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RESEARCH QUESTION: Most young female patients with large livers caused by polycystic liver disease (PLD) show progressive growth in liver size and ultimately need a liver transplant. Recent research suggests that estrogen and possibly also progesterone promote liver growth in PLD and that after spontaneous menopause polycystic livers stop growing. We question whether lowering of estrogen and progesterone levels with the GnRH analogue leuproreline may be an effective treatment.

 

HYPOTHESIS: Leuproreline will reduce liver volume and related complaints in women with large polycystic livers.

 

STUDY DESIGN: A prospective randomized open label design with blinded endpoint assessment is used (PROBE) for this early proof-of-concept study. Patients will be randomized to direct or delayed start of study medication, and be followed in one of 5 participating expert centers.

 

STUDY POPULATION: Adult female premenopausal patients with PLD, with large livers for their age.

 

INTERVENTION: leuproreline will initially be given as monthly injections, and thereafter, if tolerated, as three-monthly injections. Study medication and medication associated costs will be an (in-kind) donation by the company Abbvie, that waives IP rights and ownership of data. Leuproreline is routinely available, off-patent, relatively low in costs and side effects are well-known, which is expected to facilitate implementation.

 

OUTCOME MEASURES: Liver growth rate measured by serial MRIs, liver volume related complaints, quality of life, safety, tolerability and cost-effectiveness of treatment.

 

SAMPLE SIZE, DATA ANALYSIS: Estimated sample size is 36 patients. Data analysis will be performed by an independent statistician, that will assess differences in outcome measures between the leuproreline and control group (between group comparison), and also within individuals before and after start of leuproreline.

 

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