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MPN PROGRESSion Registry™ Glossary
Acute Myeloid Leukemia (AML) is a fast-growing blood cancer in which the bone marrow produces large numbers of immature white blood cells called blasts. These abnormal cells crowd out healthy blood cells, leading to symptoms such as fatigue, infections, easy bruising, and bleeding. AML typically develops on its own but can also arise from earlier blood conditions like myelodysplastic syndromes or myeloproliferative neoplasms (MPNs). (See the definition for secondary Acute Myeloid Leukemia)
Biomarkers are biological molecules found in blood, other body fluids, or tissues that are a sign of a normal or abnormal process, or of a condition or disease. A biomarker may be a gene, protein, or other molecule that is detected and used to see how well the body responds to a treatment for a disease or condition. Also called a molecular marker or signature molecule.
Biomarker Testing is a laboratory method that uses a sample of tissue, blood, or other body fluid to check for the presence or changes in certain genes, proteins, or other molecules that may be a sign of a disease or condition, such as cancer. It may also be used to help plan treatment, find out how well treatment is working, make a prognosis, or predict whether cancer will come back or spread to other parts of the body. Also referred to as molecular profiling or molecular testing.
Bone Marrow Transplant (BMT) is a type of hematopoietic stem cell transplant (HSCT) in which healthy stem cells are collected directly from the donor’s bone marrow, usually from the hip bone, under anesthesia. These cells are then used to replace damaged or diseased marrow and restore normal blood cell production.
Data Linkage is the process of securely combining a person’s health information from different sources, such as electronic medical records, laboratory results, and health surveys. Linking these different types of data helps researchers build a more complete and accurate picture of a person’s health and how their condition changes over time.
Electronic Health Record (EHR) is a digital record of a patient’s medical information that allows authorized healthcare providers across different care settings to access and share information. EHRs include data such as diagnoses, medications, laboratory and imaging results, immunizations, treatment plans, and visit summaries from multiple clinicians involved in a patient’s care. EHRs provide healthcare providers with a comprehensive view of a patient’s health history, which improves the coordination of care.
Electronic Medical Record (EMR) is a digital record of a patient’s medical information over time. They are used by individual doctors’ offices, clinics, and hospitals to support care. These records contain medical information such as diagnoses, medications, test results, vital signs, treatment plans, imaging reports, and visit notes, as well as administrative details like demographics and immunization history. EMRs help healthcare providers within one practice or institution track a patient’s condition, reduce medical errors, and improve care at that location.
Endpoint in clinical trials is an event or outcome used to measure whether a treatment is safe and effective. The endpoints of a clinical trial are usually included in the study objectives. Some examples of endpoints are survival, improvements in quality of life, relief of symptoms, and disappearance of the tumor.
Essential Thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by an increased number of platelets in the blood. ET may be driven by mutations in genes such as JAK2, CALR, or MPL. ET is associated with the overproduction of platelet precursors in the bone marrow and complications frequently include blood clots and/or bleeding.
Fast Healthcare Interoperability Resources (FHIR) is a modern technology standard that allows different health systems and apps to securely share medical information in a common, consistent format. FHIR makes it easier for electronic medical records (EMRs) to exchange information, even if they store or organize patient data differently. For the MPN PROGRESSion Registry, FHIR can help pull together key data, like test results, treatments, or symptoms, from many different clinics or hospitals, so researchers can get a clearer, more complete picture of how MPNs behave and respond to care in the real world.
Hematopoietic Stem Cell Transplant (HSCT) is a procedure that replaces diseased or damaged bone marrow with healthy blood-forming stem cells. These cells can come from the bone marrow, bloodstream, or umbilical cord blood. All types aim to restore normal blood cell production, but they differ in how the stem cells are collected. HSCT may be considered for people with advanced myeloproliferative neoplasms (MPNs), such as myelofibrosis or MPN in blast phase. Although it offers the possibility of long-term remission or even cure, HSCT also carries serious risks, and not all patients are eligible for this treatment.
Honest Broker is a person who helps protect your privacy when participating in a research project. Their job is to keep your personal information (like your name or date of birth) separate from the health data that researchers use. In the MPN PROGRESSion Registry, the Honest Broker makes sure your identity stays private even when your information comes from different places like medical records and surveys. Only data that cannot identify you is shared with researchers. This process follows strict privacy rules and is approved by an ethics committee called the IRB (Institutional Review Board).
Informed Consent is the process by which a person voluntarily agrees to take part in a research study after receiving clear, complete, and understandable information about what participation involves. This includes learning about the purpose of the study, what data will be collected, any potential risks or benefits, and the person’s right to say no or to withdraw at any time. Informed consent ensures that participants fully understand how their medical data will be used and that their choice to share it is entirely voluntary.
