TERMINOLOGY:
| Name | Description |
|---|---|
| Adjuvant therapy | Treatment used in addition to main definitive treatment to reduce risks of cancer recurrence. It usually refers to hormone therapy, chemo, radiation therapy, or other treatments given after surgery to increase the chances of cure. |
| Alopecia | Alopecia means hair loss. This may be an adverse effect to some, not all cancer treatments. |
| Anemia | Result of having too few red blood cells or a low red blood cell count. This is commonly measured as hemoglobin. |
| Antibody | Antibody also called immunoglobulin is somewhat Y shaped protein produced in the body which recognize a specific molecular target (called epitope) on germs and some unwanted cells. This immune recognition is a component of immune response against those targets. |
| Anti-emetic | A drug or medication that prevents or relieves nausea and vomiting. |
| Area under the curve (AUC) | Area under the curve (also called area under the concentration-time curve) reflects the total drug exposure to the body across time. It is dependant on the bioavailability, rate of elimination and dose of the drug administered. Mathematical formulas may be used to estimate AUC when several variables noted above are known. |
| Benign | Non-cancerous. A condition that is not malignant. A benign growth is a non-cancerous tumor which does not spread. |
| Blood count | Refers to number or amount of blood cells. This is commonly measured as CBC or complete blood count which includes red blood cells, white blood cells and platelets. |
| Bone marrow | A tissue present inside certain bones where blood cells are produced. |
| Cancer | A general term to indicate abnormal growth of cells many times resulting in a lump or a mass which has the ability to invade and spread locally and to other parts of the body. Some cancers such as blood or bone marrow cancers may not result in measurable lump or mass and may manifest as increased cells in the blood and/or in the bone marrow. |
| CAR-T therapy | Please see immunotherapy for details. |
| Cellular therapy | Please see immunotherapy for details. |
| Chemotherapy | Chemotherapy in general are the drugs (mostly cytotoxic) used to treat cancer. It is often abbreviated as chemo. Oxford dictionary defined chemotherapy as "The treatment of disease by the use of chemical substances, especially the treatment of cancer by cytotoxic and other drugs". |
| Chromosomes | Thread-like strands that carry genetic information. They are found in the nucleus, or center part, of a cell. Humans have 23 pairs of chromosomes, one member of each pair from the mother, the other from the father. Each chromosome can contain hundreds or thousands of individual genes. |
| Clinical trials | Medical research studies done in patient volunteers. Each study is designed to answer scientific questions and find better ways to detect, prevent, or treat cancer and/or its side effects. |
| Combination chemotherapy | The use of more than one chemo drug to treat cancer |
| Complementary and alternative medicine | Ways of dealing with disease other than those used by doctors in standard medicine. This term covers a broad range of tested and untested methods, such as herbs/vitamins/minerals, mind/body/spirit, diet and nutrition, physical touch, and biological methods. |
| Confidence Interval | Confidence Interval (CI) is used to measure the precision of statistical estimate. CI is a range of values that is likely to include the true value. Hence, if the confidence interval includes 1, then the hazard ratio is not significant. A commonly used CI is a 95% confidence interval which has a probability of containing the population mean 95% of the times. |
| Contralateral | On the opposite side |
| Cytogenetic Response | In chronic myeloid leukemia, response to treatment is assessed using several criteria with varying sensitivity to assess response. Cytogenetic response is to assess response based on percentage of Philadelphia chromosome positive (Ph+) cells (with conventional cytogenetics) or BCR-ABL1-positive cells (on FISH analysis). Various cytogenetic responses are: no response (>95 percent), minimal (66 - 95 percent), minor (36 - 65 percent), major (1 - 35 percent), and complete (0 %). |
| Disease-Free Survival | Please see "relapse-free survival" |
| Disease specific survival (DSS) | Disease specific survival is the percentage of people who have not died from the specified disease in a defined period of time. |
| Event-free survival | Event-free survival (EFS) is the length of survival after definitive cancer treatment to disease progression, death, or discontinuation of treatment for any reason or initiation of a new treatment without documented progression. EFS is not commonly used and similar to progression free survival. |
