21st Century Adult Cancer Sourcebook: Chronic Myeloproliferative Disorders (Polycythemia Vera, Myelofibrosis, Thrombocythemia, CML) - Clinical Data for Patients, Families, and Physicians
Edition 1.0 - October 2011
National Cancer Institute
Smashwords Edition
Copyright 2011 Progressive Management
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PART ONE
Chapter 1A: Chronic Myeloproliferative Disorders Patient Information
Chapter 2A: Chronic Myeloproliferative Disorders Health Professional Information
Chapter 3A: Chronic Myeloproliferative Disorders NCI Drugs
Chapter 4A: Leukemia Background Information
Chronic Myeloproliferative Disorders Clinical Trials:
Chapter 5A: Clinical Trials for Chronic Myelogenous Leukemia
Chapter 6A: Clinical Trials for Polycythemia Vera
Chapter 7A: Clinical Trials for Myelofibrosis
Chapter 8A: Clinical Trials for Essential Thrombocythemia
PART TWO
Chapter 1B: Levels of Evidence for Adult and Pediatric Cancer Treatment Studies (NCI)
Chapter 2B: Glossary of Clinical Trial Terms
Chapter 3B: Clinical Trials Background Information
Chapter 4B: Cancer Clinical Trials -The Basic Workbook
Chapter 5B: Cancer Clinical Trials - The In-Depth Program
Chapter 6B: Clinical Trials at NIH
Chapter 7B: How To Find A Cancer Treatment Trial: A Ten Step Guide
Chapter 8B: Taking Part in Cancer Treatment Research Studies
Chapter 9B: Cancer Clinical Trials
Chapter 10B: Access to Investigational Drugs
Chapter 12B: Taking Time: Support for People with Cancer
Chapter 13B: Facing Forward - Life After Cancer Treatment
Chapter 14B: Chemotherapy and You
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PART ONE
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Chapter 1A: Chronic Myeloproliferative Disorders Patient Information
Chronic Myeloproliferative Disorders Treatment
Patient Version
Last Modified: 09/27/2011
General Information About Chronic Myeloproliferative Disorders
Key Points for This Section
* Myeloproliferative disorders are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells, or platelets.
* There are 6 types of chronic myeloproliferative disorders.
* Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myeloproliferative disorders.
Myeloproliferative disorders are a group of diseases in which the bone marrow makes too many red blood cells, white blood cells, or platelets.
Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:
* Red blood cells that carry oxygen and other materials to all tissues of the body.
* White blood cells that fight infection and disease.
* Platelets that help prevent bleeding by causing blood clots to form.
Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.
In myeloproliferative disorders too many blood stem cells develop into one or more types of blood cells. The disorders usually get worse slowly as the number of extra blood cells increases.
There are 6 types of chronic myeloproliferative disorders.
The type of myeloproliferative disorder is based on whether too many red blood cells, white blood cells, or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Chronic myeloproliferative disorders include the following 6 types:
* Chronic myelogenous leukemia.
* Polycythemia vera.
* Primary myelofibrosis (also called chronic idiopathic myelofibrosis).
* Essential thrombocythemia.
* Chronic neutrophilic leukemia.
* Chronic eosinophilic leukemia.
These types are described below. Chronic myeloproliferative disorders sometimes become acute leukemia, in which too many abnormal white blood cells are made.
Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myeloproliferative disorders.
The following tests and procedures may be used:
* Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
* Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following:
* The number of red blood cells and platelets.
* The number and type of white blood cells.
* The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
* The portion of the blood sample made up of red blood cells.
* Complete blood count (CBC). Blood is collected by inserting a needle into a vein and allowing the blood to flow into a tube. The blood sample is sent to the laboratory and the red blood cells, white blood cells, and platelets are counted. The CBC is used to test for, diagnose, and monitor many different conditions.
* Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells.
* Bone marrow aspiration and biopsy. After a small area of skin is numbed, a Jamshidi needle (a long, hollow needle) is inserted into the patient’s hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope.
* Cytogenetic analysis: A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes. Certain diseases or disorders may be diagnosed or ruled out based on the chromosomal changes.
Glossary Terms
abnormal (ab-NOR-mul)
* Not normal. An abnormal lesion or growth may be cancer, premalignant (likely to become cancer), or benign (not cancer).
acute leukemia (uh-KYOOT loo-KEE-mee-uh)
* A rapidly progressing cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of white blood cells to be produced and enter the blood stream.
blood (blud)
* A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.
blood clot (blud klot)
* A mass of blood that forms when blood platelets, proteins, and cells stick together. When a blood clot is attached to the wall of a blood vessel, it is called a thrombus. When it moves through the bloodstream and blocks the flow of blood in another part of the body, it is called an embolus.
bone marrow (bone MAYR-oh)
* The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.
bone marrow aspiration (bone MAYR-oh AS-pih-RAY-shun)
* A procedure in which a small sample of bone marrow is removed, usually from the hip bone, breastbone, or thigh bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special wide needle is pushed into the bone. A sample of liquid bone marrow is removed with a syringe attached to the needle. The bone marrow is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow biopsy.
bone marrow biopsy (bone MAYR-oh BY-op-see)
* A procedure in which a small sample of bone with bone marrow inside it is removed, usually from the hip bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special, wide needle is pushed into the bone and rotated to remove a sample of bone with the bone marrow inside it. The sample is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow aspiration.
breastbone (brest...)
