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

共有29例患者入组研究方案并接受随机化。 第1组9例患者，第2组11例患者，第3组9例患者。 研究人员注意到所有三组的脂肪分数都显著增加。 脂肪分数的平均总体增加为4.35 ± 5.51（p=0.001）。 所有的增加都是在治疗的前六个月或每两周60 u/kg剂量实现的。 在随后的一个月里，在30 u/kg的脂肪分数趋于稳定。 补充卡立醇作为酶替代疗法的辅助治疗对非糖尿病患者没有影响。对于入组研究的29例患者，2年时的骨小梁密度测量和骨转换标志物尚未确定。初步结果表明血骨钙素和尿脱氧吲哚啉增加。 支持骨转换增加。

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.