AGAINST Polycystic Liver Growth: Estrogen as target for treatment

Polycystic liver disease (PLD) is a genetic disease in which cysts grow in the liver unrestrictedly. The cyst growth can lead to hepatomegaly, sometimes up to 10-15 liters, while liver functions remain unaffected. The large polycystic livers lead to several and sometimes severe complaints, such as pain, abdominal discomfort, loss of appetite, early satiety, difficulty bending forward and psychological complaints. In this thesis, we studied medical treatment options for hepatomegaly in PLD.
In the first study, we showed that the only treatment to slow liver growth currently available, somatostatin analogues (SSA), has gallstones as a side effect. In our study, 15% of the patients treated with SSA developed gallstones, while this was 1% in the control group. Of the patients with these gallstones, 50% developed severe complications, almost all (years) after discontinuation of treatment. In the next studies, a literature review, two case-report and a trial design paper, we showed the potential of anti-estrogenic treatments to slow the liver growth. The majority of patients with the disease is female, and after menopause, sometimes symptoms decrease. Therefore, we studied options to block estrogens and other female hormones and designed a randomized controlled trial, which is running now, results are to follow. In the last study of the thesis we showed that the Siemens application Volume is a fast and easy-to-use method to measure the liver volumes, with a bias and precision comparable to manual measurements (the gold standard). Till now, manual liver volume measurement costed 2-3 hours per scan. With the new methods, liver volumes can be measured easier and in approximately 20 minutes, which is important to measure disease progression, effects of treatments and trial-outcomes.