Ultrase MT12 and Ultrase MT20 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis: safety and efficacy
Ultrase MT12 and Ultrase MT20 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis: safety and efficacy
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DOI：
10.1111/j.1365-2036.2004.02261.x
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发表时间：
2004-12-01
影响因子：
7.6
通讯作者：
Colin, P
中科院分区：
文献类型：
--
作者：
Konstan, MW;Stern, RC;Colin, P
背景：囊性纤维化引起胰腺外分泌功能不全，导致吸收不良。补充胰酶治疗可减轻囊性纤维化患者的营养不良。人们认识到，患者对不同品牌的酶的临床反应存在差异。这促使美国食品和药物管理局要求酶补充剂纳入新药申请。目的：探讨补充胰酶治疗囊性纤维化患者的安全性和有效性。方法：在两项单独的安全性和有效性研究中，我们比较了两种剂量的一种肠溶胰酶制剂：Ultrase MT12（每粒胶囊12000脂肪酶单位）和Ultrase MT20（每粒胶囊20000脂肪酶单位）与安慰剂。结果：各组平均总脂肪、蛋白质和碳水化合物摄入量无显著差异。酶治疗组在脂肪和蛋白质吸收方面均有显著差异。Ultrase MT12和Ultrase MT20组的平均脂肪和蛋白质吸收量分别为79.4%和83.8%，87.3%和88.6%。未报告与研究药物相关的不良事件。结论：本研究进一步支持使用酶治疗囊性纤维化患者胰腺功能不全。极好的脂肪和蛋白质吸收，最小的不良事件和安全剂量。
Background: Cystic fibrosis causes exocrine pancreatic insufficiency, leading to malabsorption. Supplemental pancreatic enzyme therapy alleviates the concomitant malnutrition experienced by cystic fibrosis patients. It is recognized that patients experience variations in clinical response to different brands of enzymes. This has prompted the US Food and Drug Administration to require that enzyme supplements be subjected to New Drug Applications.Aim: To investigate the safety and efficacy of supplemental pancreatic enzyme therapy in cystic fibrosis subjects.Methods: We compared two doses of one formulation of enteric-coated pancreatic enzymes: Ultrase MT12 (12 000 lipase units per capsule) and Ultrase MT20 (20 000 lipase units per capsule), to placebo in two separate safety and efficacy studies.Results: Mean total fat, protein and carbohydrate intake did not differ significantly between the groups. A significant difference in both fat and protein absorption occurred with the enzyme therapy groups. The Ultrase MT12 and Ultrase MT20 groups experienced a mean fat and protein absorption 79.4% and 83.8%, and 87.3% and 88.6%, respectively. No adverse events related to study drug were reported.Conclusions: This study further supports the use of enzymes to treat pancreatic insufficiency in cystic fibrosis. Excellent fat and protein absorption was achieved with minimal adverse events and safe doses.