INFUSE: Rationale and design of a multi-center, open label, collaborative study to treat HRS-AKI with continuous terlipressin infusion.

Ascites Decompensated cirrhosis Hepatorenal syndrome Liver transplant Portal hypertension

Journal

Contemporary clinical trials communications
ISSN: 2451-8654
Titre abrégé: Contemp Clin Trials Commun
Pays: Netherlands
ID NLM: 101671157

Informations de publication

Date de publication:
Dec 2023
Historique:
received: 17 07 2023
revised: 25 08 2023
accepted: 01 10 2023
medline: 13 11 2023
pubmed: 13 11 2023
entrez: 13 11 2023
Statut: epublish

Résumé

Hepatorenal syndrome-acute kidney injury (HRS-AKI) carries significant morbidity and mortality among those with end-stage liver disease. Bolus terlipressin for treatment of HRS-AKI received FDA approval in September 2022. US implementation of terlipressin, however, is hindered by the paucity of local data on the optimal patient population and administration mode, as well as the effect on transplant priority. The INFUSE study is designed to evaluate the use of continuous terlipressin infusion among transplant candidates with advanced liver disease and HRS-AKI. Fifty prospective patients with HRS-AKI will receive a single bolus of terlipressin 0.5 mg followed by continuous infusions of terlipressin from 2 to 8 mg/day for up to 14 days. The cohort will be enriched with those listed, in evaluation, or eligible for liver transplantation, while those with ACLF grade 3, MELD ≥35, and serum creatinine >5.0 mg/dL will be excluded. Fifty patients who received midodrine plus octreotide or norepinephrine for HRS-AKI will serve as a retrospective comparator cohort. The INFUSE study aims to assess the safety and efficacy of continuous terlipressin infusion among largely transplant-eligible patients with HRS-AKI, and to provide US-based data on transplant outcomes. This novel study design simultaneously mitigates terlipressin adverse events while providing renal benefits to patients, thus addressing the unmet medical need of those with HRS-AKI who have limited treatment options and are awaiting liver transplantation in the US.

Sections du résumé

Background UNASSIGNED
Hepatorenal syndrome-acute kidney injury (HRS-AKI) carries significant morbidity and mortality among those with end-stage liver disease. Bolus terlipressin for treatment of HRS-AKI received FDA approval in September 2022. US implementation of terlipressin, however, is hindered by the paucity of local data on the optimal patient population and administration mode, as well as the effect on transplant priority. The INFUSE study is designed to evaluate the use of continuous terlipressin infusion among transplant candidates with advanced liver disease and HRS-AKI.
Methods UNASSIGNED
Fifty prospective patients with HRS-AKI will receive a single bolus of terlipressin 0.5 mg followed by continuous infusions of terlipressin from 2 to 8 mg/day for up to 14 days. The cohort will be enriched with those listed, in evaluation, or eligible for liver transplantation, while those with ACLF grade 3, MELD ≥35, and serum creatinine >5.0 mg/dL will be excluded. Fifty patients who received midodrine plus octreotide or norepinephrine for HRS-AKI will serve as a retrospective comparator cohort.
Conclusion UNASSIGNED
The INFUSE study aims to assess the safety and efficacy of continuous terlipressin infusion among largely transplant-eligible patients with HRS-AKI, and to provide US-based data on transplant outcomes. This novel study design simultaneously mitigates terlipressin adverse events while providing renal benefits to patients, thus addressing the unmet medical need of those with HRS-AKI who have limited treatment options and are awaiting liver transplantation in the US.

Identifiants

pubmed: 37953795
doi: 10.1016/j.conctc.2023.101211
pii: S2451-8654(23)00157-6
pmc: PMC10632660
doi:

Types de publication

Journal Article

Langues

eng

Pagination

101211

Informations de copyright

© 2023 The Authors.

Déclaration de conflit d'intérêts

The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Ethan Weinberg.•Mallinckodt Pharmaceuticals: study funding, DSMB/Advisory board participation•BioVie: consulting•PharmaIN: consulting•Institute for Medical and Nursing Education: lectures/presentations Stevan A. Gonzalez.•Mallinckrodt Pharmaceuticals: advisory board, consulting, Speakers bureau•Salix Pharmaceuticals: consulting, Speakers bureau•AbbVie Pharmaceuticals: Speakers bureau R. Todd Frederick.•Mallinckrodt Pharmaceuticals: consultant Raymond A. Rubin.•Mallinckrodt Pharmaceuticals: clinical trial funding, Speakers bureau William Tobin.•Services contract with UPenn to monitor study data Khurram Jamil.•Mallinckrodt Pharmaceuticals: employee, patents, stock Zachary Fricker.•Mallinckrodt Pharmaceuticals: payments to institution and travel reimbursements K. Rajender Reddy.•Grants/contracts: BMS, Intercept, Mallinckrodt, BioVie, Sequana, Grifols, Exact Sciences, HCC-TARGET, NASH-TARGET, Merck•Royalties/licenses: UpToDate•Consulting: Spark Therapeutics, Novo Nordisk, Mallinckrodt, Genfit, BioVie•DSMB/Advisory Board participation: Novartix, Astra Zeneca•Associate Editor Gastroenterology No disclosures:•Suditi Rahematpura, Manhal J. Izzy, Douglass A. Simonetto, Jade Ikahihifo-Bender, Maggie Harte, Grace Kim-Lee, Sherry Witkiewicz

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Auteurs

Ethan Weinberg (E)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Suditi Rahematpura (S)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Stevan A Gonzalez (SA)

Division of Hepatology, Simmons Transplant Institute, Baylor Scott and White All Saints Medical Center, Fort Worth, TX, USA.

Manhal J Izzy (MJ)

Department of Gastroenterology, Hepatology, and Nutrition, Vanderbilt University Medical Center, Nashville, TN, USA.

Douglas A Simonetto (DA)

Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA.

R Todd Frederick (RT)

Department of Hepatology and Liver Transplantation, California Pacific Medical Center, San Francisco, CA, USA.

Raymond A Rubin (RA)

Department of Transplantation, Piedmont Transplant Institute, Atlanta, GA, USA.

Jade Ikahihifo-Bender (J)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Maggie Harte (M)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Grace Kim-Lee (G)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Sherry Witkiewicz (S)

International Healthcare, LLC, Norwalk, CT, USA.

William Tobin (W)

International Healthcare, LLC, Norwalk, CT, USA.

Khurram Jamil (K)

Mallinckrodt Ltd, Scientific Affairs, Hampton, NJ, USA.

Zachary Fricker (Z)

Division of Gastroenterology, Hepatology, and Nutrition, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA.

K Rajender Reddy (KR)

Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

Classifications MeSH