Pancytopenia-developmental delay syndrome

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

This study is testing a new approach for people with severe lung disease (like pulmonary fibrosis or COPD) who also have bone marrow failure. First, participants receive a lung transplant to restore breathing. Then, about 8 weeks later, they get a bone marrow transplant using the…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

This study tests whether giving blood transfusions at home is practical and acceptable for cancer patients who need regular transfusions. About 30 adults with blood cancers or bone marrow failure will receive 1-5 blood product units at home. Researchers will track how often trans…

This study uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study interviews patients and families who have experience with gene therapy for rare pediatric diseases, including sickle cell disease. Researchers want to understand their beliefs, attitudes, and what information they need. The goal is to create an online platform with edu…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

This study looks at how antibiotics given during a stem cell transplant change the bacteria in your gut. Researchers will track these changes in 255 adults getting a transplant for blood cancers or bone marrow failure. The goal is to understand the link between antibiotics and gu…

This study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

This study is testing a virtual reality (VR) program called Life at 100.4 to help teens and young adults with cancer, along with their caregivers, learn about their diagnosis and treatment in an immersive way. About 45 participants will use the VR tool, and researchers will measu…

This study collects blood samples from up to 700 people, including stem-cell transplant patients and their donors, to understand how the immune system recovers after transplant. Researchers will analyze immune cells and T cell subsets to learn about immune reconstitution and graf…

This study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…