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

Silva Zupančić Šalek, Günter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Cedric Hermans, Victor Jiménez-Yuste, Rolf Ljung, Massimo Morfini, Elena Santagostino, Thierry Lambert

Research output: Contribution to journalArticle

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 13th 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 ondemand treatment for patients with inhibitors.

Original languageEnglish
Pages (from-to)77-84
Number of pages8
JournalBlood Transfusion
Volume15
Issue number1
DOIs
Publication statusPublished - Jan 1 2017

Keywords

  • Haemophilia
  • Inhibitors
  • RFVIIa
  • Short-term episodic prophylaxis

ASJC Scopus subject areas

  • Immunology and Allergy
  • Hematology

Fingerprint Dive into the research topics of 'Beyond stopping the bleed: Short-term episodic prophylaxis with recombinant activated factor FVII in haemophilia patients with inhibitors'. Together they form a unique fingerprint.

  • Cite this

    Šalek, S. Z., Auerswald, G., Benson, G., Dolan, G., Duffy, A., Hermans, C., Jiménez-Yuste, V., Ljung, R., Morfini, M., Santagostino, E., & Lambert, T. (2017). Beyond stopping the bleed: Short-term episodic prophylaxis with recombinant activated factor FVII in haemophilia patients with inhibitors. Blood Transfusion, 15(1), 77-84. https://doi.org/10.2450/2015.0127-15