Impact of prophylaxis on health-related quality of life of boys with hemophilia: An analysis of pooled data from 9 countries.
health‐related quality of life
hemophilia A
hemophilia B
outcome measures
pediatrics
Journal
Research and practice in thrombosis and haemostasis
ISSN: 2475-0379
Titre abrégé: Res Pract Thromb Haemost
Pays: United States
ID NLM: 101703775
Informations de publication
Date de publication:
Jul 2019
Jul 2019
Historique:
received:
03
12
2018
accepted:
25
02
2019
entrez:
12
7
2019
pubmed:
12
7
2019
medline:
12
7
2019
Statut:
epublish
Résumé
Prophylaxis reduces the frequency of bleeds in boys with severe hemophilia and is the standard care for their management in resource-abundant countries. The effect of prophylaxis on Health-Related Quality of Life (HRQoL) has not been established, because the sample sizes of most studies are too small to explore the relationship of multiple factors that influence HRQoL. The aim of this study was to assess the impact of hemophilia severity and treatment regimen on HRQoL and to establish the minimum important difference (MID) using the international level of score distributions. HRQoL data were pooled from 7 studies across 9 countries. HRQoL was measured using the Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT). A mixed-effect linear regression analysis was employed to assess the impact of prophylaxis on the CHO-KLAT score. Data from 401 boys with hemophilia were analyzed (57.6% severe hemophilia and 57.6% receiving prophylaxis). The model revealed that receiving prophylaxis was significantly associated with higher HRQoL (regression coefficient 8.5, 95% confidence interval [CI] 3.9-13.1). Boys with severe hemophilia had a significantly lower HRQoL as compared to boys with moderate and mild hemophilia whose CHO-KLAT scores were 7.0 and 6.6 points higher, respectively. There was a significant interaction between treatment and disease severity ( This study confirms the positive effect of prophylaxis on HRQoL in boys with hemophilia in a real-world setting and provides initial benchmarks for interpreting HRQoL scores based on use of the CHO-KLAT instrument.
Sections du résumé
BACKGROUND
BACKGROUND
Prophylaxis reduces the frequency of bleeds in boys with severe hemophilia and is the standard care for their management in resource-abundant countries. The effect of prophylaxis on Health-Related Quality of Life (HRQoL) has not been established, because the sample sizes of most studies are too small to explore the relationship of multiple factors that influence HRQoL.
METHODS
METHODS
The aim of this study was to assess the impact of hemophilia severity and treatment regimen on HRQoL and to establish the minimum important difference (MID) using the international level of score distributions. HRQoL data were pooled from 7 studies across 9 countries. HRQoL was measured using the Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT). A mixed-effect linear regression analysis was employed to assess the impact of prophylaxis on the CHO-KLAT score.
RESULTS
RESULTS
Data from 401 boys with hemophilia were analyzed (57.6% severe hemophilia and 57.6% receiving prophylaxis). The model revealed that receiving prophylaxis was significantly associated with higher HRQoL (regression coefficient 8.5, 95% confidence interval [CI] 3.9-13.1). Boys with severe hemophilia had a significantly lower HRQoL as compared to boys with moderate and mild hemophilia whose CHO-KLAT scores were 7.0 and 6.6 points higher, respectively. There was a significant interaction between treatment and disease severity (
CONCLUSIONS
CONCLUSIONS
This study confirms the positive effect of prophylaxis on HRQoL in boys with hemophilia in a real-world setting and provides initial benchmarks for interpreting HRQoL scores based on use of the CHO-KLAT instrument.
Identifiants
pubmed: 31294327
doi: 10.1002/rth2.12202
pii: S2475-0379(22)01604-1
pmc: PMC6611476
doi:
Types de publication
Journal Article
Langues
eng
Pagination
397-404Références
Haemophilia. 2017 Jul;23(4):497-510
pubmed: 28429867
N Engl J Med. 2007 Aug 9;357(6):535-44
pubmed: 17687129
Hematology Am Soc Hematol Educ Program. 2013;2013:52-5
pubmed: 24319162
Res Pract Thromb Haemost. 2019 Apr 23;3(3):397-404
pubmed: 31294327
Haemophilia. 2014 Nov;20(6):794-9
pubmed: 25273150
J Thromb Haemost. 2016 May;14(5):1105-9
pubmed: 27186714
Thromb Haemost. 2001 Mar;85(3):560
pubmed: 11307831
Haemophilia. 2003 May;9 Suppl 1:1-4
pubmed: 12709030
Haemophilia. 2016 Nov;22(6):894-897
pubmed: 27456858
Haemophilia. 2018 Jul;24(4):e179-e186
pubmed: 29855125
J Med Econ. 2015 Jun;18(6):457-65
pubmed: 25660324
Haemophilia. 2015 May;21(3):351-357
pubmed: 25471939
Lancet Haematol. 2018 Jun;5(6):e252-e260
pubmed: 29731369
Haemophilia. 2013 Jan;19(1):27-34
pubmed: 23231016
Haemophilia. 2017 May;23(3):430-436
pubmed: 28345299
Haemophilia. 2011 Jan;17(1):70-4
pubmed: 20579111
Value Health. 2013 Jul-Aug;16(5):837-41
pubmed: 23947978
Pediatr Blood Cancer. 2006 Sep;47(3):305-11
pubmed: 16206209
Haemophilia. 2017 Sep;23(5):682-688
pubmed: 28440005
Haemophilia. 2001 Jan;7(1):99-102
pubmed: 11136389
Haemophilia. 2015 Sep;21(5):e344-58
pubmed: 26390060
Med Care. 2003 May;41(5):582-92
pubmed: 12719681
Haemophilia. 2012 May;18(3):353-7
pubmed: 22103664
J Thromb Haemost. 2014 Nov;12(11):1935-9
pubmed: 25059285
Haemophilia. 2004 Mar;10 Suppl 1:34-43
pubmed: 14987247
Haemophilia. 2007 Jul;13(4):345-50
pubmed: 17610546