Month: December 2018

December 10, 2018
Emicizumab data confirm early promise

Updated data on the use of emicizumab in children with haemophilia A who have inhibitors were presented at the 60th American Society of Hematology Annual Meeting in San Diego. Manufacturer Roche says its latest analysis of the HAVEN 2 trial in children under 12 years old shows that after a median of 12 weeks of weekly …

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aPCC more cost-effective than rFVIIa in acquired bleeding

There are two options for treating bleeding in patients with acquired haemophilia A and inhibitors: activated prothrombin complex concentrate (aPCC) or recombinant activated factor VII (rFVIIa). The older technology is 7 – 8 times less expensive and, say US analysts, offers a more cost effective option (Vox Sang 2018 Nov 30. doi: 10.1111/vox.12726). Their research, …

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Kinetic profiling might not reduce joint bleeds in patients with arthropathy

People with haemophilia A who take FVIII prophylaxis can benefit from optimising their dose by pharmacokinetic profiling, especially if they also switch from a conventional FVIII to one with an extended half life (EHL). A recent study involving 39 people who underwent profiling, of whom 20 agreed to modify their treatment, confirmed that profiling was …

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New pain evaluation tool described

Researchers from Portugal have described a new tool to evaluate pain in people with haemophilia (PLoS One 2018 Nov 28;13(11):e0207939. doi: 10.1371/journal.pone.0207939). The Multidimensional Haemophilia Pain Questionnaire (MHPQ) is a questionnaire that obtains information about pain characteristics (location, duration, frequency, triggering factors, intensity), its daily impact, management and satisfaction with treatment. It can distinguish between …

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Four years’ experience of EHLs reported

Long-term outcomes of prophylaxis with the extended half-life (EHL) factors efmoroctocog alfa (Elocta) and eftrenonacog alfa (Alprolix) were reported at the 60thAnnual Meeting of the American Hematology Society in San Diego. The data come from 4-year extension studies: ASPIRE followed on from the A-LONG trials in haemophilia A and B-YOND included patients with haemophilia B …

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Bayer’s pegylated EHL now licensed

Bayer’s pegylated extended half life factor VIII damoctocog alfa pegol (Jivi) has now been formally licensed by the European Medicines Agency for the treatment and prophylaxis of bleeding in previously treated patients 12 years of age or older with haemophilia A. Approval is based on the PROTECT VIII trial, which showed that prophylaxis with damoctocog …

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ISTH rethinks definition and diagnosis of mild haemophilia A

The internationally recognised definition of mild haemophilia A – a FVIII level between of 5 and 40 IU/dL – takes no account of the assay used to measure FVIII. As a result, according to the International Society for Thrombosis and Haemostasis (ISTH), FVIII measurements can be imprecise due to variation in gene mutations (J Thromb …

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Long term study shows improvements in outcome in Denmark

The progress of people with moderate to severe haemophilia in Denmark has been followed in surveys conducted in 1988 (n=128), 2001 (n=156, 94% severe) and 2012 (n=164, 74% severe) (Acta Haematol 2018;140:240-246). The findings show that proportion of the patient population falling within the oldest age group (55 – 88 years) increased from 4% of …

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WFH publishes 2017 global survey

The World Federation for Hemophilia has published its latest survey – the 19th – of worldwide demographics and treatment data for people with bleeding disorders. The survey, available in print and online at www.wfh.org/globalsurvey, includes data on haemophilia, von Willebrand disease and rare factor deficiencies and inherited platelet disorders affecting 315,000 people in 116 countries. This …

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