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

Autor:	Barten TRM; Department of Gastroenterology and Hepatology, Radboud University Medical Center, Nijmegen, the Netherlands; European Reference Network RARE-LIVER, Germany. Electronic address: trmbarten@gmail.com., Staring CB; Department of Gastroenterology and Hepatology, Radboud University Medical Center, Nijmegen, the Netherlands., Hogan MC; Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester MN, USA., Gevers TJG; Department of Gastroenterology and Hepatology, Radboud University Medical Center, Nijmegen, the Netherlands; European Reference Network RARE-LIVER, Germany; Department of Gastroenterology and Hepatology, Maastricht University Medical Center, Maastricht, the Netherlands., Drenth JPH; Department of Gastroenterology and Hepatology, Radboud University Medical Center, Nijmegen, the Netherlands; European Reference Network RARE-LIVER, Germany.
Jazyk:	angličtina
Zdroj:	HPB : the official journal of the International Hepato Pancreato Biliary Association [HPB (Oxford)] 2023 Aug; Vol. 25 (8), pp. 890-897. Date of Electronic Publication: 2023 Apr 11.
DOI:	10.1016/j.hpb.2023.04.004
Abstrakt:	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. Competing Interests: Declaration of interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. (Copyright © 2023 The Author(s). Published by Elsevier Ltd.. All rights reserved.)
Databáze:	MEDLINE
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