Skip to main content
NCBI home page
Archives of Disease in Childhood logoLink to Archives of Disease in Childhood
. 1994 Sep;71(3):181–183. doi: 10.1136/adc.71.3.181

Organ transplantation for inherited metabolic disease.

D A Kelly 1
PMCID: PMC1029967  PMID: 7979487

Abstract

Liver transplantation for inherited metabolic disease is a therapeutic reality in the 1990s. Careful patient selection and timing and choice of operative procedure should greatly improve the quality of life and long term survival for those children with life threatening metabolic disease. The rapid expansion of molecular genetics and the development of effective gene therapy may well displace liver transplantation as appropriate treatment of these disorders in the future.

Full text

PDF
181

Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Beath S. V., Brook G. D., Kelly D. A., Cash A. J., McMaster P., Mayer A. D., Buckels J. A. Successful liver transplantation in babies under 1 year. BMJ. 1993 Oct 2;307(6908):825–828. doi: 10.1136/bmj.307.6908.825. [DOI] [PMC free article] [PubMed] [Google Scholar]
  2. Burdelski M., Rodeck B., Latta A., Latta K., Brodehl J., Ringe B., Pichlmayr R. Treatment of inherited metabolic disorders by liver transplantation. J Inherit Metab Dis. 1991;14(4):604–618. doi: 10.1007/BF01797930. [DOI] [PubMed] [Google Scholar]
  3. Delorme M. A., Adams P. C., Grant D., Ghent C. N., Walker I. R., Wall W. J. Orthotopic liver transplantation in a patient with combined hemophilia A and B. Am J Hematol. 1990 Feb;33(2):136–138. doi: 10.1002/ajh.2830330211. [DOI] [PubMed] [Google Scholar]
  4. Herbert A., Corbin D., Williams A., Thompson D., Buckels J., Elias E. Erythropoietic protoporphyria: unusual skin and neurological problems after liver transplantation. Gastroenterology. 1991 Jun;100(6):1753–1757. doi: 10.1016/0016-5085(91)90680-j. [DOI] [PubMed] [Google Scholar]
  5. Hood J. M., Koep L. J., Peters R. L., Schröter G. P., Weil R., 3rd, Redeker A. G., Starzl T. E. Liver transplantation for advanced liver disease with alpha-1-antitrypsin deficiency. N Engl J Med. 1980 Jan 31;302(5):272–275. doi: 10.1056/NEJM198001313020505. [DOI] [PMC free article] [PubMed] [Google Scholar]
  6. Kaufman S. S., Wood R. P., Shaw B. W., Jr, Markin R. S., Rosenthal P., Gridelli B., Vanderhoof J. A. Orthotopic liver transplantation for type I Crigler-Najjar syndrome. Hepatology. 1986 Nov-Dec;6(6):1259–1262. doi: 10.1002/hep.1840060606. [DOI] [PubMed] [Google Scholar]
  7. Lindstedt S., Holme E., Lock E. A., Hjalmarson O., Strandvik B. Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase. Lancet. 1992 Oct 3;340(8823):813–817. doi: 10.1016/0140-6736(92)92685-9. [DOI] [PubMed] [Google Scholar]
  8. Lund D. P., Lillehei C. W., Kevy S., Perez-Atayde A., Maller E., Treacy S., Vacanti J. P. Liver transplantation in newborn liver failure: treatment for neonatal hemochromatosis. Transplant Proc. 1993 Feb;25(1 Pt 2):1068–1071. [PubMed] [Google Scholar]
  9. Macvicar D., Dicks-Mireaux C., Leonard J. V., Wight D. G. Hepatic imaging with computed tomography of chronic tyrosinaemia type 1. Br J Radiol. 1990 Aug;63(752):605–608. doi: 10.1259/0007-1285-63-752-605. [DOI] [PubMed] [Google Scholar]
  10. Manowski Z., Silver M. M., Roberts E. A., Superina R. A., Phillips M. J. Liver cell dysplasia and early liver transplantation in hereditary tyrosinemia. Mod Pathol. 1990 Nov;3(6):694–701. [PubMed] [Google Scholar]
