Have You Been Diagnosed With Fanconi Anemia?
Fanconi anemia is a rare genetic disorder that mostly affects children and often leads to bone marrow failure. Researchers at the National Institutes of Health (NIH) are investigating a new drug to improve blood counts in individuals diagnosed with Fanconi anemia. Eltrombopag is an oral drug that mimics a special protein that causes the body to make more platelets. Currently eltrombopag is used to treat low platelet counts in patients with hepatitis C and chronic immune thrombocytopenic purpura (ITP). Researchers are evaluating if this new drug will improve blood cell counts in individuals diagnosed with Fanconi anemia.
- At least 4 years old.
- Have been diagnosed with Fanconi anemia by genetic testing.
- Have declined or not responded to treatment with danazol or oxymetholone.
- Weight more than 12 kg (26 lbs)
What is involved?
- A review of medical records, and physical exam to determine eligibility for the study.
- Participating in the study may last up to 3.5 years.
- Participants with improvement in their blood cell counts at 6 months will have the option to continue taking the medication for an additional 3 years.
- You may receive blood and or platelet transfusions.
- Tests, medication, and procedures conducted at the NIH Clinical Center are at no cost to you.
- Compensation for travel may be provided.
Location: The NIH Clinical Center is America's research hospital is located in Bethesda, Maryland, on the Metro red line (Medical Center stop).
For more information, call:
Office of Patient Recruitment
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Department of Health and Human Services
National Institutes of Health Clinical Center (CC)
National Heart, Lung, and Blood Institute (NHLBI)