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Stem cell transplantation for treating Gaucher disease

Gaucher disease is an inherited disorder caused by a deficiency of the enzyme glucocerebrosidase. This leads to storage of complex lipids in some types of blood cells. Due to these abnormal cells people with Gaucher disease will have pain, fatigue, anemia, jaundice and bone damage. Some forms of Gaucher disease may also cause neurological disorder. The treatment of Gaucher disease at present is mainly by enzyme replacement therapy which is expensive. In some severe cases stem cell transplantation is used to treat people with Gaucher disease. This is a high risk procedure some times leading to death of the individual. We have not found any trials to show the effectiveness and the risks of the procedure in people with Gaucher disease. Given there are no trials included in the review and we have not identified any relevant trials up to February 2012 we do not plan to update this review until new trials are published.

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

Treatment options for Gaucher disease

Gaucher disease, a rare disorder, is caused by inherited deficiency of the enzyme glucocerebrosidase. This defect leads to the build‐up of a fatty material called glucocerebroside in various cells in the body. Untreated individuals may suffer from anaemia, a decrease in platelet counts, massive enlargement of the liver and spleen, and damage to the bones. Two different types of treatment are available: the intravenous supplementation of the deficient protein glucocerebrosidase (enzyme replacement therapy), or the oral administration of a drug that slows down the production of the fatty material that it normally breaks down (substrate reduction therapy).

Cochrane Database of Systematic Reviews: Plain Language Summaries [Internet] - John Wiley & Sons, Ltd.

Version: 2015

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