Archives: News

January 9, 2019
Haemophilia gene therapy: we are not alone

The number of trials of cell and gene therapy carried out in the UK has increased by more than one-third in the past year, according to the Cell and Gene Therapy Catapult (https://ct.catapult.org.uk), a London-based development agency that provides support for innovators in the field. The Catapult’s latest statistics show there are 85 clinical trials …

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Risk factors in children and young people with haemophilia

Concern about the possible risk of age-related disorders among people with haemophilia has been tempered by some evidence that many do not appear to be at greater risk than the population without bleeding disorders. Recent evidence from Turkey suggests some young people with haemophilia have cardiovascular risk factors – but no more than other people …

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Untreated bleeds are common

Patient diaries show that 40% of bleeds in people with haemophilia are not treated, according to a study presented at the 60th American Society of Hematology Annual Meeting in San Diego in December 2017. The study was part of the emicizumab development programme carried out by Genentech/Roche. Children and adults with haemophilia A used a …

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Acquired haemophilia A management remains a challenge

The best way to manage acquired haemophilia A in older people is unclear, according to a systematic review by specialists in France (Semin Hematol 2018;55:197-201). Their analysis of 80 publications describing 159 patients found a that a range of interventions was used and their effectiveness is difficult to assess. Mortality was high and depended on …

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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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