Institutional Review Board (IRB) is an independent committee of experts that reviews and oversees medical research involving people to ensure it is ethical and protects participants’ rights and safety. Before and throughout a research study, the IRB reviews the research plan, including how participants will be informed and treated. All U.S.-based research involving patients, including registries like the MPN PROGRESSion Registry, must be approved by an IRB to ensure participants are treated fairly, respectfully, and with full transparency. IRBs oversee both interventional and non-interventional studies, including patient registries like the MPN PROGRESSion Registry.
Legally Authorized Representative (LAR) is a person who is legally allowed to give consent for someone else to join a research study when that person cannot make the decision for themselves. This may happen if an adult has a medical condition that affects their ability to understand or communicate clearly. Depending on state law, an LAR might be a spouse, adult child, parent, legal guardian, or another individual authorized by the courts or by regulations. LARs are only used when a participant is unable to make an informed decision and must always act in the best interest of that person.
Myelodysplastic Syndrome (MDS) is a group of blood cancers characterized by poorly functioning bone marrow stem cells that do not produce enough fully developed or healthy red blood cells, white blood cells, or platelets. This leads to symptoms like fatigue, frequent infections, and easy bruising or bleeding. Although not classified as an MPN, MDS can sometimes overlap with MPNs and may evolve into acute myeloid leukemia (AML). The condition is often diagnosed through a combination of blood tests, bone marrow biopsies, and genetic analysis to identify clonal mutations or chromosomal abnormalities.
Myelofibrosis (MF) is a myeloproliferative neoplasm (MPN) where abnormal blood-making stem cells in the bone marrow cause scarring (fibrosis) and ongoing inflammation. This disrupts normal blood cell production and may cause the spleen or other organs to take over blood formation, often leading to spleen enlargement.
Myeloproliferative Neoplasms (MPNs) are a group of rare, chronic blood cancers that begin in the bone marrow, where blood cells are made. The three classic types of MPNs are essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). In these MPNs, genetic mutations in blood-forming stem cells cause the body to produce too many mature red cells or platelets. This can lead to symptoms and complications like fatigue, itchiness, anemia, bleeding or clotting problems, or an enlarged spleen.
Myeloproliferative Neoplasm in Accelerated Phase (MPN-AP) is when an MPN has reached an advanced stage where immature blood cells, called blasts, make up 10% to 19% of the cells in the bone marrow or circulating blood. This phase signals disease progression and may include worsening symptoms such as anemia, low platelet counts, or increased spleen size. Patients with MPN-AP are at high risk of progressing to blast phase, and clinicians may suggest more intensive treatments or stem cell transplants.
Myeloproliferative Neoplasm in Blast Phase (MPN-BP) is the most advanced and aggressive stage of an MPN, defined by the presence of 20% or more blasts (immature blood cells) in the bone marrow or circulating blood. It is considered a type of acute myeloid leukemia and is typically associated with severe symptoms, low blood counts, and a poor prognosis. Treatment options may include chemotherapy, hypomethylating agents, targeted therapies, or stem cell transplant, depending on the patient’s health and genetic profile.
Myeloproliferative Neoplasm, Unclassifiable (MPN-U) or sometimes called myeloproliferative neoplasm, not otherwise specified (MPN-NOS) is a diagnosis given when a person clearly shows signs of a myeloproliferative neoplasm but doesn’t meet the full criteria for any specific MPN like essential thrombocythemia, polycythemia vera, or myelofibrosis. This may happen when the disease is in an early stage, when it has progressed to a more advanced or fibrotic form, or when another illness makes diagnosis more difficult. MPN-U can vary widely in how it appears and changes over time, and treatment is often tailored to the patient’s symptoms and clinical needs.
Overall Survival (OS) is the length of time from either the date of disease diagnosis or the start of treatment that a patient is still alive. In a clinical trial, measuring OS is one way to see how well a new treatment works.
Patient Reported Outcome (PRO) is information about a patient’s health that comes directly from the patient. Examples of PROs include a patient’s description of their symptoms, their satisfaction with care, and how a disease or treatment affects their physical, mental, emotional, and social well-being. In clinical trials, PROs may provide information about the side effects of the new treatment being studied. The use of patient-reported outcomes may help plan the best treatment and improve quality of care.
Peripheral Blood Stem Cell Transplant (PBSCT) is a type of hematopoietic stem cell transplant (HSCT) in which healthy stem cells are collected from the donor’s bloodstream after medication is given to move stem cells out of the bone marrow and into the blood. The cells are then separated through a process similar to donating blood.
Polycythemia Vera (PV) is a chronic, progressive myeloproliferative neoplasm (MPN) characterized by high amounts of red blood cells. PV may be driven by mutations in genes such as JAK2, CALR, or MPL. The disease is most common in men over the age of 60, but anyone can develop PV. PV patients typically experience an elevated leukocyte (white blood cell) count, an elevated platelet count, and an enlarged spleen.
