Giardiello F M, Hamilton S R, Krush A J, Piantadosi S, Hylind L M, Celano P, Booker S V, Robinson C R, Offerhaus G J
Department of Medicine, Johns Hopkins University School of Medicine and Hospital, Baltimore, MD.
N Engl J Med. 1993 May 6;328(18):1313-6. doi: 10.1056/NEJM199305063281805.
Familial adenomatous polyposis is an autosomal dominant disorder characterized by the formation of hundreds of colorectal adenomas and eventual colorectal cancer. Administration of the nonsteroidal antiinflammatory drug sulindac has been followed by regression of polyps in patients with this disorder, but no controlled trial of this drug in patients who have not had surgery has been reported.
We conducted a randomized, double-blind, placebo-controlled study of 22 patients with familial adenomatous polyposis, including 18 who had not undergone colectomy. The patients received sulindac at a dose of 150 mg orally twice a day for nine months or identical-appearing placebo tablets. The number and size of the polyps were evaluated every three months for one year.
A statistically significant decrease in the mean number of polyps and their mean diameter occurred in patients treated with sulindac, as compared with those given placebo. When treatment was stopped at nine months, the number of polyps had decreased to 44 percent of base-line values and the diameter of the polyps to 35 percent of base-line values (P = 0.014 and P < 0.001, respectively, for the comparison with the changes in the group given placebo). No patient had complete resolution of polyps. Three months after treatment with sulindac was stopped, both the number and the size of the polyps increased in sulindac-treated patients but remained significantly lower than the values at base line. No side effects from sulindac were noted.
Sulindac reduces the number and size of colorectal adenomas in patients with familial adenomatous polyposis, but its effect is incomplete, and it is unlikely to replace colectomy as primary therapy.
家族性腺瘤性息肉病是一种常染色体显性疾病，其特征为形成数百个结肠直肠腺瘤并最终发展为结肠直肠癌。给予非甾体抗炎药舒林酸后，患有这种疾病的患者息肉会消退，但尚未有关于该药物在未接受手术患者中的对照试验报道。
我们对22例家族性腺瘤性息肉病患者进行了一项随机、双盲、安慰剂对照研究，其中18例未接受结肠切除术。患者接受剂量为每日口服150毫克舒林酸，持续九个月，或服用外观相同的安慰剂片。在一年时间里，每三个月评估息肉的数量和大小。
与服用安慰剂的患者相比，接受舒林酸治疗的患者息肉平均数量及其平均直径出现了具有统计学意义的下降。当九个月时停止治疗，息肉数量降至基线值的44%，息肉直径降至基线值的35%（与服用安慰剂组的变化相比，P分别为0.014和P<0.001）。没有患者的息肉完全消退。停止舒林酸治疗三个月后，接受舒林酸治疗的患者息肉数量和大小均增加，但仍显著低于基线值。未观察到舒林酸的副作用。
舒林酸可减少家族性腺瘤性息肉病患者结肠直肠腺瘤的数量和大小，但效果不完全，不太可能替代结肠切除术作为主要治疗方法。
