MDS GLOSSARY COMMOnLy Used BOne MaRROw FaiLURe TeRMs the myelodysplastic syndromes foundation, inc.
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MDS GLOSSARYCommonly Used
Bone marrow FailUre Terms
the myelodysplastic syndromes foundation, inc.
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TABLE OF CONTENTS
General 2
Red Blood Cells 30
White Blood Cells 31
Bone Marrow Transplant 32
Bone Marrow Biopsy 32
Medications 36
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GENERAL
Acute Sudden, such as a sudden onset of symptoms ordiseases.
Acute Myeloid Leukemia (AML)A cancer of the blood cells. AML happens whenvery young blood cells (blasts) in the bonemarrow fail to mature normally. More blastcellsare produced than needed, so there is not enoughroom within the marrow for other normal bloodcells to develop, such as red blood cells orplatelets. Some cases of MDS may develop intoAML. However, most do not.Synonyms: acute myeloblastic leukemia,acute myelocytic leukemiaAnaphylaxis A very severe allergic reaction to a foreignprotein, such as in a bee sting, or to a medicine.This reaction causes the blood pressure to dropand may cause difficulty breathing. Emergencytreatment is required to manage these symptoms.If very severe, anaphylaxis can progress to shock.Synonym: anaphylactic shockAnemia A condition in which the number of red bloodcells is below normal. This may result in fatigue,generalized weakness and shortness of breath.
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Antibiotics Medications used to treat bacterial infections andother similar microorganisms.
Antibodies Proteins produced by plasma cells in responseto foreign substances in the body.
Apheresis A procedure in which blood is taken from aperson, and part of that blood component (suchas white blood cells, red blood cells, or plasma) isseparated out, and the remaining bloodcomponents are reinfused back into the donor.
Aplastic Anemia A rare and serious condition in which the bonemarrow does not make enough blood cells: redblood cells, white blood cells, and platelets. Theterm aplastic is a Greek word meaning not toform. Anemia is a condition that happens whenthe red blood cell count is low. Most scientistsbelieve that aplastic anemia happens when theimmune system attacks the bone marrow stemcells. Aplastic anemia can be acquired (begin anytime in life) or can be hereditary (less common,passed down from parent to child).Synonyms: acquired aplastic anemia, hereditaryaplastic anemiaApoptosis Death of a cell as a part of the normal lifecycle.
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Autoimmune Disease Any condition that happens when the immunesystem attacks the body’s own normal tissues.The immune system is a complex organizationwithin the body that is designed normally to“seek and destroy” invaders of the body,including infectious agents. Patients withautoimmune diseases frequently have unusualantibodies circulating in their blood that targettheir own body tissues.
Basic Research The study of a subject to increase knowledge andunderstanding about it. The goal of basic researchin medicine is to better understand disease. In thelaboratory, basic research scientists study changesin cells and molecules linked to disease. Basicresearch helps lead to better ways of diagnosing,treating, and preventing disease.
Benzene A chemical that is widely used by the chemicalindustry to make plastics, resins, nylon andsynthetic fibers. Benzene is found in tobaccosmoke, vehicle emissions, and gasoline fumes.Exposure to benzene may increase the risk ofdeveloping a bone marrow failure disease.Benzene can affect human health by causingbone marrow stem cells not to work correctly.
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Biologic Agent A substance made from a living system, such asa virus, and used to prevent or treat disease.Biological drugs include antibodies, globulin,interleukins, serum, and vaccines. Also called abiologic or biological drug.Synonyms: biologic, biological drugBlast Cells Immature blood cells that would normallybecome fully functional mature red cells, whitecells, or platelets. The number of blast cells inthe bone marrow helps define how severe MDSis in a person. When 20 or more out of 100 cellsin the bone marrow are blasts, this is consideredacute myeloid leukemia (AML).Synonym: precursor cellBlood Clot A clot or small cluster of blood cells that formswhen platelets stick together. A combination ofplatelets and fibrin that form a mesh with theintention of preventing bleeding in response toan injury or illness. The term thrombus describesa blood clot that develops and attaches to ablood vessel. Blood clots are more common inParoxysmal Nocturnal Hemoglobinuria (PNH) orin people with blood clotting disorders.Synonym: thrombus
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Blood Tests Blood samples drawn from the arm that areevaluated for cell counts (red cells, white cells[and their subtypes], and platelets). The blood isalso evaluated for the shape and size of thedifferent blood cells and for how various organsare functioning such as the kidneys and liver.
