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.
Accelerated Approval
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 and(...)
Actionable target
A genetic mutation that causes cancer to develop and for which a drug can block its cancer-causing activity.
Active multiple myeloma
Multiple myeloma in which the percentage of plasma cells in the bone marrow is greater than 10% and in which the patient shows one or more CRAB symptoms (see definition at CRAB).
Adaptive immunity
The part of the immune system that is composed of highly specialized cells designed to recognize foreign invaders and attack them any time they enter the body.
Adhesion molecules
Complementary molecules present on cell surfaces that allow cells to interact with each other, acting in the same way as a lock and key.
Adjuvant
(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(...)
Adriamycin (doxorubicin)
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 that occurs after a medical treatment or procedure; AEs may or may not be related to the treatment or procedure. An adverse event is a term that is a unique representation of a specific event(...)
Albumin
Major protein found in the blood; albumin level can indicate a person's overall health and nutritional status.
Aloxi® (palonosetron)
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).
Alpha interferon Cytokine produced by T cells that exhibits a variety of immunomodulating effects, including suppression of cell growth and enhancement of tumor cell killing.
Amyloid protein
Fiber-like substance, often composed of light chains and other proteins, that can accumulate in and damage various tissues and organs.
Amyloidosis
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(...)
Anemia
Decrease in the number of red blood cells in the blood.
Antibody-drug conjugate (ADC)
A monoclonal antibody that is coupled to a drug (such as a toxin, a radioactive isotope, or a chemotherapy); one example is Blenrep.
Antiemetic
Drug that prevents or relieves nausea and vomiting.
Antigen
Substance that stimulates the production of an antibody to which it subsequently binds.
Antimicrobial
Drug that kills or slows the growth of bacteria.
Anzemet® (dolasetron)
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).
Apheresis
A procedure in which blood is taken from a patient, part of the blood (such as white blood cells) is taken out, and the rest of the blood is returned to the patient.
Autoimmune diseases
Illnesses that occur when the body tissues are attacked by its own immune system.
Autologous
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 stem cell transplant (ASCT)
Procedure in which stem cells collected from a patient are transplanted back into that patient; the most common type of transplant performed in myeloma.
Autologous transplant
Transplant whereby the patient’s own cells or tissues are collected and reinfused or transplanted.
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B cell
White blood cell that gives rise to a plasma cell (plasma cells produce antibodies, which fight infections).
Beta 2-microglobulin (ß2-microglobulin or ß2-M)
Protein normally found on the surface of various cells in the body; levels of ß2M in the blood are elevated in inflammatory conditions and in certain blood cell disorders, such as multiple myeloma.
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.
Bone marrow
Soft, spongy tissue found in the center of many bones and the site of blood cell production.
Bone marrow aspiration
Removal of fluid and cells of the bone marrow via use of a needle.
Bone marrow biopsy
Removal of a sample of bone marrow for examination; performed using a needle.
Bone remodeling
Process of normal bone maintenance whereby old, worn-out bone is broken down and removed by osteoclast cells and new bone is formed by osteoblast cells.
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.
Calcium
Mineral that is important in bone formation; elevated serum levels occur when there is bone destruction.
Cancer vaccine
A cell-based or protein-based immunotherapy in which cancer cells (such as myeloma cells) are mixed with immune-stimulating agents or engineered and injected into a patient to boost the immune response.
Catheter
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.
CD34+
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+.
Checkpoint inhibitor
A naked antibody that interferes with proteins that enable a cancer cell to hide from, overpower, or resist the immune system; by blocking these proteins, the “brakes” on the immune system are released and immune cells are able to kill cancer cells.
Chemobrain
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.
Chemotherapy
The use of drugs to kill rapidly dividing cells, such as cancer cells.
Chimeric antigen receptor T (CAR T) cell therapy
A form of immunotherapy in which a patient’s immune cells (mostly T cells) are collected, engineered in a lab to be better able to identify and attack myeloma cells, and then returned to the patient; examples are Abecma and Carvykti.
Chromosomal abnormality
Defect or variation in a chromosome; in some people with multiple myeloma, a piece of one or more chromosomes may be missing or swapped with another piece from a different chromosome; deletion p13 and t(4;14) are examples of chromosomal abnormalities.
Chromosomal translocation
Chromosomal abnormality in which segments of two chromosomes switch positions; t(4;14) and t(11;14) are examples of chromosomal translocations.
Chromosome
Thread-like structure in a living cell that contains DNA (genetic information).
Clinical trial
A study of the safety and effectiveness of a therapeutic agent using consenting human subjects.
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.
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).
Conditioning regimen
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.
Consolidation Therapy
Anti-myeloma treatment given after the initial therapy in order to further reduce the number of cancer cells.
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.
Corticosteroid
Potent class of drugs that has anti-inflammatory, immunosuppressive and antitumor effects. Dexamethasone and prednisone are examples of corticosteroids.
Creatinine
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.
Cryoglobulinemia
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.
Cryopreservation
A method of freezing cells that permits storage over an extended period.
Cure
Treatment outcome where there is complete and lasting recovery from disease. This has not yet been achieved in myeloma.
Cyclosporine Immunosuppressive drug used following allogeneic transplants that helps prevent graft-versus-host disease (donor cells attacking the recipient’s cells).
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).
Demographic
A particular group within a population.
Dendritic cell
A type of immune cell that is found in tissues (such as the skin) and that boosts immune responses by showing parts of proteins on its surface to other cells of the immune system.
Dexamethasone
A steroid used in the treatment of multiple myeloma, often in combination with another anti-myeloma drug. Decadron® is one brand of dexamethasone.
Dialysis
Procedure in which blood is removed from a patient, purified, and returned to the patient; commonly used in patients with kidney disease, this procedure essentially replaces the function of the damaged or diseased kidney.
DMSO
Dimethyl sulfoxide, a colorless chemical used for cryopreservation of stem cells. When introduced into the body, may cause unpleasant or even serious toxic effects.
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 was used for many years in traditional chemotherapy regimens, including VAD (vincristine, Adriamycin, and dexamethasone) in myeloma.
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Electrolytes 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.
Electrophoresis (EP)
Laboratory test used to measure the levels of proteins in the blood or urine; uses an electrical current to sort proteins by their charge.
Emend® (aprepitant)
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).
Emerging therapy
New treatment that is being investigated in clinical trials.
Engraftment
When stem cells infused into the body start to grow and make new blood cells.
Enteritis
Stomach pain or cramps caused by an inflammation of the small intestine.
Erythropoiesis
The production of red blood cells by the bone marrow.
Erythropoietin
Growth factor that stimulates the bone marrow to produce red blood cells.
Esophagitis
Heartburn caused by an irritated esophagus (tube leading from the mouth to the stomach).
Event-free survival (EFS)
Term used in clinical trials to indicate how long a patient remains free of certain negative events, such as recurrence, progression, complications or death.
Expanded access
Refers to any of the procedures initiated by the Food and Drug Administration (i.e., compassionate use, parallel track and treatment IND) that distributes experimental drugs to patients who are failing on currently available treatments for their condition and are unable to participate in(...)
Extramedullary disease
Myeloma cells found in other organs of the body beyond the bone marrow.
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Farydak® (Panobinostat)
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 Velcade and an immunomodulatory agent. This(...)
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(...)
Febrile neutropenia
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).
Filgrastim
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) or Neupogen™.
Fluorescence in situ hybridization (FISH)
Laboratory technique used to measure the number of copies of a specific DNA segment in a cell and the structure of chromosomes.
Fluoroscope
Type of X-ray that allows viewing of bone.
Free light chain (FLC)
Short protein (immunoglobulin light chain) that is produced by myeloma cells and found in the blood.
Frontline therapy
Initial treatment given to a newly diagnosed patient (also known as induction therapy, first-line therapy, or frontline treatment).
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Grade 1 Adverse Event (AE)
A mild side effect resulting from a medical therapy. Minimal or no symptoms may be present and typically medical intervention is not required.
Grade 2 AE
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.
Grade 3 AE
A severe side effect resulting from a medical therapy. This side effect typically interferes with patient functioning and requires medical intervention.
Grade 4 AE
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.
Graft-versus-myeloma effect
Beneficial effect of allogeneic transplants resulting from the donor cells mounting an attack on the recipient’s myeloma cells.
Granulocyte
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(...)
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(...)
Growth factors
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).
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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(...)
Heavily pre-treated
Refers to patients who have recently had several prior therapies for their disease.
Heavy chain
The longer of the two protein chains that make up an immunoglobulin molecule.
Hematocrit
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.
Hematopoiesis
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 other blood cells.
Hemoglobin
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.
Heparanase
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.
Heparin
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.
Hepatic impairment
Reduced liver function that is noted by increased levels of various substances in the blood, such as bilirubin.
High-dose chemotherapy
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. After high-dose chemotherapy, a procedure called stem(...)
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 are also associated with worse prognosis.
Hypercalcemia
Condition noted by elevated levels of calcium in the blood due to increased bone destruction.
Hyperviscosity syndrome
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.
Hypothyroidism
Condition where the thyroid gland is less active than normal, resulting in symptoms such as a low metabolic rate and lack of energy.
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Immune response
Reaction occurring within the body to defend against foreign invaders.
Immune system
Network of related cells, tissues and organs that protect the body from disease organisms, other foreign bodies and cancers.
Immunoglobulin
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,(...)
Immunosuppressive drug
Drug given to suppress a patient’s immune system, such as one given to prevent rejection of transplanted tissue.
Immunotherapy
The treatment of, or prevention against, a disease achieved through manipulation of the patient’s immune system.
Induction therapy
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.
Infusion Reaction
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(...)
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.
Interferon (IFN)
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-cells.
Interleukin 6 (IL-6)
Cytokine that promotes the growth and survival of myeloma cells and normal B cells.
Interleukin 8 (IL-8)
Cytokine secreted by bone marrow stromal cells in myeloma patients that promotes formation of osteoclasts (bone degrading cells).
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Karyotyping
A test that looks at the number and structure of a patient's chromosomes to identify genetic problems.
Kepivance® (palifermin)
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.
Keratopathy
Changes to a part of the eye that result in changes in the sharpness of vision.
Kyphoplasty
Procedure used to treat spinal compression fractures; in this procedure a balloon is inserted into the area of compression and inflated to elevate the collapsed section; the resulting space is then filled with bone cement, which strengthens the area.
Kyprolis™
A new type of proteasome inhibitor. It is in the same class of drugs as Velcade.
Kytril® (granisetron)
A type of medicine used to prevent or treat nausea and vomiting as a result of cancer treatment. It is taken orally.
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Lactate dehydrogenase (LDH)
Enzyme found in body tissues; elevated levels in the blood indicate tissue damage and may occur in myeloma.
Light chain
The shorter of the two protein chains that make up an antibody, characterized as either kappa or lambda type; light chains produced by myeloma cells are also referred to as Bence-Jones proteins when they occur in the urine.
Liposome
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.
Longitudinal study
Repeated observations over a long time with a large number of patients.
Lymphocyte
A type of immune cell made up of two main types, B cells and T cells.
Lymphoma
Blood cancer that develops in the lymph nodes.
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Magnetic resonance imaging (MRI)
Imaging technique that uses magnetic energy to provide detailed images of bone and soft tissue.
Maintenance therapy
Treatment that is given to patients in remission over a long period of time, in order to reduce the risk of relapse.
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.
Melphalan
Type of chemotherapy often used in the treatment of myeloma (also known as Alkeran®).
Metastasis
The spread of tumor cells from the initial site to other areas of the body.
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.
Mini-allogeneic transplant
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.
Minimal residual disease (MRD)
The amount of myeloma cells remaining after a course of treatment, as measured by very sensitive testing; can be as low as one myeloma cell in 1 million normal cells.
Minimal response
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.
Mobilization
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.
Monoclonal antibody
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 (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. M protein levels are used to determine the effectiveness of myeloma treatments.
Monocyte/Macrophage
Type of white blood cell important in the immune response.
MP
A therapy consisting of 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.
Mucositis
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).
Myeloablation
The killing of bone marrow by radiation or chemotherapy. This term usually refers to the complete or near-complete destruction of the bone marrow.
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.
Neuropathic Pain
Chronic pain caused by damage to, or dysfunction of, nerves.
