Portal diversion for the treatment of glycogen storage disease in humans

Seven patients with Types I, III, or VI glycogen storage disease were treated with portal diversion from 5 1/2 mth to 9 1/2 yr ago. The first 2 patients had portacaval transposition with 1 early death. The last 5 patients had the technically safer procedure of end to side portacaval shunt without any deaths and with no late findings of hepatic encephalopathy. The convalescence of the patients with either kind of portal diversion was characterized by accelerated body growth and bone mineralization, incomplete relief of hypoglycemia and metabolic acidosis, striking amelioration of the hyperlipidemia of Type I disease, liver shrinkage in Types I and VI disease, and variable relief of such diverse other derangements as abnormal bleeding and elevated serum uric acid concentrations. The liver concentrations of glycogen were not affected by portal diversion. Hepatocytes were decreased in size in subsequent biopsies, thereby accounting for the reduction of liver size in most of the cases without a major alteration in glycogen. There was a high incidence of pre existing coincidental liver disease in patients, including transaminase elevations and hepatic fibrosis or even cirrhosis. These abnormalities were particularly striking in Type III patients but did not appear in any of the cases to be made worse by portal diversion. The observations in these 7 patients and in 6 more reported from other centers and followed up with personal examination indicate that portal diversion should have an important role in carefully selected cases of glycogen storage disease. Recent work was reviewed which suggests that the effects of portacaval shunt are due more to the rerouting of pancreatic hormones around the liver than to the bypassing of alimentary glucose.