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absolute neutrophil count - A measure of the actual number of mature neutrophils present in the blood per unit volume.

alemtuzumab - Alemtuzumab (Campath) is an antineoplastic or monocolonal antibody, a type of chemotherapy that inhibits or prevents the growth of diseased or cancerous cells by targeting the DNA. While alemtuzumab is approved by the FDA for treatment of certain types of leukemia, it is considered an investigational therapy for treatment of aplastic anemia.

allergen - A substance that causes an allergic reaction.

allogeneic stem cell transplant - a procedure in which a patient receives stem cells from a donor. The donor used in an allogeneic stem cell transplant can be the patient's identical twin, sibling, family member or an unrelated donor.

amifostine - Amifostine (Ethyol) is a cytoprotective agent or chemoprotectant, a drug approved by the U.S. Food and Drug Administration for use during cancer therapy to protect the body's normal cells. Amifostine is being studied in clinical trials, as a single agent or in combination with other medicines, for the treatment of MDS.

anaphylaxis - A rare but life threatening allergic reaction in which the blood pressure drops and breathing becomes difficult.

anemia - Any condition in which there are too few red blood cells in the bloodstream, resulting in insufficient oxygen to tissues and organs.

anti-thymocyte globulin - ATG (Atgam, Thymoglobulin) is an immunosuppressant, a drug therapy that reduces the body's natural immunity. It is approved by U.S. Food and Drug Administration (FDA) for treating moderate to severe aplastic anemia patients. ATG is most commonly used with cyclosporine, an oral medicine. ATG/cyclosporine combination is sometimes used for treating myelodysplastic syndromes (MDS), but is not nearly as effective against MDS as it is aplastic anemia.

antibodies - A protein produced by the immune system in response to the presence of an antigen (foreign substance).

anticoagulant - A drug that decreases the clotting ability of the blood and helps to prevent harmful clots from forming in the blood.

antigen - Protein present on the surface of cells that can stimulate an immune reaction.

aplastic - Involving the absence or defective development of a tissue or organ.

aplastic anemia - anemia that is characterized by defective function of the blood-forming organs (as the bone marrow) and is caused by toxic agents (as chemicals or X rays) or is idiopathic in origin

apoptosis - Programmed cell death.

arsenic trioxide - Arsenic trioxide (Trisenox) belongs to the general group of medicines called antineoplastics. Arsenic trioxide seems to interfere with the growth of cancer cells, which are then individually destroyed by the body. It may also affect normal cells. Researchers are studying arsenic trioxide for treating myelodysplastic syndromes (MDS) to determine whether it will be effective and safe to use.

arthritis - A condition that commonly produces pain, stiffness, inflammation and damage to joint cartilage and surrounding structures.

ascites - Extra fluid in the space between the lining of the abdomen and abdominal organs.

autoimmune - When the immune system attacks its own normal tissues by mistake.

autologous - Involving the patient serving as his/her own donor, especially in transfusions and bone marrow transplants.

azacitidine - Azacitidine (Vidaza) is a demethylating agent, a new class of drugs. Azacitidine is thought to work by restoring normal growth and differentiation of bone marrow cells. It is approved by the F.D.A. for treating myelodysplastic syndromes.

Band - An immature neutrophil.

band - An immature neutrophil. See Neutrophil.

basophil - A type of white blood cell that plays a role in allergic reactions.

bilirubin - A reddish yellow pigment that is found in the bile and blood and causes jaundice if accumulated in excess.

blast cells - Immature cells that mature into the three types of blood cells.

blasts - See Blast Cells.

blood clots - See Clot.

bone marrow - Soft tissue occupying the inner cavities of bones responsible for blood cell production.

bone marrow aspiration - A medical process in which a small amount of bone marrow is extracted through a needle inserted into the back of the hip.

bone marrow biopsy - A medical procedure in which a small piece of bone with intact bone marrow is removed for study; usually taken from the posterior Ileac (pelvis or hip bone) region.

Budd-Chiari Syndrome - A clotting of the hepatic vein (the major vein that leaves the liver).