Blood Thinner A medicine used to treat or prevent blood clots.Also called anticoagulants or blood thinners.Some common blood thinners are enoxaparin orclexaine (Lovenox or Clexane), heparin (Calciparineor Liquaemin), and warfarin (Coumadin).Synonyms: anticoagulant, anti-clottingBlood Transfusion A procedure in which whole blood or one of itscomponents is given to a person through anintravenous (IV) line into the bloodstream.A red blood cell transfusion or a platelettransfusion can provide temporary relief forsome patients with low blood counts.
Bone Marrow The soft, sponge-like tissue in the center ofbones that functions like a factory to producewhite blood cells, red blood cells, and platelets.
Bone Marrow AspirateThe bone marrow aspirate is a sample of theliquid portion of the bone marrow. It is used to
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obtain spicules — a small collection of bloodforming cells. This provides information aboutthe shape of the cells (morphology), how thecells are maturing (differentiation) and thenumber of blasts (immature cells) in the bonemarrow. The aspirate may also be used foradditional testing that may help to determine thecause of the cytopenias, such as cytogenetics.
Bone Marrow AspirationThe process of removing bone marrow from aspecific area using a small needle and syringe.Used for diagnostic purposes. Tests may also berun on the bone marrow cells to look for anygenetic abnormalities.
Bone Marrow Biopsy The bone marrow biopsy is a small core (theshape and size of a medium pencil lead) ofthe spongy center of the bone marrow. Itprovides information about the cellularity of thebone marrow (crowded=hypercellular, empty=hypocellular). It will also provide usefulinformation about iron storage, scarring (fibrosis),and the presence of any other abnormal cells.Synonym: Bone Marrow Trephine BiopsyBone Marrow Failure A condition that occurs when the bone marrowstops making enough healthy blood cells. Themost common of these rare diseases are myelo-
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dysplastic syndromes (MDS), aplastic anemia,and paroxysmal nocturnal hemoglobinuria (PNH).Bone marrow failure can be acquired (begin anytime in life) or can be hereditary (less common,passed down from parent to child).
Cellularity How much of the bone marrow volume isoccupied by various types of blood cells.
Chemotherapy The use of medicines that kill cells (cytotoxicagents). People with high-risk or intermediate-2risk myelodysplastic syndrome (MDS) may begiven chemotherapy. Chemotherapy may alsohurt healthy cells causing side-effects. Ifchemotherapy works in controlling abnormalcells, then relatively normal blood cells will startto grow again. Chemotherapy agents include:cytarabine (Ara-C) and hydroxyurea (Hydrea),daunorubicin (Cerubidine), idarubicin (Idamycin),and mitoxanrone (Novantrone).
Chronic Illness A medical condition that lasts a long time. Achronic illness can affect a person’s lifestyle,ability to work, physical abilities and independence.
Chromosomes A structure that contains your genetic infor-mation, or DNA. Normally each person has 23pairs of chromosomes.
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Clinical Trial A type of research study that tests how a drug,medical device, or treatment approach works inpeople. There are several types of clinical trials.Treatment trials test new treatment options.Diagnostic trials test new ways to diagnose adisease. Screening trials test the best way todetect a disease or health problem. Quality of life(supportive care) trials study ways to improve thecomfort of people with chronic illness. Preventiontrials look for better ways to prevent disease inpeople who have never had the disease.
Trials are in four phases:• Phase I tests a new drug or treatment in a
small group to see if it is safe.
• Phase II expands the study to a larger group ofpeople to find out if it works.
• Phase III expands the study to an even largergroup of people to compare it to the standardtreatment for the disease.
• Phase IV takes place after the drug or treatmenthas been licensed and marketed to find out thelong-term impact of the new treatment.
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Clone To make copies. Bone marrow stem cells clonethemselves all the time. The cloned stem cellsbecome mature blood cells that leave the bonemarrow and enter the bloodstream. Abnormalclones are associated with cancers, such as MDS.
Coagulate To thicken. Normal blood platelets cause the blood tocoagulate and stop bleeding.
Combination Chemotherapy The use of more than one drug during cancertreatments.
Comorbidities Additional diseases beyond MDS.
Complete Blood Count (CBC)The CBC measures the number of white bloodcells (WBC) and the number and size of redblood cells, the total amount of hemoglobin,and the fraction of the blood made up of redblood cells.
Complex Karyotype Three or more abnormalities in their chromosomes.
Conditioning Treatment Used to kill all remaining cancer cells beforestem cell transplantation.
