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

An in vitro evaluation of standard rotational thromboelastography in monitoring of effects of recombinant factor VIIa on coagulopathy induced by hydroxy ethyl starch

BACKGROUND: Rotational thromboelastography (ROTEG) has been proposed as a monitoring tool that can be used to monitor treatment of hemophilia with recombinant factor VIIa (rFVIIa). In these studies special non-standard reagents were used as activators of the coagulation. The aim of this study was to evaluate if standard ROTEG analysis could be used for monitoring of effects of recombinant factor VIIa (rFVIIa) on Hydroxy Ethyl Starch-induced dilutional coagulopathy. METHODS: The study was performed in vitro on healthy volunteers. Prothrombin time (PT) and ROTEG analysis were performed after dilution with 33% hydroxy ethyl starch and also after addition of rFVIIa to the diluted blood. RESULTS: PT was impaired with INR changing from 0.9 before dilution to 1.2 after dilution while addition of rFVIIa to diluted blood lead to an overcorrection of the PT to an International Normalized Ratio (INR) value of 0.6 (p = 0.01). ROTEG activated with the contact activator ellagic acid was impaired by hemodilution (p = 0.01) while addition of rFVIIa had no further effects. ROTEG activated with tissue factor (TF) was also impaired by hemodilution (p = 0.01) while addition of rFVIIa lead to further impairment of the coagulation (p = 0.01). CONCLUSIONS: The parameters affected in the ROTEG analysis were Clot Formation Time and Amplitude after 15 minutes while the Clotting Time was unaffected. We believe these effects to be due to methodological problems when using standard activators of the coagulation in the ROTEG analysis in combination with rFVIIa

Choice of factor VIII/IX regimen in adolescents and young adults with severe or moderately severe haemophilia. A French national observational study (ORTHem 15-25)

International audienceIntroduction: The value and challenges of long-term prophylaxis (LTP) in adolescents and young adults need further characterisation.Aim: To determine the proportions of adolescents and young adults with severe or moderately severe haemophilia in France under LTP and treatment on demand (OD).Methods: Patients 15 to 25 years old with haemophilia A or B, factor VIII/IX ≤ 2% and no current inhibitor could be included if they had been under factor VIII/IX treatment at least 12 months and kept a treatment and bleeding diary.Results: LTP was administered to 169/212 patients (79.7%) and OD treatment to 40/212 patients (18.9%). The most frequent reasons for initiating LTP were joint bleeding, target joints and frequent bleeds; whereas OD treatment was most often selected on the basis of mild bleeding phenotype or because of constraints on LTP. The mean annual bleed rate (ABR) in the OD group (6.33) was higher than in the LTP group (3.07, p  18–21 and > 21–25 years), but was significantly higher for patients with severe haemophilia (4.02) as compared to those with moderate haemophilia (1.97, p = 0.002). No significant difference was observed in mean ABR for joint bleeds between the LTP and OD groups. Physician reported LTP compliance was good or excellent in 97.0% of patients.Conclusion: LTP is the predominant factor VIII/IX treatment among adolescents and young adults with severe or moderately severe haemophilia in France. LTP was associated with low ABR and high compliance. © 2017 The Author

Malignant disease in the haemophilic population: moving towards a management consensus?

The Malignancy in Haemophilia Workshop Group convened a consensus working group of haematologists and oncologists to review topics related to malignancy in haemophilia. The treatment of malignant disease in this population is increasingly relevant as both outcome and lifespan continue to improve. Although adequate guidance exists for control of spontaneous bleeding episodes and of haemostasis in general surgery, information for management of haemostasis in patients with various malignancies is sparse. To date, no clinical guidelines exist for management of complex bleeding problems, diagnosis, therapy and follow-up of malignancies in haemophilia. Furthermore, it remains unclear whether or not morbidity and mortality outcomes associated with malignancies are affected by haemophilia or by its treatment. Through presentation of five malignancies - prostate cancer, colorectal cancer, acute leukaemia, bladder cancer and hepatocellular carcinoma - important issues are highlighted, such as risk from bleeding as a symptom of malignancy; risks from invasive screenings and how these should be handled in haemophilic individuals; the implications of chemotherapy and treatment schedules, bone marrow suppression, radiotherapy, or surgery; and the likelihood of an interaction between treatment for haemophilia and malignancy outcomes. Ultimately, the aim is to establish consensus guidelines to direct and harmonize future treatment policy for malignant disease in the haemophilic population

A systematic approach to controlling problem bleeds in patients with severe congenital haemophilia A and high-titre inhibitors

