PREPARING FOR GENE THERAPY
preparing your PATIENTS
Which patients could potentially be candidates for gene therapy?
The current gene therapy trials for haemophilia have recruited adult men aged >18 years. Because gene therapy targets the liver, patients had healthy liver function.1-3
Preparing Your Practice
Could Your Practice Become a Gene Therapy Centre?
Currently, no gene therapy has been approved for the treatment of haemophilia. However, with promising results at clinical trials,1,2,4 you may want to start thinking about gene therapy. To prepare for the possible approval of gene therapy, multiple haemophilia societies have released materials and recommendations.
The European Association for Haemophilia and Allied Disorders (EAHAD) and International Society on Thrombosis and Haemostasis (ISTH) have released a joint educational guide. These resources provide the most recent updates on the rapidly evolving field of gene therapy.5
In addition to providing your patients and team members with education about gene therapy, there are likely logistical and infrastructural aspects to consider. The EAHAD and European Haemophilia Consortium (EHC) have released a joint statement proposing a model for prescribing, administrating and monitoring patients receiving gene therapy. This model was designed to maximize the benefit from therapy and optimize safety for patients while ensuring standardisation across Europe.6
VORIGE
Gene Therapy for Haemophilia
Learn about what makes haemophilia the optimal candidate for a gene therapy.
VOLGENDE
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References
- Nathwani AC, Tuddenham EGD, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. New Engl J Med. 2011;365(25):2357-2365.
- Rangarajan S, Walsh L, Lester W, et al. AAV5-Factor VIII gene transfer in severe hemophilia A. New Engl J Med. 2017;377(26):2519-2530.
- Krumb E, Lambert C, Hermans C. Patient selection for hemophilia gene therapy: real-life data from a single center. Res Pract Thromb Haemost. 2021;5(3):390-394.
- Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.
- EAHAD and ISTH. ISTH and EAHAD educational partnership on gene therapy.[press release] https://eahad.org/isth-and-eahad-educational-partnership-on-gene-therapy/. Accessed October 20, 2021.
- Miesbach W, Chowdary P, Coppens M, et al. Delivery of AAV-based gene therapy through haemophilia centers-a need for re-evaluation of infrastructure and comprehensive care: a joint publication of EAHAD and EHC. Haemophilia. 2021;27:967-973.
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