Glossary

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

(noo-truh-PEE-nee-uh) A condition in which there is a shortage of neutrophils in the bloodstream. Neutrophils are a type of white blood cell white blood cell: Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte. . This results in a low white blood cell count.

(noo-truh-PEE-nik) A diet for a patient with very low white blood cell white blood cell: Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte. count. A neutropenic diet neutropenic diet: (noo-truh-PEE-nik) A diet for a patient with very low white blood cell count. A neutropenic diet avoids the use of certain foods that can may contain germs, such as raw meats, aged cheeses, fermented drinks, and unwashed fruits and vegetables. avoids the use of certain foods that can may contain germs, such as raw meats, aged cheeses, fermented drinks, and unwashed fruits and vegetables.

(NOO-truh-fil) The most numerous of the white blood cells, important for helping the body fight infections (particularly bacterial and fungal infections.

(NIGH-trik OK-side) A molecule that helps muscles stay smooth and relaxed. A shortage of nitric oxide nitric oxide: (NIGH-trik OK-side) A molecule that helps muscles stay smooth and relaxed. A shortage of nitric oxide can occur in paroxysmal nocturnal hemoglobinuria (PNH), causing muscle spasms in certain parts of the body. can occur in paroxysmal nocturnal hemoglobinuria paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH), causing muscle spasms in certain parts of the body.

The purpose of a non-therapeutic study non-therapeutic study: The purpose of a non-therapeutic study is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that… is to increase the understanding of a disease or condition through observation, data collection, or other methods. A non-therapeutic study does not provide treatment or intervention for participants. This type of research contributes to general knowledge that may benefit others in the future by improving treatment or developing new therapies.

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Obstruction; blockage

An approved medicine that is prescribed by a licensed doctor for a purpose other than that for which is was approved by the U.S. Food and Drug Administration (FDA).

(on-KAH-luh-jist) A doctor who specializes in the treatment and prevention of cancer.

A drug or biologic agent biologic agent: A substance made from a living system, such as a virus, and used to prevent or treat disease. Biological drugs include antibodies, globulin, interleukins, serum, and vaccines. Also called a biologic or biological drug. that treats a rare disease or condition. A disease is considered rare if fewer than 200,000 people in the United States have it.

A medicine that is available without a prescription from the doctor. Also called OTC medicine.

A shortage of all types of blood cells - red blood cells, white blood cells, and platelets.

(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria Hemoglobinuria: (hee-muh-gloe-buh-NYOOR-ee-uh) The presence of hemoglobin in the urine. means hemoglobin hemoglobin: A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body. in the urine. Hemoglobin is the red part of red blood cells. A person with PNH may have episodes of dark urine in the morning, but this symptom is not present in all PNH patients.

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

A procedure where 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… are collected from the donor's circulating (peripheral) blood. These stem cells are then given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells. Also called PBSC transplant.

(puh-TEE-kee-ie) Small, flat red or purplish spots on the skin, caused by bleeding. It is often a symptom of a low platelet platelet: The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte. count.

A type of white blood cell white blood cell: Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte. that surrounds and kills microorganisms, such as bacteria and fungi. They also remove dead cells. Monocytes, macrophages, and neutrophils are phagocytes.

A highly trained and licensed professional whose job concerns the preparation, distribution, and use of prescription drugs. A pharmacist pharmacist: A highly trained and licensed professional whose job concerns the preparation, distribution, and use of prescription drugs. A pharmacist also advises patients, as well as physicians and other health practitioners, on the selection, dosages, interactions, and side effects of medications. also advises patients, as well as physicians and other health practitioners, on the selection, dosages, interactions, and side effects of medications.

The gene that is in charge of making a protective shield of proteins on normal red blood cells. Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) occurs because of a change (mutation) in the PIG-A gene PIG-A gene: The gene that is in charge of making a protective shield of proteins on normal red blood cells. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a change (mutation) in the PIG-A gene of a single stem cell in the bone marrow. of a single stem cell in the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. .

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

The fluid part of the blood. Plasma Plasma: The fluid part of the blood. Plasma is mostly made of water with chemicals in it. These chemicals include proteins, hormones, minerals, and vitamins. is mostly made of water with chemicals in it. These chemicals include proteins, hormones, minerals, and vitamins.

The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte thrombocyte: (THROM-buh-site) See platelet. .

A procedure in which platelets are given to a person through an intravenous (IV) line into the bloodstream. Platelets are more likely than red blood cells to cause an immune response, such as chills and fever. The use of platelets from one donor (apheresis) reduces the chance of reaction to transfused platelets. Transfused platelets increase blood count and help control bruising and bleeding.

A corticosteroid, a man-made version of a hormone hormone: A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another. produced naturally by the adrenal gland. It is prescribed when the body is not producing enough of this chemical on its own. Prednisone Prednisone: Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own. How does it work? For PNH, it may be used to decrease… may be prescribed for numerous reasons. It is sometimes prescribed after an ATG treatment to counteract the effects of serum sickness serum sickness: An immune system reaction to foreign proteins in certain medicines. Serum sickness can be a side effect of ATG, causing fever, rash, joint pain, and muscle aches. . It helps by reducing the antibody production of the immune system. Prednisone is sold under a number of different brand names.