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m
- 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 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. M protein levels are used to determine the effectiveness of myeloma treatments.
- Monocyte/Macrophage
Type of white blood cell important in the immune response.
- Morphology
Overall appearance.
- 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.
- Myelodysplastic syndrome (MDS)
A group of diseases that affect the blood. It is not related to multiple myeloma.
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n
- 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.
- Nephrotoxicity
Toxicity to the kidneys.
- 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.
- Neutropenia
Below-normal number of neutrophils.
- 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.
- Newly diagnosed disease
Multiple myeloma that has not yet been treated.
- Non-myeloablative allogenic transplant
See mini-allogeneic transplant
- 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)
- Novel erythrocyte stimulating protein (NESP)
Protein 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.
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o
- 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.
- Osteoblast
Bone-forming cell.
- Osteoclast
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.
- 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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p
- 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.
- Palmar-plantar erythrodysesthesia (PPE)
See hand-foot syndrome (HFS)
- 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.
- Paraprotein
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 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
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.
- 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.
- Plasmablast
Immature plasma cell.
- 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.
- Positron emission tomography (PET)
Imaging technique in which radioactive glucose (sugar) is used to highlight cancer cells.
- 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.
- 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.
- Progressive disease
Active myeloma that is worsening; in most cases, relapsed and/or refractory disease can be considered to be progressive disease.
- 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.
- Pyrexia
Fever.
-
r
- Radiation therapy (or radiotherapy)
Use of high-energy rays to kill myeloma cells; sometimes used to relieve bone pain.
- 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.
- Red blood cell (RBC)
Blood cell that carries oxygen.
- Refractory disease
Myeloma that progresses during therapy.
- Relapsed disease
Myeloma that progresses after initially responding to therapy.
- Renal Impairment
Reduced function of the kidneys.
- Response Rate or Overall Response
The total percentage of patients who respond to a specific therapy in a clinical trial.
- Revised International Staging System (R-ISS)
System for using laboratory test results to determine the severity of multiple myeloma.
- 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).
- RNA (ribonucleic acid)
Genetic material of the cell that codes for proteins.
-
s
- Salvage therapy
Second-line therapy; used to treat disease that has not responded to initial therapy or relapsed disease.
- 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.
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