Aplastic anemia Aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… and myelodysplastic syndrome are diseases that affect the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… leading to failure of the bone marrow to produce normal blood and immune cells. These diseases are fatal if untreated. Donor bone marrow stem cell transplant (BMT) is the only cure for such bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… (BMF) conditions. However, graft versus host disease (GVHD) is a frequent side effect of BMT and can cause significant suffering and death. GVHD commonly affects skin, intestines, lungs, liver, eyes, and mouth. Success of BMT depends on successful treatment of GVHD. Treatment for GVHD involves immune suppressive medications which can also cause significant side effects. Early prediction of the severity of GVHD can be extremely helpful to adjust the doses of immune suppressive medications and improve outcomes of GVHD. There are no blood tests that can accurately predict outcomes of GVHD. Thymic stromal lymphopoietin (TSLP) is a protein secreted by the cells lining organs such as skin, intestines, and lungs, the same organs that are affected by GVHD. We therefore hypothesized that the levels of TSLP in the patients blood can reflect the degree of damage to these organs by GVHD. Our hypothesis was true, when we tested TSLP levels in the blood of patients with GVHD at Stanford University. We noted that certain types of TSLP called isoforms, were 3 to 4-times higher in patients who died from intestinal GVHD compared to those who survived. However, the number of patients in our study was small. We consulted a statistician within Stanford University, and their recommendation was that we needed blood samples from 60 patients with intestinal GVHD (30 of whom died and 30 who survived after having GVHD). In order to obtain such a large number of patient samples within a short period of time, we started a collaboration with the Mount Sinai Acute GVHD International Consortium (MAGIC) in New York. MAGIC is a group of 25 BMT institutions within the US and other countries that actively participate in GVHD research and routinely collect and store patient samples. As a result of this collaboration, we will receive 60 patient samples from MAGIC in 11/2024. These samples will be tested for TSLP isoforms and the results will be correlated with the outcomes of these patients. We will analyze these results with the expertise of our statistician. If our hypothesis holds true in the MAGIC patients, and TSLP is significantly higher in patients who succumbed to intestinal GVHD, these results can be immediately applied to patients who are undergoing BMT throughout the country and the world. The intensity of their treatment can be modified according to the TSLP levels, which will allow for lower rates of illness and death from GVHD. This study also opens an avenue to study Tezepelumab, an antibody targeting TSLP, for the prevention or treatment of GVHD. Clinical trials Clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… have already established the safety of Tezepelumab in humans.