Hemostasis and bleed prevention are essential needs for patients with hemophilia. However, care should address lifestyle and treatment concerns as well as disease burdens. Many everyday concerns remain and can negatively impact patient adherence, social lives, work, school, and ability to travel.1,2
Repeated bleeds can lead to joint damage and, over time, mobility loss. Joint damage may also lead to joint pain, and in some cases arthritis, even for hemophilia patients under the age of 24.3-6
Hip and Knee ReplacementsCompared to the general population, people with hemophilia are more likely to require hip or knee replacements. In a study, workers with hemophilia were 12.1% more likely to have had knee and hip replacements than their counterparts without hemophilia.6,7
Inhibitor DevelopmentUp to 35% of people with hemophilia A, and 3% with hemophilia B, may develop inhibitors to factor concentrate, limiting their therapy options. Patients who have developed inhibitors to factor concentrate have a higher rate of severe bleeds, hospitalization, treatment costs, and mortality. 8,9
Factor prophylaxis treatment may require frequent intravenous dosing, which may result in soreness, bruising, and physical discomfort for patients. Patients may require their doses every 2 weeks or every other day.2,10
Venous Access IssuesDue to venous access difficulty, some patients, including children, may need central venous access devices (CVADs), which are associated with risk of infection and thrombosis. Ageing veins can also become sclerotic and fragile. Intravenous infusions may not be as feasible for elderly hemophilia patients or their assisting caregiver.11,12
Adherence to RegimensAdolescents (ages 13-17) and young adults (18-30) may struggle with treatment adherence due to complications with venous access or perceived burden. The transition to self-treatment, or a move away from home, may negatively impact adherence to prophylaxis.10
Both hemophilia and prophylaxis may make work difficult—patients must consider stress on joints and care accommodations. In one study, 47.3% of patients felt their severe hemophilia affected their career choice. A majority of patients report that prophylaxis restricts their participation in sports and their ability to travel for extended periods.2,13-18
Self-Treatment TransitionMany young people with hemophilia still require parental or caretaker assistance, even in their later teenage years. Teen patients have reported another significant challenge is leaving their long-term pediatric care provider for unfamiliar adult clinics.10,19
Storage RestrictionsPatients report that prophylaxis treatment storage temperature has a significant influence on treatment preference. In one survey, participants reported that 47.4% had discarded unused product. In 26.6% of cases, this was due to storage problems.18,20
References: 1. Ozelo MC, Yamaguti-Hayakawa GG. Impact of novel hemophilia therapies around the world. Res Pract Thromb Haemost. 2022;6(3):e12695. doi:10.1002/rth2.12695 2. Brod M, Bushnell DM, Neergaard JS, Waldman LT, Busk AK. Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM). J Patient Rep Outcomes. 2023;7(1):17. doi:10.1186/s41687-023-00550-6 3. O’Hara J, Walsh S, Camp C, et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life. Health Qual Life Outcomes. 2018;16(1):84. doi:10.1186/s12955-018-0908-9 4. O’Hara S, Castro FA, Black J, et al. Disease burden and remaining unmet need in patients with haemophilia A treated with primary prophylaxis. Haemophilia. 2021;27(1):113-119. doi:10.1111/hae.14171 5. Curtis R, Baker J, Riske B, et al. Young adults with hemophilia in the U.S.: demographics, comorbidities, and health status. Am J Hematol. 2015;90(suppl 2):S11-S16. doi:10.1002/ajh.24218 6. Miesbach W, O’Mahony B, Key NS, Makris M. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia. 2019;25(4):545-557. doi:10.1111/hae.13769 7. Chu WM, Ho HE, Wang JD, et al. Risk of major comorbidities among workers with hemophilia: A 14-year population-based study. Medicine (Baltimore). 2018;97(6):e9803. doi:10.1097/MD.0000000000009803 8. Meeks SL, Batsuli G. Hemophilia and inhibitors: current treatment options and potential new therapeutic approaches. Hematology Am Soc Hematol Educ Program. 2016;2016(1):657-662. doi:10.1182/asheducation-2016.1.657 9. Soucie JM, Miller CH, Kelly FM, Oakley M, Brown DL, Kucab P. A public health approach to the prevention of inhibitors in hemophilia. Am J Prev Med. 2014;47(5):669-673. doi:10.1016/j.amepre.2014.07.007 10. Srivastava A, Santagostino E, Dougall A, et al.;WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26(suppl 6):1-158. doi:10.1111/hae.14046 11. Valentino LA, Kapoor M. Central venous access devices in patients with hemophilia. Expert Rev Med Devices. 2005;2(6):699-711. doi:10.1586/17434440.2.6.699 12. Gabriel J. Understanding the challenges to vascular access in an ageing population. Br J Nurs. 2017;26(14):S15-S23. doi:10.12968/bjon.2017.26.14.S15 13. National Hemophilia Foundation, CDC. Guidelines for growing: an action plan for teens with bleeding disorders. 2011. National Hemophilia Foundation. https://www.cdc.gov/ncbddd/hemophilia/documents/12pg_g4g_9-12.pdf 14. O’Connell D. Career decisions for teens with bleeding disorders. HemAware. July 1, 2009. Updated April 15, 2014. Accessed April 2024 https://hemaware.org/life/career-decisions-teens-bleeding-disorders. 15. Quon D, Reding M, Guelcher C, et al. Unmet needs in the transition to adulthood: 18- to 30-year-old people with hemophilia. Am J Hematol. 2015;90(suppl 2):S17-S22. doi:10.1002/ajh.24219 16. Cutter S, Molter D, Dunn S, et al. Impact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) study. Eur J Haematol. 2017;98 (suppl 86):18-24. doi:10.1111/ejh.12851 17. van Balen EC, Hassan S, Smit C, et al. Socioeconomic participation of persons with hemophilia: results from the sixth hemophilia in the Netherlands study. Res Pract Thromb Haemost. 2022;6(6):e12741. doi:10.1002/rth2.12741 18. Tischer B, Marino R, Napolitano M. Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice. Patient Prefer Adherence. 2018;12:431-441. doi:10.2147/PPA.S151812 19. Sterling LE, Nyhof-Young J, Blanchette VS, Breakey VR. Growing up with hemophilia: assessing teens’ experiences and needs during transition to adult health care. UTMJ. 2013;90(4):149-154. 20. Napolitano M, Nøhr AM. The effect of fluctuating temperature on the stability of turoctocog alfa for hemophilia A. Drugs R D. 2019;19(4):381-390. doi:10.1007/s40268-019-00290-3
