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

使用 CEREDASE 进行多剂量酶替代治疗戈谢病的效果

• 批准号: 6308037
• 负责人: HENRY J MANKIN
• 金额: $ 0.06万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-12-01 至 2000-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6308037
• 关键词: Gaucher's disease; bone disorder; clinical research; clinical trial phase I; dosage; enzyme therapy; glucosylceramidase; human subject; human therapy evaluation; macrophage; metabolism disorder chemotherapy; patient care management

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 density 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.

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。 补充钙三醇作为酶替代疗法的辅助治疗对无密度没有影响。 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。