Liver transplantation in glycogen storage disease type i

Orphanet Journal of Rare Diseases

Volumn 9, Issue 1, 2014, Pages

  Boers, Susanna J B ^a   Visser, Gepke ^a   Smit, Peter G P A ^b   Fuchs, Sabine A ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b UNIVERSITY OF GRONINGEN   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

AZATHIOPRINE; CYCLOSPORIN; STEROID; TACROLIMUS;

ACUTE GRAFT REJECTION; ACUTE KIDNEY FAILURE; ARTICLE; BRAIN DISEASE; CATCH UP GROWTH; CAUSE OF DEATH; CHRONIC GRAFT REJECTION; CHRONIC KIDNEY FAILURE; DIET RESTRICTION; DISEASE COURSE; DRUG WITHDRAWAL; FOLLOW UP; GLYCOGEN STORAGE DISEASE TYPE 1; HEPATIC ARTERY THROMBOSIS; HEPATITIS B; HUMAN; HYPERTENSION; IMMUNOSUPPRESSIVE TREATMENT; INSULIN DEPENDENT DIABETES MELLITUS; INTERPERSONAL COMMUNICATION; INVASIVE CANDIDIASIS; KIDNEY TRANSPLANTATION; LIVER CELL CARCINOMA; LIVER FUNCTION; LIVER GRAFT REJECTION; LIVER TRANSPLANTATION; LIVER VEIN OBSTRUCTION; METABOLIC REGULATION; PANCREATITIS; PHYSICIAN; PORTAL VEIN THROMBOSIS; SEPSIS; SUICIDE;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; FEMALE; GLYCOGEN STORAGE DISEASE TYPE I; HUMANS; LIVER TRANSPLANTATION; MALE; YOUNG ADULT;

EID: 84900865964     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-9-47     Document Type: Article

References (53)

