Highlights From the 17th Annual Congress of EAHAD
Stable and durable factor IX (FIX) levels over 4 years after etranacogene dezaparvovec gene therapy administration in a Phase 2b trial in patients with haemophilia B
Presenting Author: Steven W. Pipe, MD–University of Michigan, Ann Arbor, MI, USA
A. von Drygalski1, S. W. Pipe2, A. Giermasz3, E. Gomez4, P. E. Monahan5, S. Le Quellec6
- Hemophilia & Thrombosis Treatment Center, University of California, Division of Hematology/Oncology, San Diego, California
- University of Michigan, Department of Pediatrics and Pathology, Ann Arbor, Michigan
- University of California Davis, Division of Hematology/Oncology, Hemophilia Treatment Center, Sacramento, California
- Center for Inherited Blood Disorders, Orange County, California
- CSL Behring, King of Prussia, Pennsylvania, United States
- CSL Behring, Hattersheim am Main, Germany
Data Points
*Contaminated result from a blood sample obtained within 5 half-lives of previous FIX therapy
This graph shows the mean FIX activity levels over time using a one-stage aPTT assay in patients seropositive for AAV5 neutralizing antibodies who received etranacogene dezaparvovec. Post-etranacogene dezaparvovec administration, the mean FIX activity (N = 3) increased to 30.57% (6.97) at week 6. Mean FIX activity (N = 3) remained stable and within the normal range from Year 1 (40.77% [9.45; 31.3–50.2]) to Year 4 (45.00% [2.76; 42.8–48.1]).
This table shows the bleeding rate in patients who had neutralizing antibodies to AAV5 who received etranacogene dezaparvovec. No bleeding episodes were reported from Year 3 to 4 and, consequently, no FIX was infused outside of invasive procedures in these patients. ABR for the cumulative follow-up period decreased from 0.22 at Year 3 to 0.17 at Year 4.
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