Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis
Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis
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DOI：
10.1016/j.jpeds.2006.07.030
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发表时间：
2006-11-01
影响因子：
5.1
通讯作者：
Murray, Frederick T.
中科院分区：
文献类型：
--
作者：
Borowitz, Drucy;Goss, Christopher H.;Murray, Frederick T.
目标.我们研究了一种新的胰酶产品ALTU-135，一种由微生物来源的脂肪酶、蛋白酶和淀粉酶组成的专利制剂，以确定其治疗囊性纤维化（CF）患者胰腺功能不全（PI）的疗效和安全性。CF-PI的非卧床受试者（n = 117）在未接受胰酶替代治疗的住院患者中测定了基线脂肪和氮吸收系数（分别为CFA和CNA）。然后将受试者随机分配至含有5000（低）、25，000（中）或100，000（最高）单位脂肪酶（脂肪酶：蛋白酶：淀粉酶为1：1：0.15）的ALTU-135治疗28天。14天后，再次测量CFA和CNA。主要结果是治疗组间CFA和CNA较基线的变化。中剂量组和最高剂量组的治疗CFA显著高于低剂量组（分别为P = .0229和P = .0041）; CNA的结果相似。两个高剂量组中基线CFA 40%的受试者CFA平均分别增加31和8个百分点（P <0.0001）。ALTU-135在1个月的研究期间以25，000单位脂肪酶、25，000单位蛋白酶和3750单位淀粉酶的剂量有效。
Objectives. We studied a novel pancreatic enzyme product, ALTU-135, a proprietary formulation of microbially derived lipase, protease, and amylase, to determine its efficacy and safety in treatment of pancreatic insufficiency (PI) in patients with cystic fibrosis (CF).Study design. Ambulatory subjects with CF-PI (n = 117) had baseline coefficient of fat and nitrogen absorption (CFA and CNA, respectively) determined in an inpatient setting while not receiving pancreatic enzyme replacement therapy. Subjects were then randomized to treatment with ALTU-135 containing 5000 (low), 25,000 (mid), or 100,000 (highest) units of lipase (1:1:0.15 of lipase:protease:amylase) for 28 days. After 14 days, CFA and CNA were re-measured. The primary outcomes were change from baseline in CFA and CNA between treatments.Results. Treatment CFA was significantly greater in the mid and highest dose groups compared with that in the low dose group (P = .0229 and P = .0041, respectively); findings were similar for CNA. Subjects with baseline CFA 40% in the 2 higher dose groups had a mean increase of 31 and 8 percentage points in CFA, respectively (P < .0001).Conclusion. ALTU-135 was efficacious during the 1-month study period at the dose of 25,000 units of lipase, 25,000 units of protease, and 3750 units of amylase.