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Glossary of Terms

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Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (ALL) is a fast-growing cancer of the white blood cells. Lymphocytes are a type of white blood cell that the body uses to fight infections. In ALL, the bone marrow makes lots of unformed cells called blasts that normally would develop into lymphocytes. However, the blasts are abnormal. They do not develop and cannot fight infections. The number of abnormal cells (or leukemia cells) grows quickly. They crowd out the normal red blood cells, white blood cells and platelets the body needs.

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Acute Myelogenous Leukemia

Acute myelogenous leukemia (AML) is a fast-growing cancer of the blood and bone marrow. In AML, the bone marrow makes many unformed cells called blasts. Blasts normally develop into white blood cells that fight infection. However, the blasts are abnormal in AML. They do not develop and cannot fight infections. The bone marrow may also make abnormal red blood cells and platelets. The number of abnormal cells (or leukemia cells) grows quickly. They crowd out the normal red blood cells, white blood cells and platelets the body needs.

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Adult Stem Cells

Also called non-embryonic stem cells - any kind of stem cell that has been taken from an adult source. Adult stem cell sources include bone marrow cells, umbilical cord blood cells and peripheral blood stem cells (PBSC). Adult/non-embryonic stem cells are completely without controversy because they are taken from post-birth individual donors who are unharmed by the harvesting process. No embryos or unborn are involved in their collection or transplantation. Meanwhile, adult stem cells offer a gift of life to patients with leukemia, other cancers, anemia and a host of other serious illnesses.

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Allogeneic Transplantation

In an allogeneic transplant, the patient gets bone marrow/stem cell or cord blood cells from a donor. The donor's tissue type (HLA type) must closely match the patient's. The donor can be either related or unrelated. Related donors are usually a brother or sister, but only 25% of all patients usually find a match in their family, or find a donor from their community. This means 75%, an overwhelming majority, will need to find an unrelated donor. can help with finding an unrelated bone marrow/stem cell or cord blood donor.

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A simple, painless process in which blood is drawn from a patient's or donor's arm and circulated through a filter which removes stem cells from the blood. The rest of the blood is returned back to the patient or donor. Apheresis produces no side effects or discomfort for the blood stem cell donor. This is the procedure used for a Peripheral Blood Stem Cell (PBSC) Donation.

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Aplastic Anemia (Severe)

Aplastic anemia is a disease of the bone marrow. The bone marrow stops making enough red blood cells, white blood cells and platelets for the body. Any blood cells the marrow does make are normal, but there are not enough of them. Aplastic anemia can be moderate, severe or very severe. People with severe or very severe aplastic anemia are at risk for life-threatening infections or bleeding.

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Autologous Transplantation

In an autologous transplant, the patient's own blood stem cells are collected from the bone marrow or peripheral blood and frozen. The patient then receives high-doses of chemotherapy and/or radiation therapy to destroy the cancerous cells. The extracted stem cells are also treated to destroy the cancer cells, and then re-infused into the patient.

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Be The Match Foundation - National Bone Marrow Registry / National Marrow Donor Program (NMDP)

A confidential national database of potential volunteer bone marrow stem cell donors established and maintained by the National Marrow Donor Program.

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Blast Cells

Immature white blood cells. Healthy bone marrow makes stem cells that grow into red blood cells, white blood cells, and platelets. A leukemia patient's bone marrow makes too many blast cells (immature white blood cells). Leukemic blast cells remain immature and do not function like mature white blood cells, which are supposed to destroy infection by bacteria and fungi. This results in anemia and vulnerability to infection.

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Blood Stem Cell Transplant

Replacing a patient's diseased blood cells with healthy new blood stem cells from a donor; also known as a Peripheral Blood Stem Cell (PBSC) Transplant. However, these blood cells must match the patient's own cells as closely as possible, which is why HLA typing is crucial to the process.

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Blood Stem Cells

Blood stem cells are  among several types of stem cells. Healthy blood stem cells replace the body's supply of red blood cells, white blood cells, and platelets in the body that are depleted by diseases such as leukemia. Often, the only hope for a cure is a blood stem cell transplant.

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Bone Marrow

A soft, spongy tissue that fills the cavities inside most human bones. Bone marrow contains adult stem cells which generate all our red blood cells, white blood cells, and platelets. It is the most common source for stem cell transplantation.

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Bone Marrow Donation

A surgical procedure by which a person donates a portion of his/her bone marrow to a patient who has a disease which requires a bone marrow transplant.

