Standard treatment overtaken by second choice Study leads to change in treatment guideline for liver disease

The standard treatment for the liver disease autoimmune hepatitis is insufficiently effective and has many side effects. Researchers at Radboud university medical center show that a drug that is normally used as a second-line treatment is much more effective and safer. The treatment guideline will be updated based on the results of this study.

Patients with autoimmune hepatitis have an inflamed liver. As a result, they have to take medications that suppress the immune system during their whole lifetime. The standard treatment for this rare disease consists of two drugs, corticosteroids and azathioprine. However, this combination is not always effective, leading to worsening of the disease. In addition, a significant number of patients stop taking azathioprine due to side effects. That is why researchers at Radboud university medical center started looking for an alternative to azathioprine.

More effective and safer

That is where mycophenolate mofetil, or MMF, came into play. Normally, patients with autoimmune hepatitis only receive this drug if treatment with azathioprine is not effective or has too many side effects. Together with colleagues from Leiden University Medical Center, the Nijmegen scientists investigated the efficacy of MMF is as a first-line treatment. They performed their study in seventy patients who had just been diagnosed with autoimmune hepatitis. Half of them received MMF and corticosteroids, the other half azathioprine and corticosteroids.

MMF turned out to be much more effective than azathioprine. ‘The liver values in the blood after six months of treatment unequivocally showed this’, says researcher Romée Snijders. ‘They reverted to normal in more than half of the patients who received MMF. In the azathioprine group, normalization was observed in less than a third of the patients. Furthermore, one in four patients had to stop taking azathioprine due to serious side effects, such as nausea and vomiting. In the MMF-treated group, only one in twenty patients stopped taking the drug.’

Updated guideline

This appears to be good news for patients with autoimmune hepatitis. ‘Absolutely’, says study lead Joost Drenth. ‘I was hoping for a positive result, but did not expect that the difference between the drugs would be so large.’ Together with international colleagues, Drenth will now update the treatment guideline for autoimmune hepatitis so that patients can start with MMF instead of azathioprine.

However, not everyone can be treated with MMF. Snijders: ‘The drug can cause damage to the unborn child. That is why women who want to have children are not allowed to use it. But this is a fairly small group, because autoimmune hepatitis usually manifests itself at a later age.’

About the publication

This study was published in the Journal of Hepatology: An open-label randomised-controlled trial of azathioprine vs. mycophenolate mofetil for the induction of remission in treatment-naive autoimmune hepatitis. R.J.A.L.M. Snijders, A.E.C. Stoelinga, T.J.G. Gevers, S. Pape, M. Biewenga, M.E. Tushuizen, R.C. Verdonk, H.J.M. de Jonge, J.M. Vrolijk, S.F. Bakker, T. Vanwolleghem, Y.S. de Boer, M.A.M.C. Baven Pronk, U. Beuers, A.J. van der Meer, N.M.F.V. Gerven, M.G.M. Sijtsma, B.C. van Eijck, M.C. van IJzendoorn, M. van Herwaarden, F.F. van den Brand, K.S. Korkmaz, A.P. van den Berg, M.M.J. Guichelaar, A.D. Levens, B. van Hoek, J.P.H. Drenth; on behalf of the Dutch Autoimmune Hepatitis Working Group. DOI: 10.1016/j.jhep.2023.11.032.

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