* The long flat bone that forms the center front of the chest wall. The breastbone is attached to the collarbone and the first seven ribs. Also called sternum.
cell (sel)
* The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.
chromosome (KROH-muh-some)
* Part of a cell that contains genetic information. Except for sperm and eggs, all human cells contain 46 chromosomes.
chronic eosinophilic leukemia (KRAH-nik EE-oh-SIH-noh-FIH-lik loo-KEE-mee-uh)
* A disease in which too many eosinophils (a type of white blood cell) are found in the bone marrow, blood, and other tissues. Chronic eosinophilic leukemia may stay the same for many years, or it may progress quickly to acute leukemia.
chronic myelogenous leukemia (KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A slowly progressing disease in which too many white blood cells (not lymphocytes) are made in the bone marrow. Also called chronic granulocytic leukemia, chronic myeloid leukemia, and CML.
chronic neutrophilic leukemia (KRAH-nik NOO-troh-FIH-lik loo-KEE-mee-uh)
* A disease in which too many neutrophils (a type of white blood cell) are found in the blood. The extra neutrophils may cause the spleen and liver to become enlarged. Chronic neutrophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia.
complete blood count (kum-PLEET blud kownt)
* A test to check the number of red blood cells, white blood cells, and platelets in a sample of blood. Also called blood cell count and CBC.
cytogenetics (SY-toh-jeh-NEH-tix)
* The study of chromosomes and chromosomal abnormalities.
diagnosis (DY-ug-NOH-sis)
* The process of identifying a disease, such as cancer, from its signs and symptoms.
essential thrombocythemia (eh-SENT-shul THROM-boh-sy-THEE-mee-uh)
* An increased number of thrombocytes (platelets) in the blood, without a known cause. Also called essential thrombocytosis.
hemoglobin (HEE-moh-GLOH-bin)
* The substance inside red blood cells that binds to oxygen in the lungs and carries it to the tissues.
infection (in-FEK-shun)
* Invasion and multiplication of germs in the body. Infections can occur in any part of the body and can spread throughout the body. The germs may be bacteria, viruses, yeast, or fungi. They can cause a fever and other problems, depending on where the infection occurs. When the body’s natural defense system is strong, it can often fight the germs and prevent infection. Some cancer treatments can weaken the natural defense system.
lymphoid (LIM-foyd)
* Referring to lymphocytes, a type of white blood cell. Also refers to tissue in which lymphocytes develop.
myeloid (MY-eh-loyd)
* Having to do with or resembling the bone marrow. May also refer to certain types of hematopoietic (blood-forming) cells found in the bone marrow. Sometimes used as a synonym for myelogenous; for example, acute myeloid leukemia and acute myelogenous leukemia are the same disease.
myeloproliferative disorder (MY-eh-loh-proh-LIH-feh-ruh-tiv dis-OR-der)
* A group of slow growing blood cancers, including chronic myelogenous leukemia, in which large numbers of abnormal red blood cells, white blood cells, or platelets grow and spread in the bone marrow and the peripheral blood.
oxygen (OK-sih-jen)
* A colorless, odorless gas. It is needed for animal and plant life. Oxygen that is breathed in enters the blood from the lungs and travels to the tissues.
pathologist (puh-THAH-loh-jist)
* A doctor who identifies diseases by studying cells and tissues under a microscope.
physical examination (FIH-zih-kul eg-ZA-mih-NAY-shun)
* An exam of the body to check for general signs of disease.
platelet (PLATE-let)
* A tiny piece of a cell found in the blood that breaks off from a large cell found in the bone marrow. Platelets help wounds heal and prevent bleeding by forming blood clots. Also called thrombocyte.
polycythemia vera (PAH-lee-sy-THEE-mee-uh VAYR-uh)
* A disease in which there are too many red blood cells in the bone marrow and blood, causing the blood to thicken. The number of white blood cells and platelets may also increase. The extra blood cells may collect in the spleen and cause it to become enlarged. They may also cause bleeding problems and make clots form in blood vessels.
primary myelofibrosis (PRY-mayr-ee MY-eh-loh-fy-BROH-sis)
* A progressive, chronic disease in which the bone marrow is replaced by fibrous tissue and blood is made in organs such as the liver and the spleen, instead of in the bone marrow. This disease is marked by an enlarged spleen and progressive anemia. Also called agnogenic myeloid metaplasia, chronic idiopathic myelofibrosis, idiopathic myelofibrosis, and myelosclerosis with myeloid metaplasia.
protein (PROH-teen)
* A molecule made up of amino acids that are needed for the body to function properly. Proteins are the basis of body structures such as skin and hair and of substances such as enzymes, cytokines, and antibodies.
red blood cell (red blud sel)
* A cell that carries oxygen to all parts of the body. Also called erythrocyte and RBC.
stem cell (stem sel)
* A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.
tissue (TIH-shoo)
* A group or layer of cells that work together to perform a specific function.
white blood cell (hwite blud sel)
* A type of immune cell. Most white blood cells are made in the bone marrow and are found in the blood and lymph tissue. White blood cells help the body fight infections and other diseases. Granulocytes, monocytes, and lymphocytes are white blood cells. Also called leukocyte and WBC.