Neuropathy
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.
Neutrophil
Type of white blood cell that plays an important role in the immune system (e.g., can destroy 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 disease indication.
Nonsecretory myeloma
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 painkillers. There are both over-the-counter NSAIDs as well as prescription NSAIDs. Examples of over-the-counter NSAIDs include aspirin, Motrin® (ibuprofen) and Aleve® (naproxen)
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.
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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.
Orphan Drug
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.
Osteolytic lesion
Soft spot in the bone where bone tissue has been destroyed. The lesion appears as a hole on a standard bone X-ray.
Osteonecrosis
Death or destruction of bone tissue due to trauma, loss of blood supply or disease.
Osteopenia
A condition where bones are thinner than normal, but the bone loss is not severe enough to be classified as osteoporosis.
Osteoporosis
Generalized bone loss typically associated with old age.
Overall response rate (ORR)
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.
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Paget’s Disease
A bone disease that is not related to cancer. It is a chronic condition that results in enlarged and deformed bones.
Palifermin
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(...)
Palliative
Meant to reduce symptoms and relieve pain rather than to alter the course of disease.
Pamidronate (Aredia®)
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.
Partial response (PR)
Treatment outcome where there is a greater than 50% decrease in M protein and disappearance of some (but not all) signs and symptoms of cancer; also referred to as partial remission.
PCROWD study
A clinical trial conducted to identify changes in the cells of patients with myeloma precursor conditions (MGUS or SMM) (visit www.enroll.pcrowd.org).
Performance status
A measure of a patient’s ability to perform everyday functions and self-care.
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.
Peripheral edema
Abnormally large amount of fluid in the circulatory system or in tissues.
Peripheral neuropathy
Disorder of the nerves that can disrupt sensation or cause burning/tingling in the hands and feet.
Personalized medicine
Tailoring or individualizing treatment based on an individual’s biological make up.
Phase 1/2 study
A clinical study that combines a phase 1 and a phase 2 trial of the same treatment. First, the phase 1 part of the trial determines a safe dose, then more patients are treated at this dosage in the phase 2 part of the study to further evaluate safety and efficacy.
Phase 1 study
The first round of a clinical trial, conducted with a small number of participants to assess a drug’s safety and non-toxic dosage levels.
Phase 2 study
The second stage of a clinical trial, conducted with a larger number of participants to assess a drug’s effectiveness and further evaluate its safety.
Phase 3 study
The most advanced stage of drug development, conducted with a large number of participants to confirm a drug’s effectiveness, identify and monitor its side effects, compare it to commonly used treatments, and collect information that will allow the drug to be used safely; usually required for(...)
Phase 4 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 4 studies may involve a larger patient population than that studied during earlier clinical trials;(...)
Placebo
Drug or treatment that is designed to look like the medicine being tested but that does not have the active ingredient; rarely used in cancer treatment trials.
Plasma cell
Antibody-secreting immune cell that develops from a B cell; in myeloma, it is this type of cell that has become cancerous or abnormal.
Plasmablastic morphology
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.
Plasmacytoma
Tumor made up of cancerous plasma cells that occurs in bone or soft tissue; patients with a plasmacytoma may develop multiple myeloma.
Plasmapheresis
Method of removing blood plasma from the body by withdrawing blood, separating it into plasma and cells, and transfusing the cells back into the bloodstream; it is often performed when treating autoimmune conditions and may be used in myeloma.
Plateau phase
When the outcome of therapy, be it a response or stable disease, has leveled off and disease parameters remain at a stable level.
Platelets
Small cell fragments in the blood that help to clot.
Pomalyst (pomalidomide)
A type of immunomodulatory drug. It is in the same class of drugs as Revlimid and Thalomid.
Precision medicine
Highly specialized approach to myeloma therapy in which DNA test results are used to guide treatment.
Preclinical studies
Experiments conducted in the laboratory and in animals to identify a target for therapy and to confirm its anti-cancer activity.
Precursor cell
Earlier form of a cell; for example, B cells are precursors of plasma cells.
Prednisone
Type of steroid used to treat myeloma. It is frequently given in combination with a chemotherapy drug called melphalan.
Priority Review
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(...)