The presence of inhibitory antibodies to clotting factors complicates the treatment of bleeding in haemophilia patients. For patients with high-titre inhibitors, bypassing agents are essential to haemostatic management. To determine optimal treatment practices, an international panel of physicians convened to develop a systematic treatment approach for problem bleeds (i.e. bleeds that are unresponsive to initial therapy with a single agent within a reasonable amount of time) in haemophilia patients with inhibitors. AIM: The goal of this panel was to develop a consensus algorithm that would aid physicians in considering a variety of treatment approaches to optimize patient care by preventing extensive therapy with inadequate treatments that may lead to suboptimal patient outcomes and unnecessary costs. METHODS: Consensus opinions were analyzed for clinical preferences at different time periods, depending on patient response to treatment. Decision-making points were defined based on the type of bleed: every 8-12 h for the first 24 h, then every 24 h thereafter for limb-threatening bleeds; every 2-4 h for 2-7 days for life-threatening bleeds. RESULTS: The resultant consensus guidelines provide a generalized methodology to guide the treatment of problem bleeds in patients with severe haemophilia A and inhibitors, and emphasize changing treatment at the first sign of an inadequate haemostatic response. The treatment algorithms apply to both paediatric and adult patients, although the differences between the two groups were reviewed. CONCLUSION: These guidelines are focused on optimising the timing of treatment decisions, which may lead to faster responses and improved outcomes

Factor VIII light chain mutations and cysteine substitutions predispose for inhibitor development in mild and moderate haemophilia A: first results from the INSIGHT study

Clinical epidemiolog

Managing women-specific bleeding in inherited bleeding disorders: A multidisciplinary approach

Introduction: Multidisciplinary management of women-specific bleeding is important to preserve quality of life, healthy reproduction and social participation of women and girls with bleeding disorders (WBD). Aim: To support appropriate multidisciplinary care for WBD in haemophilia treatment centres. Methods: Two case examples are presented and management issues discussed from different health care perspectives, including the nurse, patient, psychologist, gynaecologist, geneticist, psychosexual therapist and haematologist. Results: Woman with bleeding disorders may experience heavy menstruation from menarche onwards. This has a physical and psychosocial impact requiring a multidisciplinary approach. If a woman with an inherited bleeding disorder desires to become pregnant, preconception counselling is essential, to discuss genetic diagnosis, state of the art treatment options for the bleeding disorder in question and possible choices to prevent having an affected child, as well as maternal bleeding risks during conception, delivery and the post-partum period. Conclusion: Adequate management and good education of WBD requires a patient-centred multidisciplinary approach with experienced specialists in a haemophilia treatment centre

Sciences et recherches participatives sur les sols en France Bilan et perspectives

International audienceregarding citizen science. They are touchable and many observa-tions can be proposed on them. They are included in multiple public policies (agroecology, bio-diversity, food, climate...). They integrate numerous impacts of human activities. Their diverse properties, physical, chemical and biological ones, to qualify soils can provide the researcher, rich and original data and the citizen who embarks, a journey of discovery. Despite that, for non-experts, working on soil remains more difficult than working on other environmental com-ponents (plants, insects, rivers…). The objective of this article is to provide i) an inventory of the projects of citizen science on soils in France from a survey of the project leaders, ii) a presenta-tion of five pilot projects, iii) lessons learned from a workshop mixing scientists and non-scientists involved in these projects. Twenty projects have been inventoried, all fairly young (the majority are less than 5 years old), mostly on soil biology and on agricultural land use. They are mainly managed by research, with the contribution of diverse partners of the society who are involved in a part of the project, most often soil data collecting. Co-building protocols, interpre-tation and feedback to non-scientists are still under developed. Five examples illustrate the diversity of the projects. This first evaluation of the projects on soil citizen science will contrib-ute to increase exchanges between projects up to create a community or a network on such transdisciplinary approach on soil.Les approches de sciences et recherches participatives (SRP) se développent, avec de plus en plus d’initiatives sur les sols. Ceux-ci sont des objets originaux vis-à-vis des SRP. Ils sont touchables, manipulables, l’enjeu de multiples politiques publiques (agroécologie, biodiversité, alimentation, climat…), intégrateurs de nombreux impacts des activités humaines. Les propriétés physiques, chimiques, biologiques pour les qualifier peuvent amener au chercheur une richesse d’information et au citoyen qui s’y engage un parcours de découverte. Les sols restent néanmoins plus difficiles à appréhender par des non-spécialistes que d’autres éléments de l’environnement (plantes, insectes, cours d’eau, …). L’objectif de cet article est de faire un état des lieux des SRP sur les sols en France, à partir i) de la présentation d’une enquête auprès de porteurs de projet à l’échelle nationale, ii) de la présentation de quelques projets pilotes, enfin iii) des enseignements tirés d’un colloque réunissant la communauté. Les 20 projets identifiés sont tous assez récents (plus de la moitié a moins de 5 ans), principalement sur la biologie des sols, et sur les sols agricoles. Ils sont majoritairement portés par la recherche, associant des acteurs assez divers de la société, le plus souvent sur une partie du projet, celle de la collectede données. La co-construction des protocoles, de l’interprétation, du retour vers les non scientifiques est encore peu développée. Cinq exemples illustrent la diversité des projets. Ce premier bilan doit permettre une plus grande mutualisation des projets, voire la création d’une communauté et d’un réseau de travail sur ces travaux transdisciplinaires sur le sol

Efficacy and safety of high ration HVWF/FVIII concentrate (VONCENTO (R)) for the treatment of bleeding epidodes in patients with WITH VON WILLEBRAND disease :s: the OPALE French experience

International audienc