| Fatigue | The feeling of being tired physically, mentally, and/or emotionally. |
| Growth factors | Growth factors (also known as colony-stimulating factors) are substances that stimulate the production of blood cells in the bone marrow. They may help prevent or recover from low blood counts as a result of chemotherapy and radiation therapy. |
| Hazard Ratio (HR) | Hazard ratio (HR), a type of relative risk, a measure of an effect of an intervention on an outcome/event over time. Hazard represents the probability of an event such as relapse after the intervention. Many survival analysis are reported as Hazard ratio. HR is the ratio of chance of an event occurring (hazard rate) in the treatment group ÷ chance of an event occurring (hazard rate) in the control group. Hence, HR of 1 indicates that the event rates are same in both treatment and control groups. HR less than one indicates less patients in the treatment group are experiencing an event compared to the control group. A hazard ratio of 0.70 may mean that the study drug provides 30% risk reduction compared to the control treatment. Similarly, 0.50 may indicate 50% reduction. HR greater than 1 indicates more patients in the treatment experienced an event compared to control arm. |
| Hormonal Therapy | Use of medications which work by increasing or decreasing hormonal functions. Several hormones make some cancer cells multiply and grow. In cancer treatment, hormonal therapy usually imply methods to reduce hormones or their function. |
| Hormone | A hormone is a chemical or a molecule which are produced in endocrine glands. These are released into blood and influence or control functions of other body organs and tissues. |
| Hormones | Natural substances released by an organ that can influence the function of other organs in the body and the growth of some types of cancer |
| Immunotherapy | Immunotherapy or immune therapy is a category of treatment where drugs or cellular therapy is used to harness, augment, enable, modulate or add additional capabilities to body’s natural immune functions to achieve a desired therapeutic effect. Cellular therapy is the use of externally stimulated, multiplied, augmented and/or genetically modified cells (mostly immune cells) rather than chemicals or drugs to achieve therapeutic objectives. CAR-T cell therapy where T-cells (immune cells) are genetically modified to express a special receptor called a chimeric antigen receptor (CAR) is a type of cellular therapy. |
| Infusion | Slow and/or prolonged intravenous (IV) delivery of a drug or fluids |
| Injection | Using a syringe and needle to push fluids or drugs into the body; often called a shot |
| Intramuscular | Inside a muscle. Usually refers to a medication administered into a muscle. |
| Intrathecal | Into the spinal fluid (also called cerebrospinal fluid or CSF) |
| Intravenous (IV) | Inside a vein. Usually refers to a medication administered inside a blood vessel (vein). |
| in vitro | A process (usually a biological process) occurring or happening outside of a living organism, such as in test tubes or tissue cultures etc. |
| in vivo | A process (usually a biological process) occurring or happening in a living organism. |
| Ipsilateral | On the same side |
| Major Response | Commonly indicate responses that include complete response (CR) as well as partial responses (PR). Some cancer response assessments include responses such as pathologic complete response (pCR), unconfirmed CR, very good partial responses (VGPR) etc. which may be included under 'major response'. Stable disease (SD) is usually not included. |
| Malignant | In cancer medicine or oncology, malignant or cancerous indicate unwanted excessive multiplication of cells which have the ability to invade and spread. Cancer is the result of malignant cells which multiply uncontrollably. |
| Median not reached (PFS, statistics) | Median is reached when 50% of patients have experienced an event. Median not reached means 50% of patients have not experienced a defined event in that group. |
| Metastasis | The spread of cancer or malignant cells to other areas of the body, often through the lymph system or bloodstream |
