21st Century Adult Cancer Sourcebook: Chronic Myelogenous Leukemia (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 Myelogenous Leukemia Patient Information
Chapter 2A: Chronic Myelogenous Leukemia Health Professional Information
Chapter 3A: Chronic Myelogenous Leukemia NCI Drugs
Chapter 4A: Leukemia Background Information
Chapter 5A: Chronic Myelogenous Leukemia Cancer Clinical Trials
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 Myelogenous Leukemia Patient Information
Chronic Myelogenous Leukemia Treatment
Patient Version
Last Modified: 09/27/2011
General Information About Chronic Myelogenous Leukemia
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.
* 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.
* Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it.
* 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, such as the Philadelphia chromosome.
* Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a 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.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) and treatment options depend on the following:
* The patient’s age.
* The phase of CML.
* The amount of blasts in the blood or bone marrow.
* The size of the spleen at diagnosis.
* The patient’s general health.
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).
anemia (uh-NEE-mee-uh)
* A condition in which the number of red blood cells is below normal.
blast (blast)
* An immature blood cell.
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.
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.
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 granulocytic leukemia (KRAH-nik GRAN-yoo-loh-SIH-tik 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 myelogenous leukemia, chronic myeloid leukemia, and CML.
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.
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.
DNA
* The molecules inside cells that carry genetic information and pass it from one generation to the next. Also called deoxyribonucleic acid.
enzyme (EN-zime)
* A protein that speeds up chemical reactions in the body.
genetic (jeh-NEH-tik)
* Inherited; having to do with information that is passed from parents to offspring through genes in sperm and egg cells.
granulocyte (GRAN-yoo-loh-SITE)
* A type of immune cell that has granules (small particles) with enzymes that are released during infections, allergic reactions, and asthma. Neutrophils, eosinophils, and basophils are granulocytes. A granulocyte is a type of white blood cell. Also called granular leukocyte, PMN, and polymorphonuclear leukocyte.
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.
leukemia (loo-KEE-mee-uh)
* Cancer that starts in blood-forming tissue such as the bone marrow and causes large numbers of blood cells to be produced and enter the bloodstream.
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.
organ (OR-gun)
* A part of the body that performs a specific function. For example, the heart is an organ.
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.
Philadelphia chromosome (FIH-luh-DEL-fee-uh KROH-muh-some)
* An abnormality of chromosome 22 in which part of chromosome 9 is transferred to it. Bone marrow cells that contain the Philadelphia chromosome are often found in chronic myelogenous leukemia.
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.
prognosis (prog-NO-sis)
* The likely outcome or course of a disease; the chance of recovery or recurrence.
progressive disease (pruh-GREH-siv dih-ZEEZ)
* Cancer that is growing, spreading, or getting worse.
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.
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.
stem cell (stem sel)
* A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.
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.
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.
Table of Links
1 http://www.cancer.gov/cancertopics/pdq/treatment/adultALL/Patient
2 http://www.cancer.gov/cancertopics/pdq/treatment/childALL/Patient
3 http://www.cancer.gov/cancertopics/pdq/treatment/adultAML/Patient
4 http://www.cancer.gov/cancertopics/pdq/treatment/childAML/Patient
5 http://www.cancer.gov/cancertopics/pdq/treatment/CLL/Patient
6 http://www.cancer.gov/cancertopics/pdq/treatment/hairy-cell-leukemia/Patient
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Stages of Chronic Myelogenous Leukemia
Key Points for This Section
* After chronic myelogenous leukemia has been diagnosed, tests are done to find out if the cancer has spread.
* There are three ways that cancer spreads in the body.
* Chronic myelogenous leukemia has 3 phases.
* Chronic phase
* Accelerated phase
* Blastic phase
After chronic myelogenous leukemia has been diagnosed, tests are done to find out if the cancer has spread.
Staging is the process used to find out how far the cancer has spread. There is no standard staging system for chronic myelogenous leukemia (CML). Instead, the disease is classified by phase: chronic phase, accelerated phase, or blastic phase. It is important to know the phase in order to plan treatment. The following tests and procedures may be used to find out the phase:
* 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, such as the Philadelphia chromosome.
* Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells.
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.
Chronic myelogenous leukemia has 3 phases.
As the amount of blast cells increases in the blood and bone marrow, there is less room for healthy white blood cells, red blood cells, and platelets. This may result in infections, anemia, and easy bleeding, as well as bone pain and pain or a feeling of fullness below the ribs on the left side. The number of blast cells in the blood and bone marrow and the severity of symptoms determine the phase of the disease.
Chronic phase
In chronic phase CML, fewer than 10% of the cells in the blood and bone marrow are blast cells.
Accelerated phase
In accelerated phase CML, 10% to 19% of the cells in the blood and bone marrow are blast cells.
Blastic phase
In blastic phase CML, 20% or more of the cells in the blood or bone marrow are blast cells. When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis.
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).
accelerated phase chronic myelogenous leukemia (ak-SEH-leh-ray-ted fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which the disease is progressing. In this phase, 10% to 19% of the cells in the blood and bone marrow are blast cells (immature blood cells).
anemia (uh-NEE-mee-uh)
* A condition in which the number of red blood cells is below normal.
blast (blast)
* An immature blood cell.
blast crisis (blast KRY-sis)
* A phase of chronic myelogenous leukemia in which tiredness, fever, and an enlarged spleen occur during the blastic phase, when more than 30% of the cells in the blood or bone marrow are blast cells (immature blood cells).
blastic phase chronic myelogenous leukemia (BLAS-tik fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which 20% or more of the cells in the blood or bone marrow are blast cells (immature blood cells). When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis.
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.
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.
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.
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 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 phase chronic myelogenous leukemia (KRAH-nik fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which fewer than 10% of the cells in the blood and bone marrow are blast cells (immature blood cells). This phase may last from several months to several years, and there may be no symptoms of leukemia.
cytogenetics (SY-toh-jeh-NEH-tix)
* The study of chromosomes and chromosomal abnormalities.
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.
leukemia (loo-KEE-mee-uh)
* Cancer that starts in blood-forming tissue such as the bone marrow and causes large numbers of blood cells to be produced and enter the bloodstream.
lymph vessel (limf ...)
* A thin tube that carries lymph (lymphatic fluid) and white blood cells through the lymphatic system. Also called lymphatic vessel.
lymphatic system (lim-FA-tik SIS-tem)
* The tissues and organs that produce, store, and carry white blood cells that fight infections and other diseases. This system includes the bone marrow, spleen, thymus, lymph nodes, and lymphatic vessels (a network of thin tubes that carry lymph and white blood cells). Lymphatic vessels branch, like blood vessels, into all the tissues of the body.
metastasis (meh-TAS-tuh-sis)
* The spread of cancer from one part of the body to another. A tumor formed by cells that have spread is called a “metastatic tumor” or a “metastasis.” The metastatic tumor contains cells that are like those in the original (primary) tumor. The plural form of metastasis is metastases (meh-TAS-tuh-SEEZ).
metastatic (meh-tuh-STA-tik)
* Having to do with metastasis, which is the spread of cancer from the primary site (place where it started) to other places in the body.
pathologist (puh-THAH-loh-jist)
* A doctor who identifies diseases by studying cells and tissues under a microscope.
Philadelphia chromosome (FIH-luh-DEL-fee-uh KROH-muh-some)
* An abnormality of chromosome 22 in which part of chromosome 9 is transferred to it. Bone marrow cells that contain the Philadelphia chromosome are often found in chronic myelogenous leukemia.
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 tumor (PRY-mayr-ee TOO-mer)
* The original tumor.
red blood cell (red blud sel)
* A cell that carries oxygen to all parts of the body. Also called erythrocyte and RBC.
solid tumor (SAH-lid TOO-mer)
* An abnormal mass of tissue that usually does not contain cysts or liquid areas. Solid tumors may be benign (not cancer), or malignant (cancer). Different types of solid tumors are named for the type of cells that form them. Examples of solid tumors are sarcomas, carcinomas, and lymphomas. Leukemias (cancers of the blood) generally do not form solid tumors.
