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Patient Recruitment

Eltrombopag for Patients with Fanconi Anemia

Have You Been Diagnosed With Fanconi Anemia?

mother and child smiling


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 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.

Eligible participants:

  • 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 three-and-a-half years.
  • Participants with improvement in their blood cell counts at six months will have the option to continue taking the medication for an additional three 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, MD, on the Metro red line (Medical Center stop).

For more information, call:
Office of Patient Recruitment
TTY: 1-866-411-1010
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Or go online:
Refer to study #17-H-0121

Department of Health and Human Services
National Institutes of Health Clinical Center
National Heart, Lung, and Blood Institute

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This page last updated on 07/14/2022

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