Rectal budesonide: A potential game changer after Kasai hepatoportoenterostomy.

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.