Long‐term outcome after liver transplantation in children with type 1 glycogen storage disease

2020 ◽  
Author(s):  
Wing Yan Yuen ◽  
Seng Hock Quak ◽  
Marion M. Aw ◽  
Sivaramakrishnan Venkatesh Karthik
Keyword(s):  
Liver Transplantation ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Term Outcome ◽  
Long Term Outcome

Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia

1999 ◽  
Vol 22 (6) ◽  
pp. 723-732 ◽  
Author(s):  
L. Faivre ◽  
D. Houssin ◽  
J. Valayer ◽  
J. Brouard ◽  
M. Hadchouel ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Type Ia

The natural history of glycogen storage disease types VI and IX: Long-term outcome from the largest metabolic center in Canada

2014 ◽  
Vol 113 (3) ◽  
pp. 171-176 ◽  
Author(s):  
Anne Roscher ◽  
Jaina Patel ◽  
Stacy Hewson ◽  
Laura Nagy ◽  
Annette Feigenbaum ◽  
...  
Keyword(s):  
Natural History ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Term Outcome ◽  
Long Term Outcome ◽  
History Of

scholarly journals Modifiable factors affecting renal preservation in type I glycogen storage disease after liver transplantation: a single-center propensity-match cohort study

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Yi-Chia Chan ◽  
Kai-Min Liu ◽  
Chao-Long Chen ◽  
Aldwin D. Ong ◽  
Chih-Che Lin ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Renal Function ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Renal Outcome ◽  
Type I ◽  
Long Term Outcome ◽  
Renal Prognosis

Abstract Background and aims Glycogen storage disease type I (GSD-I) is an autosomal recessive disorder of carbohydrate metabolism, resulting in limited production of glucose and excessive glycogen storage in the liver and kidneys. These patients are characterized by life-threatening hypoglycemia, metabolic derangements, hepatomegaly, chronic kidney disease, and failure to thrive. Liver transplantation (LT) has been performed for poor metabolic control and delayed growth. However, renal outcome was diverse in pediatric GSD patients after LT. The aim of this study was to investigate the long-term outcome of renal function in pediatric GSD-I patients after living donor LT (LDLT), and to identify modifiable variables that potentially permits LT to confer native renal preservation. Methods The study included eight GSD-Ia and one GSD-Ib children with a median age of 9.0 (range 4.2–15.7) years at the time of LT. Using propensity score matching, 20 children with biliary atresia (BA) receiving LT were selected as the control group by matching for age, sex, pre-operative serum creatinine (SCr) and pediatric end-stage liver disease (PELD) score. Renal function was evaluated based on the SCr, estimated glomerular filtration rate (eGFR), microalbuminuria, and morphological changes in the kidneys. Comparability in long-term renal outcome in terms of anatomic and functional parameters will help to identify pre-LT factors of GSD-I that affect renal prognosis. Results The clinical and biochemical characteristics of the GSD and BA groups were similar, including immunosuppressive regimens and duration of follow-up (median 15 years) after LT. Overall, renal function, including eGFR and microalbuminuria was comparable in the GSD-I and BA groups (median eGFR: 111 vs. 123 ml/min/1.73m2, P = 0.268; median urine microalbuminuria to creatinine ratio: 16.0 vs. 7.2 mg/g, P = 0.099, respectively) after LT. However, in the subgroups of the GSD cohort, patients starting cornstarch therapy at an older age (≥ 6-year-old) before transplantation demonstrated a worse renal outcome in terms of eGFR change over years (P < 0.001). In addition, the enlarged kidney in GSD-I returned to within normal range after LT. Conclusions Post-LT renal function was well-preserved in most GSD-I patients. Early initiation of cornstarch therapy before preschool age, followed by LT, achieved a good renal prognosis.

Modifiable Factors Affecting Renal Preservation in Type I Glycogen Storage Disease After Liver Transplantation: A Single-center Propensity-match Cohort Study

2021 ◽  
Author(s):  
Yi-Chia Chan ◽  
Kai-Min Liu ◽  
Chao-Long Chen ◽  
Aldwin D. Ong ◽  
Chih-Che Lin ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Renal Function ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Renal Outcome ◽  
Control Group ◽  
Type I ◽  
Long Term Outcome ◽  
Modifiable Factors

Abstract Background and aimsGlycogen storage disease type I (GSD-I) is an autosomal recessive disorder of carbohydrate metabolism, resulting in limited production of glucose and excessive glycogen storage in the liver and kidneys. These patients are characterized by life-threatening hypoglycemia, metabolic derangements, hepatomegaly, chronic kidney disease, and failure to thrive. Liver transplantation (LT) has been performed for poor metabolic control, delayed growth, and complications including liver cirrhosis or failure. However, renal outcome was diverse in pediatric GSD patients after LT. The aim of this study was to investigate the long-term outcome of renal function in pediatric GSD-I patients after living donor LT (LDLT), and to identify modifiable variables that potentially permits LT to confer native renal preservation.Methods The study included eight GSD-Ia and one GSD-Ib children with a median age of 9.0 (range 4.2-15.7) years at the time of LT. Using propensity score matching, 20 children with biliary atresia (BA) receiving LT were selected as the control group by matching for age, sex, pre-operative creatinine (Cr) and pediatric end-stage liver disease (PELD) score. Renal function was evaluated based on the serum Cr, estimated glomerular filtration rate (eGFR), microalbuminuria, and morphological changes in the kidneys. Comparability in long-term renal outcome in terms of anatomic and functional parameters will help to identify pre-LT factors of GSD-I that affect renal prognosis.Results The clinical and biochemical characteristics of the GSD and BA groups were similar, including immunosuppressive regimens and duration of follow-up (median 15 years) after LT. Overall, renal function, including eGFR and microalbuminuria was comparable in the GSD-I and BA groups (median eGFR: 111 vs. 123 ml/min/1.73m2, P=0.268; median urine microalbuminuria to creatinine ratio: 16.0 vs 7.2 mg/g, P=0.099, respectively) after LT. However, in the subgroups of the GSD cohort, patients starting cornstarch therapy at an older age (≥ 6-year-old) before transplantation demonstrated a worse renal outcome in terms of eGFR change over years (P<0.001). In addition, the enlarged kidney in GSD-I returned to within normal range after LT.Conclusions Post-LT renal function was well-preserved in most GSD-I patients. Early initiation of cornstarch therapy before preschool age, followed by LT, achieved a good renal prognosis.

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