Chronic Myelogenous Leukemia
Chronic myelogenous leukemia is a disease in which too many white blood cells are made in the bone marrow.
Polycythemia Vera
Key Points for This Section
* Polycythemia vera is a disease in which too many red blood cells are made in the bone marrow.
* Possible signs of polycythemia vera include headaches and a feeling of fullness below the ribs on the left side.
* Special blood tests are used to diagnose polycythemia vera.
Polycythemia vera is a disease in which too many red blood cells are made in the bone marrow.
In polycythemia vera, the blood becomes thickened with too many red blood cells. The number of white blood cells and platelets may also increase. These extra blood cells may collect in the spleen and cause it to swell. The increased number of red blood cells or platelets in the blood can cause bleeding problems and make clots form in blood vessels. This can increase the risk of stroke or heart attack. In patients who are older than 65 years or who have a history of blood clots, the risk of stroke or heart attack is higher. Patients also have an increased risk of developing acute myeloid leukemia or primary myelofibrosis.
Possible signs of polycythemia vera include headaches and a feeling of fullness below the ribs on the left side.
Polycythemia vera often does not cause early symptoms. It is sometimes found during a routine blood test. Symptoms may occur as the number of blood cells increases. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:
* A feeling of pressure or fullness below the ribs on the left side.
* Headaches.
* Double vision or seeing dark or blind spots that come and go.
* Itching all over the body, especially after being in warm or hot water.
* Reddened face that looks like a blush or sunburn.
* Weakness.
* Dizziness.
* Weight loss for no known reason.
Special blood tests are used to diagnose polycythemia vera.
In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a serum erythropoietin test is used to diagnose polycythemia vera. In this test, a sample of blood is checked for the level of erythropoietin (a hormone that stimulates new red blood cells to be made).
Primary Myelofibrosis
Key Points for This Section
* Primary myelofibrosis is a disease in which abnormal blood cells and fibers build up inside the bone marrow.
* Possible signs of primary myelofibrosis include pain below the ribs on the left side and feeling very tired.
* A special blood test is used to diagnose primary myelofibrosis.
* Certain factors affect prognosis (chance of recovery) and treatment options for primary myelofibrosis.
Primary myelofibrosis is a disease in which abnormal blood cells and fibers build up inside the bone marrow.
The bone marrow is made of tissues that make blood cells (red blood cells, white blood cells, and platelets) and a web of fibers that support the blood-forming tissues. In primary myelofibrosis (also called chronic idiopathic myelofibrosis), large numbers of blood stem cells develop into blood cells that do not mature properly (blasts). The web of fibers inside the bone marrow also becomes very thick (like scar tissue) and slows the blood-forming tissue’s ability to make blood cells. This causes the blood-forming tissues to make fewer and fewer blood cells. In order to make up for the low number of blood cells made in the bone marrow, the liver and spleen begin to make the blood cells.
Possible signs of primary myelofibrosis include pain below the ribs on the left side and feeling very tired.
Primary myelofibrosis often does not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by primary myelofibrosis or by other conditions. A doctor should be consulted if any of these problems occur:
* Feeling pain or fullness below the ribs on the left side.
* Feeling full sooner than normal when eating.
* Feeling very tired.
* Shortness of breath.
* Easy bruising or bleeding.
* Petechiae (flat, red, pinpoint spots under the skin that are caused by bleeding).
* Fever.
* Night sweats.
* Weight loss.
A special blood test is used to diagnose primary myelofibrosis.
In addition to a complete blood count, bone marrow aspiration and biopsy, and cytogenetic analysis, a peripheral blood smear is used to diagnose primary myelofibrosis. A peripheral blood smear is a procedure in which a sample of blood is checked for tear drop-shaped red blood cells, the number and kinds of white blood cells, the number of platelets, and the presence of blast cells.
Certain factors affect prognosis (chance of recovery) and treatment options for primary myelofibrosis.
Prognosis (chance of recovery) depends on the following:
* The age of the patient.
* The number of abnormal red blood cells and white blood cells.
* The number of blasts in the blood.
* Whether there are certain changes in the chromosomes.
* Whether the patient has symptoms such as fever, night sweats, or weight loss.
Essential Thrombocythemia
Key Points for This Section
* Essential thrombocythemia is a disease in which too many platelets are made in the bone marrow.
* Patients with essential thrombocythemia may have no symptoms.
* Certain factors affect prognosis (chance of recovery) and treatment options for essential thrombocythemia.
Essential thrombocythemia is a disease in which too many platelets are made in the bone marrow.
Essential thrombocythemia causes an abnormal increase in the number of platelets made in the blood and bone marrow.
Patients with essential thrombocythemia may have no symptoms.
Essential thrombocythemia often does not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by essential thrombocytopenia or by other conditions. A doctor should be consulted if any of these problems occur:
* Headache.
* Burning or tingling in the hands or feet.
