LOW-DOSE ENZYME REPLACEMENT THERAPY FOR GAUCHERS-DISEASE - EFFECTS OF AGE, SEX, GENOTYPE, AND CLINICAL-FEATURES ON RESPONSE TO TREATMENT
LOW-DOSE ENZYME REPLACEMENT THERAPY FOR GAUCHERS-DISEASE - EFFECTS OF AGE, SEX, GENOTYPE, AND CLINICAL-FEATURES ON RESPONSE TO TREATMENT
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DOI：
10.1016/0002-9343(94)90042-6
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发表时间：
1994-07-01
影响因子：
5.9
通讯作者：
HOROWITZ, M
中科院分区：
文献类型：
--
作者：
ZIMRAN, A;ELSTEIN, D;HOROWITZ, M
尽管阿糖酶疗法已成为有症状的戈谢病患者的首选治疗方法，但作为降低高治疗费用的手段而引入的低剂量/高频治疗方案引起了重大争议。我们评估了 29 名戈谢病患者接受低剂量阿糖酶的疗效和安全性，这些患者完成了 6 至 28 个月的治疗。所有患者均接受静脉注射阿糖酶，每月剂量为 30 单位/公斤，通常每周 3 次等量给药。所有患者对治疗反应良好。血液学改善和器官肿大减少令人满意。年龄、性别、基因型、既往脾切除术或严重程度评分指数与治疗反应之间没有发现相关性。肝肿大程度较重的患者肝脏大小往往有更明显的减小，尽管这种减小并未达到统计学显着性。我们证实，低剂量/高频阿糖酶方案在治疗戈谢病方面与高剂量/低频方案一样有效，即使对于重症患者也是如此。当成本成为问题时，我们建议使用这种低剂量方案。
Although alglucerase therapy has become the treatment of choice for symptomatic patients with Gaucher's disease, the low-dose/high-frequency regimen introduced as a means to reduce the high cost of treatment has raised major controversy. We evaluated the efficacy and safety of low-dose alglucerase in 29 patients with Gaucher's disease who completed 6 to 28 months of therapy. All received intravenous alglucerase at a monthly dose of 30 units/kg, given usually in equal doses 3 times a week. All patients responded well to treatment. The hematological improvement and the reduction in organomegaly were satisfactory. No correlation was found between age, sex, genotype, previous splenectomy, or severity score index and the response to treatment. Patients with a greater degree of hepatomegaly tended to have a more pronounced decrease in liver size, although this reduction did not reach statistical significance. We confirmed that a low-dose/high-frequency regimen of alglucerase was as effective as a high-dose/low-frequency protocol in the treatment of Gaucher's disease, even in the severely ill. Whenever cost is an issue, we recommend using this low-dose regimen.