The impact of factor infusion frequency on health-related quality of life in people with haemophilia

Gabriel Pedra; Pia Christoffersen; Kate Khair; Xin Ying Lee; Sonia O’Hara; Jamie O’Hara; John Pasi

doi:10.17225/jhp00158

Abstract

Abstract Background

Some studies suggest that people with haemophilia (PwH) who use prophylaxis value low frequency of clotting factor administration more than a lower risk of bleeding. However, more frequent infusions offer the potential of reducing joint disease and pain, which in turn may improve functioning and quality of life.

Aims

To explore the impact on health-related quality of life (HRQoL) aspects of haemophilia associated with adherence and annual infusion rate in the context of factors influencing treatment that are important to patients, including prophylaxis, chronic pain, concomitant conditions and hospital admission.

Materials and methods

HRQoL was assessed in participants with severe haemophilia in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study who were using prophylaxis. Patients using on-demand treatment were excluded. This multivariate analysis examined the interaction between factors potentially influencing treatment and HRQoL, and minor and major bleeds.

Results

From the total CHESS population (n=1,285), 338 (26%) participants provided responses for major and minor bleeds and target joints, and 145 (11%) provided EQ-5D-3L responses. Major and minor bleeds were associated with pain. Patients with severe chronic pain reported a substantial negative impact on HRQoL; but this was significantly improved by increases in the annual infusion rate. This was not apparent in participants with mild or moderate pain.

Conclusion

Increasing the frequency of prophylaxis infusions is associated with improved quality of life in PwH who have severe chronic pain. However, increasing the number of infusions per week in those with mild or moderate chronic pain with the intention of improving prophylactic effect may not have the same effect.

