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Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study

Gringeri, A.; Leissinger, C.; Cortesi, P. A.; Jo, H.; Fusco, F.; Riva, S.; Antmen, B.; Berntorp, Erik LU ; Biasoli, C. and Carpenter, S., et al. (2013) In Haemophilia 19(5). p.736-743
Abstract
Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health-related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short-Form (SF)-36 Health Survey and the EQ-5D questionnaire in subjects 14years participating in a prospective, randomized, crossover study comparing 6months of aPCC prophylaxis with 6months of on-demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on-demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in... (More)
Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health-related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short-Form (SF)-36 Health Survey and the EQ-5D questionnaire in subjects 14years participating in a prospective, randomized, crossover study comparing 6months of aPCC prophylaxis with 6months of on-demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on-demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF-36 dimensions except for vitality/energy and physical functioning. After prophylaxis, good responders,' defined as patients experiencing 50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P=0.021), role - physical (P=0.042), bodily pain (P=0.015), and social functioning (P=0.036). Similarly, the EQ-5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on-demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ-5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on-demand treatment (P=0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on-demand treatment. (Less)
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Contribution to journal
publication status
published
subject
keywords
activated prothrombin complex concentrate, health-related quality of, life, haemophilia, inhibitors, prophylaxis
in
Haemophilia
volume
19
issue
5
pages
736 - 743
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000323453200030
  • scopus:84883053042
ISSN
1351-8216
DOI
10.1111/hae.12178
language
English
LU publication?
yes
id
858ea55d-1178-4287-9efd-cc09574c741b (old id 4025672)
date added to LUP
2013-10-01 15:10:42
date last changed
2019-03-05 01:00:52
@article{858ea55d-1178-4287-9efd-cc09574c741b,
  abstract     = {Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health-related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short-Form (SF)-36 Health Survey and the EQ-5D questionnaire in subjects 14years participating in a prospective, randomized, crossover study comparing 6months of aPCC prophylaxis with 6months of on-demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on-demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF-36 dimensions except for vitality/energy and physical functioning. After prophylaxis, good responders,' defined as patients experiencing 50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P=0.021), role - physical (P=0.042), bodily pain (P=0.015), and social functioning (P=0.036). Similarly, the EQ-5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on-demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ-5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on-demand treatment (P=0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on-demand treatment.},
  author       = {Gringeri, A. and Leissinger, C. and Cortesi, P. A. and Jo, H. and Fusco, F. and Riva, S. and Antmen, B. and Berntorp, Erik and Biasoli, C. and Carpenter, S. and Kavakli, K. and Morfini, M. and Negrier, C. and Rocino, A. and Schramm, W. and Windyga, J. and Zulfikar, B. and Mantovani, L. G.},
  issn         = {1351-8216},
  keyword      = {activated prothrombin complex concentrate,health-related quality of,life,haemophilia,inhibitors,prophylaxis},
  language     = {eng},
  number       = {5},
  pages        = {736--743},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study},
  url          = {http://dx.doi.org/10.1111/hae.12178},
  volume       = {19},
  year         = {2013},
}