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

共有29名患者被纳入研究方案，并被随机分组。1组9例，2组11例，3组9例。研究人员注意到，在所有三组中，脂肪含量都显著增加。整体脂肪分数平均增加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60u /kg的剂量中实现的。在随后的一个月里，脂肪含量在30 u/kg时趋于稳定。作为酶替代疗法的辅助疗法，补充三醇对无乳症无影响。2年后的骨小梁骨密度测量和骨转换标记有待于29名参与研究的患者。初步研究结果表明，血液中的骨钙素和尿中的脱氧普林多林增加。支持增加骨转换。

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.