Glycogen storage disease type I natural history, complications and prognosis
|
Glycogen storage disease type I Microchapters |
|
Differentiating Glycogen storage disease type I from other Diseases |
|---|
|
Diagnosis |
|
Treatment |
|
Case Studies |
|
Glycogen storage disease type I natural history, complications and prognosis On the Web |
|
American Roentgen Ray Society Images of Glycogen storage disease type I natural history, complications and prognosis |
|
FDA on Glycogen storage disease type I natural history, complications and prognosis |
|
CDC on Glycogen storage disease type I natural history, complications and prognosis |
|
Glycogen storage disease type I natural history, complications and prognosis in the news |
|
Blogs on Glycogen storage disease type I natural history, complications and prognosis |
|
Directions to Hospitals Treating Glycogen storage disease type I |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Overview
Natural History
Without adequate metabolic treatment, patients with GSD I have died in infancy or childhood of overwhelming hypoglycemia and acidosis. Those who survived were stunted in physical growth and delayed in puberty because of chronically low insulin levels. Mental retardation from recurrent, severe hypoglycemia is considered preventable with appropriate treatment.
Complications
Hepatic complications have been serious in some patients. Adenomas of the liver can develop in the second decade or later, with a small chance of later malignant transformation to hepatoma or hepatic carcinomas (detectable by alpha-fetoprotein screening). Several children with advanced hepatic complications have improved after liver transplantation.
Additional problems reported in adolescents and adults with GSD I have included hyperuricemic gout, pancreatitis, and chronic renal failure. Despite hyperlipidemia, atherosclerotic complications have been infrequently reported.
Prognosis
With diagnosis before serious harm occurs, prompt reversal of acidotic episodes, and appropriate long-term treatment, most children will be healthy. With exceptions and qualifications, adult health and life span may also be fairly good, although lack of effective treatment before the mid-1970s has limited our long-term information.