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.
staging (STAY-jing)
* Performing exams and tests to learn the extent of the cancer within the body, especially whether the disease has spread from the original site to other parts of the body. It is important to know the stage of the disease in order to plan the best treatment.
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.
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.
* * * * * * * * * * * *
Relapsed Chronic Myelogenous Leukemia
In relapsed CML, the number of blast cells increases after a remission.
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Treatment Option Overview
Key Points for This Section
* There are different types of treatment for patients with chronic myelogenous leukemia.
* Six types of standard treatment are used:
* Targeted therapy
* Chemotherapy
* Biologic therapy
* High-dose chemotherapy with stem cell transplant
* Donor lymphocyte infusion (DLI)
* Surgery
* New types of treatment are being tested in clinical trials.
* 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 myelogenous leukemia.
Different types of treatment are available for patients with chronic myelogenous leukemia (CML). 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 for patients with cancer. 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.
Six types of standard treatment are used:
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 used to treat chronic myelogenous leukemia.
Imatinib mesylate, nilotinib, and dasatinib are tyrosine kinase inhibitors that may be used as initial treatment for newly diagnosed patients with chronic phase CML.
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.
Biologic therapy
Biologic therapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy.
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.
Donor lymphocyte infusion (DLI)
Donor lymphocyte infusion (DLI) is a cancer treatment that may be used after stem cell transplant. Lymphocytes (a type of white blood cell) from the stem cell transplant donor are removed from the donor’s blood and may be frozen for storage. The donor’s lymphocytes are thawed if they were frozen and then given to the patient through one or more infusions. The lymphocytes see the patient’s cancer cells as not belonging to the body and attack them.
Surgery
Splenectomy is surgery to remove the spleen.
New types of treatment are being tested in clinical trials.
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.
Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials.
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.
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.
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.
cavity (KA-vih-tee)
* A hollow area or hole. It may describe a body cavity (such as the space within the abdomen) or a hole in a tooth caused by decay.
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 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 phase chronic myelogenous leukemia (KRAH-nik fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which fewer than 10% of the cells in the blood and bone marrow are blast cells (immature blood cells). This phase may last from several months to several years, and there may be no symptoms of leukemia.
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.
dasatinib (da-SA-tih-nib)
* A drug used to treat certain types of chronic myeloid leukemia and acute lymphoblastic leukemia. Dasatinib is also being studied in the treatment of certain other blood diseases and types of cancer. Dasatinib binds to and blocks BCR-ABL and other proteins that help cancer cells grow. It is a type of tyrosine kinase inhibitor. Also called BMS-354825 and Sprycel.
diagnosis (DY-ug-NOH-sis)
* The process of identifying a disease, such as cancer, from its signs and symptoms.
donor (DOH-ner)
* In medicine, a person who gives blood, cells, tissue, or an organ for use in another person, such as in a blood transfusion or an organ transplant.
donor lymphocyte infusion (DOH-ner LIM-foh-site in-FYOO-zhun)
* A type of therapy in which lymphocytes from the blood of a donor are given to a patient who has already received a stem cell transplant from the same donor. The donor lymphocytes may kill remaining cancer cells. Donor lymphocyte infusion is used to treat chronic myelogenous leukemia (CML) that has come back and myeloma. It is being studied in the treatment of other types of cancer.
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.
first-line therapy (... THAYR-uh-pee)
* Initial treatment used to reduce a cancer. First-line therapy is followed by other treatments, such as chemotherapy, radiation therapy, and hormone therapy to get rid of cancer that remains. Also called induction therapy, primary therapy, and primary treatment.
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.
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.
imatinib mesylate (ih-MA-tih-nib MEH-zih-layt)
* A drug used to treat different types of leukemia and other cancers of the blood, gastrointestinal stromal tumors, skin tumors called dermatofibrosarcoma protuberans, and a rare condition called systemic mastocytosis. It is also being studied in the treatment of other types of cancer. Imatinib mesylate blocks the protein made by the bcr/abl oncogene. It is a type of tyrosine kinase inhibitor. Also called Gleevec and STI571.
