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Phase 1
For pediatric ages
For Seniors
Recruiting in 10s
Interventional Study
all
Recruiting

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

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. …

blood cell count

corticosteroids

eltrombopag

anemia

bone marrow biopsy

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16 Feb, 2024
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