EFFECTS OF MULTIPLE DOSE ENZYME REPLACEMENT USING CEREDASE IN GAUCHER'S DISEASE

共有29名患者参加了这项研究，并被随机分组。第1组有9名患者，第2组有11名患者，第3组有9名患者。研究人员注意到，所有三组患者的脂肪比例都有显著增加。全球平均增加的脂肪比例为4.35±5.51(p=0.001)。所有增加都是在治疗的前6个月或每两周60u/kg剂量实现的。在接下来的一个月里，脂肪含量在30u/kg时趋于稳定。补充钙化三醇作为酶替代疗法的辅助治疗对所有患者均无影响。骨小梁密度测量和两年后骨转换的标记物正在这项研究中登记的二十九名患者中待定。初步研究结果显示，血中的骨钙素和尿中的脱氧普林多林有所增加。支持增加骨骼周转率。

A total of 29 patients have been enrolled in the study protocol and were randomized. Nine patients in group 1, eleven patients in group 2 and nine patients in group 3. The investigators noted a significant increase in fat fraction in all three groups. The mean global increase in fat fraction was 4.35 + 5.51 (p=0.001). All the increase was achieved in the first six months of therapy or 60 u/kg dose every two weeks. In one subsequent months, at 30 u/kg the fat fraction leveled off. Calitriol supplementation had no effect on none demsitry as an adjunct to enzyme replacement therapy. Trabecular bone density measurements and markers of bone turnover at 2 years are pending on the twentynine patients enrolled in the study. Preliminary findings suggest an increase in blood osterocalcin and urinary deoxyprindoline. Supportive of increased bone turnover.