Steven W. Pipe, MD
Highlights From the ISTH 2021 Congress

Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B

M. Schmidt1, G.R Foster2, M. Coppens3, H. Thomsen1, D. Cooper4, R. Dolmetsch4, E.K Sawyer4, L. Heijink5, S.W Pipe6

1GeneWerk GmbH, Heidelberg, Germany

2Barts Liver Centre, Queen Mary University of London, London, United Kingdom

3Amsterdam University Medical Center, Amsterdam, Netherlands

4uniQure Inc, Lexington, United States

5uniQure BV, Amsterdam, Netherlands

6University of Michigan, Ann Arbor, United States

Key Data Points

  • 69-year-old, white non-Hispanic male with moderately severe Hemophilia B
  • 1980-Diagnosed with HBV (+ve for anti-HBs, and anti-HBc and anti-HBe antibodies)
  • 1983-Diagnosed with non-A/non-N hepatitis
  • 2003-Confirmed positive for HCV when testing available
  • 2015-Evaluated for HCV eradication therapy, genotype 1a, no significant fibrosis (Fibroscan 5.7 kPa)
  • 2015-Treated with paritaprevir/ombitasvir/ritonavir, dasabuvir, and ribavirin; achieving a sustained virologic response
  • Social history notable for prior smoking, alcohol consumption of 0-2 units/week
  • Familial history notable for cancer

Liver-related medical history of a patient with hemophilia B in the HOPE-B trial who experienced hepatocellular carcinoma while participating in the study. His medical history contains several risk factors for the development of HCC, including hepatitis B and C, advanced age, and alcohol consumption.

Summary of HCC Molecular Analyses
At 1-year post-infusion, a per protocol ultrasound revealed a subcapsular liver lesion. This table summarizes the subsequent molecular integration site analysis and whole genome sequencing done on resected tumor tissue, including expected findings if the HCC was dependent on (column 1) or independent of (column 2) AAV integration. The actual findings of the analyses are shown in column 3.

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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