CBC - See Complete Blood Count.

cellularity - The quantity, quality, or condition of cells present in the bone marrow.

chromosomes - The part of the cell that contains our DNA or genetic code.

clot - The change, especially of blood, from liquid to solid.

coagulation - A thickening of a liquid, such as blood, by chemical reaction.

colony stimulating factors - See Growth Factors.

complement - A group of proteins that is part of our properly functioning immune system.

Complete Blood Count - A group of tests performed on a small amount of blood that provides information about the quantity and quality of each type of blood cell.

CT Scan - A computer-generated noninvasive diagnostic technique that produces a three-dimentional image of a body part.

cyclophosphamide - Cyclophosphamide is an alkylating agent, a class of chemotherapy drugs which interferes with the growth of diseased cells. While cyclophosphamide is used as a treatment for certain diseases it is still an investigational therapy in Phase II, in combination with other medications, for treatment of MDS, PNH, and aplastic anemia

cyclosporine - Cyclosporine (Neoral, Sandimmune) is an immunosuppressant, a drug that reduces the body's natural immunity. Cyclosporine is used along with anti-thymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia.

cytogenetic - The branch of genetics (the study of our genes, or chromosomes) that deals with the structure and function of the cell.

cytogenetics - The branch of genetics (the study of genes, or chromosomes) that deals with the structure and function of the cell. See Chromosomes.

cytokines - Hormone-like proteins secreted by many different cell types which regulate cell proliferation and function.

cytomegalovirus - A widespread opportunistic virus that can cause disease in an immunosuppressed person.

cytopenia - A deficiency of cells in the blood.

cytotoxic - Destructive to certain cells.

d-dimer - A test that helps doctors diagnose and monitor disease related to inappropriate coagulation or clotting.

daclizumab - Daclizumab (Zenapax) is an immunosuppressant, a drug that reduces the body's natural immunity. It is approved by the FDA for use in many transplant patients to reduce the rate of organ rejection. Daclizumab is being studied in clinical trials for treating aplastic anemia.

darbepoetin alfa - Darbepoetin alfa (Aranesp) promotes red blood cell production and is known as a growth factor. Growth factors are normal chemicals in the body that encourage the bone marrow to make blood cells. Growth factors have been manufactured as drugs to stimulate the bone marrow to produce more blood cells.

dead bowel - A death of part of the intestine after its blood supply is cut off.

decitabine - Decitabine (Dacogen) belongs to a new class of drugs called demethylating agents. Decitabine is thought to work by restoring normal growth and differentiation of bone marrow cells.

deferasirox - Deferasirox is an iron chelator. Iron chelators are medicines that are used to remove excess iron from the body. Iron build-up occurs in patients who become dependent on blood transfusions. Deferasirox combines with the iron stores in the body and is removed by the kidneys.

deferiprone - Deferiprone is an iron chelator that removes excess iron from the body. Deferiprone is approved for use in Europe and is currently in Phase IV clinical trials for its use in patients with thalassemia for review by of the U.S. Food and Drug Administration. Deferiprone combines with iron in the bloodstream and both are removed from the body by the kidneys. Aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) patients are at risk for developing iron overload if they become dependent on blood transfusions.

deferoxamine - Deferoxamine (Desferal) is an iron chelator, approved by the U.S. Food and Drug Administration (FDA) to remove excess iron from the body. Deferoxamine combines with iron in the bloodstream and is removed from the body by the kidneys. Aplastic anemia, myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) patients are at risk for developing iron overload if they become dependent on blood transfusions.

Diamond-Blackfan Anemia - A rare congenital form of red cell aplasia.

differentiate - To become distinct or specialized; acquire a different characteristic or function than the original cell.

