Aapkes, S. E., Bernts, L. H. P., van den Berg, A. P., van den Berg, M., Blokzijl, H., Cantineau, A. E. P., van Gastel, M. D. A., de Haas, R. J., Kappert, P., Muller, R. U., Nevens, F., Torra, R., Visser, A., Drenth, J. P. H. and Gansevoort, R. T. (2022). Protocol for a randomized controlled multicenter trial assessing the efficacy of leuprorelin for severe polycystic liver disease: the AGAINST-PLD study. BMC Gastroenterol., 22 (1). LONDON: BMC. ISSN 1471-230X

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Abstract

Background: In patients with severe polycystic liver disease (PLD), there is a need for new treatments. Estrogens and possibly other female sex hormones stimulate growth in PLD. In some patients, liver volume decreases after menopause. Female sex hormones could therefore be a target for therapy. The AGAINST-PLD study will examine the efficacy of the GnRH agonist leuprorelin, which blocks the production of estrogen and other sex hormones, to reduce liver growth in PLD. Methods: The AGAINST-PLD study is an investigator-driven, multicenter, randomized controlled trial. Institutional review board (IRB) approval was received at the University Medical Center of Groningen and will be collected in other sites before opening these sites. Thirty-six female, pre-menopausal patients, with a very large liver volume for age (upper 10% of the PLD population) and ongoing liver growth despite current treatment options will be randomized to direct start of leuprorelin or to 18 months standard of care and delayed start of leuprorelin. Leuprorelin is given as 3.75 mg subcutaneously (s.c.) monthly for the first 3 months followed by 3-monthly depots of 11.25 mg s.c. The trial duration is 36 months. MRI scans to measure liver volume will be performed at screening, 6 months, 18 months, 24 months and 36 months. In addition, blood will be drawn, DEXA-scans will be performed and questionnaires will be collected. This design enables comparison between patients on study treatment and standard of care (first 18 months) and within patients before and during treatment (whole trial). Main outcome is annualized liver growth rate compared between standard of care and study treatment. Secondary outcomes are PLD disease severity, change in liver growth within individuals and (serious) adverse events. The study is designed as a prospective open-label study with blinded endpoint assessment (PROBE). Discussion: In this trial, we combined the expertise of hepatologist, nephrologists and gynecologists to study the effect of leuprorelin on liver growth in PLD. In this way, we hope to stop liver growth, reduce symptoms and reduce the need for liver transplantation in severe PLD.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Aapkes, S. E.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bernts, L. H. P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
van den Berg, A. P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
van den Berg, M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Blokzijl, H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Cantineau, A. E. P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
van Gastel, M. D. A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
de Haas, R. J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kappert, P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Muller, R. U.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Nevens, F.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Torra, R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Visser, A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Drenth, J. P. H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gansevoort, R. T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-683056
DOI: 10.1186/s12876-022-02142-y
Journal or Publication Title: BMC Gastroenterol.
Volume: 22
Number: 1
Date: 2022
Publisher: BMC
Place of Publication: LONDON
ISSN: 1471-230X
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
CLINICAL-PRACTICE; KIDNEY-DISEASE; WOMEN; QUESTIONNAIRE; SOMATOSTATIN; SYMPTOMS; ANALOGS; VOLUMEMultiple languages
Gastroenterology & HepatologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/68305

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