Familial adenomatous polyposis (FAP) is a hereditary disease characterized by the development of numerous colorectal adenomas in young adults. Metformin, an oral diabetic drug, has been shown to have antineoplastic effects and a favorable safety profile. We performed a randomized, double-blind, controlled trial to evaluate the efficacy of metformin on the regression of colorectal and duodenal adenoma in patients with FAP. Thirty-four FAP patients were randomly assigned in a 1:2:2 ratio to receive placebo, 500 mg metformin, or 1,500 mg metformin per day orally for 7 months. The number and size of polyps and the global polyp burden were evaluated before and after the intervention. This study was terminated early based on the results of the interim analysis. No significant differences were determined in the percentage change of colorectal and duodenal polyp number over the course of treatment among the three treatment arms ( P = 0.627 and P = 1.000, respectively). We found no significant differences in the percentage change of colorectal or duodenal polyp size among the three groups ( P = 0.214 and P = 0.803, respectively). The overall polyp burdens of the colorectum and duodenum were not significantly changed by metformin treatment at either dosage. Colon polyps removed from the metformin-treated patients showed significantly lower mTOR signal (p-S6) expression than those from patients in the placebo arm. In conclusion, 7 months of treatment with 500 mg or 1,500 mg metformin did not reduce the mean number or size of polyps in the colorectum or duodenum in FAP patients (ClinicalTrials.gov ID: [NCT01725490][1]). Prevention Relevance: A 7-month metformin treatment (500 mg or 1,500 mg) did not reduce the number or size of polyps in the colorectum or duodenum of FAP patients as compared to placebo. These results do not support the use of metformin to promote regression of intestinal adenomas in FAP patients. [1]: /lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT01725490&atom=%2Fcanprevres%2F14%2F5%2F563.atom

中文翻译：

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二甲双胍治疗家族性腺瘤性息肉病患者腺瘤的疗效

家族性腺瘤性息肉病 (FAP) 是一种遗传性疾病，其特征是在年轻人中出现大量结直肠腺瘤。二甲双胍是一种口服糖尿病药物，已被证明具有抗肿瘤作用和良好的安全性。我们进行了一项随机、双盲、对照试验，以评估二甲双胍对 FAP 患者结直肠和十二指肠腺瘤消退的疗效。34 名 FAP 患者以 1:2:2 的比例随机分配接受安慰剂、500 mg 二甲双胍或每天口服 1,500 mg 二甲双胍治疗 7 个月。在干预前后评估息肉的数量和大小以及整体息肉负担。根据中期分析结果，本研究提前终止。三个治疗组在治疗过程中结直肠和十二指肠息肉数量的百分比变化没有显着差异（分别为 P = 0.627 和 P = 1.000）。我们发现三组之间结直肠或十二指肠息肉大小的百分比变化没有显着差异（分别为 P = 0.214 和 P = 0.803）。两种剂量的二甲双胍治疗均未显着改变结直肠和十二指肠的总体息肉负担。从接受二甲双胍治疗的患者身上切除的结肠息肉显示出比安慰剂组患者显着降低的 mTOR 信号 (p-S6) 表达。总之，使用 500 mg 或 1,500 mg 二甲双胍治疗 7 个月并未减少 FAP 患者结直肠或十二指肠息肉的平均数量或大小（ClinicalTrials.gov ID：[NCT01725490][1]）。预防相关性：与安慰剂相比，7 个月的二甲双胍治疗（500 毫克或 1,500 毫克）并未减少 FAP 患者结直肠或十二指肠中息肉的数量或大小。这些结果不支持使用二甲双胍来促进 FAP 患者肠腺瘤的消退。[1]：/lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT01725490&atom=%2Fcanprevres%2F14%2F5%2F563.atom