Home treatment with intravenous enzyme replacement therapy for Gaucher
disease: an international collaborative study of 33 patients
A Zimran, CE Hollak, A Abrahamov, MH van Oers, M Kelly and E Beutler
Gaucher Clinic, Shaare-Zedek Medical Center, Jerusalem, Israel.
Intravenous enzyme replacement therapy (Alglucerase; Ceredase; Genzyme
Corp, Boston, MA) is an effective and safe treatment for patients with type
1 Gaucher disease. In an attempt to reduce its high cost, a "low- dose
high-frequency" protocol (30 U/kg/mo, 3 times a week) was introduced and
found to be as effective as the original high-dose protocol (60 U/kg every
2 weeks). Because receiving frequent infusions creates a burden for many
patients, we have implemented a program of home treatment for our patients.
We now report the safety and feasibility of low-dose/high-frequency home
intravenous enzyme- replacement therapy in 33 patients with Gaucher
disease. The chronic nature of the treatment, its safety, lack of adverse
effects, the stable condition of most patients, and the need to reduce the
high cost make enzyme replacement for Gaucher disease a good candidate for
intravenous home therapy.
Volume 82,
Issue 4,
pp. 1107-1109,
08/15/1993
Copyright © 1993 by The American Society of Hematology
