Individualised low-dose alglucerase therapy for type 1 Gaucher's disease

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From: The Lancet(Vol. 345, Issue 8963)
Publisher: Elsevier B.V.
Document Type: Article
Length: 3,629 words

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Abstract :

Individualized, low doses of alglucerase may be effective for patients with type 1 Gaucher's disease. Gaucher's disease is a disease characterized by the abnormal accumulation of lipids in the liver, spleen, lymph nodes, capillaries and bone marrow caused by an enzyme deficiency. It is associated with enlarged spleen, anemia, jaundice and bone lesions. Alglucerase is an enzyme supplementation therapy. Doctors started 25 patients with Gaucher's disease on a dose of alglucerase of 1.15 units per kilogram given three times per week and adjusted every six months based on the individual's response. After six months, 18 (72%) of the 25 patients had a moderate or good response to the drug. Sixteen patients with a moderate response and the one patient with a good response received a halved dose and one patient with a moderate response received a doubled dose because of other complications. After 12 months, six non-responding patients received double doses. At the end of 18 months, all patients showed improvement and most were on half doses.

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Gale Document Number: GALE|A17166466