Abstract
Even though significant progress has been achieved in the management of patients with glycogen storage disease type I, hepatic (mainly adenomas) and renal (proteinuria, renal failure) complications may still develop. Orthotopic liver transplantation has been reported in less than 20 patients, and, in most cases, its indications were multiple hepatic adenomas, sometimes combined with poor metabolic control and/or growth retardation. Even though short-term outcome seems to be favourable, long-term complications have been reported in several cases. Thus it appears that improved metabolic control has to be attempted before performing liver transplantation in such patients. As for renal transplantation, it has been performed in patients with terminal renal failure. It is hoped that improving longterm metabolic control will prevent renal involvement from evolving to terminal renal failure. Finally, combined liver and kidney transplantation may be indicated in a few patients.Conclusion: organ (liver/kidney) transplantation in glycogen storage disease type I may be advantageous when long-term metabolic control has been attempted. Nevertheless, post-transplantat longterm complications may still develop.
Similar content being viewed by others
Abbreviations
- GSD :
-
glycogen storage disease
- OLT :
-
orthotopic liver transplantation
References
Bianchi L (1993) Glycogen storage disease type Ia and hepatocellular tumors. Eur J Pediatr 152[Suppl 1]: S63-S70
Burdelski M, Nolkemper D, Ganschow R, Sturm E, Malago M, Rogiers X, Brolsch CE (1999) Liver transplantation in children: long-term outcome and quality of life. Eur J Pediatr 158[Suppl 2]: S34-S42.
Chen CL, Chen YS, Lin PP, Chiang YC, Cheng Yf, Huang TL, Eng HL (1997) Living related donor liver transplantation. J Gastroenterol Hepatol 12[Suppl]: 5342–5345
Chen YT (1991) Type I glycogen storage disease: kidney involvement, pathogenesis and its treatment. Pediatr Nephrol 5: 71–76.
Chen YT, Scheinman JI (1991) Hyperglycaemia associated with lactic acidaemia in a renal allograft recipient with type I glycogen storage disease. J Inherit Metab Dis 14: 80–86
Coire CI, Qizilbash AH, Castelli MF (1987) Hepatic adenomata in type I a glycogen storage disease. Arch Pathol Lab Med 111: 166–169
Emmett M, Narins RG (1978) Renal transplantation in type I glycogenosis. Failure to improve glucose metabolism. JAMA 239: 1642–1644.
Faivre L, Houssin D, Valayer J, Brouard J, Hadchouel M, Bernard O (1999) Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia. J Inherit Metab Dis 22: 723–732
Gerin I, Veiga-da-Cunha M, Achouri Y, Collet JF, Van Schaftingen E (1997) Sequence of a putative glucose-6-phosphate translocase mutated in glycogen storage disease type Ib. FEBS Lett 419: 235–238
Kay RM, Eckardt JJ, Goldstein LI, Busuttil RW (1994) Metastatic hepatocellular carcinoma to bone in a liver transplant patient. A case report. Clin Orthop 303: 237–241
Kirschner BS, Baker AL, Thorp FK (1991) Growth in adult-hood after liver transplantation for glycogen storage disease type I. Gastroenterology 101: 238–241
Koestinger A, Giller M, Chiolero R, Mosimann F, Tappy L (2000) Effect of liver transplantation on hepatic glucose metabolism in a patient with type I glycogen storage disease. Transplantation 69: 2205–2207
Labrune P, Trioche P, Duvaltier I, Chevalier P, Odièvre M (1997) Hepatocellular adenomas in glycogen storage disease type I and III: a series of 43 patients and review of the literature. J Pediatr Gastroenterol Nutr 24: 276–279
Lachaux A, Boillot O, Stamm D, Canterino I, Dumontet C, Regnier F, Floret D, Hermier M (1993) Treatment with lenograstim (glycosylated recombinant human granulocyte colony-stimulating factor) and orthotopic liver transplantation for glycogen storage disease type Ib. J Pediatr 123: 1005–1008
Lei KJ, Shelly LL, Pan CJ, Sidbury JB, Chou JY (1993) Mutations in the glucose-6-phosphatase gene that cause glycogen storage disease type Ia. Science 262: 580–583
Malatack JJ, Iwatsuki S, Gartner JC, Roe T, Finegold DN, Shaw BW, Zitelli BJ, Starzl TE (1983) Liver transplantation for type I glycogen storage disease. Lancet I: 1073–1075
Martinez-Ibanez V, Margarit C, Tormo R, Infante D, Iglesias J, Allende H, Lloret J, Jimenez A, Boix-Ochoa J (1987) Liver transplantation in metabolic diseases. Report of five pediatric cases. Transplant Proc 19: 3803–3804
Mattern D, Starzl TE, Arnaout W, Barnard J, Bynon JS, Dhawan A, Edmond J, Haagsma EB, Hug G, Lachaux A, Smit GPA, Chen YT (1999) Liver transplantation for glycogen storage disease types I, III and IV. Eur J Pediatr 158[Suppl 2]: S43-S48
Poe R, Snover DC (1988) Adenomas in glycogen storage disease type I. Two cases with unusual histologic features. am J Surg Pathol 12: 477–483
Reid CJD, Hebert D (1996) Acute renal failure complicating liver transplantation in twin sisters with glycogen storage disease type Ia. Transplant Proc 28: 3629–3631
Selby R, Starzl TE, Yunis E, Todo S, Tzakis AG, Brown BI, Kendall RS (1993) Liver transplantation for type I and type IV glycogen storage disease. Eur J Pediatr 152[Suppl 1]: S71-S76
Sokal EM, Lopez-Silvarrey A, Buts JP, Otte JB (1993) Orthotopic liver transplantation for type I glycogenosis unresponsive to medical therapy. J Pediatr Gastroenterol Nutr 16: 465–467
Tokunaga Y, Tanaka K, Uermoto S, Honnda K, Inamoto T, Yamaoka Y (1994) Living-related liver transplantation for inborn errors of metabolism. Transplant Proc 26: 2250–2251
Visser G, Rake JP, Fernandes J, Labrune P, Leonard JV, Moses S, Ullrich K, Smit GP (2000) Neutropenia, neutrophil dysfunction, and inflammatory bowel disease in glycogen storage disease type Ib: results of the European Study on Glycogen Storage Disease Type I. J Pediatr 137: 187–191
Author information
Authors and Affiliations
Additional information
Published online: 19 July 2002
Rights and permissions
About this article
Cite this article
Labrune, P. Glycogen storage disease type I: indications for liver and/or kidney transplantation. Eur J Pediatr 161, S53–S55 (2002). https://doi.org/10.1007/BF02679995
Published:
Issue Date:
DOI: https://doi.org/10.1007/BF02679995