Grabowski G A, Barton N W, Pastores G, Dambrosia J M, Banerjee T K, McKee M A, Parker C, Schiffmann R, Hill S C, Brady R O
Children's Hospital Medical Center, Cincinnati, Ohio.
Ann Intern Med. 1995 Jan 1;122(1):33-9. doi: 10.7326/0003-4819-122-1-199501010-00005.
To compare the efficacy of mannose-terminated glucocerbrosidase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Cambridge, Massachusetts) and recombinant (imiglucerase; Cerezyme, Genzyme Corp.) sources in treating type 1 Gaucher disease.
Double-blind, randomized, parallel trial.
University medical center and clinical research hospital.
15 patients (4 children and 11 adults) randomly assigned to receive Ceredase and 15 patients (3 children and 12 adults) assigned to receive Cerezyme.
Ceredase and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight.
Hemoglobin levels, platelet counts, and serum acid phosphatase and angiotensin-converting enzyme activities were monitored every 2 weeks during the trial. Hepatic and splenic volumes were assessed at the time of randomization and after 6 and 9 months of enzyme infusion. Formation of IgG antibodies to Ceredase or Cerezyme was monitored every 3 months by radioimmunoprecipitation assay.
No significant differences were found in the rate or extent of improvement in hemoglobin levels, platelet counts, serum acid phosphatase or angiotensin-converting enzyme activities, or hepatic or splenic volumes between either treatment group. The incidence of IgG antibody formation was greater in the Ceredase group (40%) than in the Cerezyme group (20%). No major immunologic adverse events occurred in either group.
Our study shows the therapeutic similarity of Ceredase and Cerezyme. Cerezyme has the advantage of being theoretically unlimited in supply and free of potential pathogenic contaminants.
比较天然来源（阿糖苷酶；思而赞，健赞公司，马萨诸塞州剑桥）和重组来源（伊米苷酶；思而停，健赞公司）的甘露糖末端葡萄糖脑苷脂酶治疗1型戈谢病的疗效。
双盲、随机、平行试验。
大学医学中心和临床研究医院。
15例患者（4名儿童和11名成人）随机分配接受思而赞，15例患者（3名儿童和12名成人）分配接受思而停。
思而赞和思而停每2周输注1次，共9个月，剂量为60 U/kg体重。
在试验期间每2周监测血红蛋白水平、血小板计数、血清酸性磷酸酶和血管紧张素转换酶活性。在随机分组时以及酶输注6个月和9个月后评估肝脏和脾脏体积。每3个月通过放射免疫沉淀试验监测针对思而赞或思而停的IgG抗体形成情况。
两个治疗组在血红蛋白水平、血小板计数、血清酸性磷酸酶或血管紧张素转换酶活性的改善速率或程度，或肝脏或脾脏体积方面均未发现显著差异。思而赞组IgG抗体形成的发生率（40%）高于思而停组（20%）。两组均未发生重大免疫不良事件。
我们的研究表明思而赞和思而停在治疗上具有相似性。思而停具有理论上供应无限且无潜在致病污染物的优势。
