Liver transplantation for Type IV glycogen storage disease

Article Abstract:

The body's two major storehouses of energy are fat and glycogen. While fat is a storehouse that may only be utilized slowly, glycogen is available for rapid conversion to glucose when needed. The molecular structure of glycogen is a mass of branching chains of glucose molecules linked with one another end-to-end. The liver and other tissues can use excess glucose to produce glycogen molecules as a reserve for future use. In patients with Type IV glycogen storage disease, however, the resulting glycogen molecules are not normal. Type IV glycogen storage disease, also called Andersen's disease or amylopectinosis, results from a deficiency in an enzyme that is responsible for building the branch points in the growing glucose chains. With fewer than normal branch points, the resulting molecule is insoluble and irritating. This form of glycogen is called amylopectin, due to its similarity to plant starch. Type IV glycogen storage disease has an autosomal recessive pattern of inheritance, and children with the disorder generally develop liver cirrhosis and die before the age of four years. In some patients, other organs develop symptoms of amylopectinosis; seizures, dementia, and myopathy may also develop. These patients generally die of heart failure. The authors present seven cases of Type IV glycogen storage disease which were treated with liver transplantation. Two patients died soon after the operation. The remaining five patients, however, have survived after periods ranging from 16 to 73 months. An unusual and unexpected finding in these cases was that the amylopectin deposits in tissues other than the liver improved after transplantation. It is to be expected that the donor liver, with its normal enzymes, would store normal glycogen. However, other tissues, such as the heart muscle, still have the defective enzyme. Nevertheless, biopsy specimens revealed a reduction in amylopectin deposits in muscle tissues after liver transplant. The reason for this improvement is not known, but the observation may have important implications for the treatment of other enzyme deficiency disorders. (Consumer Summary produced by Reliance Medical Information, Inc.)

Health aspects, Case studies, Surgery, Genetic aspects, Liver, Liver transplantation, Glycogenosis, Glycogen storage diseases, Metabolism, Inborn errors of, Inborn errors of metabolism

---

Transplantation milestones: viewed with one- and two-way paradigms of tolerance

Article Abstract:

A new paradigm of organ transplantation may make it more likely that organs will one day be transplanted without the need for drugs that suppress the immune system. From the 1960s through the early 1990s it was believed that only the patient's body mounted an immune response against a transplanted organ or bone marrow. Because of this it was assumed that a procedure known as host preconditioning was essential to successful organ transplantation. Host preconditioning involved inducing immune system tolerance to the transplanted organ by suppressing immune system functioning. However, in 1992 blood cells from donor organs were isolated in the bone marrow of some of the first liver and kidney recipients. Some of these patients had received their transplants 30 years earlier. A new paradigm of organ transplantation developed. The new paradigm recognized a bidirectional movement of cells between the donor organ and the patient. It now appears that suppression of the immune system is necessary for a time to protect the cells of both the transplanted organ and the cells of the patient. However, the immunologic confrontation appears to eventually become self-canceling.

---

Similar abstracts:

• Abstracts: Liver transplantation. Chimerism after liver transplantation for type IV glycogen storage disease and type I Gaucher's disease
• Abstracts: Estrogen replacement therapy: indications, contraindications, and agent selection. Monitoring skeletal response to estrogen
• Abstracts: Vulvar intraepithelial neoplasia and skin appendage involvement. Micropapillomatosis labialis appears unrelated to human papillomavirus
• Abstracts: Amiodarone in patients with previous drug-mediated torsade de pointes. Electrophysiologic mechanisms of the long QT interval syndromes and torsade de pointes
• Abstracts: Lupus anticoagulant: clinical significance in anticardiolipin positive patients with systemic lupus erythematosus

This website is not affiliated with document authors or copyright owners. This page is provided for informational purposes only. Unintentional errors are possible.