Living Well with Haemophilia

Professor Ulla Hedner’s pioneering haemophilia research helped to save Johann’s life in 1981 by staunching serious bleeds as he lost his milk teeth.¹,² Indeed, Professor Hedner’s work eventually led to the development of a synthetic form of Factor VIIa.¹,³ Nowadays, recombinant clotting factors are the standard instead of the human-derived forms of the 1970s. These days Johann works as an economist in Stockholm, living well with haemophilia, one of thousands who owe their lives to her pioneering research.²

 


Published 15 July 2019 | 4 min read
 


Daniel Timmermann, Corporate Vice President of Haemophilia Research at Novo Nordisk, has helped research methods develop in more recent times. For Daniel, it has always been about improving outcomes for people; “from the very beginning of my career, I have always been fascinated with the idea of employing cutting-edge science to reduce distress and make a real difference for people.”

 

The future looks bright

Someone who experiences the developments in treatment is seven-year-old Harry, who was diagnosed with haemophilia B just after birth. At the same time Harry’s mum Olivia found out she was a carrier.⁴ At first, Olivia’s reaction was of worry, but she soon found online support groups and was encouraged by treatment options available to Harry. “I linked in with quite a few other haemophilia mums and discovered that actually Harry could have quite a normal life.”⁴

 

Recommendations and approaches in haemophilia care have changed as the care landscape has progressed. Diagnosed with severe haemophilia A six decades ago, Heinz was excluded from social and physical activities with his school friends for fear of triggering a bleed.⁵ In contrast, today Harry knows that being active can help his joints and he likes to exercise. “I love riding my bike up and down the tracks. I like it because the wind goes in my face, and I get fresh air.”⁴ Harry gets his friends involved too — they look out for him in the playground, “I’ve got lots of friends at school, and they think I’ve got special blood.”⁴

 

Heinz with haemophilia sitting on a bench - smiling

60 years since diagnosis, Heinz continues to live well with haemophilia and enjoy his favourite activities.

 

Daniel recognises that despite major advances, treatments can always improve; “although the standard of care has increased dramatically in recent years, the burden of treatment is still high.” Indeed, over the years, Heinz has found lots of ways to adapt. Treatment methods of the past have affected Heinz’s mobility, but he continues to live well with haemophilia and enjoy his favourite activities. “I love reading but I have difficulties with my elbows when holding books, so I have a stand to help reduce stress on my elbows.”⁵

 

Understanding and treating haemophilia today

Treatment today can be on-demand and preventative and, with access to the right care, people living with haemophilia today can live a normal life.⁶,⁷ Bucket list adventures, such as travelling or going on holiday, are possible for people living with haemophilia today, but it used to be much more difficult. Heinz can relate, noting, “travelling in my youth was very limited; for example, my parents would only travel in selected vacation spots close to a hospital.”⁵

Nowadays, increased understanding of the condition means that the aim of treatment is to help people living with haemophilia live a full life and minimise long-term damage.⁶ Professor Hedner reflects, “I don’t think the numbers of people with haemophilia will decrease, but at least they will have longer lives and are able to contribute to society.”² Daniel is motivated by improving outcomes even further, “although the standard of care has increased dramatically in recent years, the burden of treatment is still high.” With research endeavours continuing, the future looks positive.

 


References

  1. In the blood — haemophilia. PR Monitor News release. Available at:
    http://www.prmonitor.co.za/read_story.php?st=72 [Accessed April 2019].
  2. Siegel-Itzkovich J. J Post 2014. Available at https://www.jpost.com/Health-and-Science/She-stanched-thebleeding-349920 [Accessed April 2019].
  3. Kisiel W. J Thromb Haemost 2009; 7: 1053‒1056.
  4. Inspiring Change in Haemophilia: Harry and Olivia. Novo Nordisk Changing Haemophilia. Available at:
    https://video.novonordisk.com/video/49636013/inspiring-change-in-haemophilia-1 [Accessed April 2019].
  5. Inspiring Change in Haemophilia: Heinz. Novo Nordisk Changing Haemophilia. Available at:
    https://video.novonordisk.com/video/49635758/inspiring-change-in-haemophilia [Accessed April 2019].
  6. World Federation of Haemophilia. About Bleeding Disorders: Treatment. Available at:
    https://www.wfh.org/en/page.aspx?pid=642 [Accessed April 2019].
  7. The Haemophilia Society. Understanding Haemophilia 2017. Available at:
    https://haemophilia.org.uk/wpcontent/uploads/2017/04/Understanding_haemophilia_WEB.pdf [Accessed April 2019].