BACKGROUND: The clinical characteristics and outcomes of patients with glycogen storage disease (GSD) who undergo liver transplantation (LT) have not been well defined. In this study, our objective was to determine the outcome of LT in patients with GSD and compare it with a comparable group of patients without GSD (matched controls). METHODS: UNOS data from 1986 to 2007 were used for this study. For each GSD patient (n = 95; men 62%) who was transplanted, three patients (n = 285, men 60%) without GSD (case controls) matched for age ± five yr, year of transplantation and donor risk index (DRI) ± 0.2 were identified from the UNOS database in a random manner. Unadjusted patient survival was determined by Kaplan-Meier survival analysis and significance determined by log-rank test. RESULTS: The mean age of the group was 17.9 yr. GSD patients had lower BMI (22 vs. 24, p = 0.002), lower serum bilirubin (2.7 vs. 13.5 mg/dL, p < 0.0001), higher serum albumin (3.7 vs. 3.1 g/dL, p < 0.0001), and higher wait-list time (239 vs. 74 d, p < 0.0001) compared to case controls. Recipient age and DRI were similar between the groups. Tumors were more common in GSD group (13.7% vs. 5%). Patient survival was significantly better (p = 0.024) in GSD group at one, five, and 10 yr (82%, 76%, and 64%) than non-GSD (73%, 65%, and 59%) group. CONCLUSIONS: In this matched-control study, patients who underwent LT for GSD had a better long-term survival than a comparable group of patients without GSD.
BACKGROUND: The clinical characteristics and outcomes of patients with glycogen storage disease (GSD) who undergo liver transplantation (LT) have not been well defined. In this study, our objective was to determine the outcome of LT in patients with GSD and compare it with a comparable group of patients without GSD (matched controls). METHODS: UNOS data from 1986 to 2007 were used for this study. For each GSDpatient (n = 95; men 62%) who was transplanted, three patients (n = 285, men 60%) without GSD (case controls) matched for age ± five yr, year of transplantation and donor risk index (DRI) ± 0.2 were identified from the UNOS database in a random manner. Unadjusted patient survival was determined by Kaplan-Meier survival analysis and significance determined by log-rank test. RESULTS: The mean age of the group was 17.9 yr. GSDpatients had lower BMI (22 vs. 24, p = 0.002), lower serum bilirubin (2.7 vs. 13.5 mg/dL, p < 0.0001), higher serum albumin (3.7 vs. 3.1 g/dL, p < 0.0001), and higher wait-list time (239 vs. 74 d, p < 0.0001) compared to case controls. Recipient age and DRI were similar between the groups. Tumors were more common in GSD group (13.7% vs. 5%). Patient survival was significantly better (p = 0.024) in GSD group at one, five, and 10 yr (82%, 76%, and 64%) than non-GSD (73%, 65%, and 59%) group. CONCLUSIONS: In this matched-control study, patients who underwent LT for GSD had a better long-term survival than a comparable group of patients without GSD.
Authors: Sandra Sirrs; Fady Hannah-Shmouni; Stephen Nantel; James Neuberger; Eric M Yoshida Journal: J Inherit Metab Dis Date: 2018-02-01 Impact factor: 4.982