Absolute neutrophil count (ANC)
The number of white blood cells in the blood that are neutrophils. A low ANC indicates neutropenia and a possible increased risk of infection.
Designation assigned to a drug by the Food and Drug Administration (FDA) intended to make promising products for life-threatening diseases available on the market on the basis of preliminary evidence prior to formal demonstration of patient benefit. In this case, clinical studies are designed to measure and the FDA evaluation is performed on the basis of a surrogate marker that is considered likely to predict patient benefit. The marketing approval that is granted may be considered provisional, and a company may be required to complete additional clinical studies that formally demonstrate patient benefit.
Type of immunotherapy that stimulates the body to mount an immune response; an example is a vaccine.
Complimentary molecules present on cell surfaces that allow cells to interact with each other, acting in the same way as a lock and key.
(1) When referring to vaccines, an adjuvant is a substance administered as part of, or along with, a vaccine that increases its effectiveness by strengthening the immune response against the vaccine. (2) Adjuvant therapy also refers to a treatment that is given in addition to a standard treatment regimen to increase its effectiveness.
Type of passive immunotherapy (treatment that is prepared outside the body) that involves the transfer of immune cells to a patient.
A type of chemotherapy which is given intravenously (into a vein). It is part of an older type of myeloma treatment called VAD. VAD consists of the combination of Vincristine (another chemotherapy drug), Adriamycin, and dexamethasone which is a steroid.
Adverse event (AE)
Any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medical treatment or procedure that may or may not be considered related to the medical treatment or procedure. An adverse event is a term that is a unique representation of a specific event used for medical documentation and scientific analyses.
Major protein found in the blood. A patient?s albumin level can provide some indication of overall health and nutritional status and may also be useful in staging myeloma according to a recently proposed staging system.
Allogeneic stem cell transplant
A procedure in which bone marrow or peripheral blood stem cells from a donor (usually related) are collected, stored, and infused into a patient (recipient) following high-dose chemotherapy and/or radiation therapy.
Transplant from a human donor who is not an identical genetic match.
Allogeneic stem cell transplant.
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It is given intravenously (into a vein).
Cytokine produced by T cells that exhibits a variety of immunomodulating effects, including suppression of cell growth and enhancement of tumor cell killing.
Condition related to multiple myeloma whereby a patient’s light chains can clump together to form insoluble protein fibers known as amyloid fibrils. Amyloid fibrils can accumulate in various tissues and organs, disrupting their normal functions. Rarely, patients with myeloma may also develop amyloidosis.
Fiber-like substance, often composed of light chains and other proteins, that can accumulate in and damage various tissues and organs.
Decrease in the number of red blood cells in the blood.
Growth of new blood vessels.
Class of anticancer drugs that includes doxorubicin (Adriamycin®) and related compounds such as daunorubicin and daunomycin.
Protein produced by a plasma cell that is generated in reaction to a foreign protein (antigen), thus producing an immunity against that protein; also known as an immunoglobulin.
Substance that stimulates the production of an antibody to which it subsequently binds.
Chemically-altered stretch of DNA bases designed to bind to and block the production of specific proteins.
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It is given intravenously (into a vein).
Procedure in which blood is taken from a donor, a blood component (such as white blood cells, red blood cells, or plasma) is separated out, and the remaining blood components are reinfused back into the donor.
Programmed (natural) cell death.
Marked loss of body strength.
Autologous stem cell transplant.
Illnesses that occur when the body tissues are attacked by its own immune system.
From the patient. In myeloma, this term usually refers to a commonly used type of stem cell transplantation where the patient serves as their own donor.
Autologous peripheral blood stem cell transplants
A type of stem cell transplantation where the patients own stem cells are used. In this type of transplant, stem cells are collected from the circulating blood (peripheral blood). Autologous peripheral blood stem cell transplants are the most common type of stem cell transplants performed today.
Autologous stem cell transplant
Procedure in which a patient’s own stem cells from the bone marrow or peripheral blood are collected, stored, and reinfused following high-dose chemotherapy and/or radiation therapy.
Transplant whereby the patient’s own cells or tissues are collected and reinfused or transplanted.
Basic fibroblast growth factor (bFGF)
Growth factor that promotes angiogenesis (the growth of new blood vessels); also known as FGF-2.
White blood cell that gives rise to a plasma cell after being exposed to a foreign substance; also called a B lymphocyte.
Protein made by cancer cells that is thought to block chemotherapy-induced cell death.
See Light chain.
Beta 2-microglobulin (ß2-microglobulin or ß2-M)
Protein normally found on the surface of various cells in the body. Increased serum levels can occur in patients with myeloma or kidney disease. Lower levels are associated with a more favorable prognosis in myeloma.
Type of antibiotic.
Type of drug used to treat osteoporosis and bone disease in cancer patients. Bisphosphonates work by inhibiting the activity of bone destroying cells called osteoclasts.
Set of stringent, standardized criteria developed by members of the European Group for Blood and Marrow Transplant (EBMT), International Bone Marrow Transplant Registry (IBMTR), and Autologous Blood and Marrow Transplant Registry (ABMTR) that are increasingly being used to measure response to therapy in myeloma clinical trials (Bladé et al. Br J Haematol. 1998;102(5):1115-1123.) The criteria include two assessments of M protein levels, percent plasma cells in the bone marrow, and skeletal disease performed at least 6 weeks apart.
Blood urea nitrogen (BUN)
Byproduct of protein metabolism that is normally filtered out of the blood and found in the urine. Elevated levels in the blood can indicate decreased kidney function.
See B cell.
Soft, spongy tissue found in the center of many bones where blood cells are produced.
