Weekly recombinant FIX prophylaxis for severe haemophilia B in normal clinical practice: data from UKHCDO and Finland

The haemtrack home therapy reporting system: Design, implementation, strengths and weaknesses: A report from UK Haemophilia Centre Doctors Organisation

“…Given these constraints, how can we use Haemtrack data for larger scale outcome analysis and analysis of treatment patterns and trends? To date, we have limited our analysis to patients who satisfy prestated compliance criteria (eg, >45 weeks data per year and reported use of >75% of treatment issued by the centre) . These compliance criteria can be validated centrally at NHD for patients using regular prophylaxis, but are unsuitable for validation of patients treating themselves on demand, and their data must be validated locally by their HTC.…”

“…These compliance criteria can be validated centrally at NHD for patients using regular prophylaxis, but are unsuitable for validation of patients treating themselves on demand, and their data must be validated locally by their HTC. NHD has cross‐validated data for smaller studies directly with the managing HTC …”

“…Haemtrack is being used prospectively and retrospectively in several national and international studies of treatment trends and may be customized for use in clinical trials and epidemiological research . The study population should be carefully defined and potential ascertainment bias assessed, but bias may also be minimized by careful study design.…”

“…UKHCDO, supported by NHD and Haemtrack is in a strong position, for example to conduct large‐scale longitudinal within‐patient comparisons of the effect of changing regimens or products. These include studies on the introduction of EHL products and the future introduction of factor VIII mimetics or anti‐TFPI antibodies . Using Haemtrack, we are able to report on an observation period long enough to avoid the problem of regression to the mean.…”

UK Haemophilia Centre Doctors’ Organisation guidance on the use of extended‐half‐life coagulation factor concentrates in routine clinical practice: Report of a meeting on their adoption by Belgian haemophilia treaters

“…In this study, once-weekly treatment provided effective prophylaxis for most patients in this study, with a very low annual bleed rate comparable with more frequent prophylaxis and trials of extended-half-life products. 6 Despite evidence that extravascular FIX may serve an important role in coagulation, FIX plasma concentration remains the current standard surrogate endpoint for haemostatic efficacy in haemophilia B and further work is required to fully explore the role of the extravascular store.…”