Month: May 2019

May 23, 2019
The First European Conference on Women and Bleeding Disorders: Diagnosis, Treatment, Care, Planning, Community, Psychosocial

Frankfurt, 24 – 26 May 2019. The European Hemophilia Consortium (EHC;, #WBDEHC) has been at the forefront of tackling issues that affect people with bleeding disorders who have otherwise been under-represented by the haemophilia community. This is its first formal conference to address the challenges faced by women with bleeding disorders – and perhaps the …

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May 10, 2019
Moderate haemophilia ‘neglected’

The available evidence shows that moderate haemophilia is a ‘neglected disease’, according to the eminent Swedish haemophilia specialist Eric Berntorp (Haemophilia 2019 Mar;25(2):187-188). He was commenting on the THUNDER study (Haemophilia 2019;25:205-212). Using Haemtrack data, this suggested that people with moderate haemophilia in the UK are undertreated because their annual bleeding rate and joint status …

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May 9, 2019
Haemophilia Society UK responds to emicizumab concerns

The Haemophilia Society has published advice to its members on recent reports on social media ‘about deaths relating to a new treatment for haemophilia A, Emicizumab (Hemlibra)’ Without providing details of the reports, the Society states: ‘Worldwide over 2,500 people have been using Emicizumab. Although a number of deaths have been reported, these were not …

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AAV antibodies in people with haemophilia

Most gene therapies now undergoing clinical trials rely on a viral vector to achieve transduction of liver cells so that they produce the replacement gene. The majority of vectors are derived from adeno-associated viruses (AAV). AAVs naturally cause infection in humans, provoking an immune response that generates antibodies; individuals with AAV antibodies are ineligible for …

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May 8, 2019
What people with haemophilia want from their treatment

A study from Canada reports that people with haemophilia A would like to administer their factor replacement without needing an intravenous infusion and they want longer-lasting factors (Haemophilia 2019 Apr 23. doi: 10.1111/hae.13754). Interviews and focus groups involving 20 people with haemophilia A without inhibitors and caregivers revealed that having intravenous infusions, coordinating treatment schedules …

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May 6, 2019
NHS England funds leadership development

NHS England has recognised the importance of leadership by committing £2 million to ‘kick-start or boost leadership development activities to support and inspire workforce in health systems across England from GPs, consultants and therapists to nurses, social workers and managers’. The move aims to fulfil the promise of the Long Term Plan to develop new …

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May 5, 2019
Arthrocentesis for acute hemarthrosis?

Joint aspiration enhances recovery from acute haemarthrosis, say clinicians from Spain (Blood Coagul Fibrinolysis 2019;30:111-119). They report outcomes in 33 people with haemophilia who received standard care or underwent immediate joint aspiration. There were 55 episodes of acute haemarthrosis of the elbow, knee and ankle. All patients received factor replacement and advised to rest. Join …

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May 4, 2019
NHS beefs up early access scheme

The NHS Accelerated Access Collaborative has received a shot in the arm intended to improve its performance in fast-tracking innovative technologies and provide earlier access for patients. The Department of Health and Social Care has created a new unit within NHS England and NHS Improvement. This will deliver ‘a system to identify the best new innovations …

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May 3, 2019
Impact of emicizumab on inhibitor management

Emicizumab has been called a disruptive therapy for good reason – it could transform care delivery for patients with haemophilia A who have inhibitors. Now, a multinational group has been formed to consider the implications for future management. The Future of Immunotolerance Treatment (FIT) group has published its first statement, saying that inhibitor eradication will …

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May 2, 2019
Barriers and challenges faced by women with congenital bleeding disorders

The European Health Consortium has published the findings of its survey of the experiences of women with a bleeding disorder (Haemophilia 2019 Apr 29. doi: 10.1111/hae.13722). Responses from 709 women in 32 mostly western European countries reveal that diagnostic delay is a problem: the median age at diagnosis was 16 overall but only 6 when …

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