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Cytogenetics Testing that is performed on bone marrow samplesand examines the chromosomes of the cells.Your cytogenetic results are used to identify thetype of MDS you have and to calculate theInternational Prognostic Scoring System (IPSS)and the revised IPSS (IPSS-R) risk category.Common abnormalities include:1. Deletion 5q- – deletion of chromosome 52. Deletion 20 – deletion of chromosome 203. Deletion Y – deletion of the Y chromosome4. Monosomy 7 – loss of one of the two 7
chromosomes5. Trisomy 8 – addition of a third chromosome 8Synonyms: Chromosomes, Karyotype, DNACytogenetic RemissionNo sign of previously detected abnormalchromosomes are found. This represents aresponse to treatment. When a bone marrowtest is performed on a patient with 5q deletionMDS, and there are no signs of an abnormalchromosome 5, then that patient has achieveda cytogenetic remission. Also called cytogeneticresponse.
Cytokines Proteins
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Cytopenia A deficiency of (or too few) mature cells in theblood. Deficiencies can occur in red cells, whitecells, and/or platelets.
Cytotoxic Agent A medicine that kills certain cells. Chemotherapyfor MDS patients often involves the use ofcytotoxic agents.
del(5q) Deletion in the long (q) arm of chromosome 5.
De Novo The original source of disease, somethingpresent at the start. MDS may be de novo, theoriginal source of disease, or treatment related,caused from chemotherapy or radiation givenfor other forms of cancer.
Differentiation The process of cells maturing to become healthyadult cells of a particular type (i.e. red cells,white cells, and platelets).
Dietary Supplement Vitamins, minerals, herbs and other substancesmeant to improve your nutritional intake.Dietary supplements are taken by mouth in theform of a pill, capsule, tablet or liquid.
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DNA Methylation A process that helps control gene activity, resultingin blockage of cell growth.
Dysplasia Abnormal shape and appearance or morphology,of a cell.Synonym: dysplastic
Embolus A blood clot or other foreign matter that getsinto the bloodstream and gets stuck in a bloodvessel.
Epidemiology The study of patterns and causes of disease ingroups of people. Researchers who study howmany people have a disease, how many newcases are diagnosed each year, where patients arelocated, and environmental or other factors thatinfluence disease, are known as Epidemiologists.
Erythroid Response • In patients who have not received red blood
transfusions—hemoglobin increase of 1.5 g/dl• In those who have had transfusions—reduction
in transfusions by at least four units of packedred blood cells over 8 weeks compared withthe 8 weeks before treatment
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Erythropoietin (EPO)A protein substance manufactured by the kidneysin response to low oxygen levels in body tissues.Erythropoietin stimulates the production of redblood cells in the bone marrow.
Erythropoietin-stimulating Agent(ESA)A medicine used to help the bone marrow makemore red blood cells. Epoetin alfa (Epogen,Procrit) and darbepoetin alfa (Aranesp) areerythropoiesis-stimulating agents that can helpboost the red blood cell count of some bonemarrow failure patients. Also called red bloodcell growth factor.
Etiology The cause or origin of a disease.
FAB Classification A criteria used for classifying different types ofmyelodysplastic syndromes (MDS). The FAB(French, American, British) Classification Systemwas developed by a group of French, Americanand British scientists. This system is based on 2main factors: the percentage of blast cells inbone marrow, and the percentage of blast cellsin the bloodstream. The FAB system is somewhatoutdated, but is still used by some doctors today.
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The World Health Organization (WHO) Classifi-cation System has largely replaced the FABClassification System.
Fanconi Anemia A rare inherited disorder that happens when thebone marrow does not make enough blood cells:red cells, white cells, and platelets.Fanconi anemia is diagnosed early in life. Peoplewith Fanconi anemia have a high likelihood ofdeveloping cancer. Genetic testing is used todiagnose Fanconi anemia.
Fatigue A feeling of low or no energy, general feeling oftiredness with normal activity. Rest does notnecessarily resolve fatigue.Synonyms: tired, exhaustion, lethargy, malaise
Ferritin A protein inside of cells that stores iron for lateruse by your body. Sometimes ferritin is releasedinto the blood. The ferritin level in the blood iscalled serum ferritin.
Fibrosis Scarring of tissue. Fibrosis of the bone marrowis a feature seen in some types of unclassifiedmyelodysplastic syndrome (MDS).
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Flow Cytometry A laboratory test that gives information about cells,such as size, shape, and percentage of live cells.Flow cytometry is the test doctors use to assess forspecific proteins on the surface of blood cells. It isthe standard test for confirming a diagnosis ofparoxysmal nocturnal hemoglobinuria (PNH).Synonyms: Flow, Immunophenotyping Fluor-escence-activated cell sorting (FACS)
Fluorescence In Situ Hybridization (FISH)An important laboratory test used to helpdoctors look for chromosomal abnormalities andother genetic mutations. Fluorescence in situhybridization, also called FISH, directs coloredlight under a microscope at parts of chromosomesor genes. Missing or rearranged chromosomesare identified using FISH.
