摘要：

Background and Aims Polycystic liver disease (PLD) can lead to extensive hepatomegaly. Symptom relief is the primary goal of the treatment. The role of the recently developed disease-specific questionnaires for identification of the thresholds and the assessment of therapy needs further investigation. Methods A five-year prospective multi-centric observational study in 21 hospitals in Belgium gathered a study population of 198 symptomatic PLD-patients of whom the disease-specific symptom questionnaire PLD-complaint-specific assessment (POLCA) scores were calculated. The thresholds of the POLCA score for the need for volume reduction therapy were analyzed. Results The study group consisted of mostly (82.8%) women with baseline mean age of 54.4years ±11.2, median liver volume expressed as height-adjusted total liver volume(htLV) of 1994mL (interquartile range [IQR] 1275; 3150) and median growth of the liver of +74mL/year (IQR +3; +230). Volume reduction therapy was needed in 71 patients (35.9%). A POLCA severity score (SPI)≥14 predicted the need for therapy both in the derivation (n=63) and the validation cohort (n=126). The thresholds to start somatostatin analogues (n=55) or to consider liver transplantation (n=18) were SPI scores of ≥14 and≥18 and the corresponding mean htLVs were 2902mL (IQR 1908; 3964) and 3607mL (IQR 2901; 4337), respectively. Somatostatin analogues treatment resulted in a decrease in the SPI score 6.0 versus+4.5 in patients without somatostatin analogues (p<0.01). Changes in the SPI score were significantly different between the liver transplantation group and no liver transplantation group, +4.3±7.1 versus 1.6±4.9, respectively, (p<0.01). Conclusion A polycystic liver disease-specific questionnaire can be used as a guide on when to start a volume reduction therapy and to assess the effect of treatment.

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