* Redness and warmth of the hands or feet.
* Vision or hearing problems.
Platelets are sticky. When there are too many platelets, they may clump together and make it hard for the blood to flow. Clots may form in blood vessels and there may also be increased bleeding. These can cause serious health problems such as stroke or heart attack.
Certain factors affect prognosis (chance of recovery) and treatment options for essential thrombocythemia.
Prognosis (chance of recovery) and treatment options depend on the following:
* The age of the patient.
* Whether the patient has symptoms or other problems related to essential thrombocythemia.
Chronic Neutrophilic Leukemia
Chronic neutrophilic leukemia is a disease in which too many blood stem cells develop into a type of white blood cell called neutrophils. Neutrophils are infection -fighting blood cells that surround and destroy dead cells and foreign substances (such as bacteria). The spleen and liver may swell because of the extra neutrophils. Chronic neutrophilic leukemia may stay the same or it may progress quickly to acute leukemia.
Chronic Eosinophilic Leukemia
Key Points for This Section
* Chronic eosinophilic leukemia is a disease in which too many white blood cells (eosinophils) are made in the bone marrow.
* Possible signs of chronic eosinophilic leukemia include fever and feeling very tired.
Chronic eosinophilic leukemia is a disease in which too many white blood cells (eosinophils) are made in the bone marrow.
Eosinophils are white blood cells that react to allergens (substances that cause an allergic response) and help fight infections caused by certain parasites. In chronic eosinophilic leukemia, there are too many eosinophils in the blood, bone marrow, and other tissues. Chronic eosinophilic leukemia may stay the same for many years or it may progress quickly to acute leukemia.
Possible signs of chronic eosinophilic leukemia include fever and feeling very tired.
Chronic eosinophilic leukemia may not cause early symptoms. It is sometimes found during a routine blood test. The following symptoms may be caused by chronic eosinophilic leukemia or by other conditions. A doctor should be consulted if any of these problems occur:
* Fever.
* Feeling very tired.
* Cough.
* Swelling under the skin around the eyes and lips, in the throat, or on the hands and feet.
* Muscle pain.
* Itching.
* Diarrhea.
Stages of Chronic Myeloproliferative Disorders
Key Points for This Section
* There is no standard staging system for chronic myeloproliferative disorders.
* There are three ways that cancer spreads in the body.
There is no standard staging system for chronic myeloproliferative disorders.
Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myeloproliferative disorders. Treatment is based on the type of myeloproliferative disorder the patient has. It is important to know the type in order to plan treatment.
There are three ways that cancer spreads in the body.
When cancer cells spread outside the blood, a solid tumor may form. This process is called metastasis. The three ways that cancer cells spread in the body are:
* Through the blood. Cancer cells travel through the blood, invade solid tissues in the body, such as the brain or heart, and form a solid tumor.
* Through the lymph system. Cancer cells invade the lymph system, travel through the lymph vessels, and form a solid tumor in other parts of the body.
* Through solid tissue. Cancer cells that have formed a solid tumor spread to tissues in the surrounding area.
The new (metastatic) tumor is the same type of cancer as the primary cancer. For example, if leukemia cells spread to the brain, the cancer cells in the brain are actually leukemia cells. The disease is metastatic leukemia, not brain cancer.
Treatment Option Overview
Key Points for This Section
* There are different types of treatment for patients with chronic myeloproliferative disorders.
* Ten types of standard treatment are used:
* Watchful waiting
* Phlebotomy
* Platelet apheresis
* Transfusion therapy
* Chemotherapy
* Radiation therapy
* Other drug therapy
* Surgery
* Biologic therapy
* High-dose chemotherapy with stem cell transplant
* New types of treatment are being tested in clinical trials.
* Targeted therapy
* Patients may want to think about taking part in a clinical trial.
* Patients can enter clinical trials before, during, or after starting their cancer treatment.
* Follow-up tests may be needed.
There are different types of treatment for patients with chronic myeloproliferative disorders.
Different types of treatments are available for patients with chronic myeloproliferative disorders. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.
Ten types of standard treatment are used:
Watchful waiting
Watchful waiting is closely monitoring a patient’s condition without giving any treatment until symptoms appear or change.
Phlebotomy
Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some chronic myeloproliferative disorders.
Platelet apheresis
Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream.
Transfusion therapy
Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment.
Chemotherapy
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated.
Radiation therapy
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. There are two types of radiation therapy. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated.
Radiation therapy to treat myeloproliferative disorders is usually directed at the spleen.
Other drug therapy
Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low- dose aspirin may also be used to reduce the risk of blood clots.
Thalidomide and lenalidomide are drugs that prevent blood vessels from growing into areas of tumor cells.
Surgery
Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged.
Biologic therapy
Biologic therapy is a treatment that uses the patient's immune system to fight cancer or other diseases. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against disease. This type of treatment is also called biotherapy or immunotherapy. Interferon alfa and pegylated interferon alpha are biologic agents commonly used to treat some chronic myeloproliferative disorders.
Erythropoietic growth factors are also biologic agents. They are used to stimulate the bone marrow to make red blood cells.
High-dose chemotherapy with stem cell transplant
High-dose chemotherapy with stem cell transplant is a method of giving high doses of chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
Stem cell transplant (Step 1). Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm.
Stem cell transplant (Step 2). The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown).
Stem cell transplant (Step 3). The patient receives stem cells through a catheter placed into a blood vessel in the chest.
New types of treatment are being tested in clinical trials.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI Web site 3.
Targeted therapy
Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitors are targeted therapy drugs that block signals needed for tumors to grow. JAK2 tyrosine kinase inhibitors are targeted therapy drugs being studied in clinical trials to treat primary myelofibrosis.
Patients may want to think about taking part in a clinical trial.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Patients can enter clinical trials before, during, or after starting their cancer treatment.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Follow-up tests may be needed.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.
Glossary Terms
abdomen (AB-doh-men)
* The area of the body that contains the pancreas, stomach, intestines, liver, gallbladder, and other organs.
anagrelide (an-AG-re-lide)
* A drug that is used to decrease the number of platelets in the blood in order to prevent blood clotting.
apheresis (a-feh-REE-sis)
* A procedure in which blood is collected, part of the blood such as platelets or white blood cells is taken out, and the rest of the blood is returned to the donor. Also called pheresis.
aspirin (AS-pih-rin)
* A drug that reduces pain, fever, inflammation, and blood clotting. Aspirin belongs to the family of drugs called nonsteroidal anti-inflammatory agents. It is also being studied in cancer prevention.
biologic agent (BY-oh-LAH-jik AY-jent)
* A substance that is made from a living organism or its products and is used in the prevention, diagnosis, or treatment of cancer and other diseases. Biologic agents include antibodies, interleukins, and vaccines. Also called biological agent and biological drug.
biological therapy (BY-oh-LAH-jih-kul THAYR-uh-pee)
* Treatment to boost or restore the ability of the immune system to fight cancer, infections, and other diseases. Also used to lessen certain side effects that may be caused by some cancer treatments. Agents used in biological therapy include monoclonal antibodies, growth factors, and vaccines. These agents may also have a direct antitumor effect. Also called biological response modifier therapy, biotherapy, BRM therapy, and immunotherapy.
blood (blud)
* A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.
blood chemistry study (blud KEH-mih-stree STUH-dee)
* A procedure in which a sample of blood is examined to measure the amounts of certain substances made in the body. An abnormal amount of a substance can be a sign of disease in the organ or tissue that produces it.
blood clot (blud klot)
* A mass of blood that forms when blood platelets, proteins, and cells stick together. When a blood clot is attached to the wall of a blood vessel, it is called a thrombus. When it moves through the bloodstream and blocks the flow of blood in another part of the body, it is called an embolus.
blood transfusion (blud tranz-FYOO-zhun)
* A procedure in which a person is given an infusion of whole blood or parts of blood. The blood may be donated by another person, or it may have been taken from the patient earlier and stored until needed. Also called transfusion.
blood vessel (blud VEH-sel)
* A tube through which the blood circulates in the body. Blood vessels include a network of arteries, arterioles, capillaries, venules, and veins.
bone marrow (bone MAYR-oh)
* The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.
cancer (KAN-ser)
* A term for diseases in which abnormal cells divide without control and can invade nearby tissues. Cancer cells can also spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is a cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is a cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is a cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord. Also called malignancy.
catheter (KA-theh-ter)
* A flexible tube used to deliver fluids into or withdraw fluids from the body.
CBC
* A test to check the number of red blood cells, white blood cells, and platelets in a sample of blood. Also called blood cell count and complete blood count.
cell (sel)
* The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.
cerebrospinal fluid (seh-REE-broh-SPY-nul FLOO-id)
* The fluid that flows in and around the hollow spaces of the brain and spinal cord, and between two of the meninges (the thin layers of tissue that cover and protect the brain and spinal cord). Cerebrospinal fluid is made by tissue called the choroid plexus in the ventricles (hollow spaces) in the brain. Also called CSF.
chemotherapy (KEE-moh-THAYR-uh-pee)
* Treatment with drugs that kill cancer cells.
chronic (KRAH-nik)
* A disease or condition that persists or progresses over a long period of time.
clinical trial (KLIH-nih-kul TRY-ul)
* A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.
diagnosis (DY-ug-NOH-sis)
* The process of identifying a disease, such as cancer, from its signs and symptoms.
dose (dose)
* The amount of medicine taken, or radiation given, at one time.
drug (drug)
* Any substance, other than food, that is used to prevent, diagnose, treat or relieve symptoms of a disease or abnormal condition. Also refers to a substance that alters mood or body function, or that can be habit-forming or addictive, especially a narcotic.
erythropoietin (eh-RITH-roh-POY-eh-tin)
* A substance that is naturally produced by the kidneys, and that stimulates the bone marrow to make red blood cells. When erythropoietin is made in the laboratory, it is called epoetin alfa or epoetin beta.
external radiation therapy (...RAY-dee-AY-shun THAYR-uh-pee)
* A type of radiation therapy that uses a machine to aim high-energy rays at the cancer from outside of the body. Also called external-beam radiation therapy.
