Endocrine Dysfunction in Diamond–Blackfan Anemia (DBA): A Report from the DBA Registry (DBAR)

Diamond-Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome. The mainstays of treatment involve chronic red cell transfusions, long-term glucocorticoid therapy, and stem cell transplantation. Systematic data concerning endocrine function in DBA are limited. We studied patients in the DBA Registry (DBAR) of North America to assess the prevalence of various endocrinopathies.In a pilot study, retrospective data were collected for...