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Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors

Hoots, W. K. ; Ebbesen, L. S. ; Konkle, B. A. ; Auerswald, G. K. -H. ; Roberts, H. R. ; Weatherall, J. ; Ferran, J. -M. ; Ljung, Rolf LU orcid ; Bianco, R. Perez and Ozelo, M. Castro , et al. (2008) In Haemophilia 14(3). p.466-466
Abstract
Haemophilia patients with inhibitors characteristically have impaired joint function and reduced health-related quality of life (HRQoL). This analysis examined whether secondary prophylaxis with recombinant activated factor VII (rFVIIa) improves HRQoL vs. conventional on-demand therapy in patients with haemophilia with inhibitors and frequent bleeds. After a 3-month preprophylaxis period, 22 patients received daily rFVIIa prophylaxis (90 or 270 mu g kg(-1)) for 3 months, followed by 3 months' postprophylaxis. Days of hospitalization, absence from school/work and mobility aids requirements were recorded. HRQoL was assessed by EuroQoL (EQ-5D) questionnaire, visual analogue scale (VAS), derived Time to Trade-Off (TTO) scores and Quality... (More)
Haemophilia patients with inhibitors characteristically have impaired joint function and reduced health-related quality of life (HRQoL). This analysis examined whether secondary prophylaxis with recombinant activated factor VII (rFVIIa) improves HRQoL vs. conventional on-demand therapy in patients with haemophilia with inhibitors and frequent bleeds. After a 3-month preprophylaxis period, 22 patients received daily rFVIIa prophylaxis (90 or 270 mu g kg(-1)) for 3 months, followed by 3 months' postprophylaxis. Days of hospitalization, absence from school/work and mobility aids requirements were recorded. HRQoL was assessed by EuroQoL (EQ-5D) questionnaire, visual analogue scale (VAS), derived Time to Trade-Off (TTO) scores and Quality Adjusted Life Years (QALYs). rFVIIa prophylaxis significantly (P < 0.0001) reduced bleeding frequency vs. prior on-demand therapy. Hospitalization (5.9% vs. 13.5%; P = 0.0026) and absenteeism from school/work (16.7% vs. 38.7%; P = 0.0127) decreased during prophylaxis; these effects tended to be maintained during postprophylaxis. HRQoL (evaluated by EQ-5D) tended to improve during and after rFVIIa prophylaxis. Notably, pain decreased and mobility increased in 40.9% and 27.3% of patients, respectively, at the end of the postprophylaxis period vs. preprophylaxis. Median VAS score increased from 66 to 73 (P = 0.048), and TTO scores suggested better HRQoL (0.62 vs. 0.76; P = 0.054) during postprophylaxis than preprophylaxis. Small to moderate changes in effect sizes were reported for VAS and TTO scores. Median QALYs were 0.68 (VAS) and 0.73 (TTO). Reductions in bleeding frequency with secondary rFVIIa prophylaxis were associated with improved HRQoL vs. on-demand therapy. (Less)
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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
recombinant activated factor VII, inhibitors, haemophilia, HRQoL, secondary prophylaxis
in
Haemophilia
volume
14
issue
3
pages
466 - 466
publisher
Wiley-Blackwell
external identifiers
  • wos:000255463700007
  • scopus:43149116111
ISSN
1351-8216
DOI
10.1111/j.1365-2516.2008.01654.x
language
English
LU publication?
yes
id
1b80232c-0a5e-459e-890e-0478a08febf8 (old id 1204681)
date added to LUP
2016-04-01 12:16:56
date last changed
2022-05-14 20:06:11
@article{1b80232c-0a5e-459e-890e-0478a08febf8,
  abstract     = {{Haemophilia patients with inhibitors characteristically have impaired joint function and reduced health-related quality of life (HRQoL). This analysis examined whether secondary prophylaxis with recombinant activated factor VII (rFVIIa) improves HRQoL vs. conventional on-demand therapy in patients with haemophilia with inhibitors and frequent bleeds. After a 3-month preprophylaxis period, 22 patients received daily rFVIIa prophylaxis (90 or 270 mu g kg(-1)) for 3 months, followed by 3 months' postprophylaxis. Days of hospitalization, absence from school/work and mobility aids requirements were recorded. HRQoL was assessed by EuroQoL (EQ-5D) questionnaire, visual analogue scale (VAS), derived Time to Trade-Off (TTO) scores and Quality Adjusted Life Years (QALYs). rFVIIa prophylaxis significantly (P &lt; 0.0001) reduced bleeding frequency vs. prior on-demand therapy. Hospitalization (5.9% vs. 13.5%; P = 0.0026) and absenteeism from school/work (16.7% vs. 38.7%; P = 0.0127) decreased during prophylaxis; these effects tended to be maintained during postprophylaxis. HRQoL (evaluated by EQ-5D) tended to improve during and after rFVIIa prophylaxis. Notably, pain decreased and mobility increased in 40.9% and 27.3% of patients, respectively, at the end of the postprophylaxis period vs. preprophylaxis. Median VAS score increased from 66 to 73 (P = 0.048), and TTO scores suggested better HRQoL (0.62 vs. 0.76; P = 0.054) during postprophylaxis than preprophylaxis. Small to moderate changes in effect sizes were reported for VAS and TTO scores. Median QALYs were 0.68 (VAS) and 0.73 (TTO). Reductions in bleeding frequency with secondary rFVIIa prophylaxis were associated with improved HRQoL vs. on-demand therapy.}},
  author       = {{Hoots, W. K. and Ebbesen, L. S. and Konkle, B. A. and Auerswald, G. K. -H. and Roberts, H. R. and Weatherall, J. and Ferran, J. -M. and Ljung, Rolf and Bianco, R. Perez and Ozelo, M. Castro and de Oliveira, M. Hermida and Lissitchkov, T. and Chua, M. Ng and Matysiak, M. J. and Windyga, J. and Serban, M. Andras and Arion, C. V. and Colita, D. N. and Rusen, L. and Vdovin, V. and Mahlangu, J. and Hernandez, F. and Perez, R. and Serdano, C. and Kavakli, K. and Arkin, S. and Brown, D. and Di Paola, J. and Lewis, B. and Konkle, B. A. and Leissinger, C. and Lusher, J. and Neufeld, E. and Shafer, F. and Shapiro, A. and Valentino, L. and Young, G.}},
  issn         = {{1351-8216}},
  keywords     = {{recombinant activated factor VII; inhibitors; haemophilia; HRQoL; secondary prophylaxis}},
  language     = {{eng}},
  number       = {{3}},
  pages        = {{466--466}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{Haemophilia}},
  title        = {{Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors}},
  url          = {{http://dx.doi.org/10.1111/j.1365-2516.2008.01654.x}},
  doi          = {{10.1111/j.1365-2516.2008.01654.x}},
  volume       = {{14}},
  year         = {{2008}},
}