Sideroblastic anemia
MONDO:0015194A group of rare heterogeneous inherited or acquired bone marrow disorders, isolated or part of a syndrome, characterized by decreased hemoglobin synthesis, because of defective use of iron (although plasmatic iron levels may be normal or elevated) and the presence of ringed sideroblasts in the bone marrow due to the pathologic iron overload in mitochondria as visualized by Perls' staining. The group encompasses (idiopathic) acquired sideroblastic anemia and constitutional sideroblastic anemias. The latter include syndromic sideroblastic anemias such as Pearson syndrome, mitochondrial mypathy and sideroblastic anemias, x-linked sideroblastic anemia-ataxia, thiamine responsive megaloblastic anemia syndrome and nonsyndromic sideroblastic anemias comprising x-linked and autosomal recessive sideroblastic anemias.
Also known as: anaemia sideroblastic, anemia sideroblastic, sideroblastic anemia
21 clinical trials for this condition and its sub-types.
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Broader categories
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New drug combo shows promise for MDS and AML
Disease control OngoingThis study tests two drugs, vorinostat and azacitidine, together in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to find the best dose and see if the combination is safe and effective. About 135 participants will take part in this early-…
Phase: PHASE1, PHASE2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Aug 09, 2026 00:00 UTC
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New drug combo aims to make bone marrow transplants safer for kids
Disease control OngoingThis pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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Could stem cells slow MSA? new trial tests safety and effects
Disease control OngoingThis study tests whether a person's own stem cells can be safely injected into the spinal fluid to treat multiple system atrophy (MSA), a rare and serious brain disease. About 30 adults aged 30-80 with MSA will receive the treatment. The main goal is to check for side effects, an…
Phase: PHASE1, PHASE2 • Sponsor: Mayo Clinic • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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New blood treatment could block zika in transfusions
Disease control OngoingThis study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…
Phase: PHASE3 • Sponsor: Cerus Corporation • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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New hope for anemia patients: drug may cut need for blood transfusions
Disease control OngoingThis study tests a drug called luspatercept in people with anemia caused by myelodysplastic syndromes (MDS) or beta-thalassemia who need regular blood transfusions. The goal is to see if the drug is safe and can reduce the number of transfusions needed. About 85 participants in I…
Phase: PHASE4 • Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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New drug may cut transfusions for MDS patients
Disease control OngoingThis study tests a drug called luspatercept in 70 adults with a low-risk form of MDS that causes anemia. The goal is to see if the drug can reduce the need for red blood cell transfusions. Participants receive the drug and are monitored for safety and effectiveness over time.
Phase: PHASE3 • Sponsor: GWT-TUD GmbH • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC