Zimran A, Hollak C E, Abrahamov A, van Oers M H, Kelly M, Beutler E
Gaucher Clinic, Shaare-Zedek Medical Center, Jerusalem, Israel.
Blood. 1993 Aug 15;82(4):1107-9.
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.
静脉内酶替代疗法（阿糖苷酶；思而赞；健赞公司，马萨诸塞州波士顿）是1型戈谢病患者的一种有效且安全的治疗方法。为降低其高昂成本，引入了一种“低剂量高频”方案（30 U/kg/月，每周3次），并发现该方案与最初的高剂量方案（每2周60 U/kg）效果相同。由于频繁输注给许多患者带来负担，我们已为患者实施了家庭治疗计划。我们现报告33例戈谢病患者低剂量/高频家庭静脉内酶替代疗法的安全性和可行性。该治疗的慢性性质、安全性、无不良反应、大多数患者状况稳定以及降低高昂成本的需求，使得戈谢病的酶替代疗法成为静脉内家庭治疗的良好候选方法。
