Foreword

With improved healthcare strategies and more effective treatments, patients with haemophilia now have a greater life expectancy and better quality of life than ever before. Despite many advances, remaining challenges in haemophilia care include achieving parity of treatment, increasing treatment options, optimising patient outcomes (including musculoskeletal care) and improving the management of patients with inhibitors. As in other chronic diseases, psychosocial issues have a profound impact on the well-being of people living with haemophilia and also need to be considered alongside treatment for prevention and management of bleeding episodes. The 12th Novo Nordisk Symposium on Haemostasis Management, held in Vienna, Austria, in June 2014, brought together an international faculty of haemophilia experts and healthcare professional delegates to consider how these challenges can be addressed, focusing on medical, psychological and social issues associated with rare bleeding disorders, with an emphasis on haemophilia and its management. The Symposium consisted of an introductory session with the keynote lecture ‘Improving care and treatment options for women and girls with rare bleeding disorders’, followed by a presentation outlining the work of the Novo Nordisk Haemophilia Foundation. Five main sessions comprised the Symposium, the first of which, ‘Research in the spotlight’, included a summary of recent scientific research that may influence future clinical practice. The second session, ‘New opportunities and insights for factor VIII (FVIII)’, explored the rationale behind the use of recombinant replacement therapies and described the clinical development and pivotal trial results of the recombinant FVIII product, turoctocog alfa, for the treatment of haemophilia A. The third session comprised four specialised workshops led by faculty who shared their wealth of knowledge and experiences of everyday clinical practice on the topics ‘Optimising musculoskeletal health: implications for clinical practice’, ‘Challenges in transition: case studies’, ‘Pathways to surgery: utilising multidisciplinary care for optimal outcomes’ and ‘Acquired haemophilia: integrating technology for improved diagnosis and outcomes in acquired haemophilia’. The fourth session, ‘Continuing the journey of inhibitor management’, provided an overview of efficacy and safety data derived from 18 years’ experience of using recombinant activated factor FVIIa in patients with haemophilia and inhibitors. The final session, ‘Rare bleeding disorders in focus’, summarised evidence to guide the management of acquired haemophilia and Glanzmann's thrombasthenia. To facilitate knowledge acquisition, and thus inform best practice in haemostasis management, five topics were selected from the varied Symposium programme for presentation in this supplement: ‘Improving care and treatment options for women and girls with bleeding disorders’; ‘Optimising musculoskeletal care for patients with haemophilia’; ‘Updates from guardian™: a comprehensive registration programme’; ‘Challenges in transition’; and ‘Acquired haemophilia: an overview for clinical practice’. Editorial assistance was provided by Tracey Spurway of Bioscript Medical Ltd (Macclesfield, UK) and funded by Novo Nordisk Health Care AG (Zurich, Switzerland). DL was involved in the drafting, and approval for submission, of this manuscript. None to declare. Please always refer to local prescribing information. UK prescribing information for Novo Nordisk treatment options discussed during the Symposium can be found at the end of this Supplement.

Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.