Prognosis
Prediction of the course and outcome of a disease.
Prognostic indicator
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.
PROMISE study
A clinical trial conducted to identify new ways to prevent multiple myeloma in individuals with its precursor conditions (MGUS or SMM) (visit www.enroll.promisestudy.org).
Prophylactic
Preventing the spread or occurrence of infection or disease.
Proteasome inhibitors
Drugs that slow myeloma cell growth and kill myeloma cells by interfering with processes that play a role in cell function; examples include Velcade, Ninlaro, and Kyprolis.
Protocol
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.
Pulmonary embolism
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.
Radiopharmaceutical
A radioactive drug used for diagnostic or therapeutic purposes.
Randomization
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 (e.g., the control group or the investigational group).
RANK L (also known as RANK Ligand)
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. RANK L is blocked by Xgeva, a monoclonal antibody therapy approved in myeloma, that reduces skeletal related events.
Revlimid® (lenalidomide)
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 an older myeloma drug called Thalomid® (thalidomide).
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 and size.
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 currently 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(...)
Stable disease (SD)
Treatment outcome where the disease has not responded to therapy 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 Revised International Staging System (R-ISS). Patients with Stage I disease have: ß2-M less than or equal to 3.5mg/L and albumin greater than or equal to 3.5 g/dL, Absence of higher-risk DNA [deletion(17p) and/or translocation t(4;14) and/or(...)
Stage III disease
Myeloma disease classification based on the Revised International Staging System (R-ISS) Patients with Stage III disease have: ß2-M greater than or equal to 5.5mg/L and either , Presence of higher-risk DNA abnormalities [deletion(17p) and/or translocation t(4;14) and/or translocation(...)
Standard-dose chemotherapy
Administration of chemotherapy at a dose that does not completely destroy the bone marrow; also known asAdministration of chemotherapy at a dose that does not completely destroy the bone marrow; also known as conventional chemotherapy.conventional chemotherapy.
Standard of care
Treatment that experts agree is appropriate, accepted and widely used; also called standard therapy or best practice.
Standard therapy
Treatment that has been shown to be safe and effective in clinical studies and is adopted as standard practice outside of clinical trials.
Stem cell
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.
Stevens-Johnson syndrome
A rare, but very serious type of rash, resulting from an allergic reaction. It has been rarely documented in patients who received Revlimid. 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.
Stromal cell
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.
Supportive care
Treatment that addresses the symptoms and complications of a disease rather than the disease itself. Examples in myeloma include bisphosphonates, growth factors (i.e., erythropoietin), antibiotics, orthopedic interventions and pain control measures.
Synergistic
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.
t
T cell
Type of lymphocyte (immune cell) that plays an important role in immune responses and targeted cell killing, also known as a T lymphocyte.
T lymphocyte
Type of lymphocyte (immune cell) that plays an important role in immune responses and targeted cell killing, also known as a T lymphocyte.
Tandem transplant
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.
Thalomid (thalidomide)
Older 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 Revlimid®.
Thrombocytopenia
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 progression (TTP)
A measure of time after a disease is treated until the disease starts to get worse or progress.
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.
Translocation
An abnormal change in the DNA where two segments of different chromosomes switch positions.
Treatment cycles
The period in which treatment is given (e.g., 21 days or 28 days). It is usually followed by a rest period in between treatments.
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.
Tumor microenvironment
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 several effects in myeloma, including promotion of the growth of myeloma cells and activation of osteoclasts.
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Urinalysis
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 size.
v
VAD
An older type of chemotherapy treatment. It consists of the combination of two chemotherapy drugs (Vincristine, Adriamycin) plus dexamethasone, a steroid.
Velcade® (also known as bortezomib)
A highly effective myeloma drug, known as a proteasome inhibitor. It is usually given subcutaneously either alone or in combination with other myeloma drugs.
Vertebroplasty
Procedure used to treat fractures of the spine.
Vincristine
A type of chemotherapy which is given intravenously (into a vein). It is part of an older type of myeloma treatment. See VAD.
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White blood cell (WBC)
One of the major cell types in the blood; attacks infection and cancer cells as part of the immune system.
z
Zofran® (ondansetron)
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).
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