Adherence and Quality of Life in Adult Patients With Haemophilia A, Haemophilia B and Von Willebrand Disease: A National Cross-Sectional Survey.
Von Willebrand disease
blood coagulation disorder
haemophilia A
haemophilia B
medication adherence
quality of life
Journal
European journal of haematology
ISSN: 1600-0609
Titre abrégé: Eur J Haematol
Pays: England
ID NLM: 8703985
Informations de publication
Date de publication:
10 Oct 2024
10 Oct 2024
Historique:
revised:
26
09
2024
received:
18
07
2024
accepted:
27
09
2024
medline:
11
10
2024
pubmed:
11
10
2024
entrez:
11
10
2024
Statut:
aheadofprint
Résumé
New treatments for patients with bleeding disorders (PWB) have emerged, including products with extended half-life and subcutaneous administration. These less frequent treatments can potentially enhance quality of life (QoL), but adherence becomes critically important. To investigate adherence and QoL among PWB and explore the correlation between treatment adherence and QoL in adult patients with haemophilia A (HA), haemophilia B (HB) and Von Willebrand disease (vWD) in Denmark. This survey used disease-specific patient-reported questionnaires: Veritas-PRO and Veritas-PRN to measure adherence, and Haemo-A-QoL and VWD-QoL to assess QoL. Responses were obtained from 149 patients with HA, 32 with HB and 118 with vWD. Adherence was reported by 87.1% of patients on prophylaxis and 71.2% of patients treated on demand, according to Veritas-PRO and Veritas-PRN cut-off scores. High QoL was generally reported, decreasing with age in HA and HB, but not in vWD. Danish patients with HA, HB and vWD reported high QoL and high adherence to prescribed treatments. There was no correlation between treatment adherence and QoL among the different patient groups. These findings highlight the need for further research to better understand adherence behaviours and identify opportunities to further improve QoL in PWB.
Sections du résumé
BACKGROUND
BACKGROUND
New treatments for patients with bleeding disorders (PWB) have emerged, including products with extended half-life and subcutaneous administration. These less frequent treatments can potentially enhance quality of life (QoL), but adherence becomes critically important.
AIM
OBJECTIVE
To investigate adherence and QoL among PWB and explore the correlation between treatment adherence and QoL in adult patients with haemophilia A (HA), haemophilia B (HB) and Von Willebrand disease (vWD) in Denmark.
METHOD
METHODS
This survey used disease-specific patient-reported questionnaires: Veritas-PRO and Veritas-PRN to measure adherence, and Haemo-A-QoL and VWD-QoL to assess QoL.
RESULTS
RESULTS
Responses were obtained from 149 patients with HA, 32 with HB and 118 with vWD. Adherence was reported by 87.1% of patients on prophylaxis and 71.2% of patients treated on demand, according to Veritas-PRO and Veritas-PRN cut-off scores. High QoL was generally reported, decreasing with age in HA and HB, but not in vWD.
CONCLUSION
CONCLUSIONS
Danish patients with HA, HB and vWD reported high QoL and high adherence to prescribed treatments. There was no correlation between treatment adherence and QoL among the different patient groups. These findings highlight the need for further research to better understand adherence behaviours and identify opportunities to further improve QoL in PWB.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Department of Hematology, Copenhagen University Hospital, Rigshospitalet, Denmark
Informations de copyright
© 2024 The Author(s). European Journal of Haematology published by John Wiley & Sons Ltd.
Références
A. Srivastava, E. Santagostino, A. Dougall, et al., “WFH Guidelines for the Management of Hemophilia, 3rd Edition,” Haemophilia 26, no. 6 (2020): 1–158.
M. C. Ar, C. Balkan, and K. Kavaklı, “Extended Half‐Life Coagulation Factors: A New Era in the Management of Hemophilia Patients,” Turkish Journal of Hematology 36, no. 3 (2019): 141–154.
WHO, Adherence to Long‐Term Therapies: Evidence for Action (Geneva, Switzerland: World Health Orginasation, 2013).
N. Duncan, C. Roberson, and A. Shapiro, “Comment on: Khair K. Compliance, Concordance and Adherence: What Are We Talking About? Haemophilia Sept 2014; 20(5): 601–3,” Haemophilia 20 (2014): e235–e236.
