Absolute reticulocyte count (ARC) A test that measures the number of immature red blood cells in your blood. High reticulocyte counts may be a sign of ongoing hemolysis with paroxysmal nocturnal hemoglobinuria (PNH), as the body is making more new red blood cells to replace the ones being destroyed by the immune system. C3 A protein in the blood that acts as a central point of the complement system, regulating its activation. C5 Another protein in the blood that is part of the complement system. Clinical trial A type of research study that tests how well treatments work in people, as well as possible side effects of treatments. Complement system The complement system, also referred to as the complement cascade, is an army of more than 40 different proteins that work together to get rid of dangerous cells and fight infection.
Hemolysis The breaking apart of red blood cells. It can occur when the immune system attacks these cells as though they were dangerous viruses or bacteria. When red blood cells break open, hemoglobin is released. Hemolysis causes many of the symptoms of PNH. Immune system A sophisticated defense network used to protect the body from dangers like disease and infection.
Intravascular hemolysis (IVH) The destruction of red blood cells inside a blood vessel.
Lactate dehydrogenase (LDH) An enzyme found in the blood and tissues of the body, including the heart, kidneys, brain, and lungs. Red blood cell destruction results in the release of LDH into the blood. People with PNH often have higher levels of LDH due to ongoing hemolysis.
Normalization Lab values improving to levels like those of adults who don’t have PNH.
Proteins Molecules that play many important roles in the body and can be thought of as the “workhorses” of cells. Proteins are required for the structure, function, and regulation of the body’s organs and tissues. Red blood cells A common type of blood cell. Their job is to carry oxygen, using an important molecule, hemoglobin, around the body.
Extravascular hemolysis (EVH) Destruction of red blood cells that occurs in the liver or spleen.
FACIT-Fatigue scale This tool consists of a number of questions that are used to assign a score to measure a person’s experience with fatigue.
Stabilization Lab values remaining at a stable level and not getting worse.
Globin' Acting in pursuit of higher hemoglobin levels.
U/L Some test results are reported in U/L, or units per liter. This is a measurement of how many units there are in a liter. x 10 9 cells/L A short way to write a number with 9 zeros at the end. This is useful when needing to count the billions of cells in a liter of blood.
Hemoglobin (Hb) The critical protein found inside red blood cells that enables other cells throughout the body to get the oxygen they need. Hemoglobin acts like drops of glue that can “stick” to oxygen and carry it from the lungs to other tissues. It can also “stick” to waste like carbon dioxide to help remove it from the body.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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