Elliott Vichinsky, MD Director, Hematology/Oncology Children's Hospital & Research Center at Oakland

Thalassemia is the most common, inherited single gene disorder in the world. Early comprehensive treatment has changed thalassemia from a fatal pediatric disease to one in which patients live productive lives throughout adulthood. Advances in treatment are exciting, resulting in the potential for cure and improved quality of life. However, many patients never receive the information needed to make educated decisions about treatment. Many ethnic groups are unaware of their genetic risk of thalassemia and do not receive genetic counseling or pre-natal diagnosis. Others have heard of pre-implantation genetic diagnosis as a technique to achieve a healthy newborn, but do not have access to objective medical information of its risks and benefits. Thousands of patients receive medical care outside of specialized thalassemia centers. These patients and providers need information concerning recommendations for comprehensive screening tests, access to new research protocols and treatment including transfusion therapy, new chelation therapy, stem-cell transplantation, hormone replacement, heart disease, osteoporosis and hepatitis. Quality of life for families and patients can be excellent with a future including employment, marriage and children. To achieve this, families and providers need information concerning psychological wellness, cultural issues and counseling for family planning and marriage. In summary, this site should provide the family and providers with necessary information to make informed decisions and achieve optimal quality of life. It is our hope that by providing electronic education about the disease, we can raise awareness, encourage people to get tested for trait, and spread knowledge about comprehensive treatment to the global community.