A Letter From Our Founders

The Rare Coagulation Disorders Resource Room (www.rarecoagulationdisorders.org), a dynamic, open-access website, was developed through a collaboration of the international RBDD Registry (www.eu.rbdd.org), and the Indiana Hemophilia & Thrombosis Center (www.ihtc.org), in conjunction with the many contributing authors from around the world. The Resource Room provides current and searchable information on the basic science, clinical management, available laboratory and genetic testing, clinical trials, and global research initiatives for these very rare and heterogeneous coagulation disorders.

This website was created to address the needs of both individuals with rare coagulation disorders and their health care providers. Health care professionals often have little experience treating these disorders, with limited resources for their diagnosis and treatment. Moreover, as rare coagulation disorders represent a small potential commercial market, few, if any, specific therapies exist for these conditions. As a result, affected individuals often face delayed diagnosis, insufficient laboratory evaluation, and limited treatment options.

Recent pharmaceutical efforts have focused on the development of safe and accessible products for a few of these rare disorders. In addition, global registries and networks have been developed to provide coordinated and consistent data collection to increase knowledge of these conditions, their sequelae and management.

Ongoing efforts continue to promote international data harmonization. In the United States, the American Thrombosis and Hemostasis Network (www.athn.org) has expanded its data collection system to assure inclusion of individuals affected with these rare coagulation disorders followed through the U.S. federal hemophilia treatment center network. In Europe, 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 a registry to better identify the number of affected individuals worldwide and define the clinical manifestations and sequelae of these disorders. The Rare Coagulation Disorders Resource Room is an important part of a global program to further enhance efforts to improve the health and quality of lives of individuals with rare coagulation disorders. We encourage the international rare coagulation disorders community to contribute to, and benefit from, this global endeavor.

Amy D Shapiro, MD

Medical Director

Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana
United States of Ameria

Flora Peyvandi MD, PhD

Professor of Internal Medicine

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
University of Milan
Milan, Italy