Expanding the clinical application of the polycystic liver disease questionnaire: determination of a clinical threshold to select patients for therapy.

Abstract

BACKGROUND:

Polycystic liver disease (PLD) causes symptoms resulting from cystic volume expansion. The PLD-specific questionnaire (PLD-Q) captures symptom burden. This study aims to develop a threshold to identify patients with symptoms requiring further exploration and possibly intervention.

METHODS:

We recruited PLD patients with completed PLD-Qs during their patient journey. We evaluated baseline PLD-Q scores in (un)treated PLD patients to determine a threshold of clinical importance. We assessed our threshold's discriminative ability with receiver operator characteristic statistics, Youden Index, sensitivity, specificity, positive and negative predictive value parameters.

RESULTS:

We included 198 patients with a balanced proportion of treated (n=100) and untreated patients (n=98, PLD-Q scores 49 vs 19, p<0.001; median total liver volume 5827 vs 2185 ml, p<0.001). We established the PLD-Q threshold at 32 points. A score of ≥32 differentiates treated from untreated patients with an area under the ROC of 0.856, Youden Index 0.564, sensitivity of 85.0%, specificity of 71.4%, positive predictive value of 75.2%, and negative predictive value of 82.4%. Similar metrics were observed in predefined subgroups and an external cohort.

CONCLUSION:

We established the PLD-Q threshold at 32 points with high discriminative ability to identify symptomatic patients. Patients with a score ≥32 should be eligible for treatment or inclusion in trials.

• Authors: Thijs R M Barten, Christian B Staring, Marie C Hogan, Tom J G Gevers, Joost P H Drenth, DIPAK consortium
• Journal: HPB : the official journal of the International Hepato Pancreato Biliary Association (HPB (Oxford)) Vol. 25 Issue 8 Pg. 890-897 (08 2023) ISSN: 1477-2574 [Electronic] England
• PMID: 37095030 (Publication Type: Journal Article)
• Copyright: Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved.

Topics

• Humans
• Liver Diseases (diagnosis, therapy)
• Cysts (diagnosis, therapy)
• Surveys and Questionnaires