Dr. Flora Peyvandi, under the auspices of the International Society of Thrombosis and Haemostasis, has spearheaded 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.
The hope of this international community 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.
In the last ten years we have investigated more than 400 patients with RBDs from a variety of countries (Figure 4). The continuing collaboration with different international centres will allow to further extend the knowledge of RBDs and give a platform to focus and develop further diagnostic and therapeutic research activity.
The main goals of the RBD working group are:
- To establish and implement an International Database of RBDs
Contributions to this database will allow a comprehensive analysis of the distribution of patients affected by RBDs in each region of the world. This data collection will increase our knowledge on the clinical and therapeutic aspects of RBDs
- Identification of available drugs for replacement therapy of each very rare bleeding disorder in different regions of the world
The work of this group will help to constantly follow (overhaul) drug production, cost and distribution in the world and mainly to encourage the development of drugs particularly for those deficiencies with no available therapeutic concentrate.
With these goals, an on-line International Database on RBDs was developed with the aim of efficiently collecting and extracting already available data on RBDs.
The conceptual schema of RBDD (Figure 5) was designed thanks to the collaboration of molecular, clinical and informatic expertises.
Currently the database contains clinical, genetic and therapeutic information on a large group of severely affected patients scattered in various parts of the world.
By creating a network of the Haemophilia Centres around the World, we believe that RBDD should represent a reference informative tool where knowledge and expertise on RBDs could be collected and shared.
The knowledge extracted from RBDD will allow optimisation of retrospective studies; whereas further studies are needed to ameliorate current treatments and diagnosis.
Thus the final goal is to provide evidence-based guidelines for diagnosis and management of patients affected by RBDs, coming from an unique International database formed by the implementation of all the pre-existing databases and managed by a scientific International organization, such as ISTH or WFH.