follow-up (FAH-loh-up)
* Monitoring a person's health over time after treatment. This includes keeping track of the health of people who participate in a clinical study or clinical trial for a period of time, both during the study and after the study ends.
growth factor (grothe FAK-ter)
* A substance made by the body that functions to regulate cell division and cell survival. Some growth factors are also produced in the laboratory and used in biological therapy.
high-dose chemotherapy (hy-dose kee-moh-THAYR-uh-pee)
* An intensive drug treatment to kill cancer cells, but that also destroys the bone marrow and can cause other severe side effects. High-dose chemotherapy is usually followed by bone marrow or stem cell transplantation to rebuild the bone marrow.
immune system (ih-MYOON SIS-tem)
* The complex group of organs and cells that defends the body against infections and other diseases.
infusion (in-FYOO-zhun)
* A method of putting fluids, including drugs, into the bloodstream. Also called intravenous infusion.
injection (in-JEK-shun)
* Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."
interferon (in-ter-FEER-on)
* A biological response modifier (a substance that can improve the body's natural response to infections and other diseases). Interferons interfere with the division of cancer cells and can slow tumor growth. There are several types of interferons, including interferon-alpha, -beta, and -gamma. The body normally produces these substances. They are also made in the laboratory to treat cancer and other diseases.
internal radiation therapy (in-TER-nul RAY-dee-AY-shun THAYR-uh-pee)
* A type of radiation therapy in which radioactive material sealed in needles, seeds, wires, or catheters is placed directly into or near a tumor. Also called brachytherapy, implant radiation therapy, and radiation brachytherapy.
lenalidomide (leh-nah-LIH-doh-mide)
* A drug that is similar to thalidomide, and is used to treat multiple myeloma and certain types of anemia. It is also being studied in the treatment of other types of cancer. Lenalidomide belongs to the family of drugs called angiogenesis inhibitors. Also called CC-5013 and Revlimid.
myeloproliferative disorder (MY-eh-loh-proh-LIH-feh-ruh-tiv dis-OR-der)
* A group of slow growing blood cancers, including chronic myelogenous leukemia, in which large numbers of abnormal red blood cells, white blood cells, or platelets grow and spread in the bone marrow and the peripheral blood.
NCI
* NCI, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. It conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the NCI Web site at http://www.cancer.gov. Also called National Cancer Institute.
organ (OR-gun)
* A part of the body that performs a specific function. For example, the heart is an organ.
PEG-interferon alfa-2a (peg-IN-ter-FEER-on AL-fuh ...)
* A drug used to treat hepatitis C infections. It is also being studied in the treatment and prevention of cancer. It is a cytokine that is modified in the laboratory. It is a type of biological response modifier. Also called Pegasys.
phlebotomy (fleh-BAH-toh-mee)
* The puncture of a vein with a needle for the purpose of drawing blood. Also called venipuncture.
platelet (PLATE-let)
* A tiny piece of a cell found in the blood that breaks off from a large cell found in the bone marrow. Platelets help wounds heal and prevent bleeding by forming blood clots. Also called thrombocyte.
primary myelofibrosis (PRY-mayr-ee MY-eh-loh-fy-BROH-sis)
* A progressive, chronic disease in which the bone marrow is replaced by fibrous tissue and blood is made in organs such as the liver and the spleen, instead of in the bone marrow. This disease is marked by an enlarged spleen and progressive anemia. Also called agnogenic myeloid metaplasia, chronic idiopathic myelofibrosis, idiopathic myelofibrosis, and myelosclerosis with myeloid metaplasia.
radiation (RAY-dee-AY-shun)
* Energy released in the form of particle or electromagnetic waves. Common sources of radiation include radon gas, cosmic rays from outer space, medical x-rays, and energy given off by a radioisotope (unstable form of a chemical element that releases radiation as it breaks down and becomes more stable).
radiation therapy (RAY-dee-AY-shun THAYR-uh-pee)
* The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.
radioactive (RAY-dee-oh-AK-tiv)
* Giving off radiation.
radioactive seed (RAY-dee-oh-AK-tiv...)
* A small, radioactive pellet that is placed in or near a tumor. Cancer cells are killed by the energy given off as the radioactive material breaks down and becomes more stable.
recur
* To come back or to return.
red blood cell (red blud sel)
* A cell that carries oxygen to all parts of the body. Also called erythrocyte and RBC.
regional chemotherapy (REE-juh-nul KEE-moh-THAYR-uh-pee)
* Treatment with anticancer drugs directed to a specific area of the body.
side effect (side eh-FEKT)
* A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.
spleen (spleen)
* An organ that is part of the lymphatic system. The spleen makes lymphocytes, filters the blood, stores blood cells, and destroys old blood cells. It is located on the left side of the abdomen near the stomach.
splenectomy (spleh-NEK-toh-mee)
* An operation to remove the spleen.
stage
* The extent of a cancer in the body. Staging is usually based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread from the original site to other parts of the body.
standard therapy (... THAYR-uh-pee)
* Treatment that experts agree is appropriate, accepted, and widely used. Also called best practice, standard medical care, and standard of care.
stem cell (stem sel)
* A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.
