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. 2011 Sep;159(3):442-6.
doi: 10.1016/j.jpeds.2011.02.031. Epub 2011 Apr 9.

Natural history of hepatocellular adenoma formation in glycogen storage disease type I

David Q Wang  1 , Laurie M Fiske, Caroline T Carreras, David A Weinstein
Affiliations

Natural history of hepatocellular adenoma formation in glycogen storage disease type I

David Q Wang et al. J Pediatr. 2011 Sep.

Abstract

Objective: To characterize the natural history and factors related to hepatocellular adenoma (HCA) development in glycogen storage disease type Ia (GSD Ia).

Study design: Retrospective chart review was performed for 117 patients with GSD Ia. Kaplan-Meier analysis of HCA progression among two groups of patients with GSD Ia (5-year mean triglyceride concentration ≤ 500 mg/dL and >500 mg/dL); analysis of serum triglyceride concentration, body mass index SDS, and height SDS between cases at time of HCA diagnosis and age- and sex-matched control subjects.

Results: Logrank analysis of Kaplan-Meier survival curve demonstrated a significant difference in progression to HCA between the 5-year mean triglyceride groups (P = .008). No significant difference was detected in progression to adenoma event between sexes. Serum triglyceride concentration was significantly different at time of diagnosis of adenoma (737 ± 422 mg/dL) compared with control subjects (335 ± 195 mg/dL) (P = .009). Differences in height SDS (P = .051) and body mass index SDS (P = .066) approached significance in our case-control analysis.

Conclusion: Metabolic control may be related to HCA formation in patients with GSD Ia. Optimizing metabolic control remains critical, and further studies are warranted to understand the pathogenesis of adenoma development.

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Figures

Figure 1
Figure 1
Kaplan-Meier survival curve of adenoma-free progression in GSD Ia based on five-year mean triglyceride concentration.
Figure 2
Figure 2
Box-and-whisker plots comparing serum triglyceride concentration and body mass index between case and control groups. The central box represents the values from the lower to upper quartile (25 to 75 percentile). The middle line represents the median. The horizontal line extends from the minimum to the maximum value, excluding “outside” and “far out” values which are displayed as separate points. An outside value is defined as a value that is smaller than the lower quartile minus 1.5 times the interquartile range, or larger than the upper quartile plus 1.5 times the interquartile range. A far out value is defined as a value that is smaller than the lower quartile minus 3 times the interquartile range, or larger than the upper quartile plus 3 times the interquartile range.
Figure 3
Figure 3
Kaplan-Meier survival curve of adenoma-free progression in GSD types Ia and Ib.
See this image and copyright information in PMC

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