| Molecular Response | In chronic myeloid leukemia, response to treatment is assessed using several criteria with varying sensitivity to assess response. Molecular response (MR) is used to assess response based on level of detection of BCR-ABL1 transcripts in sensitive quantitative PCR test. BCR-ABL1 transcripts are commonly reported using International Scale (IS) (ratio of BCR-ABL1 transcripts to ABL1 transcripts) on a log scale. Early Molecular Response (EMR): BCR-ABL1 transcripts (IS) ≤ 10% at 3 and 6 months. Major Molecular Response (MMR): BCR-ABL1 transcripts (IS) ≤ 0.1% or ≥ 3 log reduction in BCR-ABL1 mRNA if IS is not available. Complete Molecular Response (EMR): Depending on the level of detection and sensitivity of PCR assay, various types of complete molecular responses are defined. Below noted are from UpToDate (accessed: July 16, 2019): "* MR2 – Detectable disease at a level of ≤1 percent on the IS (≥2 log reduction from the standardized baseline). This level of response roughly corresponds to a 'complete cytogenetic response.' * MR3 – Detectable disease at a level of ≤0.1 percent on the IS (≥3 log reduction from the standardized baseline). This level of response has been termed a 'major molecular response.' * MR4 – Either detectable disease at a level of ≤0.01 percent on the IS (≥4 log reduction) or undetectable disease in cDNA with ≥10,000 ABL1 transcripts. This level of response requires that the assay being used is sensitive enough to detect a single abnormal transcript amongst 10,000 normal ABL1 transcripts. * MR4.5 – Either detectable disease at a level of ≤0.0032 percent on the IS (≥4.4 log reduction) or undetectable disease in cDNA with ≥32,000 ABL1 transcripts. This level of response requires that the assay being used is sensitive enough to detect a single abnormal transcript amongst 32,000 normal ABL1 transcripts." |
| Monoclonal antibodies | Monoclonal antibodies are antibodies which are produced by the same clone of cells that targets specific molecular target (called epitope). Monoclonal antibodies can be engineered and designed against a specific molecular target and commercially produced for treatment of diseases including cancers. These fall under targeted therapy category along with other types of targeted therapy. |
| Neoadjuvant therapy | Treatment, such as chemotherapy, hormone therapy, or radiation therapy, given before the main treatment is done |
| Objective Response rate | Is the response to a treatment as measured by the tumor measurements or other measurable parameters. |
| Odds Ratio | Odds ratio (OR) is the ratio of odds of an outcome in the presence and in the absence of intervention. Odds of an outcome is the ratio of the probability that the outcome happening / probability of the outcome not happening. OR indicates strength of association (not necessarily causation) between outcome and intervention. Odds ratio (OR) of 1 indicate no effect on the odds of outcome, >1 indicates higher odds and <1 indicates lower odds of the outcome with the intervention. |
| Oncologist | A doctor or a physician with special training in the diagnosis and treatment of cancer |
| Orally (PO) | Taken by mouth |
| Peripheral neuropathy | Damage to the nerves that usually starts in the hands and/or feet. Symptoms of neuropathy include numbness, tingling, burning, and/or weakness etc. There are several causes of neuropathy including treatment side effects. |
| Placebo | Placebo is a substance or treatment that is considered safe with no therapeutic effect. placebo effect is the benefit attributable to the psychological or idea of taking a treatment and not due to real therapeutic effect of the treatment. Placebo is sometimes used as a control while testing new drugs which may help assess and control for placebo effect and make data more meaningful. |
| Platelets | A type of blood cells that usually serve as the first responders when there is a damage to the lining of blood vessels. They help blood clot and stop bleeding. |
| Platinum sensitivity | Platinum sensitivity is helpful in the management of tumors sensitive to platinim compounds. The following terms are commonly used in the management of ovarian, fallopian tube or peritoneal cancer. 1. Platinum-sensitive (platinum-free interval of six or more months) 2. Platinum-resistant (platinum-free interval of less than six months) 3. Platinum-refractory (disease progression while on platinum based therapy). |
| Port | Also called a mediport is a devise that is inserted usually under the skin to connect to central veins (a type of central venous catheter or CVC). The port can be accessed through the skin with a special needle and provides easy and reliable access to central veins to facilitate administration of medications safely. It can also be used to draw blood, administer fluids or blood products. |
| Progression free survival (PFS) | The length of time during and after the treatment for a disease, such as cancer, that a person lives without disease getting any worse than before (without progression). Progression-free survival is one way to assess effect of new treatment on the disease condition. PFS is defined as the time until objective tumor progression or death. |