Dyskeratosis Congenita - An inherited disease that may lead to bone marrow failure.

dysplastic - Abnormal cellular development whether it is in shape, size or appearance - literally, "bad grower."

eculizumab - Eculizumab is in a class of drugs called monoclonal antibodies. This drug blocks the effects of serum proteins ("complement") and stops hemolysis, the destruction of blood cells. Eculizumab is in clinical studies to determine its effectiveness and safety in reducing the need for blood transfusions in paroxysmal nocturnal hemoglobinuria (PNH) patients.

embolism - A sudden obstruction of a blood vessel by an abnormal particle (such as an aire bubble) circulating in the blood.

engraftment - When bone marrow infused during a bone marrow transplant is accepted by the patient’s body and begins producing blood cells.

enzyme - A protein that acts as a catalyst to induce chemical changes in other substances.

eosinophil - A type of white blood cell that kills parasites and plays a role in allergic reactions.

epoetin alfa - Epoetin alfa (Epogen, Procrit) promotes red blood cell production in the bone marrow and is known as a growth factor. Growth factors are normal chemicals in the body that encourage the bone marrow to make blood cells. Growth factors have been manufactured as drugs to stimulate the bone marrow to produce more blood cells.

erythrocyte - A mature red blood cell.

FAB classification system - Criteria used for classifying leukemia and myelodysplastic syndromes that were developed and agreed upon by a group of French, American and British scientists.

Fanconi's Anemia - A distinct and rare form of inherited anemia that leads to bone marrow failure.

febrile - Feverish; involving an elevated body temperature.

ferritin test - A test used to monitor how much iron your body is storing for later use.

filgrastim - Filgrastim (G-CSF) and Sargramostim (GM-CSF) promote white cell growth in the bone marrow and are known as hematopoietic (blood forming) growth factors or colony stimulating factors. They are a man-made (synthetic) version of the colony stimulating factors produced naturally in your body. These drugs are prescribed "off label" for patients with MDS and other patients with low white cell counts (neutropenia).

flow cytometry - A laboratory technique used to identify and sort cells and their components.

folate - A B-vitamin that occurs naturally in food. Folic Acid is the man made version of Folate.

genes - The blueprints for how a cell functions, how long it will live, and how frequently it will make a copy of itself.

Graft vs. Host Disease - A complication (during a bone marrow transplant) in which the transplanted marrow reacts or rejects normal tissues in the person who has received the transplant.

grans - See Neutrophil.

granulocyte - Any one of these three types of white blood cells: neutrophils, eosinophils, and basophils; so called because they have granules that contain enzymes to help fight infection.

growth factors - Growth factors, or colony-stimulating factors, occur naturally in the body and stimulate the bone marrow to produce white and red blood cells and platelets. Synthetic or man-made versions of growth factors are treatment options that help promote production of specific cells.

Ham's Test - A test that looks at the instability of red blood cells in acid.

hematocrit - The percentage of a volume of blood occupied by red blood cells.

hematologist - A doctor who specializes in the study of blood and blood-producing organs.

hematopoiesis - The production of blood cells.

hemochromatosis - An excess of iron deposits in the body, also known as iron overload.

hemoglobin - The red blood cell protein-iron compound responsible for transporting oxygen from the lungs to the cells, and carbon dioxide from the cells to the lungs.

hemoglobinuria - The presence of hemoglobin in the urine.

hemolysis - The destruction of red blood cells.

heparin - is a natural part of our coagulation system (See Coagulation) that helps prevent blood clots from forming.

hereditary - Refers to any condition passed via genes from parent to child.

histamine - is a naturally occurring protein that is part of allergic reactions.

hormones - The part of the endocrine system that serves as the body's chemical messengers.

hypercellular - Too many cells, for example within the bone marrow.

hyperplastic - Involving an increased number of cells.

hypersensitivity - An abnormal sensitivity to a stimulus.

hypocellular - Too few cells, for example within the bone marrow.

hypoplastic - Involving a decreased number of cells.

idiopathic - Usually refers to any condition with no known cause.

immuno-compromised - Occurs when the immune system is not functioning properly, leaving the patient open to infection.

immunosuppressant - Something that inhibits the immune system.

immunosuppressive - Being capable of inhibiting immune responses.

intravenous - To inject medicine or fluids directly into a vein, allowing immediate access to the blood supply.

iron chelator - A substance that binds iron and then eliminates it from the body in the urine and stool.

irradiation - Application of radiation for therapeutic purposes.

ischemia - Occurs when the blood or oxygen supply is depleted to specific organs.