Folate A B-vitamin that is found in fresh or lightly cookedgreen vegetables. It helps the bone marrow makenormal blood cells. Most people get enoughfolate in their diet. Doctors may have people withparoxysmal nocturnal hema-globinuria (PNH)take a man-made form of folate called folic acid.
Gene Expression The process that genes use to make theirproducts, such as proteins.
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Graft-Versus-Host Disease (GVHD) Attack by transplanted cells on the recipient'sbody in which the transplanted cells causeinflammation of some normal tissues.• Acute: within 3 months of transplantation• Chronic: starting more than 3 months after
transplantation
Graft-Versus-Leukemia EffectT cells (part of the immune system) in the donatedstem cells can attack the remaining cancer cells.
Haploidentical Stem Cell TransplantationThe donor’s blood markers match half thepatient’s markers.
Hematocrit (HCT)Percent of the total blood volume that is madeup of red blood cells. In men a normalhematocrit is 40–52% while in women thenormal is 36–46%. Hematocrit is part of acomplete blood count. Also called HCT, packedcell volume, PCV. (see red blood cells)Synonyms: packed cell volume, PCV
Hematologist A doctor who specializes in the diseases anddisorders of blood.
Hematopoiesis The formation and development of blood cells.
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Hemochromatosis A condition that occurs when the body absorbsand stores too much iron. This leads to a conditioncalled iron overload. In the United States,hemochromatosis is usually caused by a geneticdisorder. Organ damage, particularly to the liverand heart, can occur if iron overload is not treated.Hemolysis The destruction of red blood cells.
Hypercellular A condition in which there are too many cellswithin the bone marrow.
Hypocellular A condition in which there are too few cells,within the bone marrow. Patients with aplasticanemia have hypocellular bone marrow.
Hypomethylating AgentA hypomethylating agent is a drug that inhibitsDNA methylation. Works by preventing certaingenes involved in controlling cancer from beingsilenced, allowing for the normal functioning ofthe tumor suppressor genes.Synonym: demethylating agent
Idiopathic Usually refers to any condition with no known cause.
Immature Blood Cells May be called stem cells, progenitor cells or blasts.
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Immune Deficiency A decreased ability of the immune system tofight infection.
Immune System The complex group of organs and cells thatdefend the body against infection and disease.
Immunocompromised Occurs when the immune system is notfunctioning properly, leaving the patient open toinfection. A person can be immunocompromiseddue to low white blood cell count or due to somemedicines.Synonym: immune compromised
Immunosuppressive DrugDrugs that lower the body’s immune responsein autoimmune diseases. These drugs may be usedto allow the bone marrow stem cells to growand make new blood cells. ATG (antithymocyteglobulin) or ALG (antilymphocyte globulin) withcyclosporine are used to treat bone marrowfailure in aplastic anemia. Immunosuppressivedrugs may help some patients with myelodysplasticsyndromes (MDS) and paroxysmal nocturnalhemoglobinuria (PNH).
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Intravenous Infusion A method of getting fluids or medicines directlyinto the bloodstream over a period of time. Alsocalled IV infusion.Synonym: IV infusion
IPSS/IPSS-R An International Prognostic Scoring System –system for grading the severity of MDS. Thesystem turns patient data into a score. The scoretells how quickly a myelodysplastic syndrome(MDS) case is progressing and helps predict whatmay happen with the patient’s MDS in the future.
Iron Chelation TherapyA drug therapy to remove extra iron from thebody. Patients with high blood iron (ferritin) levelsmay receive iron chelation therapy. The U.S. Foodand Drug Administration (FDA) has approved twoiron chelators to treat iron overload in the U.S.:deferasirox, an oral iron chelator, and deferoxamine,a liquid given by injection, these may differdepending on which country you live in.
Iron Overload A condition that occurs when too much ironaccumulates in the body. Bone marrow failuredisease patients who need regular red blood celltransfusions are at risk for iron overload. Organdamage can occur if iron overload is not treated.
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Ischemia Occurs when the blood supply to specific organor part of the body is cut off, causing a localizedlack of oxygen.
Lymphatic System A network of organs, lymph nodes, lymph ducts,and lymph vessels that help keep the body’s fluidsin balance and help the body fight infection.Minimal Residual DiseaseSmall numbers of cancer cells that stay in thebody after treatment.