Bone marrow aspiration
Removal of fluid and cells of the bone marrow via use of a needle.
Bone marrow biopsy
Removal of bone marrow tissue via the use of a needle.
Bone marrow transplant
Procedure in which stem cell-containing bone marrow is collected, stored, and infused following high-dose chemotherapy and/or radiation therapy.
Process of normal bone maintenance whereby old, worn-out bone is broken down and removed by osteoclasts and new bone is formed by osteoblasts.
The normal process of breaking down of bone by osteoclasts during bone remodeling. Bone resorption can be increased in myeloma.
Bone (skeletal) survey
Series of x-rays of the skull, spine, arms, ribs, and legs.
Mineral important in bone formation. Elevated serum levels occur when there is bone destruction.
A thin flexible tube that is inserted into the body. For example, it may be inserted into a vein in order to give drugs, blood or nutrients. Catheters are also used to take blood or empty the bladder.
Cell surface marker. CD stands for cluster of differentiation and the 34+ indicates a specific antigen for which this cell is positive. Stem cells are CD34+.
Blood test that determines levels of several chemical compounds in the blood at one time. Of particular importance in myeloma are levels of blood urea nitrogen (BUN), calcium, creatinine, and lactate dehydrogenase (LDH).
Constellation of symptoms, such as loss of memory, difficulty with language, and lack of concentration, that have been reported by cancer patients who receive chemotherapy; also known as cognitive dysfunction.
The use of drugs to treat cancer.
A structure in cells which hold the DNA (which contains the genes).
In some individuals with multiple myeloma, part of the short arm of chromosome 13 is deleted. Absence of this deletion is associated with a more favorable prognosis.
Chromosome 14 deletion
In some individuals with multiple myeloma, a piece of one or more of certain chromosomes may be missing (or deleted). A deletion in chromosome 14 may result in a more aggressive form of myeloma.
Chromosome 17 translocation
An abnormal change in the DNA where two segments switch positions. A change in chromosome 17 may result in a more aggressive form of myeloma.
Chromosome analysis (cytogenetic testing)
Laboratory test that measures the number and normalcy of chromosomes; also known as cytogenetic testing.
Colony-stimulating factor (CSF)
Protein that stimulates the development and growth of blood cells; sometimes called growth factor. Granulocyte colony-stimulating factor is a CSF that is used to mobilize stem cells from the bone marrow into the bloodstream prior to apheresis.
Complete blood count (CBC)
Blood test that measures the number of red blood cells, white blood cells, and platelets in the blood and the relative proportions of the white blood cells present.
See complete response
Complete response (CR)
A treatment outcome where there are ≤5% plasma cells in the bone marrow and no evidence of myeloma proteins in the serum or urine as measured by standard laboratory techniques.
Computed tomography (CT)
Imaging technique that uses a computer to generate 3-dimensional x-ray pictures; also known as computerized axial tomography (CT).
Computerized axial tomography (CAT)
See Computed tomography (CT).
Combination of chemotherapy and/or radiation treatments administered over a period of several days prior to stem cell transplantation in order to kill cancer cells.
Anti-myeloma treatment given after the initial therapy in order to further reduce the number of cancer cells.
Administration of chemotherapy at a dose that does not completely destroy the bone marrow; also known as standard-dose chemotherapy.
Cord blood transplant
Type of transplant where the stem cells are obtained from the umbilical cord and placenta (afterbirth) following the delivery of a baby. These cells are frozen for future use.
Potent class of drugs that has anti-inflammatory, immunosuppressive, and antitumor effects. Dexamethasone and prednisone are examples of corticosteroids.
Coumadin® (also known as warfarin)
A pill that is a type of blood thinner. It used to prevent blood clots.
C-reactive protein (CRP)
Protein produced by the liver when there is an inflammatory process occurring in the body. Serum levels of CRP are increased in various inflammatory diseases, degenerative diseases, and cancers, including myeloma. Lower levels are associated with a more favorable prognosis in myeloma.
See complete response
Product of energy metabolism of muscle that is normally filtered out of the blood and found in the urine. Elevated levels in the blood can indicate decreased kidney function.
Condition that occurs when a myeloma protein is of a specific type that comes out of solution as particles when exposed to cold temperatures. These particles may block small blood vessels and cause symptoms such as pain and numbness in the fingers and toes in cold weather.
A method of freezing cells that permits storage over an extended period of time.
Treatment outcome where there is complete and lasting recovery from disease. This has not yet been achieved in myeloma.
Cutaneous T-cell lymphoma (CTCL)
A type of non-Hodgkin’s lymphoma which is a cancer of the immune system.
A protein that acts as an enzyme to enhance the production of prostaglandins responsible for promoting inflammation.
Type of chemotherapy. Brand names include Cytoxan® and Neosar®.
Immunosuppressive drug used following allogeneic transplants that helps prevent graft-versus-host disease (donor cells attacking the recipient’s cells).
See Chromosome analysis.
Soluble factor produced by cells that has an effect on other cells.
Type of corticosteroid used in the treatment of myeloma. Decadron is part of the chemotherapy regimen called VAD.
Deep vein thrombosis (DVT)
Serious condition where a blood clot forms in one of the deep veins in the body, usually in the legs or lower abdomen. DVT is treated with a blood thinner (also called an anticoagulant).
Immune cell that plays an important role in initiating and regulating immune responses.
A steroid used in the treatment of multiple myeloma, often in combination with another anti-myeloma drug. Decadron® is one brand of dexamethasone.
The length of time during and after treatment in which a patient is living with a disease that does not get worse; also referred to as progression-free survival (PFS).
