Wolfgang Miesbach: Monitoring Patients after Etranacogene Dezaparvovec Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital and heads the Department of Haemostaseology, the Haemophilia Center of Medical Clinic, posted on LinkedIn:
“Our new research provides crucial insights for monitoring patients after etranacogene dezaparvovec (HEMGENIX) gene therapy – the EMA and FDA-approved gene therapy for haemophilia B. This study validates that existing laboratory infrastructure can effectively monitor the transgene expression. The research involved 54 patients from the pivotal HOPE-B trial, with follow-up over 24 months across >30 clinical sites worldwide.
KEY FINDINGS
- Strong correlation between different Factor IX activity assays (R² = 0.891).
- Local laboratory assays show acceptable agreement with central labs (ratios 0.789-1.021).
- Chromogenic assays consistently show lower levels than one-stage assays (40-55% of OSA values).
- FIX-Padua variant doesn’t significantly impact assay discrepancies vs wild-type.
CLINICAL IMPACT
- Provides confidence in real-world laboratory monitoring capabilities.
- Commonly available one-stage assays can reliably monitor post-gene therapy patients.
- Consistent use of the same assay platform is recommended for individual patient follow-up.”
Title: Evaluation of One-Stage Assays for the Monitoring of Recombinant Human Factor IX Padua Activity After Etranacogene Dezaparvovec Gene Therapy
Authors: Jan Astermark, Wolfgang Miesbach, Michiel Coppens, Sander Gielen, Jaap Twisk, Ricardo Dolmetsch, Stephanie Verweij, Paul E. Monahan, Bruce M. Ewenstein, Silpa Nuthalapati, Nick Galante, Guy Young
Read The Full Article at Wiley.
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