ISTH 2025 Spotlight: HOPE-B Trial Highlights Sustained Efficacy of Gene Therapy for Hemophilia B
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on X:
“ISTH 2025 Highlight: Our HOPE-B study results were brilliantly presented by Dr. Steven Pipe at ISTH 2025, showcasing 4-year durability data in haemophilia B gene therapy.
Study Overview
The phase 3 HOPE-B trial evaluated a single IV dose of etranacogene dezaparvovec (AAV5-FIX Padua) in adults with haemophilia B (FIX ≤2 IU/dL).
N = 54; Responders = 52
Key Efficacy Results
· ↓ 81% in annualized bleeding rate (ABR) (months 7–48 vs. baseline)
· ↓ 90% in ABR at Year 4 vs. lead-in period
· ↓ 98% in mean exogenous FIX use (IU/year) at Year 4
· 98% of participants remained prophylaxis-free through 4 years
· 37% required zero exogenous FIX infusions over 4 years
Factor IX (FIX) Expression
· Mean FIX activity: 37.4% at Year 4
· 83% of responders maintained FIX levels 12–100 IU/dL at 4 years
· Stable expression over 4 years → near-normal hemostasis
Safety Profile
· 96 treatment-related adverse events (TRAEs) over 4 years
· 96% occurred in first 6 months
· Most frequent: ALT↑ (18.5%), headache, flu-like illness
· No FIX inhibitors
· No thrombotic events
· No AAV5-associated genotoxicity or persistent late hepatotoxicity
Clinical Implications
· Single-dose gene therapy led to durable FIX expression and sustained reduction in bleeding and FIX use for ≥4 years
· Majority achieved and maintained prophylaxis freedom
· Favorable safety profile with minimal late events
· Ongoing follow-up remains important for long-term assessment
Congratulations to Steve Pipe and the co-authors.”
Stay updated with Hemostasis Today.
-
Oct 23, 2025, 06:03Melanie Daniel: Wonderful Presentation on Emicizumab by Mickael Rosa at ECTH
-
Oct 22, 2025, 06:17We Will Continue to Raise Awareness and Remind Rveryone That Donating Blood Means Saving Lives - Yeolyan Hematology and Oncology Center
-
Oct 21, 2025, 06:59Samwel Mikaye: Polycythemia and the Risk of Blood Clots
-
Oct 21, 2025, 06:58Samuel Nweke: Interpreting D-Dimer Results
-
Oct 21, 2025, 05:20Ewa Fiorentino Rozek: APS - When “Unprovoked” Clots And Pregnancy Loss Connect
-
Oct 23, 2025, 06:48Omid Seidizadeh Shares Deep Molecular Modeling and Mechanistic Insights into Type 2A VWD
-
Oct 23, 2025, 06:45Mélanie Roussel on D-Dimer Thresholds for Diagnosis of Pulmonary Embolism
-
Oct 23, 2025, 06:28Ramya Ramakrishnan on IDA Association with Depression
-
Oct 23, 2025, 06:07Cedric Hermans: Von Willebrand Disease Diagnosis - From Complexity to Simplicity
-
Oct 23, 2025, 05:45Andrew Larry Frelinger III on Activation of Platelets in Flow Cytometry
-
Oct 23, 2025, 07:11Sebastien Lobet Presented the ACTIVLIM-Hemo Scale
-
Oct 23, 2025, 07:05Syed Ehsan Shah on Advantages of Werfen HemoCell Automation
-
Oct 22, 2025, 08:25Johanna Nystedt: Not Every Day We Can Release a New Blood Product to Meet An Unmet Medical Need!
-
Oct 21, 2025, 06:58Arno Nierich: How Caffeine Affects Red Blood Cells and Blood Storage
-
Oct 21, 2025, 06:43Could Gene Therapy Offer Lasting Quality of Life for People with Hemophilia B?
-
Oct 23, 2025, 06:52Louise Bannon: Thrilled to Witness The Incredible Reach of This Year's World Thrombosis Day!
-
Oct 23, 2025, 06:50Caitlin Raymond to Speak at The 11th Annual Bloodless Medicine and Surgery Society Conference
-
Oct 22, 2025, 10:46Conversation with Ferran Nadeu – Touch Haematology Future Leader 2025
-
Oct 22, 2025, 10:32Lynn Malec - The New Director of the CCBD and Pharmacy at Children's Wisconsin
-
Oct 22, 2025, 09:41Deanna Joseph: Feeling Inspired and Deeply Grateful After Attending the 2025 HTRS SEARCH Workshop