Clinical Trial: Identifying Characteristics of Bone Marrow Failure Syndromes

Study Status: Active, not recruiting
Recruit Status: Unknown status
Study Type: Observational




Official Title: Screening Protocol and Longitudinal Study of Bone Marrow Failure Syndromes and Cytopenias

Brief Summary: Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells. The deficiency of blood cells, or cytopenia, caused by this malfunction leads to an assortment of diseases and disorders, all of which are characterized as BMFS. Because these diseases are rare, conducting research on them is difficult, and standards of treatment for most BMFS have yet to be developed. This study will collect clinical and laboratory data from people with BMFS to identify the characteristics and biological markers associated with these diseases over time. This information will assist doctors and researchers to develop better therapies and diagnostic tests that will help improve the management of BMFS and cytopenias.