title: Beyond stopping the bleed: short-term episodic prophylaxis with recombinant activated factor FVII in haemophilia patients with inhibitors.
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 13thZürich 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.
authors.affiliation: National Haemophilia Centre, University Hospital Centre Zagreb, Zagreb, Croatia.
authors.forename: Silva Zupančić
authors.initials: SZ
authors.lastname: Šalek
authors.affiliation: Klinikum Bremen-Mitte, Professor Hess Children's Hospital, Bremen, Germany.
authors.forename: Günter
authors.initials: G
authors.lastname: Auerswald
authors.affiliation: Northern Ireland Haemophilia Comprehensive Care Centre, Belfast, Northern Ireland, United Kingdom.
authors.forename: Gary
authors.initials: G
authors.lastname: Benson
authors.affiliation: Department of Haematology, Queens Medical Centre, Nottingham, United Kingdom.
authors.forename: Gerry
authors.initials: G
authors.lastname: Dolan
authors.affiliation: Irish Haemophilia Society, Dublin, Republic of Ireland.
authors.forename: Anne
authors.initials: A
authors.lastname: Duffy
authors.affiliation: Division of Haematology, Cliniques Saint-Luc, Brussels, Belgium.
authors.forename: Cedric
authors.initials: C
authors.lastname: Hermans
authors.affiliation: La Paz University Hospital, Coagulopathy Unit, Haematology Service, Autonoma University, Madrid, Spain.
authors.forename: Victor
authors.initials: V
authors.lastname: Jiménez-Yuste
authors.affiliation: Lund University, Departments of Clinical Sciences Lund - Paediatrics and Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden.
authors.forename: Rolf
authors.initials: R
authors.lastname: Ljung
authors.affiliation: Past President of AICE, Italian Association of Haemophilia Centre Directors, Florence, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Morfini
authors.affiliation: "Angelo Bianchi Bonomi" Haemophilia and Thrombosis Centre, IRCCS Ca' Granda Foundation, Maggiore Hospital, Milan, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Santagostino
authors.affiliation: Hemophilia Care Center, Bicêtre AP-HP Hospital and Faculty of Medicine Paris XI, Paris, France.
authors.forename: Thierry
authors.initials: T
authors.lastname: Lambert
mesh_terms: D015942:Factor VIIa / Q000008:administration & dosage / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000209:etiology* / Q000517:prevention & control*; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy*; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa