van Keimpema Loes, Nevens Frederik, Vanslembrouck Ragna, van Oijen Martijn G H, Hoffmann Aswin L, Dekker Helena M, de Man Robert A, Drenth Joost P H
Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands.
Gastroenterology. 2009 Nov;137(5):1661-8.e1-2. doi: 10.1053/j.gastro.2009.07.052. Epub 2009 Jul 29.
BACKGROUND & AIMS: Therapy for polycystic liver is invasive, expensive, and has disappointing long-term results. Treatment with somatostatin analogues slowed kidney growth in patients with polycystic kidney disease (PKD) and reduced liver and kidney volume in a PKD rodent model. We evaluated the effects of lanreotide, a somatostatin analogue, in patients with polycystic liver because of autosomal-dominant (AD) PKD or autosomal-dominant polycystic liver disease (PCLD).
We performed a randomized, double-blind, placebo-controlled trial in 2 tertiary referral centers. Patients with polycystic liver (n = 54) were randomly assigned to groups given lanreotide (120 mg) or placebo, administered every 28 days for 24 weeks. The primary end point was the difference in total liver volume, measured by computerized tomography at weeks 0 and 24. Analyses were performed on an intention-to-treat basis.
Baseline characteristics were comparable for both groups, except that more patients with ADPKD were assigned to the placebo group (P = .03). The mean liver volume decreased 2.9%, from 4606 mL (95% confidence interval (CI): 547-8665) to 4471 mL (95% CI: 542-8401 mL), in patients given lanreotide. In the placebo group, the mean liver volume increased 1.6%, from 4689 mL (95% CI: 613-8765 mL) to 4895 mL (95% CI: 739-9053 mL) (P < .01). Post hoc stratification for patients with ADPKD or PCLD revealed similar changes in liver volume, with statistically significant differences in patients given lanreotide (P < .01 for both diseases).
In patients with polycystic liver, 6 months of treatment with lanreotide reduces liver volume.
多囊肝的治疗具有侵入性、费用高昂且长期效果令人失望。生长抑素类似物治疗可减缓多囊肾病（PKD）患者的肾脏生长，并在PKD啮齿动物模型中减少肝脏和肾脏体积。我们评估了生长抑素类似物兰瑞肽对常染色体显性（AD）PKD或常染色体显性多囊肝病（PCLD）所致多囊肝患者的影响。
我们在2个三级转诊中心进行了一项随机、双盲、安慰剂对照试验。54例多囊肝患者被随机分为接受兰瑞肽（120mg）或安慰剂治疗的组，每28天给药1次，共24周。主要终点是在第0周和第24周通过计算机断层扫描测量的肝脏总体积差异。分析采用意向性分析。
两组的基线特征具有可比性，只是更多ADPKD患者被分配到安慰剂组（P = 0.03）。接受兰瑞肽治疗的患者肝脏平均体积下降了2.9%，从4606mL（95%置信区间（CI）：547 - 8665）降至4471mL（95%CI：542 - 8401mL）。在安慰剂组中，肝脏平均体积增加了1.6%，从4689mL（95%CI：613 - 8765mL）增至4895mL（95%CI：739 - 9053mL）（P < 0.01）。对ADPKD或PCLD患者进行事后分层显示肝脏体积有类似变化，接受兰瑞肽治疗的患者有统计学显著差异（两种疾病均P < 0.01）。
在多囊肝患者中，6个月的兰瑞肽治疗可减少肝脏体积。
