Excellent safety and effectiveness of high-dose myrcludex-B monotherapy administered for 48 weeks in HDV-related compensated cirrhosis: A case report of 3 patients.


Journal

Journal of hepatology
ISSN: 1600-0641
Titre abrégé: J Hepatol
Pays: Netherlands
ID NLM: 8503886

Informations de publication

Date de publication:
10 2019
Historique:
received: 01 05 2019
revised: 15 06 2019
accepted: 08 07 2019
pubmed: 16 7 2019
medline: 15 12 2020
entrez: 15 7 2019
Statut: ppublish

Résumé

Short-term administration of the entry inhibitor myrcludex-B (MyrB) has been shown to be safe and effective in phase II studies in patients coinfected with hepatitis B virus (HBV) and hepatitis delta virus (HDV). However, its effectiveness and safety are unknown during long-term and high-dose treatment of patients with compensated cirrhosis in real-life settings. Herein, we describe the first 3 European patients with HDV-related compensated cirrhosis who were treated with MyrB 10 mg/day for 48 weeks as a compassionate therapy. Liver function tests, bile acids, and virological markers were monitored every 4 weeks. HBV/HDV-specific T cell quantity (up to 48 and 36 weeks) and HBV RNA levels were also assessed in 2 cases. During MyrB treatment, HDV RNA levels progressively declined from 4.4 and 5.6 logs IU/ml to undetectability in 2 cases, and from 6.8 log copies/ml to 500 copies/ml for the other patient. Alanine aminotransferase normalised after 20, 12 and 28 weeks, respectively. A significant improvement in features of portal hypertension, liver function tests and alpha-fetoprotein levels were documented in 2 cases. In the male patient with histological and clinical stigmata of autoimmune hepatitis, IgG and immunoglobulins rapidly normalised. No significant changes in HBV surface antigen levels and circulating HBV/HDV-specific T cells were demonstrated; HBV DNA and HBV RNA levels remained undetectable throughout the study period. MyrB was well tolerated; patients remained fully asymptomatic despite a significant increase of bile acids. In conclusion, this report shows excellent safety and effectiveness of a 48-week course of MyrB 10 mg/day, combined with tenofovir disoproxil fumarate, for the treatment of HDV-related compensated cirrhosis.

Identifiants

pubmed: 31302176
pii: S0168-8278(19)30411-8
doi: 10.1016/j.jhep.2019.07.003
pii:
doi:

Substances chimiques

Antiviral Agents 0
Lipopeptides 0
RNA, Viral 0
myrcludex-B 0
Tenofovir 99YXE507IL

Types de publication

Case Reports Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

834-839

Informations de copyright

Copyright © 2019 European Association for the Study of the Liver. Published by Elsevier B.V. All rights reserved.

Auteurs

Alessandro Loglio (A)

CRC "A. M. and A. Migliavacca" Center for Liver Disease, Division of Gastroenterology and Hepatology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Peter Ferenci (P)

Department of Internal Medicine III, Division of Gastroenterology and Hepatology, Medical University of Vienna, Vienna, Austria.

Sara Colonia Uceda Renteria (SC)

Virology Unit, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Christine Y L Tham (CYL)

Program Emerging Infectious Diseases, Duke-NUS Medical School, Singapore.

Florian van Bömmel (F)

Section of Hepatology, Department of Gastroenterology, University Hospital Leipzig, Leipzig, Germany.

Marta Borghi (M)

CRC "A. M. and A. Migliavacca" Center for Liver Disease, Division of Gastroenterology and Hepatology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Heidemarie Holzmann (H)

Center for Virology, Medical University of Vienna, Vienna, Austria.

Riccardo Perbellini (R)

CRC "A. M. and A. Migliavacca" Center for Liver Disease, Division of Gastroenterology and Hepatology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Elena Trombetta (E)

Flow Cytometry Service, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Silvia Giovanelli (S)

Department of Transfusion Medicine and Hematology, Milano Cord Blood Bank, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Letizia Greco (L)

Virology Unit, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Laura Porretti (L)

Flow Cytometry Service, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Daniele Prati (D)

Department of Transfusion Medicine and Hematology, Milano Cord Blood Bank, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy.

Ferruccio Ceriotti (F)

Virology Unit, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Giovanna Lunghi (G)

Virology Unit, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy.

Antonio Bertoletti (A)

Program Emerging Infectious Diseases, Duke-NUS Medical School, Singapore.

Pietro Lampertico (P)

CRC "A. M. and A. Migliavacca" Center for Liver Disease, Division of Gastroenterology and Hepatology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy. Electronic address: pietro.lampertico@unimi.it.

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