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Bone Marrow Transplant

A simple procedure in which bone marrow is drawn from a donor's pelvic (hip) bone and injected into the bloodstream of a patient who needs to replenish a healthy supply of adult stem cells.However, the donated bone marrow cells must match the patient's own cells as closely as possible, which is why HLA typing is crucial to the process.

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Treatment of cancer or other diseases by the use of drugs that selectively destroy rapidly growing cells. Extremely high doses unfortunately also kill the patient's own stem cells in the bone marrow and peripheral blood stream. This is why chemotherapy should be done in conjunction with a bone marrow or stem cell transplant, to prevent the patient from suffering grave effects from the loss of their stem cells.

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Chronic Lymphocytic Leukemia

Chronic lymphocytic leukemia (CLL), like all leukemias, is a cancer of the blood cells. CLL affects a certain type of white blood cell, called a B lymphocyte. This disease is also called B-cell chronic lymphocytic leukemia. B cells originate in the bone marrow, mature in the lymph nodes, and then circulate in the blood to help fight infections.

In CLL, malignant (cancerous) B cells grow into mature cells, but they don't work properly. These defective B cells fight infections poorly and they can crowd out the normal lymphocytes in the marrow and lymph nodes.

CLL is primarily an adult disease; it is very rare in children and young adults. The median age of diagnosis is 72 years, and about 60% of all people getting CLL are male. In the United States, about 15,000 people are diagnosed with CLL each year. (Data from the National Cancer Institute.)

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Chronic Myelogenous Leukemia

Chronic myelogenous leukemia (CML) is a slow-growing bone marrow cancer resulting in too many white blood cells. The disease is also sometimes called chronic myeloid, chronic granulocytic or chronic myelocytic leukemia. CML is a relatively common form of leukemia, but overall it is a relatively uncommon type of cancer. In the United States, more than 20,000 people have CML and about 4,600 new cases are diagnosed each year. Most cases of CML appear in adults, but about 2-4% of CML patients are children.

CML is caused by a change in the genetic code of some of the cells in the bone marrow. In these cells, part of chromosome 9 switches places with a part of chromosome 22, a process called chromosomal translocation. This creates an abnormal chromosome called the Philadelphia chromosome. The rearranged Philadelphia chromosome signals the marrow to overproduce white blood cells. Doctors do not know what causes the Philadelphia chromosome to appear.

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The chemotherapy and/or radiation given to patients before a bone marrow/stem cell, cord blood or peripheral blood stem cell transplant. The purpose is to kill diseased cells so the new cells can grow normally.

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Cord Blood

Blood remaining in the umbilical cord immediately following the birth of a baby. It contains a rich concentration of stem cells which can save the life of a leukemia or other patient requiring a stem cell transplant. Umbilical cord blood stem cells are considered adult stem cells because they come from the mother's tissue.The baby is in no way harmed or affected by collection of umbilical cord blood and no embryos are involved. Umbilical cord blood is an alternative source of stem cells if bone marrow stem cells or bone marrow donors are not available.

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Cord Blood Bank

An organization that helps to collect and store umbilical cord blood for transplantation. Cord blood banks recruit expectant mothers who donate their baby's umbilical cord blood for stem cell transplants. The blood in the umbilical cord contains large numbers of stem cells. The cord blood banks collect, process, test and store the donated umbilical cord blood. Blood from each cord is frozen (cryopreservervation) as an individual cord blood unit that is available to transplant.

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Storage of biological materials at temperatures of -196°C (-321°F) until needed for transplant.

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DNA Based HLA Typing

Determining a person's HLA type by testing the DNA. The test is very accurate and efficient and the kit can be ordered conveniently online.

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Donor Workup

The process that a closely matched potential donor undergoes to ensure that he or she is healthy and ready to donate marrow or blood stem cells. Workup includes a detailed information session at the donor Ccnter, a complete physical examination and donation of blood samples for testing and research.

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The process by which newly transplanted stem cells migrate to and nest in the appropriate site of the recipient's body and start producing normal quantities of normal mature cells.

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A protein that helps bone marrow make more white blood cells. Filgrastim is also known as GCSF (granulocyte-colony stimulating factor) or by the trade name Neupogen®. It is given to donors who have agreed to donate peripheral blood stem cells. This moves blood stem cells from the bone marrow into the blood stream so that they can be collected by apheresis. It also helps patients increase their white blood cell count after the transplant.

