Higher need for polycystic liver disease therapy in female patients: sex-specific association between liver volume and need for therapy.

BACKGROUND AIM

Prognostic tools or biomarkers are urgently needed in polycystic liver disease (PLD) to monitor disease progression and evaluate treatment outcomes. Total liver volume (TLV) is currently used to assess cross-sectional disease severity and female patients typically have larger livers than males. Therefore, this study explores the sex-specific association between TLV and volume reducing therapy.

APPROACH RESULTS

In this prospective cohort study, we included PLD patients from European treatment centers. We explored sex-specific differences in the association between baseline TLV and initiation of volume reducing therapy and determined the cumulative incidence rates of volume reducing therapy in our cohort.We included 358 patients, of whom 157 (43.9%) received treatment. Treated patients had a higher baseline TLV (median TLV 2.16 versus 4.34 liter, p<0.001), were more frequently female (69.7% versus 89.8%, p<0.001) and had a higher risk of liver events (HR 4.381, p<0.001). The cumulative volume reducing therapy rate at 1 year of follow-up was 21.0% for females compared to 9.1% for males. Baseline TLV was associated with volume reducing therapy and there was an interaction with sex (HR females 1.202, p<0.001;HR males 1.790, p<0.001; at 1.5 liters).

CONCLUSION

Baseline TLV is strongly associated with volume reducing therapy initiation at follow-up in PLD patients, with sex-specific differences in this association. Disease staging systems should use TLV to predict need for future volume reducing therapy in PLD separately for males and females.

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