1. Glycogen storage disease type I and G6Pase-beta deficiency: etiology and therapy. Chou JY, Jun HS, Mansfield BC, Nat Rev Endocrinol 2010 6 676 688 10.1038/nrendo.2010.189 20975743
2. Glucose-6-phosphatase deficiency. Froissart R, Piraud M, Boudjemline AM, Vianey-Saban C, Petit F, Hubert-Buron A, Eberschweiler PT, Gajdos V, Labrune P, Orphanet J Rare Dis 2011 6 27-1172-6-27
3. Mutations in the glucose-6-phosphatase gene that cause glycogen storage disease type 1a. Lei KJ, Shelly LL, Pan CJ, Sidbury JB, Chou JY, Science 1993 262 580 583 10.1126/science.8211187 8211187
4. Inactivation of the glucose 6-phosphate transporter causes glycogen storage disease type 1b. Hiraiwa H, Pan CJ, Lin B, Moses SW, Chou JY, J BiolChem 1999 274 5532 5536
5. Glycogen storage disease type I: diagnosis, management, clinical course and outcome. Results of the European Study on Glycogen Storage Disease Type I (ESGSD I). Rake JP, Visser G, Labrune P, Leonard JV, Ullrich K, Smit GP, Eur J Pediatr 2002 161 Suppl 1 20 S34 12373567
6. Liver transplantation for type I glycogen storage disease. Malatack JJ, Finegold DN, Iwatsuki S, Shaw BW Jr, Gartner JC, Zitelli BJ, Roe T, Starzl TE, Lancet 1983 1 1073 1075 6133106
7. Hepatic adenomata in type Ia glycogen storage disease. Coire CI, Qizilbash AH, Castelli MF, Arch Pathol Lab Med 1987 111 166 169 3545138
8. Liver transplantation in metabolic diseases. Report of five pediatric cases. Martinez Ibanez V, Margarit C, Tormo R, Infante D, Iglesias J, Allende H, Lloret J, Jimenez A, Boix-Ochoa J, Transplant Proc 1987 19 3803 3804 3313927
9. Adenomas in glycogen storage disease type 1. Two cases with unusual histologic features. Poe R, Snover DC, Am J Surg Pathol 1988 12 477 483 10.1097/00000478-198806000-00008 2454037
10. Growth in adulthood after liver transplantation for glycogen storage disease type I. Kirschner BS, Baker AL, Thorp FK, Gastroenterology 1991 101 238 241 2044912
11. Metastatic hepatocellular carcinoma to bone in a liver transplant patient. A case report. Kay RM, Eckardt JJ, Goldstein LI, Busuttil RW, Clin Orthop Relat Res 1994 303 237 241 8194240
12. Acute renal failure complicating liver transplantation in twin sisters with glycogen storage disease type Ia. Reid CJ, Hebert D, Transplant Proc 1996 28 3629 3631 8962400
13. Liver transplantation for glycogen storage disease types I, III, and IV. Matern D, Starzl TE, Arnaout W, Barnard J, Bynon JS, Dhawan A, Emond J, Haagsma EB, Hug G, Lachaux A, Smit GP, Chen YT, Eur J Pediatr 1999 158 Suppl 2 43 S48 10603098
14. Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia. Faivre L, Houssin D, Valayer J, Brouard J, Hadchouel M, Bernard O, J Inherit Metab Dis 1999 22 723 732 10.1023/A:1005544117285 10472532
15. Effect of liver transplantation on hepatic glucose metabolism in a patient with type I glycogen storage disease. Koestinger A, Gillet M, Chiolero R, Mosimann F, Tappy L, Transplantation 2000 69 2205 2207 10.1097/00007890- 200005270-00045 10852627
16. Glycogenosis storage type I diseases and evolutiveadenomatosis: an indication for liver transplantation. Lerut JP, Ciccarelli O, Sempoux C, Danse E, de Flandre J, Horsmans Y, Sokal E, Otte JB, TransplInt 2003 16 879 884
17. Simultaneous liver-kidney transplantation for glycogen storage disease type IA (von Gierke's disease). Panaro F, Andorno E, Basile G, Morelli N, Bottino G, Fontana I, Bertocchi M, DiDomenico S, Miggino M, Saltalamacchia L, Ghinolfi D, Bonifazio L, Jarzembowski TM, Valente U, Transplant Proc 2004 36 1483 1484 10.1016/j.transproceed.2004.05.070 15251364
18. Hepatocellular carcinoma in children and effect of living-donor liver transplantation on outcome. Arikan C, Kilic M, Nart D, Ozgenc F, Ozkan T, Tokat Y, Yagci RV, Aydogdu S, Pediatr Transplant 2006 10 42 47 10.1111/j.1399-3046. 2005.00395.x 16499586
19. Orthotopic liver transplantation in glucose-6-phosphatase deficiency-Von Gierke disease-with multiple hepatic adenomas and concomitant focal nodular hyperplasia. Carreiro G, Villela-Nogueira CA, Coelho H, Basto S, Pannain VL, Caroli-Bottino A, Ribeiro Filho J, J Pediatr Endocrinol Metab 2007 20 545 549 17550220
