Archives: News

March 14, 2019
European Commission approves Roche’s Hemlibra for people with severe haemophilia A

The European Commission has approved emicizumab (Hemlibra) as prophylaxis in people with severe haemophilia A without factor VIII inhibitors. The decision endorses a positive opinion from the Agency’s Committee for Medicinal Products for Human Use on January 31st. Emicizumab was approved for prophylaxis in people with inhibitors in February 2018 and was approved for NHS …

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March 4, 2019
New NHS unit to oversee digital transformation

A new organisation is being created to drive forward the adoption of digital technology in the NHS. Christened NHSX, it will provide leadership on national policy “both by working with industry and via its own prototyping and development capability”. Its chief executive will have strategic responsibility for directing technological development and will be accountable to …

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March 3, 2019
UK haemophilia centre staff unclear about dentistry guidance

UK staff involved in the treatment of haemophilia know about guidelines for management during dental procedures but not much about what’s in them or how to apply them to assess risk, say dentists (Spec Care Dentist. 2019 Feb 17. doi: 10.1111/scd.12359). They sent questionnaires to 180 staff working in 60 UK centres, of whom 41 …

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March 2, 2019
Bleeding risk the same for vaginal and Caesarean delivery

The incidence of major bleeds and intracranial haemorrhage associated with vaginal delivery and Caesarean section are similar, a new analysis of the PedNet database has shown (Haematologica 2019 Feb 21. doi: 10.3324/haematol.2018.209619). The study included 926 neonates (786 with severe and 140 with moderate haemophilia) in hospitals in Europe and Israel. About two-thirds were delivered …

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March 1, 2019
New EHL factor VIII approved in US

The US Food and Drug Administration has approved Novo Nordisk’s extended half-life factor VIII for prophylaxis and treatment in children and adults with severe haemophilia A. Turoctocog alfa pegol (Esperoct) is a pegylated version of Novo’s recombinant factor VIII NovoEight. The company says that, compared with standard factor VIII, half-life is increased by a factor …

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February 27, 2019
Roche invests in gene therapy

Spark Therapeutics, the company at the forefront of developing gene therapy for haemophilia, has been acquired by Roche in a merger worth $4.8 billion. The deal, which is expected to be closed by the middle of the year, brings benefits to both sides. Spark gains the global resources required to fully exploit its discoveries. Roche …

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February 20, 2019
EHC announces first conference on women and rare bleeding disorders

The European Health Consortium (EHC) has announced that its first European Women & Bleeding Disorders Conference will be held in Frankfurt, Germany, on May 24th– 26th. The programme will cover issues in genetics, management of heavy menstrual bleeding, family planning, everyday coping, anaemia, oral care, impact on quality of life and pregnancy. Online registration is …

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February 19, 2019
Virtual reality distracts children during venepuncture

US researchers have developed a hands-free virtual reality environment that could help to avoid the development of needle phobia in children with haemophilia (JMIR Serious Games. 2019 Jan 9;7(1):e10902. doi: 10.2196/10902). It was no more effective than a standard technique but probably more fun. They created a wireless virtual reality platform using a disposable adjustable …

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Is fortnightly FIX feasible?

Bioverativ’s B-LONG and B-YOND studies demonstrated the long-term safety and efficacy of its EHL FIX, rFIXFc, when administered every 7 days to >14 days. Now, a post hoc analysis of the small subgroup of patients who used a dose interval of 14 days or longer has shown that control of bleeding was similar to that …

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February 18, 2019
EHL FIX review

An up to date review of evidence for the efficacy and safety of nonacog beta pegol, an extended half-life FIX for haemophilia B, covers outcomes from the PARADIGM trial programme (Drug Des Devel Ther 2018;12:2933-2943). The review is free to access at https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6141116/pdf/dddt-12-2933.pdf

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