| p-value | p-value is a statistical probability value. p-value indicates the probability of differences between two groups to be a random and/or unrelated. Smaller the value, less likely the difference to be a random occurance. Generally P < 0.05 (< 5% chance of being wrong) is considered minimal threshold to consider statistically significant and P < 0.001 as statistically highly significant. In statistics, null hypothesis is a default starting hypothesis which states there is no difference between the groups to be compared and any difference is due to some form of error. To show a meaningful difference between the groups, statistical tests/ analysis must prove that null hypotheses is worng. p-value indicates the chances that null hypothesis is true. p-value of <0.05 indicates less than 5% chances that null hypothesis is true (less than 5% chances that there is no difference between the groups). |
| Radiation therapy | The use of high-energy rays or subatomic particles to treat disease. |
| Radiographic Progression Free Survival (rPFS) | Usually indicate time from randomization to evidence of radiographic disease progression or death. |
| Red blood cells | Red blood cells or RBCs are a type of blood cells which are commonly measured as hemoglobin. These cells carry oxygen from the lungs and deliver to tissues throughout the body. Anemia is a condition of low red blood cells. |
| Relapse-free survival | Relapse-free survival also known as disease-Free Survival is the length of survival after definitive cancer treatment without any evidence of cancer relapse (or recurrence). |
| Relative risk (RR) | Relative risk (RR), also called Risk Ratio is the ratio of probability of an outcome in one group divided by the probability of the same event in the control group. This is not same as Hazard ratio (HR). However, this is used in a similar sense. Choice of HR or RR as a measure is decided by statistitians depending on the contexts of the research data. RR of 1 indicate no difference between the groups. RR of less than one indicate lesser probability of the outcome than the control group. Similarly RR of greater than one indicate greater probability than the control group. |
| Remission | The partial or complete disappearance of signs and symptoms of disease |
| Residual cancer burden (RCB) | Residual cancer burden is a measure of pathologic response to assess effectiveness of pre-surgical therapy. Factors used in calculating RCB include pathologic size and cellularity of primary tumor as well as number and size of lymph node metastases. Depending on the amount of residual disease, RCB is categorized 0 (no residual disease or pathologic complete response) or RCB I, II or III (extensive residual disease). |
| Stomatitis | Sores on the lining of the mouth |
| Surgery | Surgery is a medical treatment which involves use of anesthesia and cutting open body parts. In cancer treatment surgery is done to remove cancerous growth with an effort when possible to remove all the known cancer growths with good negative margins. |
| Targeted therapy | Targeted therapy is a broad category which indicate use of drugs that work by targeting specific molecular targets. These molecular targets could be inside or outside of the cells including non cellular molecules of the body. |
| Time to Progression (TTP) | Time to progression (TTP) is defined as the time until objective tumor progression. TTP does not include deaths. TTP does not count patients who die from other causes but is otherwise a close equivalent to PFS |
| Topical | Usually refers to a medication which is applied on the surface of the skin. |
| Tumor | An abnormal growth (lump or mass) of cells or tissues. Tumors are either benign (not cancer) or malignant (cancer). |
| White blood cells (WBCs) | The blood cells that fight infection |
ABBREVIATIONS:
| Name | Description |
|---|---|
| 5FU | 5-fluorouracil or fluorouracil is an injectable chemotherapy agent. |
| ADT | Androgen Deprivation Therapy |
| ALK | Anaplastic Lymphoma Kinase |
| ALL | Acute Lymphoblastic Leukemia |
| AML | Acute Myeloid Leukemia disease |
| AR | Androgen Receptor |
| ASCT | Autologous Stem Cell Transplant |
| AUC | Area Under the concentration-time Curve (pharmacokinetics) |
| AUC | Area Under the Curve (area under the plasma drug concentration time curve) represent total drug exposure across time. AUC is also used to indicate target drug concentration (Ex: carboplatin) to be achieved in the body which can be calculated. |
| BSC | Best Supportive Care. |
| CBR | Clinical Benefit Rate. Used similar to DCR (disease control rate) to indicate responses to treatment including complete response AND partial response AND stable disease. |
| CCyR | Complete Cytogenetic Response. Used in CML to indicate reduction of Philadelphia chromosome positive (Ph+) cells to 0 percent. |
| CHF | Congestive Heart Failure |
| CHOP | Abbreviation for chemotherapy regimen: Cyclophosphamide+Adriamycin+Vincristine+Prednisone Drg regimen |
| CHR | Complete Hematologic Response. Used to assess response in hematologic malignancies. In CML, used to indicate normalization of blood counts and no abnormal immature cells seen in blood. |