Monosomy 7 Describes the loss of one of the two number 7chromosomes. “Mono” means one and “somy”comes from the word chromosome. Bonemarrow samples are used to detect monosomy7 and other genetic abnormalities. Monosomy 7can occur in adult patients with MDS and canoccur in childhood bone marrow failure diseases.Morphology The study of the structure and form of anorganism or one of its parts.
Multilineage Dysplasia Abnormalities in more than one type of blood cell
Mutation Any change or alteration in a gene. A mutationmay cause disease or may be a normal variation.
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Myelo A Greek word meaning marrow.
Myelodysplastic Syndromes (MDS)The Myelodysplastic Syndromes (MDS) are agroup of bone marrow failure disorders. Myelorefers to the bone marrow. Dysplastic meansabnormal growth or development. In MDS, thebone marrow does not make blood cellsnormally. The result is too few cells or low bloodcounts (cytopenias) and cells that do notfunction properly.The most common cytopenias include:Anemia: low red blood cells (oxygen carrying cells)Thrombocytopenia: low platelets (cells thathelp to clot the blood)Leukopenia: low white blood cells (WBC) (helpto fight infection)Neutropenia: low neutrophils (most importanttype of WBC for fighting infection)
Natural Killer Cells A type of cell that lacks B-cell and T-cell receptorsand attacks mutant and virus-infected cells.
Neutropenia A deficiency (below-normal number) of maturewhite blood cells called neutrophils that assist infighting bacterial infections.
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Neutropenic Diet A diet for a patient with very low white bloodcell count. A neutropenic diet avoids the use ofcertain foods that can contain bacteria or fungus,such as raw meats, unpasteurized dairy products,aged cheeses, fermented drinks, and unwashedfruits and vegetables.
Oncologist A doctor who specializes in the treatment andprevention of cancer.
Over-the-Counter (OTC) MedicineA medicine that is available without a prescriptionfrom the doctor. Also called OTC medicine.
Packed RBCs A concentrated blood product in which most ofthe plasma, the fluid part of blood, is removed tomake red blood cell transfusions easier and faster.
Pancytopenia A reduced number of all types of blood cells –red blood cells, white blood cells, and platelets.
Paroxysmal Nocturnal Hemoglobinuria(PNH)A rare and serious blood disease that causes redblood cells to break apart. Paroxysmal meanssudden and irregular. Nocturnal means at night.Hemoglobinuria means hemoglobin in the urine.
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Hemoglobin is the red part of red blood cells. Aperson with PNH may have episodes of darkurine in the morning, but this symptom is notpresent in all PNH patients.
Pathophysiology Abnormal function or processes that cause orare associated with disease or injury.
Pediatric MDS MDS is rare in children; but it does happen. Mostpatients are 60 years old or older.
Peripheral Blood Stem Cell (PBSC)TransplantA procedure where stem cells are collected fromthe donor’s circulating (peripheral) blood. Thesestem cells are then given to the patient throughan intravenous (IV) line. In time, donated stemcells start making new, healthy blood cells. Alsocalled PBSC transplant.
Petechiae Small, flat red or purplish spots caused bypinpoint bleeding into the skin. It is often a signof a low platelet count.
Pharmacist A highly trained and licensed professional whosejob concerns the preparation, distribution, anduse of prescription drugs. A pharmacist also
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advises patients, as well as physicians and otherhealth practitioners, on the selection, dosages,interactions, and side effects of medications.
Placebo A placebo is an inactive pill, liquid, or powderthat has no treatment value. Placebo use inclinical trials is extremely uncommon today.
Platelets Irregularly shaped, colorless cells that arepresent in blood. Their sticky surface lets them,along with other substances, form clots to stopbleeding. Also called thrombocytes.
Platelet Transfusion A procedure in which platelets are given to aperson through an intravenous (IV) line into theblood-stream. Platelets are more likely than redblood cells to cause an immune response, suchas chills and fever. The use of platelets from onedonor (apheresis) reduces the chance of reactionto transfused platelets. Transfused plateletsincrease the blood platelet count and helpcontrol bruising and bleeding.
Prophylactic Something that prevents or protects. Forexample, blood thinners may be given as aprophylactic measure to prevent blood clots inhigh risk patients.
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Pure Red Cell Aplasia (PRCA)A condition that occurs when the bone marrowstem cells do not make red blood cells. Redblood cell counts are low. White blood cell andplatelet counts are normal.
Red Blood Cell (RBC) Transfusion A procedure in which packed red blood cells aregiven to a person through an intravenous (IV) lineinto the bloodstream. Transfused red blood cellsincrease the blood count and help improvesymptoms of anemia. Before transfused blood isgiven, donated blood is typed and cross matchedto the recipient’s blood. Also called RBC transfusion.
