Steven W. Pipe, MD
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

  1. Hemophilia & Thrombosis Treatment Center, University of California, Division of Hematology/Oncology, San Diego, California
  2. University of Michigan, Department of Pediatrics and Pathology, Ann Arbor, Michigan
  3. University of California Davis, Division of Hematology/Oncology, Hemophilia Treatment Center, Sacramento, California
  4. Center for Inherited Blood Disorders, Orange County, California
  5. CSL Behring, King of Prussia, Pennsylvania, United States
  6. CSL Behring, Hattersheim am Main, Germany

Data Points

Figure 1: Mean FIX activity levels over time

*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]).

Table 1: Annualized Bleeding Rate (ABR) and FIX Consumption

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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