| CI | Confidence interval (to measure the precision of statistical estimate) |
| CLL | Chronic Lymphocytic Leukemia |
| CML | Chronic Myeloid Leukemia disease |
| CNS | Central Nervous System |
| CPS | Combined Positive Score, a PD-L1 scoring method. |
| CR | Complete Response |
| CR1 | 1st Complete Response |
| CR2 | Complete Response after treatment for relapse |
| CRC | Colorectal cancer disease |
| CRh | Complete response with partial hematologic recovery (definitions may vary) |
| CRi | Complete Remission with incomplete marrow recovery (definitions may vary) |
| CRPC | Castration Resistant Prostate Cancer |
| CRR | Complete Response Rate |
| CRu | CR (complete response) unconfirmed |
| Cmax | Maximum (or peak) serum concentration of a drug after the drug has been administered. |
| Ctrough | Trough (or lowest) serum concentration of a drug before the next dose is administered |
| DCR | Disease Control Rate. Used similar to CBR (clinical Benefit rate) to indicate responses to treatment including complete response AND partial response AND stable disease. |
| DFS | Disease Free Survival (Survival without any evidence of cancer recurrence) |
| DFS | Disease free survival (also known as relapse-free survival) |
| DLBCL | Diffuse Large B Cell lymphoma disease |
| DoR | Duration of Response |
| DSS | Disease specific survival |
| EFS | Event Free Survival |
| EGFR | Epidermal Growth Factor Receptor |
| ER | Estrogen Receptor |
| FACT score | Functional Assessment of Cancer Therapy. These are questionnaire used to assess patient reported quality of life in patients undergoing cancer therapy. FACT-G ((FACT-General) can be used to assess patients of any tumor type. There are several different sets of questionatres designed to assess quality of life for specific cancers, specific treatment etc. FACT questionnaires generally use 5 point patient scale ranging from 0 (Not at all) to 4 (Very much). |
| FGFR | Fibroblast Growth Factor Receptor |
| FGFR1 | Fibroblast Growth Factor Receptor 1 |
| FIGO | International Federation of Gynecology and Obstetrics |
| FIGO staging | FIGO (International Federation of Gynecology and Obstetrics) cancer staging is commonly used in gynecological malignancies. |
| FL | Follicular Lymphoma |
| FOLFIRI | Abbreviation used to indicate chemotherapy regimen incorporating 5FU with leucovorin (folinic acid) with irinotecan. FOL (folinic acid or leucovorin)+F (fluorouracil or 5-FU)+ IRI (irinotecan). |
| FOLFOX | Abbreviation used to indicate chemotherapy regimen incorporating 5FU with leucovorin (folinic acid) with oxaliplatin. FOL (folinic acid or leucovorin)+F (fluorouracil or 5-FU)+ OX (oxaliplatin). |
| G-CSF | granulocyte-colony stimulating factor |
| HER-2 | Human Epidermal growth factor Receptor-2 |
| HPF | High Power Field (usually 40x magnification in microscope) |
| HR | Hazard Ratio (Statistical method) |
| HR | Hormone Receptor |
| HRD | Homologous Recombination Deficiency. Homologous recombination is a process involved in repair of double-stranded DNA breaks and during cell division. Multiple gene mutations including BRCA1 and BRCA2 etc. may result in HRD. |
| iDFS | Invasive Disease Free Survival. Similar to DFS. Useful when the cancer included invasive and non-invasive types (Ex. Breast cancer). |
| IGVH | IGVH of IgVH. Immunoglobulin heavy-chain variable region. |
| IHC | Immuno-Histo-Chemistry. Immunohistochemistry is a method of staining pathoogy specimen for detection of molecular markers based on antibody binding. |
| IM | Intramuscular injection |
| INI1 | INI1 (INtegrase Interactor 1) also known as SMARCB1 (SWI/SNF related matrix‐associated actin dependent regulator of chromatin subfamily B member 1) is a part of an essential chromatin remodeling complex involved in multiple pathways that affect DNA structure and function. Gene encoding for INI1 is considered a tumor supressor gene. |
| IQR | InterQuartile Range (statistics) |
| IRC | Independent Review Committee |
| IV | Intravenous (route of administration of a drug) |
| KIT | KIT (also called CD117, Stem Cell Factor Receptor) is a surface protein receptor tyrosine kinase which mediate actions of stem cell factor (SCF). |
| KRAS | KRAS is a growth promoting protein enzyme (GTPase) that belongs to Ras superfamily of proteins. Name originated from: Kirsten RAt Sarcoma virus. |
| LRC | Local regional control (locoregional control) |
| LVEF | Left Ventricular Ejection Fraction |
| MaHR | Major Hematologic Response. Used in CML to indicate normalization of blood counts. |
| MCyR | Major Cytogenetic Response. Used in CML to indicate reduction of Philadelphia chromosome positive (Ph+) cells to 1 to 35 percent. |
| MDS | Myelodysplastic syndromes disease |
