotopic liver transplantation for urea cycle enzyme deficiency.  Hepatology. 1992;  15 419-422
• 216 Santos S ilva E, Martins E, Cardoso M L. et al . Liver transplantation in a case of argininaemia.  J Inherit Metab Dis. 2001;  24 885-887
• 217 Saudubray J M, Touati G, Delonlay P. et al . Liver transplantation in urea cycle disorders.  Eur J Pediatr. 1999;  158 (Suppl 2) S55-59
• 218 Whitington P F, Alonso E M, Boyle J T. et al . Liver transplantation for the treatment of urea cycle disorders.  J Inherit Metab Dis. 1998;  21 (Suppl 1) 112-118
• 219 Ikeda S, Yazaki M, Takei Y. et al . Type II (adult onset) citrullinaemia: clinical pictures and the therapeutic effect of liver transplantation.  J Neurol Neurosurg Psychiatry. 2001;  71 663-670
• 220 Takashima Y, Koide M, Fukunaga H. et al . Recovery from marked altered consciousness in a patient with adult-onset type II citrullinemia diagnosed by DNA analysis and treated with a living related partial liver transplantation.  Intern Med. 2002;  41 555-560
• 221 Leonard J V, Walter J H, McKiernan P J. The management of organic acidaemias: the role of transplantation.  J Inherit Metab Dis. 2001;  24 309-311
• 222 van’t H off W, McKiernan P J, Surtees R A. et al . Liver transplantation for methylmalonic acidaemia.  Eur J Pediatr. 1999;  158 (Suppl 2) S70-74
• 223 Saudubray J M, Touati G, Delonlay P. et al . Liver transplantation in propionic acidaemia.  Eur J Pediatr. 1999;  158 (Suppl 2) S65-69
• 224 Wendel U, Saudubray J M, Bodner A. et al . Liver transplantation in maple syrup urine disease.  Eur J Pediatr. 1999;  158 (Suppl 2) S60-64
• 225 DuCerf C, Bancel B, Caillon P. et al . Orthotopic liver transplantation for type 1 Gaucher’s disease.  Transplantation. 1992;  53 1141-1143
• 226 Smanik E J, Tavill A S, Jacobs G H. et al . Orthotopic liver transplantation in two adults with Niemann-Pick and Gaucher’s diseases: implications for the treatment of inherited metabolic disease.  Hepatology. 1993;  17 42-49
• 227 Starzl T E, Demetris A J, Trucco M. et al . Chimerism after liver transplantation for type IV glycogen storage disease and type 1 Gaucher’s disease.  N Engl J Med. 1993;  328 745-749
• 228 Kale A S, Ferry G D, Hawkins E P. End-stage renal disease in a patient with cholesteryl ester storage disease following successful liver transplantation and cyclosporine immunosuppression.  J Pediatr Gastroenterol Nutr. 1995;  20 95-97
• 229 Daloze P, Delvin E E, Glorieux F H. et al . Replacement therapy for inherited enzyme deficiency: liver orthotopic transplantation in Niemann-Pick disease type A.  Am J Med Genet. 1977;  1 229-239
• 230 Sokal E M, Sokol R, Cormier V. et al . Liver transplantation in mitochondrial respiratory chain disorders.  Eur J Pediatr. 1999;  158 (Suppl 2) S81-84
• 231 Rake J P, van Spronsen F J, Visser G. et al . End-stage liver disease as the only consequence of a mitochondrial respiratory chain deficiency: no contra-indication for liver transplantation.  Eur J Pediatr. 2000;  159 523-526
• 232 Dubern B, Broue P, Dubuisson C. et al . Orthotopic liver transplantation for mitochondrial respiratory chain disorders: a study of 5 children.  Transplantation. 2001;  71 633-637
• 233 Gilli S C, Boin I F, Sergio L eonardi L. et al . Liver transplantation in a patient with S(beta)o-thalassemia.  Transplantation. 2002;  74 896-898
• 234 Olivieri N F, Liu P P, Sher G D. et al . Brief report: combined liver and heart transplantation for end-stage iron-induced organ failure in an adult with homozygous beta-thalassemia.  N Engl J Med. 1994;  330 1125-1127
• 235 Salviati L, Sacconi S, Mancuso M. et al . Mitochondrial DNA depletion and dGK gene mutations.  Ann Neurol. 2002;  52 311-317
• 236 Braegger C P, Belli D C, Mentha G. et al . Persistence of the intestinal defect in abetalipoproteinaemia after liver transplantation.  Eur J Pediatr. 1998;  157 576-578
• 237 Hadzic N, Pagliuca A, Rela M. et al . Correction of the hyper-IgM syndrome after liver and bone marrow transplantation.  N Engl J Med. 2000;  342 320-324
• 238 Broering D C, Sterneck M, Rogiers X. Living donor liver transplantation.  J Hepatol. 2003;  38 (Suppl 1) S119-135
• 239 Strassburg C P, Manns M P. Partielle Lebertransplantation und Lebendspende aus der Sicht des Internisten.  Internist. 2002;  43 1551-1558
• 240 Kobayashi S, Ochiai T, Hori S. et al . Copper metabolism after living donor liver transplantation for hepatic failure of Wilson’s disease from a gene mutated donor.  Hepatogastroenterology. 2001;  48 1259-1261
• 241 Komatsu H, Fujisawa T, Inui A. et al . Hepatic copper concentration in children undergoing living related liver transplantation due to Wilsonian fulminant hepatic failure.  Clin Transplant. 2002;  16 227-232
• 242 Kasahara M, Ohwada S, Takeichi T. et al . Living-related liver transplantation for type II citrullinemia using a graft from heterozygote donor.  Transplantation. 2001;  71 157-159
• 243 Broelsch C E, Frilling A, Testa G. et al . Living donor liver transplantation in adults.  Eur J Gastroenterol Hepatol. 2003;  15 3-6
• 244 Florman S, Shneider B. Living-related liver transplantation in inherited metabolic liver disease: feasibility and cautions.  J Pediatr Gastroenterol Nutr. 2001;  33 520-521
• 245 Fung J, Rao A, Starzl T. Clinical trials and projected future of liver xenotransplantation.  World J Surg. 1997;  21 956-961
• 246 Kanazawa A, Platt J L. Prospects for xenotransplantation of the liver.  Semin Liver Dis. 2000;  20 511-522
• 247 Petersen J, Ott M, von Weizsäcker F. Zelltherapie in der Hepatologie.  Z Gastroenterol. 2001;  39 975-980
• 248 Grompe M. Liver repopulation for the treatment of metabolic diseases.  J Inherit Metab Dis. 2001;  24 231-244
• 249 Fox I J, Chowdhury J R, Kaufman S S. et al . Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation.  N Engl J Med. 1998;  338 1422-1426
• 250 Muraca M, Gerunda G, Neri D. et al . Hepatocyte transplantation as a treatment for glycogen storage disease type 1 a.  Lancet. 2002;  359 317-318
• 251 Horslen S P, McCowan T C, Goertzen T C. et al . Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder.  Pediatrics. 2003;  111 1262-1267
• 252 Wilson J M. Adenovirus-mediated gene transfer to liver.  Adv Drug Deliv Rev. 2001;  46 205-209
• 253 Lee B, Goss J. Long-term correction of urea cycle disorders.  J Pediatr. 2001;  138 S62-71
• 254 Shiratori Y, Kanai F, Ohashi M. et al . Strategy of liver-directed gene therapy: present status and future prospects.  Liver. 1999;  19 265-274