胰脂肪酶与安慰剂治疗临床外分泌性胰腺功能不全的囊性纤维化患者脂肪泻的疗效及耐受性比较。

A comparison of the efficacy and tolerance of pancrelipase and placebo in the treatment of steatorrhea in cystic fibrosis patients with clinical exocrine pancreatic insufficiency.

作者信息

Stern R C, Eisenberg J D, Wagener J S, Ahrens R, Rock M, doPico G, Orenstein D M

机构信息

Rainbow Babies & Children's Hospital, Cleveland, Ohio 44106, USA.

出版信息

Am J Gastroenterol. 2000 Aug;95(8):1932-8. doi: 10.1111/j.1572-0241.2000.02244.x.

DOI:10.1111/j.1572-0241.2000.02244.x

PMID:10950038

Abstract

OBJECTIVE

The safety and efficacy of Minimicrospheres, which are enteric-coated, delayed-release pancrelipase capsules, on fat absorption in pediatric/adolescent and adult cystic fibrosis (CF) patients was assessed. Exocrine pancreatic insufficiency, common in CF patients, causes steatorrhea due to insufficient release of pancreatic enzymes.

METHODS

In the open-label phase, 97 CF patients with pancreatic insufficiency and steatorrhea were stabilized on a high-fat diet and administered pancrelipase. Seventy-four patients with >80% coefficient of fat absorption received placebo or pancrelipase in the double-blind phase. Fat intake and excretion, stool frequency and consistency, and clinical global improvement were recorded.

RESULTS

Average daily fat intake was comparable between treatment groups within each age group (adults vs pediatric/adolescent), but placebo patients had a significant (p < 0.001) mean decrease in coefficient of fat absorption (adult, 36.9 percentage points; pediatric/adolescent, 34.9 percentage points) from open-label to double-blind treatment compared to pancrelipase patients (adult, 2 percentage points; pediatric/adolescent, 3.25 percentage points); this difference was caused by a greater (p < or = 0.001) increase in mean fecal fat excretion (grams per day) in the placebo groups compared to pancrelipase groups (adult: 61.9 vs 2.3; pediatric/adolescent: 45.4 vs 4.1). Change in mean stool frequency from open-label to double-blind phases was significantly different (p < or = 0.002) between treatment groups, with increases in placebo groups and no difference (adult) or decrease (pediatric/adolescent) in pancrelipase groups. Pancrelipase patients' stool consistency remained about the same from open-label to double-blind. Placebo patients' stool consistency decreased (became softer) from open-label pancrelipase to double-blind placebo. Clinical global improvement data showed that > or =83% of pancrelipase patients improved or remained unchanged.

CONCLUSIONS

Enteric-coated, delayed-release (Minimicrospheres) pancrelipase capsules are an effective treatment for steatorrhea associated with pancreatic insufficiency in patients with cystic fibrosis.

摘要

目的

评估肠溶包衣、缓释胰酶胶囊（微小球）对儿科/青少年及成年囊性纤维化（CF）患者脂肪吸收的安全性和有效性。外分泌性胰腺功能不全在CF患者中很常见，由于胰腺酶释放不足导致脂肪泻。

方法

在开放标签阶段，97例伴有胰腺功能不全和脂肪泻的CF患者采用高脂饮食并给予胰酶进行稳定治疗。在双盲阶段，74例脂肪吸收系数>80%的患者接受安慰剂或胰酶治疗。记录脂肪摄入与排泄、大便频率和性状以及临床整体改善情况。

结果

各年龄组（成人与儿科/青少年）内治疗组间平均每日脂肪摄入量相当，但与胰酶治疗患者相比（成人，2个百分点；儿科/青少年，3.25个百分点），安慰剂组患者从开放标签治疗到双盲治疗脂肪吸收系数平均显著下降（成人，36.9个百分点；儿科/青少年，34.9个百分点）（p<0.001）；与胰酶组相比（成人：61.9对2.3；儿科/青少年：45.4对4.1），这种差异是由安慰剂组平均粪便脂肪排泄量（克/天）更大幅度增加（p≤0.001）所致。治疗组间从开放标签阶段到双盲阶段平均大便频率变化有显著差异（p≤0.002），安慰剂组增加，胰酶组无差异（成人）或下降（儿科/青少年）。胰酶治疗患者从开放标签到双盲阶段大便性状基本保持不变。安慰剂组患者大便性状从开放标签胰酶治疗到双盲安慰剂治疗时变差（变稀）。临床整体改善数据显示，≥83%的胰酶治疗患者病情改善或维持不变。