20. Liver transplantation in children with glycogen storage disease: controversies and evaluation of the risk/benefit of this procedure. Davis MK, Weinstein DA, Pediatr Transplant 2008 12 137 145 10.1111/j.1399-3046.2007.00803. x 18307661
21. Liver transplantation for glycogen storage disease type Ia. Reddy SK, Austin SL, Spencer-Manzon M, Koeberl DD, Clary BM, Desai DM, Smith AD, Kishnani PS, J Hepatol 2009 51 483 490 10.1016/j.jhep.2009.05.026 19596478
22. Glycogen Storage Disease type 1a-a secondary cause for hyperlipidemia: report of five cases. Carvalho PM, Silva NJ, Dias PG, Porto JF, Santos LC, Costa JM, J Diabetes Metab Disord 2013 12 25-6581-12-25
23. Orthotopic liver transplantation for type I glycogenosis unresponsive to medical therapy. Sokal EM, Lopez-Silvarrey A, Buts JP, Otte JB, J Pediatr Gastroenterol Nutr 1993 16 465 467 10.1097/00005176-199305000-00021 8315558
24. Glycogen storage disease type I: indications for liver and/or kidney transplantation. Labrune P, Eur J Pediatr 2002 161 Suppl 1 53 S55 12373572
25. Long-term results of living donor liver transplantation for glycogen storage disorders in children. Iyer SG, Chen CL, Wang CC, Wang SH, Concejero AM, Liu YW, Yang CH, Yong CC, Jawan B, Cheng YF, Eng HL, Liver Transpl 2007 13 848 852 10.1002/lt.21151 17539004
26. Combined liver-kidney transplantation in glycogen storage disease Ia: a case beyond the guidelines. Belingheri M, Ghio L, Sala A, Menni F, Trespidi L, Ferraresso M, Berardinelli L, Rossi G, Edefonti A, Parini R, Liver Transpl 2007 13 762 764 10.1002/lt.21147 17457869
27. Preemptive living donor liver transplantation in glycogen storage disease Ia: case report. Kaihara S, Ushigome H, Sakai K, Yoshizawa A, Nobori S, Suzuki T, Okamoto M, Ochiai T, Yoshimura N, Transplant Proc 2008 40 2815 2817 10.1016/j.transproceed.2008.07.026 18929868
28. Treatment with lenograstim (glycosylated recombinant human granulocyte colony-stimulating factor) and orthotopic liver transplantation for glycogen storage disease type Ib. Lachaux A, Boillot O, Stamm D, Canterino I, Dumontet C, Regnier F, Floret D, Hermier M, J Pediatr 1993 123 1005 1008 10.1016/S0022-3476(05)80403-2 7693904
29. Living related liver transplantation across ABO blood groups. Tanaka A, Tanaka K, Kitai T, Yanabu N, Tokuka A, Sato B, Mori S, Inomoto T, Shinohara H, Uemoto S, Tokunaga Y, Inomata Y, Yamaoka Y, Transplantation 1994 58 548 553 10.1097/00007890-199409150-00004 7522363
30. Liver transplantation for type Ibglycogenosis with reversal of cyclic neutropenia. Martinez-Olmos MA, López-Sanromán A, Martín-Vaquero P, Molina-Pérez E, Bárcena R, Vicente E, Candela A, Pallardo-Sánchez LF, Clin Nutr 2001 20 375 377 10.1054/clnu.2001.0432 11478837
31. Improved neutrophil function in a glycogen storage disease type 1b patient after liver transplantation. Adachi M, Shinkai M, Ohhama Y, Tachibana K, Kuratsuji T, Saji H, Maruya E, Eur J Pediatr 2004 163 202 206 10.1007/s00431-004-1405-1 14872340
32. The benefits of liver transplantation in glycogenosis type Ib. Bhattacharya N, Heaton N, Rela M, Walter JH, Lee PJ, J Inherit Metab Dis 2004 27 539 540 15334736
33. Successful staged kidney and liver transplantation for glycogen storage disease type Ib: a case report. Martin AP, Bartels M, Schreiber S, Buehrdel P, Hauss J, Fangmann J, Transplant Proc 2006 38 3615 3619 10.1016/j.transproceed. 2006.10.160 17175348
34. Glycemic management in living donor liver transplantation for patients with glycogen storage disease type 1b. Karaki C, Kasahara M, Sakamoto S, Shigeta T, Uchida H, Kanazawa H, Kakiuchi T, Fukuda A, Nakazawa A, Horikawa R, Suzuki Y, Pediatr Transplant 2012 16 465 470 10.1111/j.1399-3046.2012.01705.x 22574785
35. Living donor liver transplantation for glycogen storage disease type Ib. Kasahara M, Horikawa R, Sakamoto S, Shigeta T, Tanaka H, Fukuda A, Abe K, Yoshii K, Naiki Y, Kosaki R, Nakagawa A, Liver Transpl 2009 15 1867 1871 10.1002/lt.21929 19938129