Article

View Full Article

References

1. Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al; Treatment Guidelines Working Group on Behalf of The World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1–47. doi: https://doi.org/10.1111/j.1365-2516.2012.02909.x.
2. Ling G, Nathwani AC, Tuddenham EGD. Recent advances in developing specific therapies for haemophilia. Br J Haematol 2018; 181: 161–172. doi: https://doi.org/10.1111/bjh.15084.
3. Kruse-Jarres R, Oldenburg J, Santagostino E, et al. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non-interventional study in a real-world setting. Haemophilia 2019; 25(2): 213–220. doi: https://doi.org/10.1111/hae.13655.
4. Kalnins W, Schelle G, Jost K, et al. Pain therapy in haemophilia in Germany. Patient survey (BESTH study). Hamostaseologie 2015; 35(2): 167–73. doi: https://doi.org/10.5482/HAMO-14-03-0021.
5. Forsyth AL, Witkop M, Lambing A, et al. Associations of quality of life, pain, and self-reported arthritis with age, employment, bleed rate, and utilization of hemophilia treatment center and health care provider services: results in adults with hemophilia in the HERO study. Patient Prefer Adherence 2015; 9: 1549–60. doi: https://doi.org/10.2147/PPA.S87659.
6. Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017; 23: 105–114. doi: https://doi.org/10.1111/hae.13111.
7. Collins P, Chalmers E, Chowdary P, et al. The use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDO. Haemophilia 2016; 22(4): 487–98. doi: https://doi.org/10.1111/hae.13013.
8. den Uijl IE, Fischer K, Van Der Bom JG, et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia 2011; 17(1): 41–4. doi: https://doi.org/10.1111/j.1365-2516.2010.02383.x.
9. Furlan R, Krishnan S, Vietri J. Patient and parent preferences for characteristics of prophylactic treatment in hemophilia. Patient Prefer Adherence 2015; 9: 1687–94. doi: https://doi.org/10.2147/PPA.S92520.
10. Steen Carlsson K, Andersson E, Berntorp E. Preference-based valuation of treatment attributes in haemophilia A using web survey. Haemophilia 2017; 23(6): 894–903. doi: https://doi.org/10.1111/hae.13322.
11. von Mackensen S, Kalnins W, Krucker J, et al. Haemophilia patients’ unmet needs and their expectations of the new extended half-life factor concentrates. Haemophilia 2017; 23(4): 566–574. doi: https://doi.org/10.1111/hae.13221.
12. Witkop M, Guelcher C, Forsyth A, et al. Treatment outcomes, quality of life, and impact of hemophilia on young adults (aged 18–30 years) with hemophilia. Am J Hematol 2015; 90 Suppl 2: S3–10. doi: https://doi.org/10.1002/ajh.24220.
13. Soucie JM, Grosse SD, Siddiqi AE, et al; Hemophilia Treatment Centers Network. The effects of joint disease, inhibitors and other complications on health-related quality of life among males with severe haemophilia A in the United States. Haemophilia 2017; 23(4): e287–e293. doi: https://doi.org/10.1111/hae.13275.
14. O’Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis 2017; 12: 106. doi: https://doi.org/10.1186/s13023-017-0660-y.
15. Camp C, O’Hara J, Hughes D, et al. The relationship between bleeding and EQ-5D in severe haemophilia. Poster P-T-62. WFH World Congress, 24–28 July 2016, Orlando, USA. Available from https://www.postersessiononline.eu/173580348_eu/congresos/WFH2016/aula/-PP-T_62_WFH2016.pdf (accessed 9 July 2020).
16. Fischer K. Low-dose prophylaxis for severe haemophilia: a little goes a long way. Haemophilia 2016; 22(3): 331–3. doi: https://doi.org/10.1111/hae.12853.
17. Mulder K, Llinás A. The target joint. Haemophilia 2004;10 Suppl 4:152–6. doi: https://doi.org/10.1111/j.1365-2516.2004.00976.x.
18. The EuroQol Group. EuroQol – a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199–208. doi: https://doi.org/10.1016/0168-8510(90)90421-9.
19. Brooks R. EuroQol: the current state of play. Health Policy 1996; 37: 53–72. doi: https://doi.org/10.1016/0168-8510(96)00822-6.
20. Hauck JW, Donner A. Wald's test as applied to hypotheses in logit analysis. J Am Stat Assoc 1977; 72: 851–53. doi: https://doi.org/10.2307/2286473.
21. Nugent D, O’Mahony B, Dolan G; International Haemophilia Access Strategy Council. Value of prophylaxis vs on-demand treatment: application of a value framework in hemophilia. Haemophilia 2018; 24(5): 755–765. doi: https://doi.org/10.1111/hae.13589.
22. Chowdary P, Carcao M, Holme PA, et al. Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A. Res Pract Thromb Haemost 2019; 3: 542–554. doi: https://doi.org/10.1002/rth2.12220.
23. Fischer K, van der Bom JG, Mauser-Bunschoten EP, et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001; 7(5): 446–52. doi: https://doi.org/10.1046/j.1365-2516.2001.00545.x.
24. Lambing A, Nichols CD, Munn JE, et al. Patient, caregiver, and provider perceptions of pain and pain management in adolescents and young adults with bleeding disorders. Haemophilia 2017; 23(6): 852–860. doi: https://doi.org/10.1111/hae.13293.
25. Witkop M, Lambing A. Knowledge and attitudes survey in bleeding disorders providers regarding pain. Haemophilia 2015; 21(6): e465–71. doi: https://doi.org/10.1111/hae.12749.
26. Tagliaferri A, Franchini M, Rivolta GF, et al. Pain assessment and management in haemophilia: A survey among Italian patients and specialist physicians. Haemophilia 2018; 24(5): 766–773. doi: https://doi.org/10.1111/hae.13600.
27. Forsyth AL, Gregory M, Nugent D, et al. Haemophilia Experiences, Results and Opportunities (HERO) Study: survey methodology and population demographics. Haemophilia 2014; 20(1): 44–51. doi: https://doi.org/10.1111/hae.12239.
28. Pérez-Robles T, Romero-Garrido JA, Rodriguez-Merchan EC, et al. Objective quantification of adherence to prophylaxis in haemophilia patients aged 12 to 25 years and its potential association with bleeding episodes. Thromb Res 2016; 143: 22–7. doi: https://doi.org/10.1016/j.thromres.2016.04.015.
29. Guedes VG, Corrente JE, Farrugia A, et al. Comparing objective and self-reported measures of adherence in haemophilia. Haemophilia 2019; 25(5): 821–830. doi: https://doi.org/10.1111/hae.13811.
30. Duncan N, Kronenberger W, Roberson C, et al. VERITAS-Pro: a new measure of adherence to prophylactic regimens in haemophilia. Haemophilia 2010; 16(2): 247–55. doi: https://doi.org/10.1111/j.1365-2516.2009.02129.x.

PDF Download

Download PDF

Open in full-page viewer

Authors

Gabriel Pedra

gabriel.pedra@hcdeconomics.com

HCD Economics, Daresbury, UK
Pia Christoffersen

Novo Nordisk A/S, Søborg, Denmark
Kate Khair
Xin Ying Lee

Novo Nordisk A/S, Søborg, Denmark
Sonia O’Hara

HCD Economics, Daresbury, UK
Jamie O’Hara

Faculty of Health and Social Care, University of Chester, UK
John Pasi

Haemophilia Centre, Royal London Hospital, UK