| MFS | Metastasis Free Survival. A duration from
treatment or randomization to first evidence of metastasis or death. |
| MMR | Mismatch Repair (gene) |
| MMR | Major Molecular Response (see "Molecular Response" under Terminology link) |
| MPN | Myeloproliferative Neoplasms. Also refered to as myeloproliferative diseases (MPDs) are a group of bone marrow malignant disorders including chronic myelogenous leukemia, polycythemia vera, essential thrombocythemia and other conditions. disease |
| MRD | Minimal Residual Disease |
| mRECIST | mRECIST is modified RECIST developed for the assessment of hepatocellular carcinoma. |
| MRI | Magnetic Resonance Imaging |
| MSI | Microsatellite Instability |
| muts/Mb | Mutations per megabase (of DNA) |
| MZL | Marginal Zone Lymphoma disease |
| NCCN | National Comprehensive Cancer Network |
| NED | No Evidence of Diseae |
| NHL | Non-Hodgkin's Lymphoma |
| NI | Non-inferiority or Non-inferior. It is generally used to indicate that the treatment studied is statistically NOT inferior to comparator treatment. |
| NI | Not-inferior/ Non - inferiority |
| NOS | Not Otherwise Specified |
| NS | Statistically NOT significant |
| NSAI | Non-steroidal aromatase inhibitors (Ex: letrozole or anastrozole)
|
| NSCLC | Non Small Cell Lung Cancer disease |
| ORR | Objective response rate |
| OS | Overall Survival |
| pCR | Pathologic Complete Response |
| PD1 | Programmed cell death protein 1 (surface protein receptor) involved in immune response. |
| PDGFRA | Platelet-derived growth factor receptor alpha (PDGFRA or PDGFRα) is a cell surface receptor which binds to and mediate actions of platelet-derived growth factors (PDGFs). |
| PD-L1 | Programmed death ligand-1, a protein that binds to PD1 receptor. |
| PET | Positron Emission Tomography (scan) |
| PFS | Progression Free Survival |
| Ph+ | Philadelphia chromosome positive. Philadelphia chromosome is abnormal chromosome 22 in which part of chromosome 9 (containing ABL1 gene) is transferred to chromosome 22 (containing BCR gene) resulting in fusion gene called BCR-ABL1. |
| PI | Probability interval |
| PR (PRR) | Partial response (Partial Response Rate) |
| PSA | Prostate-specific antigen |
| PSMA | Prostate Specific Membrane Antigen (a transmembrane protein that
is expressed in prostate cancer) |
| p-value | Statistical probability value (see more info under "Terms and Meaning" link |
| q | Every |
| q14d | Every 14 days |
| q2wk | Every 2 weeks |
| q3wk | Every 3 weeks |
| q.d | Every day |
| q.o.d | Every other day |
| QOL | Quality of Life |
| q.wk / qw | Once per week |
| R0 (resection) | R0 refers to complete surgical resection or removal of the tumor without clinical, gross or microscopic evidence of disease/tumor. |
| R1 (resection) | R1 refers to surgical resection with microscopic residual disease/tumor. |
| R2 (resection) | R2 refers to surgical resection with macroscopic or obvious residual disease/tumor. |
| RAI | Radioactive Iodine. Used in the treatment of some thyroid cancers. |
| RBC | Red Blood Cell |
| RCB | Residual Cancer Burden (a measure of residual cancer at surgery after pre-surgical treatment) |
| RCT /RCTs | Randomized Controlled Trial / Randomized Controlled Trials |
| RECIST | Response Evaluation Criteria In Solid Tumors (RECIST) is a set of criteria to assess response of treatment developed and published in 2000. |
| RECIST 1.1 | Is the revised RECIST guielines updated in 2009 to assess response |
| RET gene | Rearranged during Transfection (RET) gene |
| RFS | Relapse (recurrence) Free Survival (also known as disease-free survival) |
| rPFS | Radiographic Progression Free Survival |
| RR | Relative Risk (Statistical method) |
| RS | May indicate Recurrence Score (RS) based on on Oncotype Dx test result. |
| SCLC | Small Cell Lung Cancer disease |
| sCR | Stringent Complete Response. In multiple myeloma: "In addition to complete response criteria, these patients have a normal free light chain (FLC) ratio and have no clonal cells by bone marrow immunohistochemistry or immunofluorescence. The latter is achieved if there is a kappa/lambda ratio of ≤4:1 or ≥1:2 after examination of a minimum of 100 plasma cells."(Reference: UpToDate). |
| SD | Stable Disease. The disease that has remained stable without progression. |
| SQ | Subcutaneous injection |
| TKI | Tyrosine Kinase Inhibitor (TKI). TKIs are drugs that work by inhibiting various tyrosine kinase enzymes. |
| TTF | Time-to-treatment failure (TTF) is variably defined. It may be used to indicate an interval from initiation of treatment to its premature discontinuation. The treatment may be defined as first or subsequent treatment. Discontinuation could be due to several reasons including toxicity or death. |
| TTP | Time to progression. May also be used to indicate a disease condition thrombotic thrombocytopenic purpura.