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"Graft Versus Host Disease" (GVHD) - A potential complication of transplants associated with the use of blood or tissue from a different person (allogeneic). In GVHD, the transplanted cells reject the recipient's tissue as foreign and attack the tissue. GVHD in stem cell transplants appears to be less severe with umbilical cord blood, which appears to be more tolerant of the new body's environment. To reduce the risk of GVHD or tissue rejection, HLA testing is required.

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Hematopoietic Stem Cells

The "Master" (blood forming) stem cells that are capable of recreating themselves and creating all of the other types of blood cells. They number 1 in 10,000 cells in bone marrow, and 1 in 1,000 cells in umbilical cord blood.

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"Human Leukocyte Antigens" (HLA) - Proteins on white blood cells that make each person's tissue unique. The HLA A, B, C and DR proteins are important in matching patients and donors for a bone marrow or blood stem cell transplant. This matching process is known as HLA typing. The more closely matched the recipient and donor's HLAs are, the more likely the transplanted tissue will be compatible with and tolerated by the recipient.

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HLA Typing

A person's own specific HLA A, B, C and DR proteins. Patients are matched with stem cell donors or cord blood units by testing and comparing their HLA tissue types. Siblings are the first candidates to be tested for a match, since a brother or sister may have inherited the same HLA type. But even with siblings, there is only a 25% chance for a match, leaving 75% of all patients seeking a life-saving match from an unrelated donor.

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"Hematopoietic stem cell transplantation" (HSCT) - Entails the use of multipotent hematopoietic stem cells, typically from bone marrow, peripheral blood, or umbilical cord blood. HSCT is often a treatment of choice for patients with multiple myeloma, leukemia and other cancers of the blood or bone marrow. In these procedures, patients first undergo radiation or chemotherapy to wipe out their immune system. Next, they receive HSCT cells from a matching donor identified through lab results or Registries with data bases of volunteer donors.

Throughout this website we will refer to the different transplants as HSCT.

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Inherited Immune System Disorders

Inherited immune system disorders are diseases in which children are born without an immune system or with an incomplete immune system. People with an immune system disorder are less able to fight infections. While these disorders are rare, there are about 100 different types, which range from mild to severe. About 50,000 people in the United States have some type of inherited immune system disorder.

Inherited immune system disorders are also called primary immune deficiency disorders. They are called "primary" because they are caused by a gene defect that affects the immune system. Genes carry an inherited code of instructions that tells the body how to make every cell and protein in the body. (Secondary immune deficiencies are caused by something outside the body, such as chemotherapy or infection with a virus.)

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Inherited Metabolic Storage Disorders

Inherited metabolic storage disorders are a group of inherited diseases in which the lack of an enzyme affects various organs and tissues, including the brain. Enzymes are proteins that play many roles, including to metabolize (break down) substances in the body.

In metabolic storage disorders, the body lacks an enzyme needed to metabolize a substance, such as a sugar. Instead, the substance builds up in the body, where it can damage the brain, nervous system, bones, organs and other tissues. Different metabolic disorders affect different enzymes and cause different types and levels of damage.

Metabolic storage disorders are caused by a mutation (mistake) in a gene that affects metabolism. Genes carry an inherited code of instructions that tells the body how to make every cell and substance in the body.

These disorders are rare. Many of them appear in childhood, although some can also appear in adults. A bone marrow or cord blood transplant (also called a BMT) is a treatment option for some of these disorders.

Mucopolysaccharidoses, also called MPS disorders, are a subgroup of metabolic disorders. Some MPS disorders have been treated with transplant:

  • Hurler's syndrome (MPS I) — this is the form of MPS that doctors have the most experience treating with transplant, since 1980
  • Maroteaux-Lamy syndrome (MPS VI)
  • Sly syndrome (MPS VII)
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Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are a group of diseases that affect the bone marrow and blood. Some types of MDS are mild and easily managed, while other types are severe and life-threatening. Mild MDS can grow more severe over time. It can also develop into a fast-growing, severe leukemia called acute myelogenous leukemia.

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Non-embryonic stem cells

Synonymous with adult stem cells, i.e. stem cells donated by fully grown adults who are unharmed in the process. No embryos or unborn are involved in their collection or transplantation.

