Since 1996 the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre of Milan, Italy is a national and international reference Centre for diagnosis and management of patients affected with Rare Bleeding Disorders (RBDs). A large and regularly followed up patient population coming from different countries allowed to extend the knowledge on different aspects of RBDs and give a platform to focus and develop further diagnostic and therapeutic research activity. In 2004, under the auspices of the International Society of Thrombosis and Haemostasis, the experiences gained has enabled Dr. Flora Peyvandi to spearhead the development of an international network of care providers and national representatives to work together to discuss the prevalence, clinical manifestations, and need for coordinated and consistent data collection on RBDs. The hope of this international community was and still is to better identify the number of affected individuals throughout the world, define the clinical manifestations and sequelae associated with these disorders, create a network of individuals who care for these patients that are able to share diagnostic and treatment expertise, and provide potential centers where specific products once developed may be utilized in clinical trials.
The main goals of the RBD working group are:
- to establish and implement an International registry to homogenously collect data on different aspects of the disorders - to provide evidence-based guidelines for diagnosis and management - to identify available drugs for replacement therapy of each very rare bleeding disorder in different regions of the world - to encourage the development and the amelioration of drugs.
