Diamond-Blackfan Anemia
Steven and Alexandra Cohen Children's Medical Center has been designated by congress
as the primary resource for treating Diamond-Blackfan Anemia patients
and has treated hundreds of patients.
Bill
passed by the House of Representatives
Diamond Blackfan Anemia (DBA) is an extremely
rare, severe anemia of childhood. It is estimated that there are
only 25-30 new cases a year in the US and Canada. As such, there
are gaps in the understanding of the natural history of this disorder.
For this reason, the Diamond Blackfan Anemia Registry of North
America was established in 1993 to collect accurate clinical and
demographic data on DBA patients and their families.
Headquartered at Steven and Alexandra Cohen Children's Medical Center in New York,
directed
by physician-researchers Adrianna Vlachos, MD, and Jeffrey Lipton,
MD, PhD, the DBAR is
a research organization dedicated to acquiring, analyzing and
disseminating information on Diamond Blackfan anemia to affected
individuals, their families and medical professionals. The DBAR
has collected information on over 500 patients in its database.
Most DBA patients are diagnosed
within the first year of life. Potentially, DBA patients can live
long and healthy lives with appropriate medical treatment. The
two most common forms of treatment are blood transfusion therapy
and corticosteroid medication. Some DBA patients are in remission
and need no medical treatment related to their DBA. Other DBA
patients may choose to undergo the risky procedure of a stem cell
transplant (also referred to as a bone marrow transplant).
In recent years, the scientific community has discovered that
DBA is a ribosomal protein disease. This is a very exciting discovery
for the DBA community, as it has resulted in increased scientific
attention to this rare disease.
Diamond Blackfan Anemia is the preferred name for this disorder
but other names for DBA include:
• Blackfan Diamond syndrome
• Congenital pure red cell aplasia
• Congenital hypoplastic anemia
• Aase syndrome (felt to be a subset of DBA in which there
is a finger-like thumb, and not a distinct disorder)
If you have questions feel free to contact our
DBA Nurse at 1-877-DBA-NURSe (1-877-322-6877).
PATIENT RESOURCES:
Diamond Blackfan Anemia Registry
Daniella Maria Arturi Foundation
Diamond Blackfan Anemia Foundation
CDC-Blood
disorders-DBA
