title: Consensus perspectives on prophylactic therapy for haemophilia: summary statement.
abstract: Participants in an international conference on prophylactic therapy for severe haemophilia developed a consensus summary of the findings and conclusions of the conference. In the consensus, participants agreed upon revised definitions for primary and secondary prophylaxis and also made recommendations concerning the need for an international system of pharmacovigilance. Considerations on starting prophylaxis, monitoring outcomes, and individualizing treatment regimens were discussed. Several research questions were identified as needing further investigation, including when to start and when to stop prophylaxis, optimal dosing and dose interval, and methods for assessment of long-term treatment effects. Such studies should include carefully defined cohorts, validated orthopaedic and quality-of-life assessment instruments, and cost-benefit analyses.
authors.affiliation: Department of Coagulation Disorders, Malmö University Hospital, Malmö, Sweden. erik.berntorp@medforsk.mas.lu.se
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
authors.forename: J
authors.initials: J
authors.lastname: Astermark
authors.forename: S
authors.initials: S
authors.lastname: Björkman
authors.forename: V S
authors.initials: VS
authors.lastname: Blanchette
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.forename: P L F
authors.initials: PL
authors.lastname: Giangrande
authors.forename: A
authors.initials: A
authors.lastname: Gringeri
authors.forename: R C
authors.initials: RC
authors.lastname: Ljung
authors.forename: M J
authors.initials: MJ
authors.lastname: Manco-Johnson
authors.forename: M
authors.initials: M
authors.lastname: Morfini
authors.forename: R F
authors.initials: RF
authors.lastname: Kilcoyne
authors.forename: P
authors.initials: P
authors.lastname: Petrini
authors.forename: E C
authors.initials: EC
authors.lastname: Rodriguez-Merchan
authors.forename: W
authors.initials: W
authors.lastname: Schramm
authors.forename: A
authors.initials: A
authors.lastname: Shapiro
authors.forename: H M
authors.initials: HM
authors.lastname: van den Berg
authors.forename: C
authors.initials: C
authors.lastname: Hart
mesh_terms: D003245:Consensus Statements as Topic; D003362:Cost-Benefit Analysis; D003906:Developing Countries; D006467:Hemophilia A / Q000000981:diagnostic imaging / Q000191:economics / Q000517:prevention & control*; D006801:Humans; D017410:Practice Guidelines as Topic; D011859:Radiography; D012307:Risk Factors; D013997:Time Factors