lactose dehydrogenase - An enzyme that is present in many of our organs. LDH is abundant in red blood cells. It can function as a marker for hemolysis which can cause elevated LDH.

latched related donor - A donor that is a sibling or has another familial relation to the patient (recipient).

lenalidomide - Lenalidomide (Revlimid) is a member of a new class of drugs called immunomodulatory drugs (IMiDs), drugs that can modify or regulate the functioning of the immune system. IMiDs are a group of oral drugs that are chemically similar to thalidomide. Lenalidomide is an investigative drug being studied for it's safety and effectiveness in treating myelodysplastic syndromes.

leukaporfilters - a pre-transfusion filter that reduces the amount of leukocytes present in the blood.

leukocyte - White blood cells, important in defending against infection and clearing the body of harmful material (see also granulocytes, monocytes and lymphocytes).

lineages - Refers to the different lines of blood cells within the bone marrow and the rest of the body, i.e. red blood cells, white blood cells and platelets.

lupus - An autoimmune disease that causes the immune system to attack the body's own tissue and organs.

lymph - A clear, transparent filtrate of plasma that is collected from tissues throughout the body and eventually flows to the lymphatic system.

lymph node - See Lymphatic System.

lymphatic system - The tissues and organs (including the bone marrow, spleen, thymus and lymph nodes) that produce and store cells that fight infection and the network of vessels that carry lymph.

lymphocyte - A type of white blood cell that directs the body’s immune response; divided into two forms, B cells and T cells.

magnetic resonance imaging (MRI) - A noninvasive diagnostic technique that produces computerized images of internal body tissues.

megakaryocyte - A large cell in the bone marrow from which platelets are formed.

meningococcal infection - Infections caused by a group of bacteria that are uncommon, but potentially fatal.

monoclonal antibodies - Identical antibodies produced by one type of immune cell.

monocyte - A type of white blood cell that plays a role in immune and inflammatory responses.

morphology - The study of the structure and form of an organism or one of its parts.

multiple sclerosis - An autoimmune disease that affects the central nervous system (brain,spinal cord, and optic nerves) associated with partial or complete paralysis and jerking muscle tremor.

mycophenolate mofetil - Mycophenolate mofetil is an immunosuppressive agent, a drug that reduces the body's natural immunity in patients who receive organ transplants. It is currently in Phase II of the Food and Drug Adminstration's clinical trials for treatment of patients with myelodysplastic syndromes (MDS) or aplastic anemia.

myelodysplastic syndromes - A group of bone marrow failure disorders that occur when bone marrow stem cells malfunction, resulting in the production of too many defective blood cells not enough normal blood cells (the typical way MDS is first diagnosed)

natural killer cells - a type of cell that lacks B-cell and T-cell receptors and attacks mutant and virus-infected cells

neutropenia - A deficiency of neutrophils in the blood.

neutrophil - The most numerous of the white blood cells, important for helping the body fight infections (particularly bacterial and fungal infections; see also bands, PMNs, polys, stabs, and segs).

nitric oxide - a molecule that is a product of cellular metabolism, nitric oxide is involved in oxygen transport to the tissues

nocturnal - Occuring at night.

packed RBCs - A concentrated blood product in which plasma has been removed from the blood to make transfusions easier and faster.

pancytopenia - A deficiency of all types of blood cells, red blood cells, white blood cells, and platelets

paroxysmal - A sudden recurrence of symptoms or an intensification of existing symptoms.

paroxysmal nocturnal hemoglobinuria - A rare but potentially serious blood disorder that causes the destruction of red blood cells.

pathophysiology - Functional changes in the bodies that are associated with or result from disease or injury.

petechiae - Small red or purplish spots on the skin or a mucous membrane, caused by very small hemorrhages.

phagocytize - To engulf and destroy dangerous microorganisms or cells, a function performed by certain white blood cells.

phagocytosis - The process by which white blood cells (specifically neutrophils) engulf and destroy microorganisms or cells.

placebo - A placebo is an inactive pill, liquid, or powder that has no treatment value.

plasma - The fluid (noncellular) portion of the circulating blood.

platelet - The smallest cells in the blood, essential for blood clotting.

platelets - The smallest cells in the blood, essential for blood clotting. Also, called thrombocytes.

