Diamond-Blackfan anemia
MONDO:0015253A congenital aregenerative and often macrocytic anemia with erythroblastopenia.
Also known as: Aase syndrome, Aase-Smith II syndrome, Blackfan-Diamond anaemia, Blackfan-Diamond anemia, DBA, Diamond Blackfan Anemia, Diamond-Blackfan anemia, chronic constitutional pure red cell anemia
24 clinical trials for this condition and its sub-types.
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Broader categories
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Blood transfusion thresholds tested to save elderly surgical patients
Disease control CompletedThis study tests whether giving blood transfusions more liberally (when hemoglobin drops to 9 g/dL) versus more restrictively (when it drops to 7.5 g/dL) reduces death and serious complications like heart attack, stroke, or kidney injury in patients aged 70 and older undergoing n…
Phase: PHASE3 • Sponsor: Johann Wolfgang Goethe University Hospital • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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New hope for rare blood disorder: experimental pill reduces need for transfusions
Disease control CompletedThis study tested a drug called bitopertin in 15 adults with Diamond-Blackfan anemia (DBA), a rare inherited condition that causes severe anemia. Participants took the pill daily for 8 months, with doses possibly increasing over time. The goal was to see if the drug could raise h…
Phase: PHASE1, PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New transplant cocktail aims to cut complications in bone marrow failure
Disease control CompletedThis phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…
Phase: PHASE2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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One donor, two transplants: could this end lifelong Anti-Rejection drugs?
Disease control CompletedThis pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…
Phase: NA • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Anemia in kids may be tied to bigger hearts, study finds
Knowledge-focused CompletedThis study explored whether low hemoglobin (anemia) is linked to an enlarged heart (cardiomegaly) in children up to 18 years old. Researchers used chest X-rays and echocardiograms to check for heart enlargement in 56 anemic children. The goal was to better understand how anemia a…
Sponsor: Universitas Sumatera Utara • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC