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

Autor: Billiet A; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., Temmerman F; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., Coudyzer W; Department of Radiology, University Hospitals KU Leuven, Leuven, Belgium., Van den Ende N; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., Colle I; Department of Gastroenterology and Hepatology, Algemeen Stedelijk Ziekenhuis Aalst, Aalst, Belgium., Francque S; Department of Gastroenterology and Hepatology, Antwerp University Hospital, Antwerp, Belgium., De Maeght S; Department of Gastroenterology and Hepatology, Grand Hôpital De Charleroi Saint-Joseph, Charleroi, Belgium., Janssens F; Department of Gastroenterology and Hepatology, Jessa Ziekenhuis, Hasselt, Belgium., Orlent H; Department of Gastroenterology and Hepatology, AZ Sint Jan Brugge, Brugge, Belgium., Sprengers D; Department of Gastroenterology and Hepatology, GZA Antwerp, Antwerpen, Belgium., Delwaide J; Department of Gastroenterology and Hepatology, C.H.U. de Liège, Liège, Belgium., Decock S; Department of Gastroenterology and Hepatology, AZ Sint Lucas Brugge, Brugge, Belgium., De Vloo C; Department of Gastroenterology and Hepatology, AZ Delta, Roeselare, Belgium., Moreno C; Department of Gastroenterology and Hepatology, ULB Erasme, Brussels, Belgium., van Malenstein H; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., van der Merwe S; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., Verbeek J; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium., Nevens F; Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, European Reference Network on liver disease (ERN Rare-Liver), Leuven, Belgium.
Jazyk: angličtina
Zdroj: United European gastroenterology journal [United European Gastroenterol J] 2023 Sep; Vol. 11 (7), pp. 633-641. Date of Electronic Publication: 2023 Jun 05.
DOI: 10.1002/ueg2.12387
Abstrakt: 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.
(© 2023 The Authors. United European Gastroenterology Journal published by Wiley Periodicals LLC on behalf of United European Gastroenterology.)
Databáze: MEDLINE