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Enzyme replacement therapy for Gaucher disease
E Beutler, A Kay, A Saven, P Garver, D Thurston, A Dawson and B Rosenbloom
Department of Molecular and Experimental Medicine, Research Institute of
Scripps Clinic, La Jolla, CA 92037.
Four patients with moderately severe type I Gaucher disease were treated
with commercially available mannose terminated glucocerebrosidase
(Ceredase; Genzyme, Boston, MA) for up to 13 months. The enzyme was
administered at the rate of three to four times weekly at one fourth the
total recommended dosage, greatly decreasing the cost. Marked regression of
hepatomegaly and improvement in liver function tests, peripheral blood
counts, and serum angiotensin- converting enzyme levels were documented.
The two patients with pulmonary involvement manifested improvement in
pulmonary function tests. Skeletal disease remained unchanged.
Volume 78,
Issue 5,
pp. 1183-1189,
09/01/1991
Copyright © 1991 by The American Society of Hematology
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