Editorial: “real world data” of AIH—time to connect!

Autoimmune hepatitis (AIH) is a rare liver disease with a prevalence of approximately 16-18/100.000 in Europe.1 However, two separate recent studies from Denmark and Germany showed a significant rise of AIH incidence during the last decades,2, 3 which might also apply to other countries. The disease is basically characterised by a chronic immune-mediated inflammatory response against the patient's own liver. However, the phenotype is quite diverse, which can make diagnosis difficult.1 Untreated AIH can progress to cirrhosis with all its complications including liver transplantation and death.4 Goal of treatment is to achieve remission by normalisation of aminotransferases and immunoglobulin G, which can be achieved in 25%-80% of the patients in published different cohorts.4, 5 Treatment options are still limited, in particular for second-line therapies. Standard of care6 is treatment with predniso(lo)ne, alternatively budesonide in non-cirrhotics, with or without azathioprine.1, 4 However, management of AIH differs significantly even among expert centers.7 Therefore, the study by Dyson et  al, published in a recent issue is an important contribution to gain further insight into real-world management of AIH in particular since the study provides data not only from large academic centers.8 Interestingly, the reported remission rates in this study are lower than those reported in several publications. This might be either due to variations in expert knowledge or due to variations in patient characteristics. Furthermore, treatment options are limited, especially for second-line therapies of patients with prior treatment failure or intolerance. In detail, the study reports on a wide range of treatment regimes. The overall-use of steroids in the study was quite high, which is in contrast to present guidelines recommending to minimise the use of steroids in long-term therapy.1

So far there is no approved second-line therapy for AIH available; randomised prospective trials are missing.9 Mycophenolate seems to be an alternative option for patients intolerant to azathioprine.1 Calcineurin inhibitors (CNI) in combination with predniso(lo)ne also showed good results inducing clinical and biochemical remission.9 In contrast to other publications,10 the current study reports a better outcome with mycophenolate than with CNI-containing regimens. Furthermore, patients treated at transplant centers showed higher remission rates compared to patients treated in nontransplant centers. Although this was not associated with reduced cirrhosis rates, it highlights the need for specialist care for patients with rare liver diseases.

In summary, the current retrospective study by Dyson and colleagues contributes significantly on “real world management” of AIH. It emphasises the importance of adhering to current clinical practice guidelines and underlines the value of multicenter registries as well as the necessity of expert centers to collaborate at an international level and thus join forces in particular when studying rare diseases like AIH. The European Union (EU) sponsored European Reference Networks (ERNs) such as the one on rare liver disease (https://www.rare-liver.eu/) provide an excellent platform to study and develop new treatment options for rare and potentially live threatening diseases such as AIH.

FUNDING INFORMATION

None.