NEJM:依洛尼塞+舒林酸未改善家族性腺瘤性息肉病进展
创作:Lexi 审核:Lexi 2020年09月14日
  • 对171名家族性腺瘤性息肉病患者随机分配接受依洛尼塞、舒林酸或两药联合治疗;
  • 患者疾病进展率分别为32%(联合用药组)、38%(舒林酸组)和40%(依洛尼塞组);
  • 37例直肠切除术前患者中,疾病进展率分别为17%、46%和42%;
  • 34例结肠切除术后直肠或回肠袋息肉患者中,疾病进展率分别为36%、18%和42%;
  • 100例十二指肠息肉病患者中,疾病进展率分别为36%、41%和39%;
  • 三组患者不良反应和严重不良反应事件相似。
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Lexi
与单独使用依洛尼塞或舒林酸相比,两种药物联合治疗在延缓家族性腺瘤性息肉病患者病情进展方面的有效性和安全性尚不清楚。最新发表在New England Journal of Medicine的研究根据息肉负荷最高的解剖部位和手术情况对171名家族性腺瘤性息肉病患者进行分层,1:1:1随机分配接受依洛尼塞、舒林酸或两药联合治疗,并比较三组患者疾病进展和不良反应。该研究发现,与单独使用任一药物相比,联合使用依洛尼塞和舒林酸后患者疾病进展发生率并没有显著降低。
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Eflornithine plus Sulindac for Prevention of Progression in Familial Adenomatous Polyposis

依洛尼塞加舒林酸预防家族性腺瘤性息肉病的进展

10.1056/NEJMoa1916063

2020-09-10, Article

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BACKGROUND: The efficacy and safety of combination therapy with eflornithine and sulindac, as compared with either drug alone, in delaying disease progression in patients with familial adenomatous polyposis are unknown.
METHODS: We evaluated the efficacy and safety of the combination of eflornithine and sulindac, as compared with either drug alone, in adults with familial adenomatous polyposis. The patients were stratified on the basis of anatomical site with the highest polyp burden and surgical status; the strata were precolectomy (shortest projected time to disease progression), rectal or ileal pouch polyposis after colectomy (longest projected time), and duodenal polyposis (intermediate projected time). The patients were then randomly assigned in a 1:1:1 ratio to receive 750 mg of eflornithine, 150 mg of sulindac, or both once daily for up to 48 months. The primary end point, assessed in a time-to-event analysis, was disease progression, defined as a composite of major surgery, endoscopic excision of advanced adenomas, diagnosis of high-grade dysplasia in the rectum or pouch, or progression of duodenal disease.
RESULTS: A total of 171 patients underwent randomization. Disease progression occurred in 18 of 56 patients (32%) in the eflornithine–sulindac group, 22 of 58 (38%) in the sulindac group, and 23 of 57 (40%) in the eflornithine group, with a hazard ratio of 0.71 (95% confidence interval [CI], 0.39 to 1.32) for eflornithine–sulindac as compared with sulindac (P=0.29) and 0.66 (95% CI, 0.36 to 1.24) for eflornithine–sulindac as compared with eflornithine. Among 37 precolectomy patients, the corresponding values in the treatment groups were 2 of 12 patients (17%), 6 of 13 (46%), and 5 of 12 (42%) (hazard ratios, 0.30 [95% CI, 0.07 to 1.32] and 0.20 [95% CI, 0.03 to 1.32]); among 34 patients with rectal or ileal pouch polyposis, the values were 4 of 11 patients (36%), 2 of 11 (18%), and 5 of 12 (42%) (hazard ratios, 2.03 [95% CI, 0.43 to 9.62] and 0.84 [95% CI, 0.24 to 2.90]); and among 100 patients with duodenal polyposis, the values were 12 of 33 patients (36%), 14 of 34 (41%), and 13 of 33 (39%) (hazard ratios, 0.73 [95% CI, 0.34 to 1.52] and 0.76 [95% CI, 0.35 to 1.64]). Adverse and serious adverse events were similar across the treatment groups.
CONCLUSIONS: In this trial involving patients with familial adenomatous polyposis, the incidence of disease progression was not significantly lower with the combination of eflornithine and sulindac than with either drug alone.

First Authors:
Carol A Burke,Evelien Dekker,Patrick Lynch,N Jewel Samadder

Correspondence Authors:
Alfred Cohen

All Authors:
Carol A Burke,Evelien Dekker,Patrick Lynch,N Jewel Samadder,Francesc Balaguer,Robert Hüneburg,John Burn,Antoni Castells,Steven Gallinger,Ramona Lim,Elena M Stoffel,Samir Gupta,Alex Henderson,Frank G Kallenberg,Priyanka Kanth,Victorine H Roos,Gregory G Ginsberg,Frank A Sinicrope,Christian P Strassburg,Eric Van Cutsem,James Church,Fiona Lalloo,Field F Willingham,MPH,Paul E Wise,William M Grady,Molly Ford,Jennifer M Weiss,Robert Gryfe,Anil K Rustgi,Sapna Syngal,Alfred Cohen

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