J Haem Pract 2014; 2(1):J Haem Pract 2015; 2(1): 6-8. doi: 10.17225/jhp00040

Authors: John Morris

John Morris
Patient Advisor
Katharine Dormandy Haemophilia Centre & Thrombosis Unit, Ground floor, Royal Free Hospital, Pond Street, London NW3 2QG, UK. Email:


The investigators behind the first gene therapy trial with adeno-associated virus 8 (AAV8) Factor IX appointed a patient ombudsperson to help ensure participants were able to give truly informed consent. The experiences and challenges of the ombudsperson, who met with the first six UK-based patients, are described. It was stressed to potential participants that altruism, rather than any expectation of clinical benefit, should be the primary motivation to taking part. At the same time a sober assessment of the potential risks to their safety needed to be made.


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  1. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357-65. doi: 10.1056/NEJMoa1108046.
  2. Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994-2004. doi: 10.1056/NEJMoa1407309.

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