February 17, 2019
Is EHL factor replacement therapy cost effective?

Health economists continue to grapple with the issue of cost effectiveness of extended half-life (EHL) factors compared with the established recombinant factors. A report from the United States suggests that, for people with haemophilia A, they are not (Manag Care 2018;27:39-50). Analysis of data over a two-year period before and after a switch to an …

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February 14, 2019
Obesity and haemophilia

It’s becoming increasingly clear that, despite some obvious differences directly attributable to their bleeding disorder, people with haemophilia share many of the problems that plague the population in general. A survey of seven haemophilia centres in Germany has shown that the prevalence of overweight and obesity is similar to that in the population as a …

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February 9, 2019
Phase III gene therapy trial begins and Phase II trial updated

The first patient in uniQure’s HOPE B trial of its gene therapy AMT-061 has been treated, the company has announced ( HOPE B is an non-blinded Phase III trail to determine the efficacy and safety of AMT-061 in men with haemophilia B; it will eventually involve a total of 56 patients.The trial comprises a six-month …

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February 7, 2019
Roche joins WFH Humanitarian Aid Program

Roche has joined the World Federation of Hemophilia’s Humanitarian Aid Program with a donation of emicizumab and funds to support training to build expertise in economically developing countries ( The donation will provide prophylaxis with emicizumab for up to 1,000 people in over five years, focusing on high-need patients such as adults with inhibitors and …

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February 5, 2019
Calling all high-flying researchers

Funding of up to £2 million is being made available to researchers ‘with compelling track records in the health sector’ with ‘potentially high-risk projects… grounded in considerable external evidence’. The Peter Sowerby Foundation is inviting applications from organisations with a turnover of more than £2 million with projects ‘such as primary medical care and research …

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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 (, 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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