Glycogen storage disease type I surgery
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Anmol Pitliya, M.B.B.S. M.D.[2]
Overview
Liver transplantation is the final treatment for patients with metabolic disease associated associated with GSD type 1. Indications for liver transplantation include patients with multifocal lesions, growing lesions that do not regress with improved dietary regimens, and patients who do not have evidence of distant metastatic disease. There is resolution of metabolic derangements (correction of hypoglycemia, lactic acidosis, hyperuricemia, and hyperlipidemia) after liver transplantation in patients with GSD type 1.
Indications
Indications for liver transplantation include:[1]
- Patients with multifocal lesions
- Growing lesions that do not regress with improved dietary regimens
- Patients who do not have evidence of distant metastatic disease
Surgery
Liver transplantation
- Liver transplantation may improve the survival of patients with glycogen storage disease type 1.
- Survival rates of patients after liver transplantation are:[2]
- 1 year survival rate of 82%
- 5 year survival rate of 76%
- 10 year survival rate of 64%
- There is resolution of metabolic derangements (correction of hypoglycemia, lactic acidosis, hyperuricemia, and hyperlipidemia) after liver transplantation in patients with GSD type 1.[3][4][5]
- "Model for end-stage liver disease” (MELD) score is used to govern priority for liver transplantation.[6]
- The score range between 6 and 40 and is calculated using a logarithmic assessment of three objective and reproducible variables including:
- Total serum bilirubin
- Creatinine concentrations
- International normalized ratio
- As hepatic abnormalities in GSD type 1 are due to single-gene and cell-autonomous defect, the recurrence of primary liver disease in the transplanted allograft in not possible.
References
- ↑ Kishnani, Priya S.; Austin, Stephanie L.; Abdenur, Jose E.; Arn, Pamela; Bali, Deeksha S.; Boney, Anne; Chung, Wendy K.; Dagli, Aditi I.; Dale, David; Koeberl, Dwight; Somers, Michael J.; Burns Wechsler, Stephanie; Weinstein, David A.; Wolfsdorf, Joseph I.; Watson, Michael S. (2014). "Diagnosis and management of glycogen storage disease type I: a practice guideline of the American College of Medical Genetics and Genomics". Genetics in Medicine. doi:10.1038/gim.2014.128. ISSN 1098-3600.
- ↑ Maheshwari A, Rankin R, Segev DL, Thuluvath PJ (2012). "Outcomes of liver transplantation for glycogen storage disease: a matched-control study and a review of literature". Clin Transplant. 26 (3): 432–6. doi:10.1111/j.1399-0012.2011.01549.x. PMID 22066793.
- ↑ O'Leary JG, Lepe R, Davis GL (2008). "Indications for liver transplantation". Gastroenterology. 134 (6): 1764–76. doi:10.1053/j.gastro.2008.02.028. PMID 18471553.
- ↑ 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 (6): 723–32. PMID 10472532.
- ↑ Iyer SG, Chen CL, Wang CC, Wang SH, Concejero AM, Liu YW; et al. (2007). "Long-term results of living donor liver transplantation for glycogen storage disorders in children". Liver Transpl. 13 (6): 848–52. doi:10.1002/lt.21151. PMID 17539004.
- ↑ Wiesner R, Edwards E, Freeman R, Harper A, Kim R, Kamath P; et al. (2003). "Model for end-stage liver disease (MELD) and allocation of donor livers". Gastroenterology. 124 (1): 91–6. doi:10.1053/gast.2003.50016. PMID 12512033.