Refractory Not responsive to treatment or cure. For example,refractory anemia is a low red blood cell countthat doesn’t respond to standard treatments.
Reticulocyte An immature red blood cell. Reticulocytes arenormally found in the bone marrow. They are presentin the bloodstream only in very low numbers.
Remission Disappearance of the signs and symptoms ofcancer. A remission may be complete (CR) orpartial (PR).
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Revised IPSS (IPSS-R) Takes more information into account than theIPSS and categorizes patients into five riskgroups instead of four.
Ring Sideroblast A red blood cell that has too much iron. The irontypically forms a ring around the cell’s nucleus.
Secondary MDS A type of MDS that is caused by a previoustreatment for another disorder or disease. Treat-ments typically associated with secondary MDSinclude radiation therapy and chemotherapyused to treat cancer. Also called therapy-relatedMDS, T-MDS.Synonyms: T-MDS, therapy-related MDS
Serum Erythropoietin Amount of erythropoietin that is presentnormally in an individual’s blood.
Serum Sickness An immune system reaction to foreign proteinsin certain medicines. Serum sickness can be aside effect of ATG, a medication used in thetreatment of aplastic anemia, causing fever,rash, joint pain, and muscle aches.
Single Lineage Dysplasia Abnormalities in only one type of blood cell.
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Somatic Mutation Change in a gene that happens after conceptionin a patient’s cells, is not inherited, and is notpassed on to the patient’s children.
Stem Cells Cells in the body that develop into other cells. Adultstem cells in the body repair and maintain theorgan or tissue in which they are found. Blood-forming (hemapoietic) stem cells are found in thebone marrow. These cells make copies of them-selves and develop into red cells, white cells, andplatelets. Embryonic stem cells come from humanembryos and may be used in medical research.
Subcutaneous InjectionA method of giving medicine in the fatty tissuearea under the skin using a short needle.Synonyms: shot, injection
Supportive Care Care given to improve the quality of life, orcomfort, of a person with a chronic illness.Supportive care treats the symptoms rather thanthe underlying cause of a disease. The goal is tohelp the patient feel better. Patients with lowblood counts may be given blood transfusions assupportive care to help manage the symptomsof their disease. Also called palliative care,symptom management.Synonyms: palliative care, symptom management
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Therapy Related MDS See Secondary MDS
Thrombus A blood clot that develops and attaches to ablood vessel.
Thrombosis The process of forming a blood clot.
ThrombocytopeniaA condition in which the number of matureplatelets, or thrombocytes, is below normal.When severe, the tendency to bruise and bleedmore easily can occur.
Transfusion Process by which blood or one of its components(e.g., red blood cells, plasma, platelets) is delivereddirectly into the bloodstream by vein (intravenousof IV), similar to other IV medications.
Transfusion Independence No longer needing any type of blood transfusion.
Treatment Failure Occurs when a patient does not respond to thetreatment, responds only temporarily, or has tostop the treatment because of side effects.
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RED BLOOD CELLS
ErythrocyteA red blood cell. It carries oxygen to body cellsand carbon dioxide away from the cells. (See red blood cells)
Red Blood Cells (RBC)These are cells that carry oxygen to your tissues.(See erythrocyte)
White Blood Cells (WBC)Cells produced in the bone marrow and lymphnodes. White cells are key cells in the immunesystem that prevent or fight infection.
World Health Organization (WHO)ClassificationThe most current system for classifying leukemiaand myelodysplastic syndromes (MDS), it wasdeveloped by the World Health Organization(WHO). This system is based on patient datafrom around the world and on the most up-to-date knowledge of MDS. WHO Classification ofMDS consists of many subtypes based on testsof the blood and bone marrow.
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ANC (absolute neutrophil count) A measure of the actual number of matureneutrophils in a given volume of blood.
BasophilType of white blood cell that plays a role in allergicreactions and asthma.
EosinophilType of white blood cell that kills parasites andplays a role in allergic reactions.
GranulocyteA term for any of the white blood cell types thathave granules containing enzymes to help fightinfection: neutrophils, eosinophils and basophils.
LymphocytesSmall white blood cells produced in the lymphoidorgans (the lymph nodes, spleen, thymus, andtonsils) or bone marrow that are essential fornormal function of the immune system.
MonocyteA white blood cell that helps the body fightinfections from some bacteria such as tuberculosis.NeutrophilA type of white blood cell that functions todestroy bacteria. When the number of neutrophilsis too low, the body is at greater risk fordeveloping an infection.
