A natural history of haemophilia treatments
By Rich Gorman
In a recent paper in The Journal of Haemophilia Practice, Tyler Buckner and colleagues describe the development of ‘ATHN 7’. This might sound like the codename for a mission to space, but ATHN 7 is a long-term study that compares the outcomes across people with haemophilia over time. In doing so, it aims to provide ongoing evaluation of how different haemophilia therapies are being used in real-world settings. This is particularly important given how treatment options have expanded for people affected by haemophilia in recent years.
Natural history?
The study is organised and run by the American Thrombosis and Hemostasis Network (ATHN). It involves collecting data from across 26 different Haemophilia Treatment Centres in the United States, following 397 people with haemophilia for four years. It is described as a ‘natural history study’ – that is, a research project collecting health information to understand how a condition develops. In fact, it’s designed to be the largest natural history study of this kind in the US! The ATHN 7 team are collecting demographic, clinical, and patient-reported data through interviews and reviews of medical records at a baseline, and then every 3 months. This information can help scientists and health care professionals understand the safety of different treatments for people with haemophilia.
Treatment in the real world
The primary goal of ATHN 7 is to determine treatment safety, but the study also aims to improve understanding about what scientists call the “real-world use of haemophilia therapies”. This includes things like knowing more about the effectiveness of different treatments (for example, by recording how many bleeds people have), recording the frequency and dosage of different products used by people with haemophilia, and also how people ‘switch’ treatments. ATHN 7 also intends to understand more about people’s ‘target joints’, and how these develop or resolve.
Most excitingly though, the study team want to understand more about ‘patient reported outcomes’ – that is, a person’s account of how a condition or therapy has an impact on their wellbeing and daily life. This means considering the things like the number of times they a person has to visit a hospital per year, and their views on their mobility, ability to take part in activities, pain/discomfort, and mental health.
Looking at the long term
Broadly speaking, ATHN 7 is gathering together comprehensive data about treatment safety, effectiveness, target joints, and patient-reported outcomes for people with haemophilia in the US. By doing so, it has the potential to inform the development of guidelines for people with haemophilia in the future. Tyler and his colleagues explain that ATHN 7 was highly successful in getting people with haemophilia to volunteer to take part in the study and share their data. This is perhaps no surprise – as I said earlier, we all want to know that our treatment is safe and effective!
Following in the footsteps of ATHN 7, the American Thrombosis and Hemostasis Network has launched another initiative known as ‘ATHN Transcends’. This aims to build on the successes of ATHN 7, including all inherited and acquired bleeding disorders, and following people for an even longer period of time – 15 years.
ATHN Transcends is still open for enrolment. So, if you’re reading this from the US – speak to your clinician about it!
Further reading
Buckner TW, Daoud N, Miles M, O’Neill C, Watson C, Recht M. Designing ATHN 7: Safety, effectiveness and practice of treatment of people with haemophilia in the United States through a natural history cohort study. J Haem Pract 2022; 9(1): 147-155. doi: 10/2478/jhp-2022-0020
About the author
Rich Gorman lives with severe haemophilia A. He works as a researcher at Brighton and Sussex Medical School.
For more information about The Journal of Haemophilia Practice or Haemnet’s publishing activities, email us at publishing@haemnet.com
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