Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Status:
Recruiting
Trial end date:
2027-06-30
Target enrollment:
Participant gender:
Summary
Background:
Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids,
or steroids don t work. Other patients must get blood transfusions regularly which are time
consuming and can have significant side effects. The drug eltrombopag can increase red blood
cells. Researchers want to see if it can help people with DBA and, if so, for how long.
Objective:
To study the safety and efficacy of eltrombopag in people with DBA who have not responded to
steroids or could not take them.
Eligibility:
People ages 2 and older with DBA who did not respond to steroids or could not take them, or
their disease has returned despite taking them
Design:
Participants will be screened with:
Medical and medicine history
Physical exam
MRI: Participants will lie in a machine that takes pictures of the liver.
Blood and urine tests
Bone marrow biopsy: A thin needle will remove a marrow sample from the participant s hip
bone.
Electrocardiogram
Participants will take eltrombopag pills once daily for 24 weeks. They will have blood taken
every 2 weeks.
Participants will have visits 6 months. At 6 months, they will repeat all the screening tests
and also have:
Quality-of-life questionnaire
Neurodevelopmental test (for participants younger than 18 years)
If participants blood cell counts improve, they may keep taking eltrombopag for up to 3 more
years. If so, they will have blood taken every 4 weeks. They will visit NIH every 6 months
and repeat the above tests.
Participants will be monitored for up to 3 years after they stop taking eltrombopag. They
will visit NIH 6 months after treatment ends. If participants blood counts go down after
treatment ends, they may restart the drug....