|
| TTR | Time to response |
| VEGF | Vascular Endothelial Growth Factor |
| VGPR | Very Good Partial Response. Commonly used to assess response in multiple myeloma. |
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TUMOR RESPONSE ASSESSMENT AND CLINICAL ENDPOINTS IN CANCER RESEARCH
Contents:
Introduction
Effectiveness of cancer treatment is usually established based on outcomes assessed in clinical research. The benefits of cancer treatment are assessed using one or more measurable parameters (clinical endpoints).
A clinical endpoint is an event or an outcome that can be measured objectively to determine if a treatment or any intervention is beneficial. The intended purpose of a treatment may vary and may include shrinkage of the tumor, improvement in survival, decrease the probability of cancer progression, quality of life, improvements in symptom, cure from cancer etc. One or more such benefits may be assessed in a research trial. Since the anticipated benefits of a treatment may vary, clinical endpoints used are not always the same for different clinical studies.
Each aspect of benefit may be assessed by one or more specific clinical endpoints. Hence, every clinical trial includes one or more clinical endpoints to measure the benefits. Research scientists identify and decide on the specific clinical endpoints to be used to assess depending on the intended benefit of the treatment, type of cancer, previously available data, etc.
Even though each clinical endpoint used in a study provides a measurable parameter, this should be viewed in a narrow sense as specified in the definition of that parameter. Benefits identified based on one clinical endpoint may not mean a benefit in another related endpoint. For example, treatment may improve survival without causing shrinkage of tumor. Similarly, a treatment that reduces the tumor size may not improve overall survival. Some treatments may improve the symptoms without acting on the cancer. FDA has published a guidance for the industry on clinical trial endpoints for the approval of cancer treatments1.
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Types and Phases of Clinical Research:
Clinical trials provide us with measurable evidence if a treatment or an intervention is beneficial. Study may also provide additional information such as side effects and safety information. Clinical trials may be performed to identify a new treatment, a new preventive method, methods to improve quality of life etc. Each study has to be designed specifically with specific sets of endpoints depending on the purpose of the study.
Generally, pre-clinical (laboratory) studies are done to identify a potential new medicine. Once a promising molecule or method is identified, clinical studies are done to demonstrate benefits in humans. These clinical trials are done in a stepwise fashion with different purposes at each phase. Each early phase provides key complimentary information such as toxicity data, preliminary evidence of a benefit, tolerance etc. which are needed prior to proceeding with more advanced and expensive later phases of research. Clinical trials phases generally are grouped as Phases 0 to 4. Phases 0 and 1 are done in a small number of individuals to answer key questions such as safety and the most appropriate dose. Phase II studies may provide evidence of a treatment benefit. Phase III studies compare new treatment against the current treatment. Phase IV studies are done on the treatments that are already in clinical use and assess their safety over time.
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Comparative Vs. Noncomparative Studies:
Comparative studies are designed to compare endpoints between 2 or more interventions. An example of a comparative study is a controlled study, where one of the comparative arms includes a control group which could be a current standard treatment or a placebo (controlled study). Comparisons may also be between different doses, regimens etc.
While controlled studies provide a higher level of evidence, it is not always possible, feasible or required. These trial designs may be used when use of placebo is not ethical or when natural history of the condition is well established. These types of studies are commonly done in oncology field. Single arm trial is an example of a Noncomparative study where participants are given the experimental therapy and then followed over time to assess response.
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Randomized Vs. Non-randomized Studies:
Randomized Vs. Non-randomized refers to assigning patients or participants to different arms of the study in a comparative study. In a Non-randomized trial people are allocated to different interventions using methods that are not random. Randomized studies randomly assign participants in a balanced manner to different treatment or groups. This reduces several biases such as selection bias and allocation bias.
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Open Vs. Blinded trial:
An open trial is where information about the treatment and group allocation is open to both the researchers and the participants in a comparative study. Blinding refers to withholding certain information from the individuals involved in the study that may somehow influence them in various ways and may create potential bias.