Whether a patient has already received therapy for their myeloma disease and if so, what was the outcome.
Dimethyl sulfoxide, a colorless chemical used for cryopreservation of stem cells. When introduced into the body, may cause unpleasant or even serious toxic effects.
DNA (deoxyribonucleic acid)
Genetic material of the cell located in the chromosomes.
Donor Lymphocyte Infusion (DLI)
Experimental therapy used following high-dose chemotherapy and allogeneic stem cell transplant; involves the administration of additional immune cells from the same allogeneic donor to help attack myeloma cells and control the disease.
Doxil® (doxorubicin HCl liposome injection)
A chemotherapy drug used in cancer treatment. It is a reformulated version of doxorubicin (Adriamycin® ), a cancer drug that has been used for many years in traditional chemotherapy regimens, including VAD (vincristine, Adriamycin, and dexamethasone) in myeloma.
Electrolytes are minerals that are present in the body such as sodium, potassium, chloride, and bicarbonate. The balance of electrolytes is essential for the normal function of organs. Diarrhea may cause electrolyte depletion.
Laboratory test used to measure the levels of various proteins in the blood or urine. Uses an electrical current to sort proteins by their molecular size.
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It may be given either orally or intravenously (into a vein).
New treatment that is being investigated in clinical trials.
Type of cell that lines blood vessels.
Process in which stem cells in transplanted bone marrow or blood migrate to the bone marrow and begin to grow and produce new white blood cells, red blood cells, and platelets.
Stomach pain or cramps caused by an inflammation of the small intestine.
The production of red blood cells by the bone marrow.
Growth factor that stimulates the bone marrow to produce red blood cells.
Heartburn caused by an irritated esophagus (tube leading from the mouth to the stomach).
Event-free survival (EFS)
Term used in oncology clinical trials to denote the length of time that a patient remains free of certain negative events, such as cancer recurrence or progression, complications from the disease, or death from any cause. The specific events, as well as to when timing begins, may vary from trial to trial.
Refers to any of the procedures initiated by the Food and Drug Administration (ie, compassionate use, parallel track, and treatment IND) that distributes experimental drugs to patients who are failing on currently available treatments for their condition and also are unable to participate in ongoing clinical trials.
Enzyme involved in a signaling pathway that causes cancer cells to grow.
Farnesyl transferase inhibitor (FTI)
Drug that inhibits farnesyl transferase, an enzyme involved in a signaling pathway that causes cancer cells to grow.
Farydak® is a histone deacetylase inhibitor that is administered in combination with Velcade® (bortezomib) and dexamethasone. It is indicated for the treatment of patients with multiple myeloma who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent. This indication was approved by the FDA on February 23, 2015 under accelerated approval based on progression free survival.
Fast Track designation
Status assigned by the Food and Drug Administration (FDA) to a drug or product in clinical trials signifying that the FDA will facilitate and expedite the development and review of the application for the approval of the new drug. Fast track status is usually reserved for drugs that are intended for the treatment of serious or life-threatening conditions and which demonstrate the potential to address an unmet medical need.
Presence of a low neutrophil count in the blood that is associated with fever; may indicate the presence of infection (that may be serious or life-threatening).
Fibroblast growth factor 2 (FGF-2)
Growth factor that promotes angiogenesis (the growth of new blood vessels); also known as basic fibroblast growth factor (bFGF).
A type of growth factor that stimulates the growth of white cells in the bone marrow; also known as G-CSF (granulocyte colony stimulating factor).
Fluorescence in situ hybridization (FISH)
Type of chromosome analysis that detects abnormalities of specific chromosomes.
Type of x-ray that allows viewing of bone.
A mild side effect or resulting from a medical therapy. Minimal or no symptoms may be present and typically medical intervention is not required.
A moderate side effect resulting from a medical therapy. Depending on the nature of the side effect, medical intervention may or may not be needed.
A severe side effect resulting from a medical therapy. This side effect typically interferes with patient functioning and requires medical intervention.
A life-threatening or disabling side effect resulting from a medical therapy. Immediate medical intervention is required.
Graft-versus-host disease (GVHD)
Complication of allogeneic transplants resulting from donor immune cells recognizing the recipient’s cells as foreign and mounting an attack against them.
Beneficial effect of allogeneic transplants resulting from the donor cells mounting an attack on the recipient’s myeloma cells
Type of white blood cell important in fighting infection. Neutrophils are the most abundant type of granulocyte.
Granulocyte colony-stimulating factor (G-CSF)
A drug that stimulates the production of infection-fighting white blood cells known as granulocytes. G-CSF is used to help prevent low white blood cell counts and infection in patients receiving chemotherapy and/or stem cell transplants and is also used to help mobilize stem cells prior to stem cell transplant. In addition, it may be used to stimulate white blood cells production in patients whose white blood cell levels drop as a side effect of an anti-myeloma treatment.
Granulocyte-macrophage colony-stimulating factor (GM-CSF)
A drug that may be used as an alternate to G-CSF (see above). It stimulates the production of infection-fighting white blood cells known as granulocytes and macrophages. Similar to G-CSF, GM-CSF is used to help prevent low white blood cell counts and infection in patients receiving chemotherapy and/or stem cell transplants and is also used to help mobilize stem cells prior to stem cell transplant.
Drugs that are used to stimulate the production of certain types of cells. The most commonly used growth factors are red blood cell growth factors (also known as erythropoietin) and white blood cell growth factors(also known as colony stimulating factors or CSFs)
Hand-foot syndrome (HFS)
Skin condition noted by tingling or burning, redness, flaking, bothersome swelling, small blisters, or small sores on the palms of the hands or soles of the feet. HFS is a common side effect of certain types of chemotherapy, such as doxorubicin and liposomal doxorubicin (Doxil), and is also known as palmar-plantar erythrodysesthesia (PPE).
Refers to patients who have recent several prior therapies for their disease.
The longer of the two protein chains that make up an immunoglobulin molecule.
A laboratory test used to determine if anemia is present; refers to the percentage of the volume of a blood sample that consists of red blood cells.
Pertaining to the blood.
Formation and development of blood cells in the bone marrow.
Hematopoietic stem cells
Cells found in the bone marrow or the circulating blood that are responsible for the production of red blood cells.
A substance found within red blood cells that carries oxygen from the lungs to the tissues in the body. Low hemoglobin levels are an indicator of anemia.
Enzyme produced by tumor cells that promotes angiogenesis by loosening the “glue” that holds cells of blood vessel walls together. This, in combination with angiogenic growth factors, allows the sprouting of new blood vessels.
A type of blood thinner used to prevent blood clots. It is given as an injection. Some types may be self-administered by patients at home.
reduced liver function that is noted by increased levels of various substances in the blood, such as bilirubin.
Administration of very high doses of chemotherapy that may be more effective in eliminating myeloma cells than standard treatments. High-dose chemotherapy destroys the bone marrow, which is responsible for the production of blood cells. Therefore a procedure called stem cell transplant is required to replenish blood-forming bone marrow cells.
High risk myeloma
A type of myeloma where there is some feature that indicates a worse prognosis. For example, certain types of abnormalities in the DNA are associated with more aggressive disease. In addition, elevated blood levels of a protein called beta 2-microglobulin is also associated with worse prognosis.
Condition noted by elevated levels of calcium in the blood due to increased bone destruction.
Hypercalcemia of malignancy (HCM)
Condition occurring in various forms of cancer noted by elevated levels of calcium in the blood due to increased bone destruction; also known as tumor-induced hypercalcemia (TIH).
Condition that can occur in myeloma whereby the protein concentration in the blood becomes very high and the blood becomes very thick and sticky. Symptoms can include shortness of breath, confusion, and chest pain.
Condition where the thyroid gland is less active than normal, resulting in symptoms such as a low metabolic rate and lack of energy.
Property of an antibody molecule that is unique to that antibody.
Type of vaccine that uses a patient’s M protein as the antigen with which to stimulate an immune response.
Interaction of an antigen with lymphocytes to induce the formation of antibodies.
Network of related cells, tissues, and organs that protect the body from disease organisms, other foreign bodies, and cancers.
See Immunofixation electrophoresis.
Immunofixation electrophoresis (IFE)
Type of electrophoresis that uses a special antibody staining technique to identify specific types of immunoglobulin and light chains; also called immunoelectrophoresis.
Protein produced by a plasma cell that is generated in reaction to a foreign protein (antigen), thus producing an immunity against that protein; also known as an antibody. Immunoglobulins are made up of 2 heavy chains and 2 light chains that form a “Y”-like structure and can be of the IgA, IgD, IgE, IgG, or IgM class.
Drug that affects, enhances, or suppresses the immune system.
Immunomodulatory drugs or IMiDs®
Drugs that work to fight cancer cell growth by impacting the functioning of the immune system.
Drug given to suppress a patient’s immune system, such as one given to prevent rejection of transplanted tissue.
The treatment of, or prevention against, a disease achieved through manipulation of the patient’s immune system.
Proposed classification of myeloma that includes monoclonal gammopathy of undetermined significance (MGUS), smoldering myeloma, and Stage 1 disease.
Indolent multiple myeloma (IMM)
Type of asymptomatic myeloma noted by small amounts of monoclonal protein or increased numbers of plasma cells in the bone marrow. Rarely there can be mild anemia or a few bone lesions. Patients with indolent myeloma are monitored and only treated if the disease progresses.
Initial therapy for myeloma. This term also refers to the use of anti-myeloma drugs prior to high-dose chemotherapy and stem cell transplant in order to reduce the tumor burden.
Possible side effect seen with some drugs that are given intravenously. Symptoms are usually mild to moderate and most commonly include chills, fever, nausea, weakness, headache, skin rash, and/or itching. In most cases, these reactions can be managed by either slowing or stopping the infusion. Although rare, severe reactions such as difficulty breathing or low blood pressure may occur which require treatment.
Institutional Review Board (IRB)
Board designed to oversee the research process in order to protect participant safety. Made up of researchers, ethicists, and lay people from the community, the board must review clinical trial protocols and the informed consent forms participants sign.
Substance naturally produced in the body by virus-infected cells that protects noninfected cells from viral infection. Interferon also has various effects on the immune system and is used in the treatment of several cancers and infectious diseases.
Interleukin 1 beta (IL-1b)
Cytokine that enhances the growth and survival of B cells and myeloma cells and promotes inflammation.
Interleukin 2 (IL-2)
Cytokine (growth factor) produced by T-cells that stimulates the growth of T cells and B cells.
Interleukin 4 (IL-4)
Cytokine that enhances the immune system’s ability to fight tumor cells.
Interleukin 5 (IL-5)
A potent T-cell derived factor that stimulates the growth of B-cell growth factor 2.
Interleukin 6 (IL-6)
Cytokine that promotes the growth and survival of myeloma cells and normal B cells.
Interleukin 10 (IL-10)
Cytokine involved in the growth and survival of myeloma cells.
Interleukin 12 (IL-12)
Cytokine that promotes T cell function and tumor cell killing.
Kaposi’s Sarcoma-associated Herpes virus (KSHV)
Virus of the herpesvirus family, also known as herpesvirus 8, that has been identified in bone marrow dendritic cells in some patients with myeloma. Whether this virus is involved in the disease is controversial.
Medication that is used to protect against the development of mouth sores that are a side effect of high-dose chemotherapy. It may also reduce the duration and severity of mouth sores if they do occur.
Procedure used to treat spinal compression fractures whereby a balloon in inserted into the compressed vertebra and inflated to elevate the collapsed section. The cavity is filled with bone cement, stabilizing and preserving the re-established height.
A new type of proteasome inhibitor. It is in the same class of drugs as Velcade.
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It is taken orally.
Lactate dehydrogenase (LDH)
Enzyme found in body tissues. Elevated blood levels occur when there is tissue damage and may occur in myeloma, where they reflect tumor-cell burden.
A brand of Granulocyte colony-stimulating factor (G-CSF) that stimulates the growth of white blood cells in the bone marrow.
The shorter of the two protein chains that make up an immunoglobulin molecule. May be of the kappa or lambda type. Light chains produced by myeloma cells are also referred to as Bence-Jones proteins.
Bubble- or onion-like particle made out of lipids (fats) that is used to encapsulate drugs. Encapsulation helps the drug remain in the body for a longer period of time and may reduce toxicity.
Small white blood cell essential for normal function of the immune system; may be 1 of 2 types: a T lymphocyte or B lymphocyte.
Magnetic resonance imaging (MRI)
Imaging technique that uses magnetic energy to provide detailed images of bone and soft tissue.
Treatment that is given to patients in remission over a long period of time, in order to reduce the risk of relapse.
Cancerous, continuing to divide.
Marketing Authorization Application (MAA)
Compilation of information on the safety and efficacy of a new drug that is submitted to the European regulatory agency in order to request approval to market the drug as therapy for a particular disease indication; similar to a New Drug Application (NDA), which is submitted in the United States.
Matrix metalloproteinases (MMPs)
Enzymes that break down the structure of connective tissue.
Type of chemotherapy often used in the treatment of myeloma (also known as Alkeran®).
The spread of tumor cells from the initial site to other areas of the body, particularly the lungs.
Microvascular density (MVD)
Measure of the number of blood vessels in the bone marrow as an indication of the degree of angiogenesis (growth of new blood vessels). A value of less than 6 blood vessels per field when viewing bone marrow cells at 400x magnification is associated with a more favorable prognosis.
Type of allogeneic stem cell transplant that uses lower doses of chemotherapy and thus does not completely destroy the bone marrow; also known as mini-transplant or non-myeloablative transplant.
See mini-allogeneic transplant.
Treatment outcome where there is less than 50% decrease in M protein; also known as minor response. Some myeloma groups consider minimal response to be part of the definition of stable disease.
Administration of colony-stimulating factors or chemotherapy to help move stem cells from the bone marrow into the blood stream to increase the number of peripheral blood stem cells collected for a stem cell transplant.
Molecular complete response
Treatment outcome where there is no evidence of disease using sensitive molecular techniques; also known as molecular complete remission.
Type of man-made antibody that is used in the diagnosis and treatment of various diseases. All monoclonal antibodies of a specific type are identical to each other.
Monoclonal gammopathy of undetermined significance (MGUS)
Precancerous and asymptomatic condition noted by the presence of M protein in the serum or urine. MGUS may progress to myeloma.
Monoclonal (M) protein
A type of protein made by myeloma cells, used to estimate the extent of myeloma disease. It is an abnormal type of antibody (or immunoglobulin) and is found in the blood or urine to estimate. M protein levels are used to determine the effectiveness of myeloma treatments.
Type of white blood cell important in the immune response.
The combination of melphalan, a chemotherapy drug, and prednisone, a steroid. MP has been used in myeloma treatment for many years. Today, it is usually given in combination with one of the newer agents, such as Revlimid, Velcade or Thalomid.
Inflammation of mucous membranes lining the digestive tract; a common and painful side effect of intensive chemotherapy or radiotherapy that can result in sores and infection.
Mycophenolate mofetil (CellCept®)
Immunosuppressive drug used following allogeneic transplants that helps prevent graft-versus-host disease (donor cells attacking the recipient’s cells).
The killing of bone marrow by radiation or chemotherapy. This term usually refers to the complete or near-complete destruction of the bone marrow.
Myelodysplastic syndrome (MDS)
A group of diseases that affect the blood. It is not related to multiple myeloma.
Near Complete Response (nCR)
A treatment outcome when there are ≤5% plasma cells in the bone marrow but there are detectable myeloma proteins in the serum or urine as measured by standard laboratory techniques.
Natural killer (NK) cell
Type of white blood cell important in killing tumor cells.
Near complete response (near CR)
Response to therapy where M protein is no longer detectable in the blood and/or urine using conventional tests, but is detectable with the more sensitive immunofixation test, and there are less than 5% plasma cells in the bone marrow.
Toxicity to the kidneys.
Chronic pain caused by damage to, or dysfunction of, nerves.
Disorder of the nerves that can result in abnormal or decreased sensation, or burning/tingling. When the hands and feet are affected, it is referred to as peripheral neuropathy.
Below-normal number of neutrophils.
Type of white blood cell that plays an important role in the immune system (eg, destroys bacteria).
New Drug Application (NDA)
Compilation of information on the safety and efficacy of a new drug that is submitted to the U.S. Food and Drug Administration (FDA) in order to request approval to market the drug as therapy for a particular disease indication.
Newly diagnosed disease
Myeloma disease that has not yet been treated.
Non-myeloablative allogenic transplant
See mini-allogeneic transplant
Rare form of myeloma affecting about 1% of myeloma patients where the malignant plasma cells do not secrete M protein or light chains.
Nonsteroidal anti-inflammatory drugs (NSAIDs)
Common types of pain killers. They are both over-the-counter NSAIDs as well as prescription NSAIDs. Examples of over-the-counter NSAIDs include aspirin, Motrin® (ibuprofen), and Aleve® (naproxen)
Novel erythrocyte stimulating protein (NESP)
Protein under investigation that stimulates the bone marrow to produce red blood cells.
Nuclear factor ?B (NF-?B)
Key survival protein found within the cell that acts as a messenger. When a cell receives an external signal, such as a growth factor, NF-kB transfers the message to the nucleus of the cell, causing some type of response, such as cell growth.
Office for Human Research Protections (OHRP)
Government office that safeguards participants in federally funded research and provides unity and leadership for many federal departments and agencies that carry out research involving human participants.
Term referring to a product that treats a rare disease affecting fewer than 200,000 Americans. The Food and Drug Administration (FDA) provides this designation and incentives for companies making orphan drugs to stimulate the research, development, and approval of products that treat rare diseases.
Bone-destroying cell that works in conjunction with bone-forming cells to repair bone.
Osteoclast-activating factor (OAF)
Substance released by tumor cells that promotes the activity of bone-destroying osteoclasts. Examples of OAFs include parathyroid hormone-related peptide (PTHrP), growth factors, and cytokines.
Soft spot in the bone where bone tissue has been destroyed. The lesion appears as a hole on a standard bone x-ray.
Death or destruction of bone tissue due to trauma, loss of blood supply, or disease.
A condition where bones are thinner than normal, but the bone loss is not severe enough to be classified as osteoporosis.
Generalized bone loss typically associated with old age.
Overall response rate
Typically the percentage of patients who respond to a specific therapy in a clinical trial with a partial response or better.
Overall survival (OS)
Term used in oncology clinical trials to denote the length of time a patient survives.
A bone disease that is not related to cancer. It is a chronic condition that results in enlarged and deformed bones.
Medication that stimulates the growth of cells found in the oral cavity and skin and reduces the duration and severity of oral mucositis (inflammation of the lining of the digestive tract) after intensive chemotherapy and radiotherapy for hematologic cancers. The brand name for palifermin is Kepivance™ (Amgen).
Meant to reduce symptoms and relieve pain rather than to alter the course of disease.
Palmar-plantar erythrodysesthesia (PPE)
Skin condition noted by tingling or burning, redness, flaking, bothersome swelling, small blisters, or small sores on the palms of the hands or soles of the feet. PPE is a common side effect of certain types of chemotherapy, such as doxorubicin and liposomal doxorubicin, and is also known as hand-foot syndrome.
A type of bisphosphonate. Bisphosphonates are drugs that are used to treat osteoporosis as well as to prevent and treat bone problems in myeloma patients.
See Monoclonal protein.
Parathyroid hormone-related peptide (PTHrP)
Hormone produced by certain tumors that promotes the activity of bone-destroying osteoclasts and causes increased calcium in the blood (hypercalcemia).
Partial response (PR)
Treatment outcome where there is a greater than 50% decrease in M protein; also referred to as partial remission.
Type of immunotherapy that is prepared outside the body and administered to a patient, such as antibodies, serum, or monoclonal antibodies.
When a polyethylene glycol molecule is added on to a drug molecule. Pegylation helps a drug remain in the body for a longer period of time.
A measure of a patient’s ability to perform everyday functions and self-care.
The blood that circulates throughout the body.
Peripheral blood stem cell (PBSC)
Stem cells collected from the blood. The term “peripheral” means that the cells come from outside the bone marrow.
Peripheral blood stem cell (PBSC) transplant
Procedure in which blood containing mobilized stem cells is collected by apheresis, stored, and infused following high-dose chemotherapy and/or radiation therapy.
Disorder of the nerves that can result in abnormal or decreased sensation, or burning/tingling in the hands and feet.
Phase I Study
An early, small clinical study which is used to determine a drug’s safety and dosage levels in humans.
Phase I/II Study
A clinical study that combines a Phase I and a Phase II trial of the same treatment. First, the Phase I part of the trial determines a safe dose, then more patients are treated at this dose in the Phase II part of the study to further evaluate safety and efficacy.
Phase II Study
Phase of clinical testing where a new treatment is evaluated for activity. In cancer trials, the new treatment will be tested against a certain type of cancer.
Phase III study
A large clinical study (or trial) conducted in order to determine the effectiveness and safety of new treatments. Usually, the new treatment is compared to an established treatment (not placebo).
Phase IV study
Clinical study conducted after a drug is approved by the Food and Drug Administration (FDA); may be conducted at the request of the FDA or by the sponsoring company for various reasons. Phase IV studies may involve a larger patient population than that studied during earlier clinical trials; they help monitor the safety of the drug and provide ongoing technical support.
Drug or treatment that is designed to look like the medicine being tested but doesn’t have the active ingredient. Placebos are rarely used in cancer treatment trials.
Immature plasma cell.
When the shape and appearance of myeloma cells are more similar to that of early immature plasma cells (plasmablasts). Absence of plasmablastic morphology typically indicates a more favorable prognosis.
Antibody-secreting immune cell that develops from a B cell.
Plasma cell labeling index (PCLI)
The percentage of plasma cells that are actively dividing. A low PCLI may be associated with a more favorable prognosis in myeloma.
Single tumor comprised of malignant plasma cells that occurs in bone or soft tissue. Patients with a plasmacytoma may develop myeloma.
Procedure in which blood is taken from a donor, the plasma is separated out, and the remaining blood cells are reinfused back into the donor; fluids are sometimes administered to replace the removed plasma. In myeloma patients, this technique may be used to remove excess monoclonal protein in the blood.
When the outcome of therapy, be it a response or stable disease, has leveled off and disease parameters remain at a stable level.
Small cell fragments in the blood that help blood clotting.
A new type of immunomodulatory drug. It is in the same class of drugs as Revlimid and Thalomid.
Earlier form of a cell; for example, B cells are precursors of plasma cells.
Type of steroid used to treat myeloma. It is frequently given in combination with a chemotherapy drug called melphalan.
Designation assigned to a drug or product by the Food and Drug Administration (FDA) stating that the FDA will review the application for approval within a target date of 6 months rather than the standard review target date of 10 months. A Priority designation is intended for drugs and products that address unmet medical needs.
The forecast or likely outcome of a disease. A patient’s prognosis is usually based on the extent of disease as noted by the existence of different signs, symptoms, and circumstances, and clinical or laboratory findings.
Clinical or laboratory finding that helps determine prognosis. In myeloma, a prognostic indicator may help determine how fast the tumor is growing, the extent of disease, tumor cell biology, response to therapy, overall health status of the patient, and when treatment should begin.
Progression-free survival (PFS)
The length of time during and after treatment in which a patient is living with a disease that does not get worse; also referred to as disease-free survival.
Active myeloma that is worsening (ie, increasing M protein and worsening end organ damage). In most cases, relapsed and/or refractory disease can be considered to be progressive disease.
Complex of enzymes found within cells that play a key role in the regulation of cell function and growth. Proteasomes break down and clear out proteins after they’ve done their job and are no longer needed. Some cancer cells appear to be particularly dependent on proteasomes to grow and survive and undergo programmed cell death (apoptosis) if proteasome activity is blocked.
Action plan for a clinical trial that includes detailed description of patients who may join the trial, the therapy that will be given, and the care the patients will receive during and after the trial.
A sudden blockage in a lung artery, usually due to a blood clot that traveled to the lung from a vein in the leg. This is a very serious condition which may be life-threatening.
A radioactive drug used for diagnostic or therapeutic purposes.
Radiation therapy; the use of high-energy particles or rays to damage cancer cells and prevent them from growing.
Method used to prevent bias in research studies; a computer or a table of random numbers generates treatment assignments, and participants have an equal chance to be assigned to one of two or more groups (eg, the control group or the investigational group).
Red blood cell (RBC)
Oxygen-transporting blood cell.
Myeloma that does not respond to the first therapy given and the number of myeloma cells continues to increase in spite of treatment.
Myeloma disease that initially responded to therapy but then begins to progress again.
Period during which no evidence of disease is present.
Reduced kidney function. A type of protein called creatine, found in the blood, is used to monitor kidney function.
A decrease in the amount of myeloma cells as a result of treatment. Response is measured by the amount of M protein in the blood or urine.
Response Rate or Overall Response
The total percentage of patients who respond to a specific therapy in a clinical trial.
Myeloma that is responding to therapy with a decrease in M protein of at least 50%. Some myeloma groups consider a decrease in M protein between 25% and 50% to be a minimal response.
Oral drug with multiple anti-myeloma effects. Revlimid has been shown to be effective in newly-diagnosed and relapsed or refractory myeloma alone and in combination with other drugs. It is chemically similar to another myeloma drug called Thalomid® (thalidomide).
Second-line therapy; used to treat disease that has not responded to initial therapy or relapsed disease.
Therapeutic radioisotope under investigation that is linked to a diphosphonate compound, which concentrates in bone.
Serum protein electrophoresis (SPEP)
Test used to measure the levels of various proteins in the blood or serum. Uses an electrical current to sort proteins by their charge.
Smoldering multiple myeloma (SMM)
Type of asymptomatic myeloma representing about 5% of cases and noted by monoclonal protein and slightly increased numbers of plasma cells in the bone marrow. Patients with SMM are monitored and only treated if their disease progresses.
Special Protocol Assessment (SPA)
Procedure by which the U.S. Food and Drug Administration (FDA) evaluates and provides guidance on proposed protocols for pivotal Phase III clinical trials. The SPA provides the trial sponsors with a binding written agreement that the design and analysis of the study are adequate to support an application submission if the study is performed according to the SPA.
Stable disease (SD)
Treatment outcome where the disease has not responded to therapy (no change in Myeloma protein) but has not progressed. It also refers to disease that initially responded to therapy and remains stable after treatment is stopped.
Stage I disease
Myeloma disease classification based on the Durie and Salmon staging system. Patients with Stage I disease have a low tumor cell burden, hemoglobin levels >10 g/dL, normal serum calcium levels, normal bone x-rays, and low M protein levels. Stage I disease is considered to be the most mild form of disease and may be asymptomatic. According to a newly proposed myeloma staging system, Stage I disease can be considered to be inactive or asymptomatic disease.
Stage II disease
Myeloma disease classification based on the Durie and Salmon staging system. Patients with Stage II disease have active, symptomatic disease, noted by an intermediate tumor cell burden and other laboratory measurements and clinical criteria that fall between what is seen in Stage I and III disease.
Stage III disease
Myeloma disease classification based on the Durie and Salmon staging system. Patients with Stage III disease have active, symptomatic disease, noted by a high tumor cell burden, hemoglobin levels <8.5 g/dL, elevated serum calcium, advanced bone lesions, and high M protein levels.
Standard of care
Treatment that experts agree is appropriate, accepted, and widely used; also called standard therapy or best practice.
