Featured Studies

Severe aplastic anemia is a rare and serious blood disorder. Patients with severe aplastic anemia commonly receive Cyclosporine to help improve blood counts. When cyclosporine treatment stops, the disease may return in one in three people. Researchers at the National Institutes of Health (NIH) Clinical Center are studying if sirolimus, an immunosuppressant, can help prevent a relapse in patients diagnosed with severe aplastic anemia following cyclosporine treatment.

Treatment of severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) usually involves a bone marrow transplant. Researchers at the National Institutes of Health (NIH) are testing ways to make transplant using unrelated donor cells from umbilical cord blood safer and more effective. We hope these procedures will reduce post-transplant complications and improve transplant outcomes.

Fanconi anemia is a rare genetic disorder that mostly affects children and often leads to bone marrow failure. Researchers at the National Institutes of Health (NIH) are investigating a new drug to improve blood counts in individuals diagnosed with Fanconi anemia. Eltrombopag is an oral drug that mimics a special protein that causes the body to make more platelets. Currently eltrombopag is used to treat low platelet counts in patients with hepatitis C and chronic immune thrombocytopenic purpura (ITP). Researchers are evaluating if this new drug will improve blood cell counts in individuals diagnosed with Fanconi anemia.

Researchers at the National Institutes of Health (NIH) are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease. In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more about the role of different doses of danazol on telomere disease as well as its effects on secondary diseases.

The research study sets out to learn about changes in the genetic makeup of CLL during the early phase of treatment with venetoclax. The initial phase of venetoclax therapy can be medically and logistically challenging. Patients will receive expert medical care at the National Institutes of Health (NIH).

Doctors at the National Institutes of Health (NIH) are conducting a study on patients with clonal hematopoiesis of indeterminate potential (CHIP). Patients with CHIP have a genetic mutation that has been associated with blood cancer. The study will look at CHIP and try to determine its relationship to blood cancers and heart disease. The study will also try to discover other new organs or diseases that may be linked with.

Doctors at the National Institutes of Health (NIH) are conducting a study on patients with Clonal Cytopenia of Uncertain Significance (CCUS). Patients with CCUS have low blood counts and a normal bone marrow exam but more advanced genetic testing shows the presence of a genetic mutation that is likely causing the low counts. The study will look at CCUS and try to determine its relationship to blood cancers and heart disease. The study will also try to discover other new organs or diseases it may be linked with.

Researchers at the National Institutes of Health (NIH) are studying if low dose CT scans are as effective as regular CT scans to monitor your disease status. Very low-dose scanning uses about 1/30th the amount of radiation as a regular CT scan.

Cystic fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and over time limits the ability of individuals to breathe. Researchers at the National Institutes of Health (NIH) are studying the infection, disease progression, and abnormal genes responsible for cystic fibrosis to better understand the disease process and its effects on breathing.

NIH study # 000479-H investigates the impact of treatments intended to cure sickle cell disease (SCD) on organ function. If you or your loved one has SCD, we invite you to call us to learn more about this observational study.