L. H. Schrijvers, M. Beijlevelt‐van der Zande, M. Peters, et al., “Adherence to Prophylaxis and Bleeding Outcome in Haemophilia: A Multicenter Study,” British Journal of Haematology 174 (2016): 454–460.
K. Mulder and A. Linás, “The Target Joint,” Haemophilia 10 (2004): 152–156.
S. B. van Os, N. A. Troop, K. R. Sullivan, and D. P. Hart, “Adherence to Prophylaxis in Adolescents and Young Adults With Severe Haemophilia: A Quantitative Study With Patients,” PLoS One 12, no. 1 (2017): e0169880.
M. Witkop, J. M. McLaughlin, T. L. Anderson, J. E. Munn, A. Lambing, and B. Tortella, “Predictors of Non‐Adherence to Prescribed Prophylactic Clotting‐Factor Treatment Regimens Among Adolescent and Young Adults With a Bleeding Disorder,” Haemophilia 22 (2016): e245–e250.
L. H. Schrijvers, M. C. Kars, M. Beijlevelt‐van der Zande, M. Peters, M. J. Schuurmans, and K. Fischer, “Unravelling Adherence to Prophylaxis in Haemophilia: A Patients' Perspective,” Haemophilia 21 (2015): 612–621.
K. Lindvall, L. Colstrup, I. M. Wollter, et al., “Compliance With Treatment and Understanding of Own Disease in Patients With Severe and Moderate Haemophilia,” Haemophilia 12 (2006): 47–51.
G. Puthenveetil and D. Nugent, “Hemophilia—Impact of Recent Advances on Management,” Indian Journal of Pediatrics 87 (2019): 134–140.
F. Peyvandi, I. Garagiola, M. Boscarino, A. Ryan, C. Hermans, and M. Makris, “Real‐Life Experience in Switching to New Extended Half‐Life Products at European Haemophilia Centres,” Haemophilia 25 (2019): 1–7.
M. Hacker, S. Geraghty, and M. Manco‐Johnson, “Barriers to Compliance With Prophylaxis Therapy in Haemophilia,” Haemophilia 12 (2001): 392–396.
L. Schrijvers, M. H. Cnossen, M. Beijlevelt‐Van der Zande, M. Peters, M. J. Schuurmans, and K. Fischer, “Defining Adherence to Prophylaxis in Haemophilia,” Haemophilia 22 (2016): 311–314.
S. Du Triel, J. Rice, and C. Leissinger, “Quantifying Adherence to Treatment and Its Relationship to Quality of Life in a Well‐Characterized Haemophilia Population,” Haemophilia 13 (2007): 493–501.
E. M. de Wee, E. P. Mauser‐Bunschoten, J. G. van der Bom, et al., “Health‐Related Quality of Life Among Adult Patients With Moderate and Severe Von Willebrand Disease,” Journal of Thrombosis and Haemostasis 8 (2010): 1492–1499.
C. Schnohr, O. Ekholm, L. H. Poulsen, et al., “Health and Quality of Life of Patients With Haemophilia: A National Study of 124 Danish Men,” Haemophilia 29, no. 2 (2023): 538–544.
S. Von Mackensen, A. Gringeri, and Haema‐A‐QoL Study Group, “Development and Pilot Testing of a Disease‐Specific Quality of Life Questionnaire for Adult Patients With Haemophilia (Haem‐A‐QoL),” Blood 104, no. 11 (2004): 2214.
L. Chevallet, J. Weatherall, and S. von Mackensen, “Linguistic Validation of the Haemo‐QoL and Haem‐A‐QoL for Use in International Studies,” Value in Health 11 (2008): A165.
S. Von Mackensen, “Quality of Life in Women With Bleedning Disorders,” Haemophilia 17 (2011): 33–37.
A. Borel Derlon, J. Goudemand, D. Deprez, et al., “WISH‐QoL Study: Assessment of Health‐Related Quality of Life and Health‐Economic Aspects in Patients With Von Willebrand Disease in France: Results From the 2nd Interim Analysis on the 355 Enrolled Patients,” Blood 128, no. 22 (2016): 1395, https://abstracts.isth.org/abstract/wish‐qol‐study‐assessment‐of‐health‐related‐quality‐of‐life‐and‐health‐economic‐aspects‐in‐patients‐with‐von‐willebrand‐disease‐in‐france‐results‐from‐the‐2nd‐interim‐analysis‐on‐the‐355‐enrolled‐pa/.
