Aplastic anemia

This study tests a new approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Patients receive a combination of umbilical cord blood and stem cells from a family member, with the cord blood chosen based on specific genetic markers rather than …

This phase II trial is investigating whether eltrombopag, a drug that stimulates platelet production, can help children with severe aplastic anemia when combined with standard immunosuppressive therapy. The study will enroll children with newly diagnosed, relapsed, or refractory …

This 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…

This study tested whether low doses of the hormone drug danazol can slow the shortening of telomeres—the protective caps on DNA—in people with telomere diseases. These conditions can cause bone marrow failure, lung scarring, and liver damage. Eighteen participants took danazol fo…

This phase 2 trial tested a combination of rabbit antithymocyte globulin (Thymoglobuline) and ciclosporin in 35 people with acquired aplastic anemia, a condition where the bone marrow stops making enough blood cells. The goal was to see if this rabbit-based immune-suppressing tre…

This study tested whether adding eltrombopag to standard immunosuppressive therapy improves early recovery in patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. 202 participants received either standard treatment (horse a…

This study tested the drug eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Eltrombopag is a pill that mimics a natural protein to stimulate blood cell production. Researchers measured whether it safely imp…

This study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…

This study tested a personalized geriatric optimization plan (GO!) in 30 adults aged 60 and older who were getting a donor stem cell transplant for blood cancers or bone marrow failure. The plan included tailored advice on diet, sleep, activity, and medications to improve physica…

This study tested a stem cell transplant from a half-matched family donor for people with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. The goal was to see if this approach could improve survival one year after the transplant. The tria…

This 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…

This study reviewed medical records of 13 Korean children with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Researchers wanted to see if adding eltrombopag to standard immunosuppressive therapy was safe and effective. Over 6 months, t…

This study tested a new 9-day treatment plan for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The treatment combines three drugs (rabbit antithymocyte globulin, cyclosporine, and levamisole) to suppress the immune sys…

This study tested whether adding eltrombopag (a platelet booster) to standard immune-suppressing drugs (r-ATG and cyclosporine A) helps patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. Thirty-six East-Asian adults and c…

This study tested whether a low dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (GVHD) — a common and serious complication where donor cells attack the patient's body. The trial involved 35 adults with various blood dis…

This study tested a drug called eltrombopag in 20 Chinese adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants had not responded to previous treatments. The drug was given daily for up to a year to see if it could imp…

This study tested a two-step stem cell transplant for people with high-risk blood cancers like leukemia and lymphoma. First, patients got low-dose chemotherapy and radiation to prepare the body. Then, they received donor stem cells and immune cells to help destroy remaining cance…

This study reviews a modified transplant conditioning regimen for severe aplastic anemia in 72 patients across two hospitals. The approach uses a combination of fludarabine, melphalan, and cyclophosphamide to prepare the body for a stem cell transplant. Researchers looked at how …

This phase 2 trial tested whether adding luspatercept to standard cyclosporine treatment helps people with non-severe aplastic anemia. The study enrolled 58 newly diagnosed adults who had low blood counts but did not yet need transfusions. The goal was to see if the combination c…

This study tests whether moxibustion, a traditional therapy that burns herbs near the skin, can prevent hemorrhagic cystitis (painful bladder inflammation) in 266 patients receiving stem cell transplants. Participants are randomly assigned to receive either standard care or stand…

This study looks back at medical records of people who had an allogeneic stem cell transplant to see whether romiplostim helped their platelets recover faster. Platelets are blood cells that help clotting, and low counts are common after transplant. The researchers will compare p…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This completed registry study followed 80 children and young adults with severe immune cytopenias, including immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome. Researchers observed participants over up to 4 years to improve diagnosis and track the disease c…