PMN - A mature neutrophil.

PMN - A mature neutrophil. See Neutrophil.

poly - A mature neutrophil.

prednisone - Prednisone, a corticosteroid, is a man-made (synthetic) version of a hormone produced naturally by your adrenal gland. It is prescribed when your body is not producing enough of this chemical on its own.

prednisone (pred' ni sone) - Prednisone, a corticosteroid, is a man-made (synthetic) version of a hormone produced naturally by your adrenal gland. It is prescribed when your body is not producing enough of this chemical on its own. Prednisone may be prescribed for numerous reasons. It is sometimes prescribed after an ATG treatment to counteract the effects of serum sickness; it helps by reducing the antibody production of the immune system. Prednisone is sold under a number of different Brand Names, please speak with your pharmacist for more information.

proliferation - Growth by reproduction of similar cells.

prophylactic - A safeguard to prevent unwanted consequences.

pulmonary embolism - An obstruction of an artery that carries blood to the lungs. See Embolism.

R115777 - R115777, a farnesyl transferase inhibitor, blocks naturally occuring enzymes from activating harmful proteins, which occurs in roughly 25% of myelodysplastic syndromes (MDS) patients.

red blood cells - The oxygen carrying cells in our body. These bring oxygen to our tissues, and are the most numerous of the blood cells.

refractory - resistant to treatment or cure; unresponsive to stimulus

Reticulocyte - An immature red blood cell normally restricted to the bone marrow and present in the bloodstream only in very low numbers.

reticulocytosis - A condition in which there is an increase in the number of circulating reticulocytes (immature red blood cells).

rheumatoid arthritis - See Arthritis.

seg - A mature neutrophil. See Neutrophil.

shwachman-diamond syndrome - A rare inherited disease that affects many organs and may cause bone marrow failure.

spasm - An involuntary and abnormal contraction of muscle, muscle fibers, or a hollow organ (such as an artery, the colon, or the esophagus).

stab - An immature neutrophil. See Neutrophil.

stem cells - Cells that are produced in the bone marrow and differentiate into red cells, white cells, and platelets.

subcutaneous - Beneath the skin.

synthesis - A building up, putting together, or composition.

t-lymphocyte - A type of lymphocyte that is important in the immune response, but in aplastic anemia suppresses the stem cells; also known as a T cell lymphocyte.

thalidomide - Thalidomide (Thalomid) is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects. Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy. Thalidomide is being studied, either as a single agent or in combination with other medicines, for treating of myelodysplastic syndromes (MDS).

therapy-related MDS - A type of MDS that is caused by the treatment of another disorder, for example with the use of radiation therapy (e.g., for prostate cancer) or certain types of chemotherapy (e.g., for breast cancer)

thrombocytopenia - A decrease in the number of platelets in the blood, resulting in the potential for increased bleeding and decreased ability for clotting.

TLK-199 - TLK-199 is a small molecule drug that stimulates the bone marrow to produce blood cells. Currently TLK-199 is in Phase II of the Food and Drug Administration’s (FDA’s) clinical trial for the treatment of myelodsyplasitc syndromes (MDS).

transferrin - A protein that binds iron and thus regulates iron absorption and transports iron in the body.

transfusion - The process by which blood or one of its components is delivered directly into the bloodstream.

ulcerative colitis - A chronic inflammatory bowel disease (IBD) of the colon that causes inflammation and sores (called ulcers) in the lining of the intestines, usually in the rectum and lower colon.

unrelated donor - A donor that is not a sibling or other familial relation of the patient (recipient).

vital signs - A person’s temperature, pulse, respiration, and blood pressure.

VNP40101M - VNP4010M is a sulfonylhydrazine agent, which works by damaging DNA. It is currently in Phase II of the Food and Drug Adminstration's clinical trials for treatment of myelodysplastic syndromes (MDS) patients.

white blood cells - An important part of our body’s defense against infection and clearing the body of harmful toxins - our immune system.

WHO Classification System - Criteria used for classifying leukemia and myelodysplastic syndromes that were developed by the World Health Organization (WHO).