Researchers at the National Institutes of Health in Bethesda, MD, are conducting an investigational treatment study with eltrombopag to help patients with Diamond-Blackfan anemia (DBA). Eligible volunteers must be diagnosed with DBA, especially DBA that has not responded to or recurred despite standard therapies. DBA is an inherited disease that can lead to anemia and ultimately bone marrow failure, with increased risks of cancer. Eltrombopag is a drug that can help patients with aplastic anemia, an acquired bone marrow failure syndrome. Whether it can also help patients with different, inherited bone marrow failures is a point of ongoing research at the NIH. This study will attempt to determine whether eltrombopag is safe and effective for patients with DBA.
Though considered primarily a blood disease, DBA may affect all systems of the body, especially as the treatments involved (chronic transfusions and/or steroids) have wide-ranging effects. Eltrombopag has been shown to help reduce the iron that can build up in DBA patients, and consequently may help reduce some of these side effects. In addition to investigating whether eltrombopag can improve blood cell counts in DBA patients, this study will help answer this question. Travel, food and lodging may be provided consistent with guidelines of the National Heart, Lung and Blood Institute policies.
Study Design:
- One to two visits to the NIH Clinical Center in Bethesda, MD, before starting drug and then every six months while on drug
- Patients will take eltrombopag over a six-month period
- Patients who exhibit improvements after six months of taking eltrombopag may be eligible to continue taking eltrombopag for up to three additional years
Who Can Particpate:
- Patients 2 years or older diagnosed with DBA
- Patients diagnosed with DBA who have not responded to therapy, have relapsed, and/or are unable to tolerate current therapy
- Patients willing to take eltrombopag for a minimum of 6 months
- Patients willing to have their blood drawn every two weeks by your home physician
The NIH Clinical Center is America's Research Hospital and is located in Bethesda, MD, on the Metro Red line (Medical Center stop).
For more information:
NIH Clinical Center Office of Patient Recruitment
800-411-1222
(TTY users dial 7-1-1)
Se habla español
Email: ccopr@nih.gov
Or go online:
https://go.usa.gov/xdNyc
Refer to study # 20-H-0021
Department of Health and Human Services
National Institutes of Health Clinical Center
National Heart, Lung and Blood Institute