WHITE BLOOD CELLS
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BiopsyA medical procedure to remove a small piece ofsolid bone marrow using a needle that goes intothe marrow of the hip bone. The solid bonemarrow is examined for cell abnormalities, thenumber of different cells, and checked for scarringof the bone marrow.
Iliac CrestThe hip bone area from which bone marrowsamples are most commonly taken.
BONE MARROW BIOPSY
Allogeneic Stem Cell TransplantationA procedure in which matched bone marrow orperipheral blood stem cells from a donor(usually related) are collected, stored, andinfused into a patient (recipient) following high-dose chemotherapy with or without radiationtherapy. In time, donated stem cells given tothe patient begin making new, healthy bloodcells (known as engraftment).
AllograftAn allogeneic stem cell collection used fortransplant.
BONE MARROW TRANSPLANT
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AutograftAn autologous stem cell collection used fortransplant.
Autologous Stem Cell TransplantationA procedure in which a patient’s own stem cellsfrom bone marrow or peripheral blood arecollected, stored, and reinfused following high-dose chemotherapy or radiation therapy. Intime, donated stem cells given to the patientbegin making new, healthy blood cells (known asengraftment).
Bone Marrow TransplantA procedure in which high doses of chemotherapyor radiation therapy are used to eradicate diseasein the bone marrow and lymphatic system andthen are replaced with healthy bone marrow froma donor or the patient.
Cord Blood TransplantA procedure where umbilical cord stem cells aregiven to the patient through an intravenous (IV)line. Stem cells are collected from an umbilical cordright after the birth of a baby. They are kept frozenuntil needed. In time, donated stem cells given tothe patient begin making new, healthy blood cells.
EngraftmentRefers to how well the donor cells (graft) areaccepted by the patient’s immune system (host)after a bone marrow or stem cell transplant.
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Several factors contribute to better engraftment:physical condition of the patient, how severe thedisease is, type of donor available, age of patient.Successful engraftment results in new bonemarrow that produces healthy blood cells (newwhite blood cells, red blood cells, and platelets).
Graft-Versus-Host Disease (GVHD)GVHD is a common complication of allogeneicbone marrow/stem cell transplantation. It iscaused when the donor’s immune cells, now inthe patient, begin to see the patient’s body asforeign and mount an immune response. GVHDmost commonly affects the recipient’s skin,intestines, or liver. Severity can range from mildto very severe. In some cases, GVHD can beprevented or treated with specific drugs tosuppress the body’s immune cells (immuno-suppressive drug therapy).
Human Leukocyte Antigen (HLA)One of a group of proteins found on the surfaceof white blood cells and other cells. These antigensdiffer from person to person. A human leukocyteantigen test is done before a stem cell transplantto closely match a donor and a recipient.
Matched Related DonorBone marrow/stem cell donor that is a sibling oranother blood relative to the patient.
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Mini-TransplantSee Non-Myeloablative Transplant
MyeloablationThe killing of bone marrow by radiation orchemotherapy. This term usually refers to thecomplete or near-complete destruction of thebone marrow.
Non-Myeloablative TransplantType of allogeneic stem cell or bone marrowtransplant that uses lower doses of chemotherapy.This reduces side effects caused by chemo-therapy, making it more tolerable for olderadults. It does not reduce the risk of graft-versus-host disease. Also called nonmyeloablativetransplant or reduced intensity transplant.
Reduced Intensity TransplantProcedure similar to standard bone marrowtransplant. The reduced intensity transplant usesa mild form of chemotherapy pre-treatment.This reduces side effects caused by chemotherapy,making it more tolerable for older adults. It doesnot reduce the risk of graft-versus-host disease.Also called nonmyeloablative transplant.
Unrelated DonorA donor that is not a sibling or other familialrelation of the patient (recipient).
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Adverse Event (AE)Any undesired actions or effects of a drug ortreatment.Synonyms: side effect, toxicityAntibiotic Therapy (AB)Used to treat bacterial infections or preventrecurrence of bacterial infections.
Antithymocyte Globulin (ATG)An immunosuppressive medication that eliminatesabnormally proliferating white blood cells calledT lymphocytes which disrupt normal blood cellgrowth. This may restore normal production ofred blood cells which may lead to transfusionindependence. The three brand-name drugs areThymoglobulin®, Lymoglobulin®, and Atgam®.
Colony-Stimulating Factor (CSF)Protein that stimulates the development and growthof blood cells; sometimes called growth factor.Granulocyte colony-stimulating factor is a CSFthat is used to stimulate stem cells from the bonemarrow into the bloodstream prior to apheresis.