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Non-Hodgkin Lymphoma

Non-Hodgkin lymphoma (NHL) is a cancer of a type of white blood cell called lymphocytes. Lymphocytes are part of the immune system that protects the body from infection and disease. NHL is a group of 30 or more cancers that begin in the lymphocytes.

These cancers are similar in some ways but have many differences in the way they affect people. They are all called non-Hodgkin lymphoma to distinguish them from another cancer of the lymphocytes called Hodgkin lymphoma.

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"Peripheral Blood Stem Cells" (PBSC) - Blood derived from a patient's blood stream, as opposed to blood in the bone marrow where it is made. Blood stem cell production is stimulated in the donor by administration of the drug Filgrastim and then harvested painlessly from the donor through a medical procedure called apheresis.

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Peripheral Blood Stem Cell (PBSC) Donation

Hematopoietic stem cells are collected from a donor's circulating blood through an apheresis procedure after accelerating production from the bone marrow with Filgrastim. These adult stem cells are then transplanted into the recipient patient. However, these blood stem cells must match the patient's own cells as closely as possible, which is why HLA typing is crucial to the process.

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Pre-transplant Conditioning

A regimen of chemotherapy (with or without radiation therapy) that destroys a patient's diseased bone marrow stem cells. The bone marrow cells are then restored by transplanting new adult stem cells via bone marrow/stem cell or cord blood transplant.

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Radiation therapy

Radiation is used to kill rapidly growing cancer cells or other malignancies. Unfortunately, high doses of radiation, especially in combination with chemotherapy, also kill the body's adult stem cells in the bone marrow and peripheral blood stream. This is why a bone marrow/stem cell or cord blood stem cells transplant is performed, to replenish the patient with new, healthy cells.

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Severe Combined Immunodeficiency (SCID)

Severe combined immunodeficiency (SCID) is the name for a group of inherited immune system disorders. SCID disorders are the most severe of the inherited immune system disorders. Babies are born with these disorders, which can become life-threatening within the first year of life. SCID is rare. About 1 in 200,000 babies are born with SCID.

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Sickle Cell Anemia

Sickle cell anemia (also called sickle cell disease or SCD) is an inherited disease in which a person does not have enough healthy red blood cells to carry oxygen throughout the body.

Sickle cell disease is the most common genetic disease in the world and is most common in people whose families come from Africa, South or Central America (especially Panama), Caribbean islands, Mediterranean countries (such as Turkey, Greece, and Italy), India, and Saudi Arabia.

In the United States, about 1 in every 500 African Americans are born with the disease. Hispanic Americans also are affected at a rate of about 1 in 1,000 to 1,400.

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Stem Cell Transplant

The process of infusing healthy blood stem cells into persons who have undergone high-dose chemotherapy for one of many forms of leukemia, immunodeficiency, lymphoma, anemias, metabolic disorders and many other diseases. Healthy stem cells are collected from bone marrow, peripheral blood, or umbilical cord blood. Once the donor's healthy stem cells are infused into the patient's blood stream, the cells move from the blood vessels to the center of the bones, where they begin making new blood cells.

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Stem Cells

Certain cells in the body that can grow into other kinds of cells. There are two kinds of stem cells - embryonic stem cells and non-embryonic (adult) stem cells. does not deal in any way with embryonic stem cells taken from the unborn. Our focus is on testing adult stem cell donors for patients suffering from leukemia and other diseases whose lives can be saved via adult stem cell donation. Adult stem cells include bone marrow stem cells, umbilical cord blood stem cells, and peripheral blood stem cells (PBSC).

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The process of transferring tissues or cells from a healthy person to an ill person to treat their disease. The tissue or cells transplanted may come from the same patient (autologous) or from another person (allogeneic).

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Umbilical Cord Blood Stem Cells

Stem cells from the blood of the umbilical cord or placenta after the birth of a baby. These cells have the potential to produce all the components of blood in the same manner as stem cells derived from bone marrow. Umbilical cord blood cells are not embryonic. No harm occurs to the child or mother during the collection process; meanwhile the cord blood can help save others' lives.

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Wiskott-Aldrich Syndrome

Wiskott-Aldrich syndrome (WAS) is an inherited immune system disorder that can lead to frequent infections and problems with bleeding too easily. WAS occurs only in males and appears mostly in children but sometimes does not appear until adulthood. About 4 out of every 1 million boys born in the United States has WAS. The only treatment that can cure WAS is a bone marrow or cord blood transplant (also called a BMT).

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