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Liver transplantation for glycogen storage disease types I, III, and IV.

Publication ,  Journal Article
Matern, D; Starzl, TE; Arnaout, W; Barnard, J; Bynon, JS; Dhawan, A; Emond, J; Haagsma, EB; Hug, G; Lachaux, A; Smit, GP; Chen, YT
Published in: Eur J Pediatr
December 1999

UNLABELLED: Glycogen storage disease (GSD) types I, III, and IV can be associated with severe liver disease. The possible development of hepatocellular carcinoma and/or hepatic failure make these GSDs potential candidates for liver transplantation. Early diagnosis and initiation of effective dietary therapy have dramatically improved the outcome of GSD type I by reducing the incidence of liver adenoma and renal insufficiency. Nine type I and 3 type III patients have received liver transplants because of poor metabolic control, multiple liver adenomas, or progressive liver failure. Metabolic abnormalities were corrected in all GSD type I and type III patients, while catch-up growth was reported only in two patients. Whether liver transplantation results in reversal and/or prevention of renal disease remains unclear. Neutropenia persisted in both GSDIb patients post liver transplantation necessitating continuous granulocyte colony stimulating factor treatment. Thirteen GSD type IV patients were liver transplanted because of progressive liver cirrhosis and failure. All but one patient have not had neuromuscular or cardiac complications during follow-up periods for as long as 13 years. Four have died within a week and 5 years after transplantation. Caution should be taken in selecting GSD type IV candidates for liver transplantation because of the variable phenotype, which may include life-limiting extrahepatic manifestations. It remains to be evaluated, whether a genotype-phenotype correlation exists for GSD type IV, which may aid in the decision making. CONCLUSION: Liver transplantation should be considered for patients with glycogen storage disease who have developed liver malignancy or hepatic failure, and for type IV patients with the classical and progressive hepatic form.

Duke Scholars

Published In

Eur J Pediatr

DOI

ISSN

0340-6199

Publication Date

December 1999

Volume

158 Suppl 2

Issue

Suppl 2

Start / End Page

S43 / S48

Location

Germany

Related Subject Headings

  • Prognosis
  • Pediatrics
  • Neutropenia
  • Male
  • Liver Transplantation
  • Liver Diseases
  • Humans
  • Glycogen Storage Disease Type IV
  • Glycogen Storage Disease Type III
  • Glycogen Storage Disease Type I
 

Citation

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Matern, D., Starzl, T. E., Arnaout, W., Barnard, J., Bynon, J. S., Dhawan, A., … Chen, Y. T. (1999). Liver transplantation for glycogen storage disease types I, III, and IV. Eur J Pediatr, 158 Suppl 2(Suppl 2), S43–S48. https://doi.org/10.1007/pl00014320
Matern, D., T. E. Starzl, W. Arnaout, J. Barnard, J. S. Bynon, A. Dhawan, J. Emond, et al. “Liver transplantation for glycogen storage disease types I, III, and IV.Eur J Pediatr 158 Suppl 2, no. Suppl 2 (December 1999): S43–48. https://doi.org/10.1007/pl00014320.
Matern D, Starzl TE, Arnaout W, Barnard J, Bynon JS, Dhawan A, et al. Liver transplantation for glycogen storage disease types I, III, and IV. Eur J Pediatr. 1999 Dec;158 Suppl 2(Suppl 2):S43–8.
Matern, D., et al. “Liver transplantation for glycogen storage disease types I, III, and IV.Eur J Pediatr, vol. 158 Suppl 2, no. Suppl 2, Dec. 1999, pp. S43–48. Pubmed, doi:10.1007/pl00014320.
Matern D, Starzl TE, Arnaout W, Barnard J, Bynon JS, Dhawan A, Emond J, Haagsma EB, Hug G, Lachaux A, Smit GP, Chen YT. Liver transplantation for glycogen storage disease types I, III, and IV. Eur J Pediatr. 1999 Dec;158 Suppl 2(Suppl 2):S43–S48.
Journal cover image

Published In

Eur J Pediatr

DOI

ISSN

0340-6199

Publication Date

December 1999

Volume

158 Suppl 2

Issue

Suppl 2

Start / End Page

S43 / S48

Location

Germany

Related Subject Headings

  • Prognosis
  • Pediatrics
  • Neutropenia
  • Male
  • Liver Transplantation
  • Liver Diseases
  • Humans
  • Glycogen Storage Disease Type IV
  • Glycogen Storage Disease Type III
  • Glycogen Storage Disease Type I