CorticosteroidsAlso called “steroids,” corticosteroids arepowerful anti-inflammatory medicines used totreat many diseases and conditions. They aresimilar to a protein called cortisol that is made
MEDICATIONS
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in the adrenal glands. Names of corticosteroidsinclude prednisone and dexamethasone.Synonym: steroidsDacogen™ (decitabine)A medication used in treating some types of MDSand AML. Dacogen works by preventing certaingenes involved in controlling cancer from beingsilenced, allowing for the normal functioning ofgenes within the body. It is a DNA hypomethylatingagent that is administered intravenously (IV).
Desferal® (deferoxamine)A medication that binds to iron and promotes itsremoval from the body for treatment of transfusion-dependent iron overload. It is an iron-chelating drugthat is administered subcutaneously (under the skin).
Erythropoietin (EPO)A “recombinant” form of a natural growth factorused to treat symptoms associated with anemia.It stimulates the bone marrow to produce redblood cells. The three brand-name drugs areAranesp®, Epogen®, and Procrit®. These drugs areadministered intravenously or subcutaneously.
Exjade® (deferasirox)A medication that binds to iron and promotesits removal from the body for treatment oftransfusion-dependent iron overload. It is aniron-chelating drug that is administered orally.
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Growth Factors (hematopoietic)A substance made by the body that stimulates thebone marrow to produce blood cells. Some growthfactors are man-made in the laboratory and usedfor treating low blood counts. These include redblood cell growth factors called erythropoietin(EPO) and darbepoetin, and white blood cell growthfactors called granulocyte colony stimulatingfactors (GCSF) and granulocyte macrophage colonystimulating factors (GMCSF). Also called cytokines.Synonym: cytokineHycamtin® (topotecan hydrochloride)A chemotherapy agent that may result inremission of MDS. It is administered intravenously.JADENU™ (deferasirox)A medication that binds to iron and promotesits removal from the body for treatment oftransfusion-dependent iron overload. It is an iron-chelating drug that is administered orally. It is anew oral formulation of Exjade tablets. Whereasthe Exjade tablet must be mixed in liquid andtaken on an empty stomach, Jadenu can be takenin a single step, with or without a light meal,simplifying administration of treatment forchronic iron overload.Leukine® (sargramostim)A growth factor, granulocyte macrophagecolony-stimulating factor (GM-CSF), used forthe treatment of neutropenia. It increases
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white cell production, which may help toreduce the likelihood of additional infection. Itis administered subcutaneously.Neupogen® (filgrastim)A growth factor, granulocyte colony-stimulatingfactor (G-CSF), used for the treatment of neutro-penia. It increases white cell production, whichmay help to reduce the likelihood of additionalinfection. It is administered subcutaneously.
PrednisoneA corticosteroid that is used for many reasons.It is prescribed when the body is not producingenough of this chemical on its own. It issometimes prescribed with ATG treatment toreduce the risk of anaphylaxis or serum sickness.It helps by reducing the antibody production ofthe immune system and in treating variousallergic conditions. There are many brand namesof prednisone. (See corticosteroid)Pyridoxine (Vitamin B6)A vitamin needed to make red blood cells. It canbe useful in improving red blood cell counts insideroblastic anemia by increasing the red bloodcell production.
Revlimid® (lenalidomide)A medication that works by stimulating theimmune system, preventing new blood vesselgrowth, and stimulating cell death. It is categorizedas an immunomodulatory agent and is taken orally.
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Telintra™ (TLK199)A medication that inhibits a key enzyme(glutathione S-transferase P1-1 or GST P1-1)involved in cell growth and proliferation; thisresults in normal blood cell production. It isgiven intravenously (IV).
Thalomid® (thalidomide)A medication that reduces the blood supply in themarrow, thereby working to limit the growth ofabnormal blood cells. It also acts to interfere withother proteins (cytokines) that promote prematuredeath of cells in the bone marrow. It is taken orally.
Trisenox® (arsenic trioxide)A medication that inhibits new blood vesselgrowth and stimulates cell death of abnormalcells. It may increase transfusion independence.It is administered as an intravenous infusion (IV).
Vidaza™ (azacitidine, 5-azacytidine)A medication that works by preventing a cellularprocess (methylation) that silences the genesinvolved in controlling the development of cancer.It may increase red blood cells, transfusionindependence, hemoglobin, white blood cells,platelets, and/or decreases the amount of blastcells within the bone marrow. It is categorized asa DNA hypomethylating agent and can beadministered intravenously (IV) or subcutaneously(under the skin).
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