stem cell transplant (stem sel tranz-plant)
* A method of replacing immature blood-forming cells in the bone marrow that have been destroyed by drugs, radiation, or disease. Stem cells are injected into the patient and make healthy blood cells. A stem cell transplant may be autologous (using a patient’s own stem cells that were saved before treatment), allogeneic (using stem cells donated by someone who is not an identical twin), or syngeneic (using stem cells donated by an identical twin).
surgery (SER-juh-ree)
* A procedure to remove or repair a part of the body or to find out whether disease is present. An operation.
symptom (SIMP-tum)
* An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.
systemic chemotherapy (sis-TEH-mik KEE-moh-THAYR-uh-pee)
* Treatment with anticancer drugs that travel through the blood to cells all over the body.
targeted therapy (TAR-geh-ted THAYR-uh-pee)
* A type of treatment that uses drugs or other substances, such as monoclonal antibodies, to identify and attack specific cancer cells. Targeted therapy may have fewer side effects than other types of cancer treatments.
thalidomide (tha-LIH-doh-MIDE)
* A drug that is used to treat multiple myeloma in patients who have just been diagnosed, and a painful skin disease related to leprosy. It is also being studied in the treatment of other types of cancer. Thalidomide belongs to the family of drugs called angiogenesis inhibitors. Also called Thalomid.
therapy (THAYR-uh-pee)
* Treatment.
transfusion (tranz-FYOO-zhun)
* A procedure in which a person is given an infusion of whole blood or parts of blood. The blood may be donated by another person, or it may have been taken from the patient earlier and stored until needed. Also called blood transfusion.
tumor (TOO-mer)
* An abnormal mass of tissue that results when cells divide more than they should or do not die when they should. Tumors may be benign (not cancer), or malignant (cancer). Also called neoplasm.
tyrosine kinase inhibitor (TY-ruh-seen KY-nays in-HIH-bih-ter)
* A drug that interferes with cell communication and growth and may prevent tumor growth. Some tyrosine kinase inhibitors are used to treat cancer.
vein (vayn)
* A blood vessel that carries blood to the heart from tissues and organs in the body.
watchful waiting (WACH-ful WAY-ting)
* Closely watching a patient’s condition but not giving treatment unless symptoms appear or change. Watchful waiting is used in conditions that progress slowly, are hard to diagnose, or may get better without treatment. It is also used when the risks of treatment are greater than the possible benefits. During watchful waiting, patients may be given certain tests and exams. Watchful waiting is sometimes used in prostate cancer. It is a type of expectant management.
white blood cell (hwite blud sel)
* A type of immune cell. Most white blood cells are made in the bone marrow and are found in the blood and lymph tissue. White blood cells help the body fight infections and other diseases. Granulocytes, monocytes, and lymphocytes are white blood cells. Also called leukocyte and WBC.
x-ray (EX-ray)
* A type of high-energy radiation. In low doses, x-rays are used to diagnose diseases by making pictures of the inside of the body. In high doses, x-rays are used to treat cancer.
Table of Links
1 http://www.cancer.gov/cancertopics/druginfo/myeloproliferativedisorders
2 http://www.cancer.gov/cancertopics/pdq/treatment/myeloproliferative/Patient/Table1
3 http://cancer.gov/clinicaltrials
* * * * * * * * * * * *
Treatment Options for Chronic Myeloproliferative Disorders
Chronic Myelogenous Leukemia
Polycythemia Vera
The purpose of treatment for polycythemia vera is to reduce the number of extra blood cells. Treatment of polycythemia vera may include the following:
* Phlebotomy.
* Chemotherapy with or without phlebotomy.
* Biologic therapy using interferon alfa or pegylated interferon alpha..
* Low- dose aspirin.
* A clinical trial of a new treatment.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with polycythemia vera. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Primary Myelofibrosis
Treatment of primary myelofibrosis in patients without symptoms is usually watchful waiting.
Treatment of primary myelofibrosis in patients with symptoms may include the following:
* Transfusion of red blood cells to relieve symptoms and improve quality of life.
* Biologic therapy using interferon alfa or erythropoietic growth factors.
* Splenectomy.
* Radiation therapy to the spleen.
* Chemotherapy.
* Donor stem cell transplant.
* Thalidomide or lenalidomide.
* A clinical trial of a targeted therapy.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with primary myelofibrosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Essential Thrombocythemia
Treatment of essential thrombocythemia in patients younger than 60 years who have no symptoms and an acceptable platelet count is usually watchful waiting. Treatment of other patients may include the following:
* Chemotherapy.
* Anagrelide therapy.
* Biologic therapy using interferon alfa or pegylated interferon alpha.
* Platelet apheresis.
* A clinical trial of a new treatment.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with essential thrombocythemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Chronic Neutrophilic Leukemia
Treatment of chronic neutrophilic leukemia may include the following:
* Donor bone marrow transplant.
* Chemotherapy.
* Biologic therapy using interferon alfa.
* A clinical trial of a new treatment.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with chronic neutrophilic leukemia. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site.
Chronic Eosinophilic Leukemia
Treatment of chronic eosinophilic leukemia may include the following:
* Bone marrow transplant.
* Biologic therapy using interferon alfa.
* A clinical trial of a new treatment.
