Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, The Netherlands.
Gastroenterology. 2013 Aug;145(2):357-65.e1-2. doi: 10.1053/j.gastro.2013.04.055. Epub 2013 May 7.
BACKGROUND & AIMS: Clinical trials have shown that in patients with polycystic liver disease (PLD), short-term treatment with somatostatin analogues (SAs) reduces liver volumes by 4.5%-5.9%, compared with placebo. However, the effects of SA therapy vary among individuals. We collected data from individual patients with PLD to identify subgroups that benefit most from SA therapy.
We analyzed data from 107 patients with PLD from 3 randomized placebo-controlled trials (67 received SAs, 52 received placebo). We used multiple linear regression analysis to determine the effects of SAs based on patients' age, sex, baseline liver volume, and diagnosis (autosomal dominant polycystic liver or kidney disease). The primary outcome was change in liver volume after 6-12 months of treatment.
The effects of SA therapy did not differ significantly among patients with different diagnoses or baseline liver volumes; the overall difference in liver volume between groups receiving SAs therapy vs placebo was 5.3% (P < .001). Among subjects given placebo, young women (48 years old or younger) had the greatest increase in polycystic liver volume (4.8%; 95% confidence interval: 2.2%-7.4%), and mean liver volumes did not increase in older women and men. Women 48 years old or younger had a greater response to therapy (a reduction in liver volume of 8.0% compared with placebo; P < .001) than older women (a reduction in liver volume of 4.1% compared with placebo; P = .022).
Based on a pooled analysis of data from individual patients with PLD, treatment with somatostatin analogues is equally effective for patients with autosomal dominant polycystic kidney disease or polycystic liver disease; efficacy does not depend on size of the polycystic liver. Young female patients appear to have the greatest benefit from 6-12 months of SA therapy, which might avert the progressive course of the disease in this specific group.
临床试验表明，在多囊性肝病（PLD）患者中，与安慰剂相比，短期使用生长抑素类似物（SAs）可使肝体积减少 4.5%-5.9%。然而，SA 治疗的效果因人而异。我们收集了 PLD 患者的个体数据，以确定从 SA 治疗中获益最大的亚组。
我们分析了来自 3 项随机安慰剂对照试验的 107 例 PLD 患者的数据（67 例接受 SAs，52 例接受安慰剂）。我们使用多元线性回归分析，根据患者的年龄、性别、基线肝体积和诊断（常染色体显性多囊肾病或多囊肝病）来确定 SAs 的作用。主要结局是治疗 6-12 个月后肝体积的变化。
不同诊断或基线肝体积的患者之间，SA 治疗的效果无显著差异；接受 SAs 治疗与安慰剂组之间的肝体积总体差异为 5.3%（P<.001）。在接受安慰剂的患者中，年轻女性（48 岁或以下）多囊性肝体积增加最多（4.8%；95%置信区间：2.2%-7.4%），而年长女性和男性的肝体积没有增加。年轻女性（与安慰剂相比，肝体积减少 8.0%；P<.001）比年长女性（与安慰剂相比，肝体积减少 4.1%；P=.022）对治疗的反应更大。
基于对个体 PLD 患者数据的汇总分析，生长抑素类似物治疗对常染色体显性多囊肾病或多囊肝病患者同样有效；疗效不取决于多囊肝的大小。年轻女性患者似乎从 6-12 个月的 SA 治疗中获益最大，这可能会阻止该特定人群疾病的进展。
