Effects of the GnRH agonist leuprorelin on liver volume in premenopausal women with severe polycystic liver disease.
Inge G Wijbenga, Renee Duijzer, Paul Geertsema et al.
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In 30 seconds
This prospective, open-label randomized trial evaluated leuprorelin in premenopausal women aged 18–45 years with severe polycystic liver disease (PLD). Six participants began treatment immediately and eight received standard care with delayed treatment. At 18 months, median liver growth rates were 4.04 [-4.62 to 6.84] %/year with leuprorelin and 11.0 [0.70-13.2] %/year with standard care (p=0.15), with no significant difference. PLD-related quality of life did not improve, while menopausal side effects and adverse events were substantial; the trial was terminated early.
Key findings
- Median liver growth rates were 4.04 [-4.62 to 6.84] %/year with immediate leuprorelin versus 11.0 [0.70-13.2] %/year with standard care (p=0.15).
- Liver volume continued to increase in half of participants receiving leuprorelin, and PLD-related quality of life did not improve.
- There were 38 adverse events in the direct-start group versus 9 with standard care (p<0.001); one participant stopped treatment early because of severe menopausal side effects.
Why it matters
The trial tested estrogen suppression as a potential approach to severe PLD, but found no significant difference in liver growth and no quality-of-life improvement alongside substantial treatment burden. These results highlight the importance of weighing potential disease effects against menopausal symptoms and adverse events when evaluating hormonal approaches for PLD.
What to keep in mind
The trial was small and open-label, and it was terminated early after an interim analysis prompted by notable side effects.