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直肠布地奈德:在葛西肝门肠吻合术后可能改变局面的因素。

Rectal budesonide: A potential game changer after Kasai hepatoportoenterostomy.

作者信息

Langreen Stefanie, Pauer Nathalie, Pfister Eva D, Junge Norman, Baumann Ulrich, Madadi-Sanjani Omid, Petersen Claus, Dingemann Jens, Schukfeh Nagoud

机构信息

Department of Pediatric and Adolescent Surgery, Hannover Medical School, Hannover, Germany.

Department of Kidney, Liver and Metabolic Disease, Division of Pediatric Gastroenterology and Hepatology, Hannover Medical School, Hannover, Germany.

出版信息

J Pediatr Gastroenterol Nutr. 2025 Sep;81(3):626-633. doi: 10.1002/jpn3.70147. Epub 2025 Jul 2.

Abstract

OBJECTIVES

Intravenous or oral steroid administration in patients with biliary atresia (BA) after Kasai hepatoportoenterostomy (HPE) is commonly practiced, however, the benefits remain controversial. Some studies suggest no positive effects while risking steroid associated side effects. Rectal application of glucocorticoids has so far only been assessed by our group with promising short-term results. We now aim to evaluate the impact of rectal budesonide administration on long-term native liver survival in patients undergoing HPE for BA.

METHODS

We performed a retrospective cohort study and included all patients after HPE who received rectal budesonide (2 mg) for 3 months from 2011 to 2022, compared to a historical control group without any glucocorticoid treatment. Jaundice-free native liver survival (jfNLS) was assessed at 6 months, 2 years, 5 years, and 10 years post-Kasai. Serious adverse effects of budesonide were documented.

RESULTS

Our analysis confirmed our previously published improvements in jfNLS at 6 months (53% vs. 39%) and 2 years (45% vs. 22%), while revealing sustained benefits at 5 years (40% vs. 23%) and 10 years (32% vs. 13%). However, these benefits were exclusive to patients with nonsyndromic BA. No serious budesonide-associated adverse side effects were found.

CONCLUSIONS

Our findings support the efficacy of rectal budesonide application in enhancing long-term outcomes, providing a safe therapeutic approach and improving jfNLS after HPE for BA, without severe adverse effects. Prospective randomized controlled trials are required to further evaluate its potential post-Kasai benefits and compare it to systemic glucocorticoid therapy.

摘要

目的

在Kasai肝门肠吻合术(HPE)后,对胆道闭锁(BA)患者进行静脉或口服类固醇给药是常见做法,然而,其益处仍存在争议。一些研究表明没有积极效果,同时存在类固醇相关副作用的风险。迄今为止,糖皮质激素的直肠应用仅由我们团队进行了评估,短期结果令人鼓舞。我们现在旨在评估直肠应用布地奈德对接受BA的HPE患者长期自体肝存活的影响。

方法

我们进行了一项回顾性队列研究,纳入了2011年至2022年期间所有在HPE后接受3个月直肠布地奈德(2毫克)治疗的患者,并与未接受任何糖皮质激素治疗的历史对照组进行比较。在Kasai术后6个月、2年、5年和10年评估无黄疸自体肝存活(jfNLS)情况。记录布地奈德的严重不良反应。

结果

我们的分析证实了我们之前发表的在6个月时(53%对39%)和2年时(45%对22%)jfNLS的改善,同时显示在5年时(40%对23%)和10年时(32%对13%)有持续益处。然而,这些益处仅适用于非综合征性BA患者。未发现与布地奈德相关的严重不良副作用。

结论

我们的研究结果支持直肠应用布地奈德在改善长期预后方面的疗效,为BA患者HPE后提供了一种安全的治疗方法并改善了jfNLS,且无严重不良反应。需要进行前瞻性随机对照试验以进一步评估其Kasai术后的潜在益处,并将其与全身糖皮质激素治疗进行比较。

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