Bone Marrow Failure Disease Consortium

About the Bone Marrow Failure Disease Consortium

BMFDC Patient Contact Registry

BMFDC Protocols

About the Bone Marrow Failure Disease Consortium

To address the challenges in diagnosing and treating bone marrow diseases, the National Institutes of Health (NIH), through its Office of Rare Diseases has awarded a $4.5 million grant to a group of expert physicians and their teams called the Bone Marrow Failure Disease Consortium (BMFDC).  These distinguished and recognized authorities in treating bone marrow diseases are holding clinical studies at four centers of excellence across the US to find advances in treatment options for patients.  The BMFDC functions under the umbrella of the NIH's Rare Disease Clinical Research Network (RDCRN) along with nine other consortium which are part of this $51 million grant to study numerous other rare diseases. 

"Funding research on rare diseases is a vital aspect of the NIH mission," said NIH Director Elias A. Zerhouni, M.D. "By encouraging cooperative partnerships among the investigators at these centers we hope to accelerate the development of diagnostics and treatments that will benefit these important patients."

The BMFDC studies aplastic anemia, myelodysplastic syndromes, paroxysmal nocturnal hemoglobinuria, and single lineage cytopenias such as large granular lymphocyte leukemia and pure red cell aplasia.  While these and other rare diseases affect an estimated 25 million people in the U.S., the patient populations are widely distributed geographically.  Research therefore requires significant collaboration among scientists and research centers, one of the primary ideas behind the RDCRN and BMFDC.

"The network [RDCRN] will facilitate increased collaboration and data sharing between investigators and patient support groups working to improve the lives of those affected by these diseases and potentially prevent or eliminate these diseases in the future," said Stephen Groft, Pharm.D., director of NIH's Office of Rare Diseases.

Another important goal of the BMFDC is to enhance the availability, accessibility, and quality of information offered to patients and health care professionals.  To coordinate efforts in this area, AA&MDSIF serves as a clearinghouse for patient education about clinical research studies and for information about current advances in research.  For this role, the AA&MDSIF draws upon its wide reach and extensive information resources as a patient advocacy group focused on bone marrow diseases. 

BMFDC Patient Contact Registry

The BMFDC created a Patient Contact Registry where patients and their families can sign up to be contacted in the future about clinical research opportunities.  Every patient who is considering participating in a clinical study may want to explore this opportunity.

Join the BMFDC Patient Contact Registry by:

• mailing or faxing the attached form
• calling 866.313.9879 and registering using the telephone automated system
• visiting http://rarediseasesnetwork.epi.usf.edu/bmfdc/index.htm and completing the online registration form (click Join the Contact Registry)

If you have questions or would like to discuss the BMFDC Contact Registry, please call 800.747.2820 or email clinicaltrials@aamds.org.  

Click here to learn more about the Bone Marrow Failure Disease Consortium.