  11. Mason A. L., Marsh W., Alpers D. H. Intractable neurological Wilson's disease treated with orthotopic liver transplantation. Dig Dis Sci. 1993 Sep;38(9):1746–1750. doi: 10.1007/BF01303186. [DOI] [PubMed] [Google Scholar]
  12. Mieles L. A., Esquivel C. O., Van Thiel D. H., Koneru B., Makowka L., Tzakis A. G., Starzl T. E. Liver transplantation for tyrosinemia. A review of 10 cases from the University of Pittsburgh. Dig Dis Sci. 1990 Jan;35(1):153–157. doi: 10.1007/BF01537237. [DOI] [PMC free article] [PubMed] [Google Scholar]
  13. Mieles L. A., Orenstein D., Teperman L., Podesta L., Koneru B., Starzl T. E. Liver transplantation in cystic fibrosis. Lancet. 1989 May 13;1(8646):1073–1073. doi: 10.1016/s0140-6736(89)92464-1. [DOI] [PubMed] [Google Scholar]
  14. Otto G., Herfarth C., Senninger N., Feist G., Post S., Gmelin K. Hepatic transplantation in galactosemia. Transplantation. 1989 May;47(5):902–903. doi: 10.1097/00007890-198905000-00033. [DOI] [PubMed] [Google Scholar]
  15. Paradis K., Weber A., Seidman E. G., Larochelle J., Garel L., Lenaerts C., Roy C. C. Liver transplantation for hereditary tyrosinemia: the Quebec experience. Am J Hum Genet. 1990 Aug;47(2):338–342. [PMC free article] [PubMed] [Google Scholar]
  16. Rela M., Heaton N. D., Vougas V., McEntee G., Gane E., Farhat B., Chiyende J., Mieli-Vergani G., Mowat A. P., Portmann B. Orthotopic liver transplantation for hepatic complications of Wilson's disease. Br J Surg. 1993 Jul;80(7):909–911. doi: 10.1002/bjs.1800800738. [DOI] [PubMed] [Google Scholar]
  17. Smanik E. J., Tavill A. S., Jacobs G. H., Schafer I. A., Farquhar L., Weber F. L., Jr, Mayes J. T., Schulak J. A., Petrelli M., Zirzow G. C. Orthotopic liver transplantation in two adults with Niemann-Pick and Gaucher's diseases: implications for the treatment of inherited metabolic disease. Hepatology. 1993 Jan;17(1):42–49. [PubMed] [Google Scholar]
  18. Song H. S., Ku W. C., Chen C. L. Disappearance of Kayser-Fleischer rings following liver transplantation. Transplant Proc. 1992 Aug;24(4):1483–1485. [PubMed] [Google Scholar]
  19. Soubrane O., Gauthier F., DeVictor D., Bernard O., Valayer J., Houssin D., Chapuis Y. Orthotopic liver transplantation for Byler disease. Transplantation. 1990 Nov;50(5):804–806. doi: 10.1097/00007890-199011000-00012. [DOI] [PubMed] [Google Scholar]
  20. Terpstra O. T. Auxiliary liver grafting: a new concept in liver transplantation. Lancet. 1993 Sep 25;342(8874):758–758. doi: 10.1016/0140-6736(93)91537-v. [DOI] [PubMed] [Google Scholar]
  21. Todo S., Starzl T. E., Tzakis A., Benkov K. J., Kalousek F., Saheki T., Tanikawa K., Fenton W. A. Orthotopic liver transplantation for urea cycle enzyme deficiency. Hepatology. 1992 Mar;15(3):419–422. doi: 10.1002/hep.1840150311. [DOI] [PMC free article] [PubMed] [Google Scholar]
  22. Watts R. W., Morgan S. H., Danpure C. J., Purkiss P., Calne R. Y., Rolles K., Baker L. R., Mansell M. A., Smith L. H., Merion R. M. Combined hepatic and renal transplantation in primary hyperoxaluria type I: clinical report of nine cases. Am J Med. 1991 Feb;90(2):179–188. [PubMed] [Google Scholar]
  23. Whitington P. F., Emond J. C., Heffron T., Thistlethwaite J. R. Orthotopic auxiliary liver transplantation for Crigler-Najjar syndrome type 1. Lancet. 1993 Sep 25;342(8874):779–780. doi: 10.1016/0140-6736(93)91543-u. [DOI] [PubMed] [Google Scholar]

Articles from Archives of Disease in Childhood are provided here courtesy of BMJ Publishing Group

RESOURCES