Advertisement

Liver-Related Aspects of Gene Therapy for Haemophilia: Call to Action for Collaboration between Haematologists and Hepatologists

  • Wolfgang Miesbach
    Correspondence
    Corresponding author. Medical Clinic 2 / Institute of Transfusion Medicine and Immunohaematology University Hospital Frankfurt, Germany. Phone: +49 69 6301-5051 Fax: +49 69 6301-6738
    Affiliations
    Medical Clinic 2 / Institute of Transfusion Medicine and Immunohaematology, University Hospital Frankfurt, Germany
    Search for articles by this author
  • Graham R. Foster
    Affiliations
    Department of Hepatology, Queen Mary University of London, United Kingdom
    Search for articles by this author
  • Flora Peyvandi
    Affiliations
    Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy

    Università Degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy
    Search for articles by this author
Published:November 24, 2022DOI:https://doi.org/10.1016/j.jhep.2022.11.014
      WM received honoraria from Bayer, Biomarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, sobi, Takeda/Shire, uniQure.
      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Journal of Hepatology
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Yang X.
        • Jeong K.
        • Yabes J.G.
        • Ragni M.V.
        Prevalence and risk factors for hepatocellular carcinoma in individuals with haemophilia in the era of direct-acting antiviral agents: a national inpatient sample study.
        Haemophilia. 2022 Jun 21; (Epub ahead of print. PMID: 35727998)https://doi.org/10.1111/hae.14607
        • Leebeek F.W.G.
        • Miesbach W.
        Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues.
        Blood. 2021 Sep 16; 138 (PMID: 34232980): 923-931https://doi.org/10.1182/blood.2019003777
      1. Srivastava A, Santagostino E, Dougall A, Kitchen S, Sutherland M, Pipe SW, et al. WFH guidelines for the management of hemophilia panelists and co-authors. WFH guidelines for the management of hemophilia, 3rd ed. Haemophilia. 2020 Aug;26 Suppl 6:1-158. doi: 10.1111/hae.14046. Epub 2020 Aug 3. Erratum in: Haemophilia. 2021 Jul;27(4):699. PMID: 32744769

        • Schnepp B.C.
        • Chulay J.D.
        • Ye G.-J.
        • Flotte T.R.
        • Trapnell B.C.
        • Johnson P.R.
        Recombinant adeno-associated virus vector genomes take the form of long-lived, transcriptionally competent episomes in human muscle.
        Human Gene Therapy. 2015; 27: 32-42
        • Nguyen G.N.
        • Everett J.K.
        • Kafle S.
        • et al.
        A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
        Nature Biotechnology. 2021; 39: 47-55
        • de Jong Y.P.
        • Herzog R.W.
        AAV and hepatitis: cause or coincidence?.
        Mol Ther. 2022 Sep 7; 30 (Epub 2022 Aug 17. PMID: 35981546; PMCID: PMC9482007): 2875-2876https://doi.org/10.1016/j.ymthe.2022.08.001
        • Wang M.
        • Negrier C.
        • Driessler F.
        • Goodman C.
        • Skinner M.W.
        The hemophilia gene therapy patient journey: questions and answers for shared decision-making.
        Patient Prefer Adherence. 2022 Jun 9; 16 (PMID: 35707346; PMCID: PMC9191577): 1439-1447https://doi.org/10.2147/PPA.S355627
      2. Laffan M, Rangarajan S, Lester W, Symington E, Madan B, Hart D, et al. Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A [abstract]. https://abstracts.isth.org/abstract/hemostatic-results-for-up-to-6-years-following-treatment-with-valoctocogene-roxaparvovec-an-aav5-hfviii-sq-gene-therapy-for-severe-hemophilia-a/

        • Nathwani A.C.
        • Reiss U.
        • Tuddenham E.
        • et al.
        Adeno-associated medi- ated gene transfer for hemophilia B:8 Year follow up and impact of removing “empty viral particles” on safety and efficacy of gene transfer.
        Blood. 2018; 132 (Supplement 1): 491
        • Ozelo M.C.
        • Mahlangu J.
        • Pasi K.J.
        • Giermasz A.
        • Leavitt A.D.
        • Laffan M.
        • et al.
        GENEr8-1 trial group. Valoctocogene roxaparvovec gene therapy for hemophilia A.
        N Engl J Med. 2022 Mar 17; 386 (PMID:35294811): 1013-1025https://doi.org/10.1056/NEJMoa2113708
      3. Miesbach, W., at al. Final analysis from the pivotal phase 3 HOPE-B Gene Therapy trial: Stable steady-state efficacy and Safety of etranacogene dezaparvovec in adults with severe or moderately severe haemophilia B [abstract EAHAD 2022 and CSL Behring Press Release 4th Feb 2022]. 22.07.2022]; Available from: https://eahad.org/eahad-2022-latest-clinical-trial-results-session-press-release/; https://www.cslbehring.com/newsroom/2022/hope-b-data-presented-at-eahad2022

        • Fong S.
        • Yates B.
        • Sihn C.R.
        • Mattis A.N.
        • Mitchell N.
        • Liu S.
        • et al.
        Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.
        Nat Med. 2022 Apr; 28 (Epub 2022 Apr 11. PMID: 35411075; PMCID: PMC9018415): 789-797https://doi.org/10.1038/s41591-022-01751-0
      4. Schmidt, M.R., et al. Liver safety case report from the phase 3 HOPE-B gene therapy trial in adults with hemophilia B [abstract, ISTH 2021]. 2021 22.07.2022]; Available from: https://abstracts.isth.org/abstract/liver-safety-case-report-from-the-phase-3-hope-b-gene-therapy-trial-in-adults-with-hemophilia-b/

        • George L.A.
        • Ragni M.V.
        • Rasko J.E.J.
        • Raffini L.J.
        • Samelson-Jones B.J.
        • Ozelo M.
        • et al.
        Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B.
        Mol Ther. 2020 Sep 2; 28: 2073-2082
        • Miesbach W.
        • Chowdary P.
        • Coppens M.
        • Hart D.P.
        • Jimenez-Yuste V.
        • Klamroth R.
        • et al.
        Delivery of AAV-based gene therapy through haemophilia centres-A need for re-evaluation of infrastructure and comprehensive care: a Joint publication of EAHAD and EHC.
        Haemophilia. 2021 Nov; 27 (Epub 2021 Sep 22. PMID: 34553460): 967-973https://doi.org/10.1111/hae.14420
        • Konkle B.
        • Pierce G.
        • Coffin D.
        • Naccache M.
        • Clark R.C.
        • George L.
        • Iorio A.
        • et al.
        ISTH subcommittee on Factor VIII, Factor IX, rare bleeding disorders. Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: communication from the SSC of the ISTH.
        J Thromb Haemost. 2020 Nov; 18 (PMID: 33463024; PMCID: PMC7756325): 3074-3077https://doi.org/10.1111/jth.15023
        • Chowdary P.
        • Shapiro S.
        • Makris M.
        • Evans G.
        • Boyce S.
        • Talks K.
        • et al.
        Phase 1-2 trial of AAVS3 gene therapy in patients with hemophilia B.
        N Engl J Med. 2022 Jul 21; 387 (PMID:35857660): 237-247https://doi.org/10.1056/NEJMoa2119913