36. Preemptive liver-kidney transplantation in von Gierke disease: a case report. Marega A, Fregonese C, Tulissi P, Vallone C, Gropuzzo M, Toniutto PL, Baccarani U, Bresadola F, Toso F, Montanaro D, Transplant Proc 2011 43 1196 1197 10.1016/j.transproceed.2011.03.003 21620087
37. Results of combined and sequential liver-kidney transplantation. Demirci G, Becker T, Nyibata M, Lueck R, Bektas H, Lehner F, Tusch G, Strassburg C, Schwarz A, Klempnauer J, Nashan B, Liver Transpl 2003 9 1067 1078 10.1053/jlts.2003.50210 14526402
38. Living related donor liver transplantation. Chen CL, Chen YS, Liu PP, Chiang YC, Cheng YF, Huang TL, Eng HL, J Gastroenterol Hepatol 1997 12 342 S345 10.1111/j.1440-1746.1997.tb00519.x 9407356
39. Outcome of living donor liver transplantation for glycogen storage disease. Liu PP, de Villa VH, Chen YS, Wang CC, Wang SH, Chiang YC, Jawan B, Cheung HK, Cheng YF, Huang TL, Eng HL, Chuang FR, Chen CL, Transplant Proc 2003 35 366 368 10.1016/S0041-1345(02)03951-9 12591443
40. Living donor liver transplantation for pediatric patients with inheritable metabolic disorders. Morioka D, Kasahara M, Takada Y, Corrales JP, Yoshizawa A, Sakamoto S, Taira K, Yoshitoshi EY, Egawa H, Shimada H, Tanaka K, Am J Transplant 2005 5 2754 2763 10.1111/j.1600-6143.2005.01084.x 16212637
41. Renal function in glycogen storage disease type I, natural course, and renopreservative effects of ACE inhibition. Martens DH, Rake JP, Navis G, Fidler V, van Dael CM, Smit GP, Clin J Am Soc Nephrol 2009 4 1741 1746 10.2215/CJN.00050109 19808227
42. Neutropenia in type Ib glycogen storage disease. Chou JY, Jun HS, Mansfield BC, Curr Opin Hematol 2010 17 36 42 10.1097/MOH.0b013e328331df85 19741523
43. Survival, but not maturation, is affected in neutrophil progenitors from GSD-1b patients. Visser G, de Jager W, Verhagen LP, Smit GP, Wijburg FA, Prakken BJ, Coffer PJ, Buitenhuis M, J Inherit Metab Dis 2012 35 287 300 10.1007/s10545-011-9379-4 21863279
44. Chimerism after liver transplantation for type IV glycogen storage disease and type 1 Gaucher's disease. Starzl TE, Demetris AJ, Trucco M, Ricordi C, Ildstad S, Terasaki PI, Murase N, Kendall RS, Kocova M, Rudert WA, Zeevi A, Van Thiel D, N Engl J Med 1993 328 745 749 10.1056/NEJM199303183281101 8437594
45. Human hepatocyte transplantation: worldwide results. Fisher RA, Strom SC, Transplantation 2006 82 441 449 16926585
46. Liver and liver cell transplantation for glycogen storage disease type IA. Muraca M, Burlina AB, Acta Gastroenterol Belg 2005 68 469 472 16433006
47. Human fetal liver-derived stem cell transplantation as supportive modality in the management of end-stage decompensated liver cirrhosis. Khan AA, Shaik MV, Parveen N, Rajendraprasad A, Aleem MA, Habeeb MA, Srinivas G, Raj TA, Tiwari SK, Kumaresan K, Venkateswarlu J, Pande G, Habibullah CM, Cell Transplant 2010 19 409 418 20447340
48. Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy. Yiu WH, Lee YM, Peng WT, Pan CJ, Mead PA, Mansfield BC, Chou JY, MolTher 2010 18 1076 1084
49. Glucose-6-phosphate transporter gene therapy corrects metabolic and myeloid abnormalities in glycogen storage disease type Ib mice. Yiu WH, Pan CJ, Allamarvdasht M, Kim SY, Chou JY, Gene Ther 2007 14 219 226 10.1038/sj.gt. 3302869 17006547
50. Induced pluripotent stem cells for modelling human diseases. Unternaehrer JJ, Daley GQ, Philos Trans R Soc Lond B Biol Sci 2011 366 2274 2285 10.1098/rstb.2011.0017 21727133
51. Treatment of newborn G6pc(-/-) mice with bone marrow-derived myelomonocytes induces liver repair. Resaz R, Emionite L, Vanni C, Astigiano S, Puppo M, Lavieri R, Segalerba D, Pezzolo A, Bosco MC, Oberto A, Eva C, Chou JY, Varesio L, Barbieri O, Eva A, J Hepatol 2011 55 1263 1271 10.1016/j.jhep.2011. 02.033 21703205
52. Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy. Lee YM, Jun HS, Pan CJ, Lin SR, Wilson LH, Mansfield BC, Chou JY, Hepatology 2012 56 1719 1729 10.1002/hep.25717 22422504
53. Hepatocellular carcinoma in glycogen storage disease type Ia: a case series. Franco LM, Krishnamurthy V, Bali D, Weinstein DA, Arn P, Clary B, Boney A, Sullivan J, Frush DP, Chen YT, Kishnani PS, J Inherit Metab Dis 2005 28 153 162 10.1007/s10545-005-7500-2 15877204