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Unmet needs of people living with haemophilia

While advances in care and treatment have improved the lives of people with haemophilia, they still face many challenges.1

Research has shown that there are six major unmet needs which need to be addressed to further improve the lives of people with haemophilia.

What are the six major unmet needs?

Bleed protection

Bleeds are a common concern for people with haemophilia. Even with regular treatment, some people with haemophilia still experience bleeds, mainly into the joints and muscles.2,3,4,5

Bleeds can be particularly challenging for people who develop inhibitors (where the body has an immune response to treatment, which means ‘standard’ treatment becomes ineffective), who are twice more likely to have a major bleed.6

Joint health

Some people with haemophilia still face problems with their joints and can experience pain and irreversible damage.

Some people have a ‘target joint’ (a term used for a joint that has more than three bleeds in six months).7,8 Target joints are a major cause of joint disease and severe pain,9 which can worsen with age.10 A European study found that over 40% of target joints eventually require surgery.11

Physical freedom

Some people with haemophilia have issues with the ability to move freely and easily, which limits their ability to participate in daily activities and sports.9

While physical activity offers many benefits, such as increased strength and muscle growth, people with haemophilia are less likely to participate in physical activity compared to the general public due to the fear of self-injury.12

Quality of life

Living with haemophilia can affect a person’s ability to live their life to the fullest. It can cause physical, mental, and social challenges, affecting a person’s overall well-being.13,14,15

Mental health challenges are more common in people with haemophilia compared to the general public.14 A US study reported that half of people with moderate haemophilia experience moderate-to-severe problems with anxiety and depression.16

Treatment preparation and administration

Managing haemophilia can be cumbersome, making adherence challenging.17

Some haemophilia treatments need frequent intravenous (IV) infusions, which can take a long time to administer. Finding a vein can also be difficult, which may lead to pain, scarring, and infection.17 Both elements make it more challenging to adhere to treatment.

School and work

Living with haemophilia can impact activities like school, work, and daily tasks.

Haemophilia can affect a young person’s education, leading to missed days or being unable to complete a formal education.18,19 Adults can experience lifestyle and work challenges due to bleeds, pain, problems with mobility or because of treatment. A US study found that 75% of adults with haemophilia reported that pain has interfered with normal work or housework.20

Why do we need to take action?

While advances in care and treatment have improved the lives of people with haemophilia, they still face many challenges. Together, we can work towards a better future with:

  • Fewer spontaneous bleeds and ultimately striving for zero spontaneous bleeds
  • More freedom from joint damage and disabling pain
  • Improved mental health and minimised impact on day-to-day life and activities

By coming together to take action, we can improve the quality of life for people with haemophilia.

Help us spread the word

Together, with you and our community partners, we champion everyone to learn more and to raise awareness of the unmet needs that impact people with haemophilia.  

References
  1. Srivastava A et al. Haemophilia. 2020;26(Suppl 6):1–158
  2. Chhabra A et al. Blood Coagul Fibrinolysis. 2020;31:186–92
  3. Lewandowska M et al. Res Pract Thromb Haemost 2020;4(Suppl.1)
  4. van Leeuwen et al. J Thromb Haemost. 2023;21(5),1156-63
  5. Wilkins RA et al. BMJ Open. 2022;12:e052358
  6. Oladapo et al. Orphanet J Rare Dis. 2018 Nov 9;13(1):198
  7. Kizilocak et al. Clinical Advances in Hematology & Oncology. 2019;17(6):344-351
  8. Hanley et al. Haemophilia. 2017;1-10
  9. O'Hara S et al. Haemophilia. 2021;27:113-119
  10. Curtis R et al. J Blood Med. 2022 May 9;13:229-241
  11. O'Hara et al. Health Economics Review. 2018;8:1
  12. Goto M et al. J Blood Med. 2016 May 17;7:85-98
  13. Krumb et al. Res Pract Thromb Haemost. 2021;5:e12567
  14. Al-Huniti A et al. Haemophilia. 2020 May;26(3):431-442
  15. Wheeler et al. Blood. 2023;142:508-510
  16. Witkop et al. Haemophilia. 2021;27 Suppl 1:8-16
  17. Thornburg D et al. Patient Prefer Adherence. 2017;11:1677–86
  18. Mahlangu J et al. Haemophilia. 2019;25:382–91
  19. Cutter et al. Eur J Haematol. 2017;98:18–24
  20. Buckner TW et al. Eur J Haematol. 2018;100(Suppl. 1):5–13

Date of preparation: April 2024. HQ24CH00006