Medical menopause did not shrink polycystic livers

Design

Randomised, open-label, terminated early

Population

14 premenopausal women aged 18–45 with severe polycystic liver disease

Primary outcome

Liver growth rate at 18 months

Effect

Median 4.04% vs 11.0% per year, p=0.15; adverse events 38 vs 9, p<0.001

Because oestrogen drives cyst growth in polycystic liver disease, investigators randomised 14 premenopausal women aged 18–45 with severe disease to leuprorelin 11.25 mg every three months straight away (6) or standard care with a delayed start at 18 months (8). The trial was open-label.

Liver volume still grew in half of the treated women. Median growth was 4.04% a year with leuprorelin against 11.0% with standard care, a difference that was not statistically significant (p=0.15). Liver-related quality of life did not improve, menopausal symptoms were severe, and adverse events numbered 38 against 9 (p<0.001). The study was stopped early.

The numbers are tiny, so a modest effect cannot be excluded. But the trial argues against using a GnRH agonist off label for this indication, and gynaecologists asked to induce menopause for liver disease have a direct answer to give.

• Leuprorelin-induced menopause did not significantly slow liver growth in severe polycystic liver disease.
• Treated women had four times as many adverse events, mostly menopausal, and one stopped early.
• Liver-related quality of life did not improve with treatment.
• Oestrogen-containing contraception and HRT remain a concern in these women, which this trial does not change.

Why it matters

A biologically plausible hormonal strategy failed, and the side effects were heavier than the benefit.

Don't overread it

With 14 women the trial could miss a small benefit; it shows no worthwhile effect, not proof of none.

The statistics, in plain English

The growth rates point in favour of leuprorelin, but with so few women the p-value of 0.15 means a gap this size could easily arise by chance. The adverse event difference, by contrast, is large and unlikely to be chance.

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