Standard risk disease
Indicates myeloma with an average prognosis.
Treatment that has been shown to be safe and effective in clinical studies and is adopted as standard practice outside of clinical trials.
Administration of chemotherapy at a dose that does not completely destroy the bone marrow; also known as conventional chemotherapy.
Parent cell that grows and divides to produce red blood cells, white blood cells, and platelets. Found primarily in the bone marrow, but also in the peripheral blood.
Stem cell transplant
Therapeutic procedure in which bone marrow or peripheral blood stem cells are collected, stored, and infused into a patient following high-dose chemotherapy to restore blood cell production.
A rare, but very serious type of rash, resulting from an allergic reaction. It requires immediate medical attention.
Stringent complete response
A treatment outcome where there are no detectable plasma cells in the bone marrow or myeloma proteins in the serum or urine using very sensitive techniques. A test known as free light chain ratio is also normal.
Structural cells of the bone marrow that help support and nourish the blood-producing cells.
Supplemental New Drug Application (sNDA)
Compilation of information on the safety and efficacy of a marketed drug that is submitted to the FDA in order to request approval to market the drug for another indication.
Treatment that addresses the symptoms and complications of a disease rather than the disease itself. Examples in myeloma include bisphosphonates, growth factors (ie, erythropoietin), antibiotics, orthopedic interventions, and pain control measures.
Acts in combination with another agent such that the activity is greater than the simple additive effect of the two agents.
Syngeneic stem cell transplant
Procedure in which bone marrow or peripheral blood stem cells from a patient’s identical twin are collected, stored, and infused into the patient following high-dose chemotherapy or radiation therapy.
Type of lymphocyte (immune cell) that plays an important role in immune responses and targeted cell killing, also known as a T lymphocyte.
Cell of the immune system that plays a key role in immune responses and targeted cell killing; also known as a T-cell.
Type of transplantation technique where a patient receives two planned transplants within a short period of time. Patients may receive 2 autologous transplants or an autologous stem cell transplant followed by a mini-transplant two to four months afterward.
Relating to or causing birth defects.
Oral drug with multiple anti-myeloma effects. Thalomid has been shown to be effective in newly-diagnosed and relapsed or refractory myeloma alone and in combination with agents such as dexamethasone. It is chemically similar to a newer drug called Revlimid®.
Decrease in the number of platelets (small cell fragments in the blood that help it to clot). A low level of platelets may increase the chance of bleeding.
Time-to-disease progression (TTP)
A measure of time after a disease is treated until the disease starts to get worse or progress.
Tissue-type plasminogen activator (t-PA)
Enzyme that helps dissolves blood clots and also inhibits angiogenesis.
TNF-a-induced Interleukin 8 (IL-8)
Cytokine that acts as chemoattractant for neutrophils, basophils and T-cells and promotes degranulation of neutrophils
Total body irradiation (TBI)
Administration of radiation to the entire body with the purpose of destroying tumor cells in preparation for a stem cell transplant.
Toxic epidermal necrosis (TEN)
A rare but life-threatening skin reaction, where skin turns very red and peels off, similar to what you would see with a severe burn. It typically occurs as a side effect to certain drugs, but may also appear as a result of infection or suppression of the immune system.
Tumor lysis syndrome
A condition that can occur during cancer treatment when large numbers of cancer cells die; the breaking up of the cells and release of the material into the blood can cause organ damage.
The normal cells, molecules, and blood vessels that surround and feed a tumor cell. A tumor can change its microenvironment, and the microenvironment can affect how a tumor grows and spreads.
Tumor necrosis factor-alpha (TNF-a)
Cytokine with a number of effects in myeloma, including promotion of the growth of myeloma cells and activation of osteoclasts.
Tumor necrosis factor-related activation-induced cytokine (TRANCE)
Substance produced by bone marrow stromal cells and bone-forming osteoblasts that induces the development and growth of osteoclasts and contributes to the bone disease of myeloma.
Tumor-induced hypercalcemia (TIH)
Condition occurring in various forms of cancer noted by elevated levels of calcium in the blood due to increased bone destruction; also known as hypercalcemia of malignancy (HCM).
The period in which treatment is given (eg, 21 days or 28 days). It is usually followed by a rest period in between treatments.
Flexible cord-like structure containing blood vessels that connects the fetus to the placenta during pregnancy.
Test that determines the level of protein and various chemical compounds in the urine.
Urine electrophoresis (UEP)
Test used to detect and measure the levels of various proteins in the urine, especially Bence-Jones protein. Uses an electrical current to sort proteins by their charge.
An older type of chemotherapy treatment. It consists of the combination of two chemotherapy drugs (Vincristine, Adriamycin) plus dexamethasone, a steroid.
Vascular endothelial growth factor (VEGF)
One of the major growth factors that promotes the growth of new blood vessels (angiogenesis).
Velcade® (also known as bortezomib)
A highly effective myeloma drug, known as a proteasome inhibitor. It is given intravenously either alone or in combination with other myeloma drugs.
Procedure used to treat spinal compression fractures whereby cement is injected into the affected vertebrae to stabilize it.
Very good partial response (VGPR)
Treatment outcome where there is a greater than 90% decrease in M protein; also known as very good partial remission.
A type of chemotherapy which is given intravenously (into a vein). It is part of an older type of myeloma treatment called, VAD. VAD consists of the combination of Vincristine, Adriamycin, (another chemotherapy drug) and dexamethasone, a steroid.
White blood cell (WBC)
One of the major cell types in the blood that is responsible for immune defenses; also called a leukocyte.
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It is given either orally or intravenously (into a vein