C. V. Denis, S. Susen, and P. J. Lenting, “Von Willebrand Disease: What Does the Future Hold, in Blood,” Blood 137 (2021): 2299–2306.
N. Duncan, W. Kronenberger, C. Roberson, and A. Shapiro, “VERITAS‐Pro: A New Measure of Adherence to Prophylactic Regimens in Haemophilia,” Haemophilia 16 (2010): 247–255.
N. Duncan, W. G. Kronenberger, C. P. Roberson, and A. D. Shapiro, “VERITAS‐PRN: A New Measure of Adherence to Episodic Treatment Regimens in Haemophilia,” Haemophilia 16 (2010): 47–53.
J. Oldenburg, H. Tran, F. Peyvandi, et al., “Health‐Related Quality of Life and Health Status in Adolescent and Adult People With Haemophilia A Without Factor VIII Inhibitors—A Non Interventional Study,” Haemophilia 27 (2021): 398–407.
S. Haowei, M. Yang, M. C. Poon, et al., “The Impact of Extended Half‐Life Factor Concentrates on Patient Reported Health Outcome Measures in Persons With Hemophilia A and Hemophilia B,” Research & Practice in Thrombosis & Haemostasis 5 (2021): e12601.
R. Barr, J. Sek, J. Horsman, et al., “Health Status and Health‐Related Quality of Life Assosiated With Von Willebrand Disease,” American Journal of Hematology 73 (2003): 108–114.
I. Govorov, L. Ekelund, R. Chaireti, et al., “Heavy Menstrual Bleeding and Health‐Assosiated Quality of Life in Women With Von Willebrand's Disease,” Experimental and Therapeutic Medicine 11 (2016): 1923–1929.
Y. Xu, M. Deforest, J. Grabell, W. Hopman, and P. James, “Relative Contributions of Bleeding Scores and Iron Status on Health‐Related Quality of Life in Von Willebrand Disease: A Cross‐Sectional Study,” Haemophilia 23 (2017): 115–121.
K. P. M. van Galen, K. Meijer, H. C. Vogely, et al., “Joint Surgery in Von Willebrand Disease: A Multicentre Cross‐Sectional Study,” Haemophilia 22 (2016): 256–262.
D. Q. Tran, V. Barry, A. Antun, M. Ribeiro, S. Stein, and C. L. Kempton, “Physician Trust and Depression Influence Anherence to Factor Replacement: A Single Center Cross‐Sectional Study,” Haemophilia 23 (2017): 98–104.
J. M. McLaughlin, M. L. Witkop, A. Lambing, T. L. Anderson, J. Munn, and B. Tortella, “Better Adherence to Prescribed Treatment Regimen Is Related to Less Chronic Pain Among Adolescents and Young Adults With Moderate or Severe Haemophilia,” Haemophilia 20 (2014): 506–512.
F. Saleem, M. A. Hassali, A. A. Shafie, et al., “Does Treatment Adherence Correlates With Health Related Quality of Life? Findings From a Cross Sectional Study,” BMC Public Health 12 (2012): 318.
J. Hoefnagels, M. C. Kars, K. Fischer, R. E. G. Schutgens, and L. H. Schrijvers, “The Perspectives of Adolescents and Young Adults on Adherence to Prophylaxis in Hemophilia: A Qualitative Study,” Patient Preference and Adherence 14 (2020): 163–171.
P. de Moerloose, W. Urbancik, H. M. van den Berg, and M. Richards, “A Survey of Adherence to Haemophilia Therapy in Six European Countries: Results and Recommendations,” Haemophilia 14 (2008): 931–938.
L. Palareti, G. Melotti, F. Cassis, S. J. Nevitt, A. Iorio, and Cochrane Cystic Fibrosis and Genetic Disorders Group, “Psychological Interventions for People With Hemophilia,” Cochrane Database of Systematic Reviews 3 (2020): 68.
J. Hoefnagels, K. Fischer, R. A. T. Bos, et al., “A Feasibility Study on Two Tailored Interventions to Improve Adherence in Adults With Haemophilia,” Pilot and Feasibility Studies 6 (2020): 189.
V. Guedes, J. E. Corrente, A. Farrugia, S. Thomas, P. A. Wachholz, and E. I. de Oliveira Vidal, “Comparing Objective and Self‐Reported Measures of Adherence in Haemophilia,” Haemophilia 25 (2019): 821–830.