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.