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阿利卡福森,一种细胞间黏附分子-1的反义抑制剂,用于治疗左侧溃疡性结肠炎和溃疡性直肠炎。

Alicaforsen, an Antisense Inhibitor of Intercellular Adhesion Molecule-1, in the Treatment for Left-Sided Ulcerative Colitis and Ulcerative Proctitis.

作者信息

Greuter Thomas, Vavricka Stephan R, Biedermann Luc, Pilz Julia, Borovicka Jan, Seibold Frank, Sauter Bernhard, Rogler Gerhard

机构信息

Department of Gastroenterology and Hepatology, University Hospital Zurich, Zurich, Switzerland.

Division of Gastroenterology and Hepatology, Triemli Hospital Zurich, Zurich, Switzerland.

出版信息

Dig Dis. 2018;36(2):123-129. doi: 10.1159/000484979. Epub 2017 Dec 5.

DOI:10.1159/000484979
PMID:29207381
Abstract

BACKGROUND

Data on the efficacy of intercellular adhesion molecule-1 antisense oligonucleotide alicaforsen in ulcerative colitis (UC) is inconsistent.

METHODS

All patients, who had received at least one dose of alicaforsen, were analyzed retrospectively. Alicaforsen's efficacy was assessed in patients treated for left-sided UC and proctitis by comparing clinical and (if applicable) endoscopic disease activity before/after treatment.

RESULTS

Twelve patients were treated for left-sided UC or proctitis. Eleven patients received a 6-week course of a once-daily 240 mg alicaforsen enema formulation. In 1 patient, treatment was discontinued, because it was found to be inefficient. Disease activity measured by the partial Mayo score and 6-point symptom score was significantly reduced after treatment (6.0 vs. 2.4, p = 0.011 and 3.7 vs. 1.4, p = 0.008). Faecal calprotectin showed a trend towards reduction (484.4 vs. 179.5 μg/g, p = 0.063). Clinical improvement was achieved in 10 patients (83.3%). In 7 patients, a relapse occurred (70%). Median duration of clinical improvement was 18.0 weeks (range 1-112). Three patients showed an ongoing improvement of >9 months. No adverse events were reported.

CONCLUSIONS

A 6-week course of alicaforsen seemed to be safe and efficacious in inducing clinical improvement in patients with left-sided UC and proctitis. Prolonged clinical improvement was observed in many but not all patients.

摘要

背景

细胞间黏附分子-1反义寡核苷酸阿利卡福森治疗溃疡性结肠炎(UC)的疗效数据并不一致。

方法

对所有接受过至少一剂阿利卡福森治疗的患者进行回顾性分析。通过比较治疗前后左侧UC和直肠炎患者的临床及(如适用)内镜下疾病活动度,评估阿利卡福森的疗效。

结果

12例患者接受了左侧UC或直肠炎的治疗。11例患者接受了为期6周、每日一次240毫克阿利卡福森灌肠剂的疗程。1例患者因治疗无效而停药。治疗后,用部分梅奥评分和6分症状评分衡量的疾病活动度显著降低(6.0对2.4,p = 0.011;3.7对1.4,p = 0.008)。粪便钙卫蛋白呈下降趋势(484.4对179.5μg/g,p = 0.063)。10例患者(83.3%)实现了临床改善。7例患者复发(70%)。临床改善的中位持续时间为18.0周(范围1 - 112周)。3例患者持续改善超过9个月。未报告不良事件。

结论

为期6周的阿利卡福森疗程似乎对诱导左侧UC和直肠炎患者的临床改善安全有效。许多但并非所有患者都观察到了长期的临床改善。

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