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Beyond stopping the bleed: short-term episodic prophylaxis with recombinant activated factor FVII in haemophilia patients with inhibitors.

Šalek, Silva Zupančić; Auerswald, Günter; Benson, Gary; Dolan, Gerry; Duffy, Anne; Hermans, Cedric; Jiménez-Yuste, Victor; Ljung, Rolf LU ; Morfini, Massimo and Santagostino, Elena, et al. (2017) In Blood Transfusion 15. p.77-84
Abstract
Preventing haemarthroses and arthropathy is a major challenge in patients with haemophilia and inhibitors, as treatment options are limited. One potential strategy is short-term episodic prophylaxis, which extends bypassing agent therapy beyond the resolution of bleeding to include the post-bleed inflammatory phase. At the 13(th) Zürich Haemophilia Forum, an expert panel reviewed the rationale behind this strategy, explored its current use with recombinant activated factor VII (rFVIIa) and considered treatment monitoring and optimisation. Two protocols are currently used for short-term episodic prophylaxis, both of which stipulate on-demand rFVIIa until resolution of bleeding, followed by daily dosing for ≥3 days to prevent re-bleeds.... (More)
Preventing haemarthroses and arthropathy is a major challenge in patients with haemophilia and inhibitors, as treatment options are limited. One potential strategy is short-term episodic prophylaxis, which extends bypassing agent therapy beyond the resolution of bleeding to include the post-bleed inflammatory phase. At the 13(th) Zürich Haemophilia Forum, an expert panel reviewed the rationale behind this strategy, explored its current use with recombinant activated factor VII (rFVIIa) and considered treatment monitoring and optimisation. Two protocols are currently used for short-term episodic prophylaxis, both of which stipulate on-demand rFVIIa until resolution of bleeding, followed by daily dosing for ≥3 days to prevent re-bleeds. Short-term episodic prophylaxis should be individualised to optimise outcomes, perhaps through early treatment initiation or by combining rFVIIa with other treatments (e.g. factor VIII, tranexamic acid). Encouraging treatment compliance can also improve outcomes. Additionally, there is a need to develop objective clinical outcome measures, biomarkers and imaging protocols that can monitor treatment outcomes and joint disease in patients with inhibitors. A proactive approach incorporating a systematic package of care is needed. Currently, short-term episodic prophylaxis with rFVIIa may be an alternative treatment option to on-demand treatment for patients with inhibitors. (Less)
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Blood Transfusion
volume
15
pages
77 - 84
publisher
SIMITI SERVIZI SRL
external identifiers
  • pmid:26674816
  • scopus:85012146303
ISSN
1723-2007
DOI
10.2450/2015.0127-15
language
English
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yes
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5ed8bdf9-6d4d-42f3-ac21-df9ab8449648 (old id 8504543)
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http://www.ncbi.nlm.nih.gov/pubmed/26674816?dopt=Abstract
date added to LUP
2016-01-05 20:27:34
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2017-04-09 04:32:23
@article{5ed8bdf9-6d4d-42f3-ac21-df9ab8449648,
  abstract     = {Preventing haemarthroses and arthropathy is a major challenge in patients with haemophilia and inhibitors, as treatment options are limited. One potential strategy is short-term episodic prophylaxis, which extends bypassing agent therapy beyond the resolution of bleeding to include the post-bleed inflammatory phase. At the 13(th) Zürich Haemophilia Forum, an expert panel reviewed the rationale behind this strategy, explored its current use with recombinant activated factor VII (rFVIIa) and considered treatment monitoring and optimisation. Two protocols are currently used for short-term episodic prophylaxis, both of which stipulate on-demand rFVIIa until resolution of bleeding, followed by daily dosing for ≥3 days to prevent re-bleeds. Short-term episodic prophylaxis should be individualised to optimise outcomes, perhaps through early treatment initiation or by combining rFVIIa with other treatments (e.g. factor VIII, tranexamic acid). Encouraging treatment compliance can also improve outcomes. Additionally, there is a need to develop objective clinical outcome measures, biomarkers and imaging protocols that can monitor treatment outcomes and joint disease in patients with inhibitors. A proactive approach incorporating a systematic package of care is needed. Currently, short-term episodic prophylaxis with rFVIIa may be an alternative treatment option to on-demand treatment for patients with inhibitors.},
  author       = {Šalek, Silva Zupančić and Auerswald, Günter and Benson, Gary and Dolan, Gerry and Duffy, Anne and Hermans, Cedric and Jiménez-Yuste, Victor and Ljung, Rolf and Morfini, Massimo and Santagostino, Elena and Lambert, Thierry},
  issn         = {1723-2007},
  language     = {eng},
  pages        = {77--84},
  publisher    = {SIMITI SERVIZI SRL},
  series       = {Blood Transfusion},
  title        = {Beyond stopping the bleed: short-term episodic prophylaxis with recombinant activated factor FVII in haemophilia patients with inhibitors.},
  url          = {http://dx.doi.org/10.2450/2015.0127-15},
  volume       = {15},
  year         = {2017},
}