A study could be blinded study where various aspects of the treatment are hidden from the participant, researcher, or individual measuring the response during the study and will be revealed at the conclusion of the study. A study could be a single blind, double blind, or a triple blind study. Blinding is commonly employed in Randomized Controlled Trials (RCTs) which are historically considered the gold standard in clinical evidence.
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Assessing Tumor Response:
Clinical endpoints includes tumor response assessments (size, amount and activity of tumor) as well as various types of survival and adverse effects data. Tumor response to cancer treatment may be assessed using several different parameters such as anatomic size, biochemical parameters, quantifying tumor cells in the blood or bone marrow etc depending on the caner type being studied. Several different endpoints may be used in combination.
Anatomic Tumor Response:For solid tumors the size of which can be measured, imaging modalities such as CT scan, MRI scans etc are used to measure size of the tumor before, during and after treatment to assess measurable response. When there at=re multiple cancer lesions, a limited number of target lesions may be selected for measurements. To ensure uniformity, various standards for measurements such as RECIST (Response Evaluation Criteria in Solid Tumors), modified RECIST, WHO criteria etc are established. These criteria may be periodically updated to improve accuracy of interpretation (Ex: RECIST version 1.02, RECIST version 1.13).
Biochemical and molecular Response:For some cancer which are associated with measurable tumor markers or other biochemical abnormalities, response can be assessed based on improvement in those parameters. Prostate cancer response can be assessed based on improvements in PSA (prostate specific antigen) level. Several blood/bone marrow malignancies are assessed based on abnormal proteins produced (Ex. 'M' protein, free light chains, immunoglobulins in myeloma and some types of lymphoma) or measurements of abnormal clones in blood or bone marrow (Ex: molecular response assessed using BCR-ABL transcripts in Chronic Myeloid Leukemia)
Survival Data:In some situations, objective evidence of tumor shrinkage to a treatment may not translate into a survival improvement. This may happen if adverse effects of the treatment may cause or contribute for the reduced survival. Hence, while objective response (tumor shrinkage) is important, overall survival is considered the most important endpoint in cancer clinical trials. However, estimating overall survial may be impractical in several situations where a slow growing cancer is associated with a long survival or if several other treatments are available which may be used after the study which may affect overall survival. Hence, all the clinical endpoints have their limitations. When a clinical endpoint is difficult to measure, a surrogate endpoints may be used. A list of adult surrogate endpoint table is available in FDA website (Accessed: November 6, 2022)4.
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| Clinical Endpoint | Description* |
|---|---|
| Overall Survival (OS) | The length of time from either the date of diagnosis or the start of treatment to death from any cause. |
| Progression-free Survival (PFS) | The length of time during and after the treatment for a disease, such as cancer, that a person lives without disease getting any worse than before (without progression). PFS is defined as the time until objective tumor progression or death. |
| Radiographic Progression-free Survival (rPFS) | Time from randomization to evidence of radiographic disease progression or death. |
| Relapse free Survival (RFS) or Ddisease-Free Survival (DFS) | Length of survival after definitive cancer treatment without any evidence of cancer relapse (or recurrence). |
| Event-free Survival (EFS) | Length of survival after definitive cancer treatment to disease progression, death, or discontinuation of treatment for any reason or initiation of a new treatment without documented progression. EFS is not commonly used and similar to progression free survival. |
| Time to Progression (TTP) | Length of time until objective tumor progression. TTP does not include deaths. TTP does not count patients who die from other causes but is otherwise a close equivalent to PFS. |
| Objective Response Rate (ORR) | Percentage of patients who had measurable response (as specified in the study) to the treatment. Responses could be complete (Complete Response) or partial (Partial Response). Stable disease (SD) includes patients whose disease remains stable without a response or progression. The term ‘Major Response” commonly indicate responses that include complete response (CR) as well as partial responses (PR). Some cancer response assessments include specified responses such as pathologic complete response (pCR), very good partial responses (VGPR), cytogenetic response, molecular response etc. |
| Duration of Response (DoR)) | Length of time from randomization to disease progression or death in patients who achieve response (complete or partial). |
* The definitions of clinical end points may slightly vary from one study to another.
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References:
1. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/clinical-trial-endpoints-approval-cancer-drugs-and-biologics (Accessed: November 7, 2022)
2. Therasse P, Arbuck SG, Eisenhauer EA, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst 2000; 92:205.
3. Eisenhauer E, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer 2009; 45:228.
4. https://www.fda.gov/drugs/development-resources/table-surrogate-endpoints-were-basis-drug-approval-or-licensure (Accessed: November 6, 2022)
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