Journal List > Korean J Pediatr Gastroenterol Nutr > v.6(2) > 1110369

Korean J Pediatr Gastroenterol Nutr. 2003 Sep;6(2):129-139. Korean.
Published online Sep 30, 2003.  https://doi.org/10.5223/kjpgn.2003.6.2.129
Copyright © 2003 The Korean Society of Pediatric Gastroenterology, Hepatology and Nutrition
Long-term Outcome of Glycogen Storage Disease Type 1; Analysis of Risk Factors for Hepatic Adenoma
Hye Ran Yang and Jeong Kee Seo
Department of Pediatrics, Seoul National University College of Medicine, Seoul, Korea.
Abstract

PURPOSE

The aim of the study was to evaluate the long-term outcome of glycogen storage disease (GSD) type 1 with particular reference to hepatic adenoma and hepatocellular carcinoma, and to analyze risk factors affecting the development of hepatic adenoma in GSD type 1.

METHODS

Forty-three GSD type 1 patients (31 males and 12 females, mean age 13.9±6.4 years) were analyzed retrospectively. Hepatic adenoma was detected on abdominal USG and diagnosed on histologic examination. Clinical profiles were compared between patients with hepatic adenoma (n=16) and age-matched controls without hepatic adenoma (n=16).

RESULTS

1) Of 43 GSD type 1 patients, 16 (37.2%) had hepatic adeoma. Hepatic adenoma was detected at the age of mean 14.2±4.1 years (range: 7.9~25.7 years). Fourteen (87.5%) adenomas were multiple at detection. 2) Comparison of the clinical profiles between adenoma group and non-adenoma group revealed that age at first introduction of uncooked cornstarch treatment was significantly late in adenoma group compared with non-adenoma group (9.1±5.2 years vs. 3.0±1.8 years, p=0.003). Portocaval shunt surgery was performed in 11 (68.8%) patients in adenoma group and 3 (18.8%) in non-adenoma group (p=0.004). Hepatic adenoma developed mean 5.8±4.2 years after shunt operation. 3) One patient was diagnosed as hepatocellular carcinoma at the age of 25.7 years.

CONCLUSION

Hepatic adenoma is an important late complication of GSD type 1 with the risk of malignant transformation. Early introduction of cornstarch therapy with strict metabolic control is needed to prevent the development of hepatic adenoma in GSD type 1.

Keywords: Glycogen storage disease type 1; Prognosis; Hepatic adenoma; Hepatocellular carcinoma