Abstract
Based on the hypothesis that autoimmunological factors coregulate the pathomechanism in biliary atresia (BA), adjuvant therapy with steroids has become routine, although its efficacy has never been proven. In 2010, a study on the advantages of budesonide compared to prednisolone in autoimmune hepatitis gave rise to experimental therapy using budesonide as an adjuvant BA treatment. Ninety-five BA patients prospectively received a budesonide 2 mg/dose rectal foam daily for three months (SG). A case-matched control group (CG: 81) was retrospectively recruited. The outcome measures were survival with native liver (SNL), determined at six months and two years after the Kasai procedure. The follow-up rate was 100%. At six months, SNL was statistically not different but became so after two years (SG: 54%; CG: 32%; p < 0.001). No steroid-related side effects were observed, except for eight patients with finally caught-up growth retardation. This study demonstrates for the first time a significantly longer survival with native liver in patients with BA after adjuvant therapy. However, indication, dosage, and duration of any budesonide application is not given in neonates with BA. Hence, we suggest extending the postoperative use of budesonide in a multicenter observational study with a clearly defined follow-up protocol, particularly in terms of potentially underestimated side effects.
Highlights
The most frequent indication of pediatric liver transplantation (LTx) is given in patients with biliary atresia (BA) within the course of an unfavorable outcome after the Kasai procedure [1]
Twenty-two newborns, which have been operated by the Kasai procedure during this period could not be considered because their parents did not agree to the experimental treatment with budesonide
Two parameters had a predictive value for all patients in terms of survival over all (SOA): the survival with native liver was worse in patients with the syndromic form of BA, but the jfSNL was higher in those patients who had been operated on between days 31 and 60 (Supporting Information Tables S1 and S2)
Summary
The most frequent indication of pediatric liver transplantation (LTx) is given in patients with biliary atresia (BA) within the course of an unfavorable outcome after the Kasai procedure [1]. After reviewing the first cases, we were already observing an increasing number of patients whose bilirubin turned normal, which encouraged us to pursue this attempt. This trend stabilized during the following years and, from an ethical point of view, we realized that we could not deprive future cases of this option. We continued with the protocol of rectally applied budesonide after KPE until passing the number of more than 120 patients We reviewed their data in the context of survival with a native liver and the need for liver transplantation at defined and reproducible reference dates
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