Questionnaire PLD-complaint-specific assessment identifies need for therapy in polycystic liver disease: A multi-centric prospective study

Abstract

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 54.4 years ±11.2, median liver volume expressed as height-adjusted total liver volume(htLV) of 1994 mL (interquartile range [IQR] 1275; 3150) and median growth of the liver of +74 mL/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 2902 mL (IQR 1908; 3964) and 3607 mL (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.

Keywords: health-related quality of life; liver transplantation; liver volume; polycystic liver disease; somatostatin analogues.

FIGURE 1

(a) Receiver operating characteristic (ROC) curve of the POLCA severity of perceived illness score (POLCA SPI) score and the need for volume reducing therapy in the training cohort (n = 63). (b) ROC curve of the POLCA SPI score and the need for volume reducing therapy in the validation cohort (n = 126). In (a) ROC analysis showed that the accuracy of the POLCA SPI score in predicting the need for therapy was 80.0% (69.1%–90.0%). In (b) ROC analysis showed that the accuracy of the POLCA SPI score in predicting the need for therapy was 87.0% (80.8%–93.3%).

FIGURE 2

Receiver operating characteristic (ROC) curve of the POLCA severity of perceived illness score (POLCA SPI) score and the need for liver transplantation (n = 18). ROC analysis showed that the AUROC of the POLCA SPI score for predicting the need for liver transplantation (LT) was 86.0% (76.7%–95.3%) and a severity of perceived illness (SPI) score ≥18 predicted the need of LT with a sensitivity of 83.3% (60.8%–94.2%) and a specificity of 83.0% (76.7%–87.9%) and a likelihood ratio of 4.9.