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ISTH 2024 | Addressing the risk of cancer after AAV vector dosing in patients with hemophilia
Radek Kaczmarek, PhD, Indiana University School of Medicine, Indianapolis, IN, provides an insight into the development of cancer after adeno-associated virus (AAV) vector dosing for the treatment of hemophilia. Although the risk of malignancy with this therapeutic approach is low and has only been observed in pre-clinical models, there is evidence of some integration of AAV vectors into the genome, which could potentially trigger oncogenesis (although evidence of this in humans does not yet exist). Therefore, malignancy surveillance should be conducted in patients receiving this gene therapy, and Dr Kaczmarek highlights the steps that should be taken if cancer is identified. This interview took place at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Bangkok, Thailand, and virtually.
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Disclosures
Consulting and/or speaker fees from BioMarin, Pfizer, Bayer, Spark and Novo Nordisk
Research funding from Bayer