Pre-fibrotic Primary Myelofibrosis (pre-pMF) is an early stage of the rare, chronic blood cancer primary myelofibrosis (pMF), classified as a myeloproliferative neoplasm (MPN). It is marked by the abnormal growth and development of platelet-producing cells (called megakaryocytes) in the bone marrow. These changes may be driven by mutations in genes such as JAK2, CALR, or MPL. Unlike overt pMF, pre-pMF has little to no marrow scarring (fibrosis) at diagnosis, though fibrosis can develop over time. Some people with pre-pMF report fatigue, night sweats, or spleen enlargement, and are at an increased risk of blood clots.
Primary Myelofibrosis (pMF) is a rare, chronic myeloproliferative neoplasm (MPN) characterized by the overproduction of blood-making cells in the bone marrow. It is often related to mutations in genes such as JAK2, CALR, or MPL. Over time, the marrow becomes scarred (fibrotic), leading to anemia, an enlarged spleen, and other symptoms that affect quality of life. Unlike secondary myelofibrosis, which occurs after other MPNs such as essential thrombocythemia or polycythemia vera, pMF arises on its own (de novo). Although its origin differs, the clinical presentation and treatment approach to primary myelofibrosis is generally like that of secondary myelofibrosis (sMF).
Principal Investigator (PI) is the lead researcher responsible for the overall design, conduct, and oversight of a clinical study or research project. Their responsibilities include ensuring ethical standards, regulatory compliance, data integrity, and participant safety throughout the course of a study.
Progression (Disease Progression) refers to the worsening or advancement of a disease over time, often marked by new symptoms, changes in laboratory results, or the spread of disease to other parts of the body. In myeloproliferative neoplasms (MPNs), progression can include a shift from one phase or disease state to another — such as the development of overt myelofibrosis or acute myeloid leukemia. Signs of progression in MPNs include changes in or increased symptoms like anemia, bone fibrosis, enlarged spleen, or blood clotting or bleeding problems.
Progression-Free Survival (PFS) is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease, but it does not get worse. In a clinical trial, measuring progression-free survival is one way to see how well a new treatment works.
Real World Evidence (RWE) is information obtained from doctor’s offices or clinics about how diseases progress and treatments perform, based on data gathered during routine patient care. RWE can reveal how patients respond to treatments over time, how symptoms progress, and how care is delivered within or outside of clinical trials.
Secondary Myelofibrosis (sMF) is a progressive form of myelofibrosis that develops from other myeloproliferative neoplasms (MPNs), specifically essential thrombocythemia (post-ET MF) or polycythemia vera (post-PV MF). Like primary myelofibrosis, sMF is marked by bone marrow scarring, anemia, spleen enlargement, and symptoms such as fatigue, night sweats, and weight loss. Although its origin differs, the clinical presentation and treatment approach is a lot like those of primary myelofibrosis.
Secondary Acute Myeloid Leukemia (sAML) is when AML develops in people who already have another blood disorder, such as myeloproliferative neoplasm (MPN) or myelodysplastic syndrome. Secondary AML can be harder to treat than other types of AML and have a poorer prognosis. In people with MPNs, secondary AML typically occurs after the disease has worsened, progressing through more advanced stages such as blast phase. (See the definition for Acute Myeloid Leukemia and Myeloproliferative Neoplasm in Blast Phase)
Stem Cells are biological cells found in all multicellular organisms, that can divide and differentiate into diverse specialized cell types and can self-renew to produce more stem cells. In adult organisms, stem cells and progenitor cells act as a repair system for the body, replenishing adult tissues.
Stem Cell Transplant (SCT) is a medical procedure that replaces the cells of diseased or damaged bone marrow with healthy blood-forming stem cells, usually from a donor. It may be considered for people with advanced myeloproliferative neoplasms (MPNs), such as myelofibrosis or MPN in blast phase. Although it offers the possibility of long-term remission, it also carries serious risks, and not all patients are eligible for this treatment.
Umbilical Cord Blood Transplant (CBT) is a type of of hematopoietic stem cell transplant (HSCT) that uses stem cells collected from the umbilical cord blood of a newborn, immediately after birth. These cells are stored in a cord blood bank and can later be used for transplantation. Cord blood transplants are often an option when a fully matched donor is not available.
United States Core Data for Interoperability (USCDI) is a list of important health information like medications, allergies, lab results, and immunizations that all electronic medical record systems are required to include. By using the same set of information across different hospitals and clinics, USCDI helps make it easier and safer to share medical records. For the MPN PROGRESSion Registry, the use of USCDI means researchers can collect consistent, complete data to better understand MPNs and improve care.
World Health Organization (WHO) is an agency within the United Nations, composed of 194 member countries, dedicated to coordinating the world’s response to health emergencies while promoting well-being, disease prevention, and access to health care. The WHO also establishes disease classification systems, including for hematologic malignancies such as MPNs.
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