AdriannaVlachosMDProfessor (PAR), Johns Hopkins Univ

Summary

• I am a pediatric hematologist/oncologist and stem cell transplanter with a particular interest in bone marrow failure disorders, both inherited and acquired. I specifically care for patients with acquired aplastic anemia and inherited bone marrow failure syndromes such as Diamond Blackfan anemia, Fanconi anemia, Shwachman Diamond syndrome, and others. I was Head of the Bone Marrow Failure Program at Cohen Children's Medical Center and am presently Division Chief of Hematology at Johns Hopkins All Children's Hospital Cancer and Blood Disorders Institute.
  The goal of my long-standing research has been to promote the understanding of the rare disorder Diamond Blackfan Anemia (DBA) by studying the epidemiology, pathophysiology, and genetics of the syndrome. As such, I established the Diamond Blackfan Anemia Registry (DBAR) of North America in 1991 and to date there are over 800 patients enrolled, making this the largest of all the worldwide DBA registries. The DBAR has received funding from the National Institutes of Health/National Heart, Lung and Blood Institute and the Centers for Disease Control. These data along with the DBA DNA Biorepository have been instrumental in the discovery of 11 of the 28 confirmed DBA genes. I have been the Director of the DBAR since its inception. As such I am referred patients from throughout the United States, Canada and from abroad. I was the Principal Investigator of three Phase I/II clinical trials for DBA which are now closed. We anticipate more such therapeutic trials in the future. We are also presently working on gene therapy trials.