* * * * * * * * * * * *
Chapter 2A: Chronic Myeloproliferative Disorders Health Professional Information
Chronic Myeloproliferative Disorders Treatment
General Information About Chronic Myeloproliferative Disorders
The chronic myeloproliferative disorders consist of chronic myelogenous leukemia, polycythemia vera (p. vera), primary myelofibrosis, essential thrombocythemia, chronic neutrophilic leukemia, and chronic eosinophilic leukemia. All of these disorders involve dysregulation at the multipotent hematopoietic stem cell (CD34), with one or more of the following shared features:
• Overproduction of one or several blood elements with dominance of a transformed clone.
• Hypercellular marrow/marrow fibrosis.
• Cytogenetic abnormalities.
• Thrombotic and/or hemorrhagic diatheses.
• Extramedullary hematopoiesis (liver/spleen).
• Transformation to acute leukemia.
• Overlapping clinical features.
Patients with p. vera and essential thrombocythemia have marked increases of red blood cell and platelet production, respectively. Treatment is directed at reducing the excessive numbers of blood cells. Both p. vera and essential thrombocythemia can develop a spent phase late in their courses that resembles primary myelofibrosis with cytopenias and marrow hypoplasia and fibrosis.[1-3] A specific point mutation in one copy of the Janus kinase 2 gene (JAK2), a cytoplasmic tyrosine kinase, on chromosome 9, which causes increased proliferation and survival of hematopoietic precursors in vitro, has been identified in most patients with p. vera, essential thrombocythemia, and idiopathic myelofibrosis.[4-9] Researchers are pursuing specific targeting of this aberrant protein.
1. Schafer AI: Bleeding and thrombosis in the myeloproliferative disorders. Blood 64 (1): 1-12, 1984.
2. Barosi G: Myelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelines. J Clin Oncol 17 (9): 2954-70, 1999.
3. Tefferi A: Myelofibrosis with myeloid metaplasia. N Engl J Med 342 (17): 1255-65, 2000.
4. Kralovics R, Passamonti F, Buser AS, et al.: A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med 352 (17): 1779-90, 2005.
5. Baxter EJ, Scott LM, Campbell PJ, et al.: Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 365 (9464): 1054-61, 2005 Mar 19-25.
6. James C, Ugo V, Le Couedic JP, et al.: A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature 434 (7037): 1144-8, 2005.
7. Levine RL, Wadleigh M, Cools J, et al.: Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell 7 (4): 387-97, 2005.
8. Scott LM, Tong W, Levine RL, et al.: JAK2 exon 12 mutations in polycythemia vera and idiopathic erythrocytosis. N Engl J Med 356 (5): 459-68, 2007.
9. Campbell PJ, Green AR: The myeloproliferative disorders. N Engl J Med 355 (23): 2452-66, 2006.
Key Points for This Section
* Chronic myelogenous leukemia is a disease in which the bone marrow makes too many white blood cells.
* Possible signs of chronic myelogenous leukemia include tiredness, night sweats, and fever.
* Most people with CML have a gene mutation (change) called the Philadelphia chromosome.
* Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myelogenous leukemia.
* Certain factors affect prognosis (chance of recovery) and treatment options.
Chronic myelogenous leukemia is a disease in which the bone marrow makes too many white blood cells.
Chronic myelogenous leukemia (also called CML or chronic granulocytic leukemia) is a slowly progressing blood and bone marrow disease that usually occurs during or after middle age, and rarely occurs in children.
Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:
* Red blood cells that carry oxygen and other materials to all tissues of the body.
* Platelets that help prevent bleeding by causing blood clots to form.
* Granulocytes (white blood cells) that fight infection and disease
Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.
In CML, too many blood stem cells develop into a type of white blood cell called granulocytes. These granulocytes are abnormal and do not become healthy white blood cells. They may also be called leukemic cells. The leukemic cells can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur.
Possible signs of chronic myelogenous leukemia include tiredness, night sweats, and fever.
These and other symptoms may be caused by CML. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:
* Feeling very tired.
* Weight loss for no known reason.
* Night sweats.
* Fever.
* Pain or a feeling of fullness below the ribs on the left side.
Sometimes CML does not cause any symptoms at all.
Most people with CML have a gene mutation (change) called the Philadelphia chromosome.
Every cell in the body contains DNA (genetic material) that determines how the cell looks and acts. DNA is contained inside chromosomes. In CML, part of the DNA from one chromosome moves to another chromosome. This change is called the “ Philadelphia chromosome.” It results in the bone marrow making an enzyme, called tyrosine kinase, that causes too many stem cells to develop into white blood cells (granulocytes or blasts).
The Philadelphia chromosome is not passed from parent to child.
Philadelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The bcr-abl gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome.
Tests that examine the blood and bone marrow are used to detect (find) and diagnose chronic myelogenous leukemia.
The following tests and procedures may be used:
* Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease such as an enlarged spleen. A history of the patient’s health habits and past illnesses and treatments will also be taken.
* Complete blood count (CBC): A procedure in which a sample of blood is drawn and checked for the following:
* The number of red blood cells, white blood cells, and platelets.
* The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
* The portion of the sample made up of red blood cells.