The Second Team Haemophilia Education Meeting, 2016, Frankfurt, Germany

The first Team Haemophilia Education (THE) Meeting was held on 7-8 May 2015 in Amsterdam, The Netherlands. It aimed to promote the optimal care of patients with haemophilia through education of the multidisciplinary treatment team. This was achieved by reviewing the latest developments in haemophilia management, considering how these can be implemented in the clinic to improve patient care and providing a platform for networking and debate for all haemophilia treatment team members. The second THE Meeting was held on 19-20 May in Frankfurt, Germany, and participants included doctors, nurses, physiotherapists, patient representatives and data management staff from 20 different countries. Topics covered the role of the multidisciplinary team in delivering the best haemophilia care, challenges in the management of haemophilia across Europe, available clotting factor treatments, future treatments and the use of genetics in advising carriers of haemophilia. This report is a summary of the key developments in haemophilia care presented by various investigators and healthcare professionals at THE Meeting 2016.info:eu-repo/semantics/publishedVersio

Educational content and challenges encountered when training service user representatives as peer researchers in a mixed study on patient experience of hospital safety

International audienceAbstract Background and objectives In France, following the passing of a 2002 law, service user representatives (SURs) are part of hospital committees in charge of care quality and safety issues. Ten service user representatives (SURs) were recruited and trained as “peer researchers” to participate in all phases of a study aimed at outlining how patients experience hospital safety. This article aims to describe the study protocol and how peer researchers training was designed and implemented to prepare them to drive a qualitative and quantitative research. It also examines the challenges related to collaborative research and how these were resolved. Methods The way our training was conceived belongs to the field of “design-based research”, known for its pragmatic and collaborative scope, in which viewpoints of all participants are included. Our training was therefore based on peer researchers and research sponsors expectations, as well as on recommendations of the literature. Results A 45-h training was held. While the program was meant to train peer researchers to respect scientific norms, it also aimed to improve their sense of self-legitimacy as they navigated their new role. Peer researchers were particularly eager to understand meaning behind the instructions, especially in the field of ethical and scientific norms. Various challenges occurred related to project organization, recruitment and peer researchers involvement. Some issues were overcome by learning how to share control over the research process. Conclusion This experiment highlights the importance of a training program’s duration and quality to prepare SURs for their roles as peer investigators and to create a group dynamic around a research project, even with SURs familiar with patient involvement and our research theme (safety issues). Trainers overcame hurdles by being adaptive and by using educational approaches. They also learned to include trainees’ input, even when it forced them to reconsider their own assumptions

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table.

INTRODUCTION: With approval of gene therapy for haemophilia likely in the near future, policy frameworks are needed to guide the path forward for this disruptive and novel therapeutic advance. AIM: The WFH has initiated a series of multi-stakeholder Gene Therapy Round Tables (GTRT) to better understand where guidance is needed and develop initial consensus statements to inform policy. METHODS: The first day of the 2nd GTRT was devoted to didactic presentations on models of access to gene therapy, payment and health technology assessment considerations, regulatory issues and the generation of evidence on safety and durable efficacy of gene therapy products. On the second day, participants were tasked with developing and voting on consensus statements that reflected the information presented and multi-stakeholder views expressed during discussions in the 1st and 2nd WFH GTRTs. The statements covered global access to gene therapy for all people with haemophilia (PWH), collection of long-term safety and efficacy data, ensuring gene therapy is available for all subgroups of PWH including those who have been largely excluded from clinical trials and characterizing acceptable and ideal factor expression levels for gene therapy products. RESULTS: The first 3 statements achieved consensus (at least 80% agreement) by this group of experts. The statement on identifying an ideal and an acceptable factor level expression elicited a lively discussion but failed to achieve consensus by this group. CONCLUSIONS: This issue of ideal and acceptable factor level expression and other unresolved issues will be brought to the 3rd WFH GTRT in 2020

À propos du non-profit : rapport du quatrième symposium annuel d’éthique transfusionnelle de l’Institut national de la transfusion sanguine [Not-for-profit: A report from the fourth annual symposium of ethics held by the National Institute for Blood Transfusion (France)]

The not-for-profit issue has been debated in November 2016 in Paris; this issue is one of the four canonical pillars of ethical blood donation. It is intimately bound to benevolence though it is distinct, as not-for-profit calls for institutions while benevolence calls for individuals. It is indeed intended that voluntary blood donors do not benefit from their donation and are thus non-remunerated. Not-for-profit is essential since it refers to the public character of blood as a putative public resource aimed at being shared as a tribute of solidarity. A central question however is linked to the capacity- or not -of public sectors to ensure that blood components are universally available, with special mention to plasma derived drugs, without the contribution of the for profit, private sector

Scintilla European project, the successful research results

Conference of 4th International Conference on Advancements in Nuclear Instrumentation Measurement Methods and their Applications, ANIMMA 2015 ; Conference Code:121554International audienceThe Scintilla FP7 project is ended in December 2014, the fruitful results of 3 years development and tests will be presented. SCINTILLA offers the capacity to finding a reliable alternative to Helium-3 based detection systems since the gas which is predominantly used in nuclear safeguards and security applications has now become very expensive, rare and nearly unavailable. SCINTILLA benchmarks results are based on international standards. Radiation Portal tests were carried out at the Joint Research Centre (JRC) in Ispra (Italy). The scintilla project addresses few main issues. The first is to develop neutron detectors for Radiation Portal Monitor (RPM) and the second is the need of new wearable integrated solutions for Spectrometric Personal Radiation Monitor (SPRM). The partners which provide technical systems of the scintilla project are INFN-ANSALDO, CEA, SYMETRICA and SAPHYMO. For RPM, the objective is to find reliable alternatives to Helium-3 historical neutron detector and provide technical solutions which cope with tests for reliable mobile and cost effective. For Spectrometric Personal Radiation Monitor (SPRM), SCINTILLA is innovating in technology areas that offer complementary capabilities for detecting and identifying gamma Two CZT (Cadmium Zinc Telluride) addressing contexts of used by first responder technologies, one is a wearable detector and the second is a gamma camera complemented by advanced image processing technologies