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."
lymphocyte (LIM-foh-site)
* A type of immune cell that is made in the bone marrow and is found in the blood and in lymph tissue. The two main types of lymphocytes are B lymphocytes and T lymphocytes. B lymphocytes make antibodies, and T lymphocytes help kill tumor cells and help control immune responses. A lymphocyte is a type of white blood cell.
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.
nilotinib (ny-LOH-tih-nib)
* A drug used to treat certain types of chronic myelogenous leukemia (CML). It is used in some newly diagnosed patients. It is also used in patients who have not gotten better after treatment with other anticancer drugs or who are not able to take imatinib mesylate. It is also being studied in the treatment of other types of cancer. Nilotinib blocks a protein called BCR-ABL, which may help keep cancer cells from growing. It is a type of tyrosine kinase inhibitor. Also called Tasigna.
organ (OR-gun)
* A part of the body that performs a specific function. For example, the heart is an organ.
recur
* To come back or to return.
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 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.
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.
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.
Table of Links
1 http://www.cancer.gov/cancertopics/druginfo/leukemia#dal4
2 http://www.cancer.gov/cancertopics/pdq/treatment/CML/Patient/Table1
3 http://cancer.gov/clinicaltrials
Treatment Options for Chronic Myelogenous Leukemia
Chronic Phase Chronic Myelogenous Leukemia
Treatment of chronic phase chronic myelogenous leukemia may include the following:
* Targeted therapy with a tyrosine kinase inhibitor.
* High-dose chemotherapy with donor stem cell transplant.
* Biologic therapy (interferon) with or without chemotherapy.
* Chemotherapy.
* Splenectomy.
* A clinical trial of lower- dose chemotherapy with donor stem cell transplant.
* 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 phase chronic myelogenous leukemia 1. 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 2.
Accelerated Phase Chronic Myelogenous Leukemia
Treatment of accelerated phase chronic myelogenous leukemia may include the following:
* Stem cell transplant.
* Targeted therapy with a tyrosine kinase inhibitor.
* Biologic therapy (interferon) with or without chemotherapy.
* High-dose chemotherapy.
* Chemotherapy.
* Transfusion therapy to replace red blood cells, platelets, and sometimes white blood cells, to relieve symptoms and improve quality of life.
* 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 accelerated phase chronic myelogenous leukemia 3. 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 2.
Blastic Phase Chronic Myelogenous Leukemia
Treatment of blastic phase chronic myelogenous leukemia may include the following:
* Targeted therapy with a tyrosine kinase inhibitor.
* Chemotherapy using one or more drugs.
* High-dose chemotherapy.
* Donor stem cell transplant.
* Chemotherapy as palliative therapy to relieve symptoms and improve quality of life.
* 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 blastic phase chronic myelogenous leukemia 4. 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 2.
Relapsed Chronic Myelogenous Leukemia
Treatment of relapsed chronic myelogenous leukemia may include the following:
* Targeted therapy with a tyrosine kinase inhibitor.
* Donor stem cell transplant.
* Donor lymphocyte infusion.
* Biologic therapy (interferon).
* A clinical trial of new types or higher doses of targeted therapy and donor stem cell transplant.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with relapsing chronic myelogenous leukemia 5. 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 2.
Glossary Terms
accelerated phase chronic myelogenous leukemia (ak-SEH-leh-ray-ted fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which the disease is progressing. In this phase, 10% to 19% of the cells in the blood and bone marrow are blast cells (immature blood cells).
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.
blastic phase chronic myelogenous leukemia (BLAS-tik fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which 20% or more of the cells in the blood or bone marrow are blast cells (immature blood cells). When tiredness, fever, and an enlarged spleen occur during the blastic phase, it is called blast crisis.
chemotherapy (KEE-moh-THAYR-uh-pee)
* Treatment with drugs that kill cancer cells.
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 phase chronic myelogenous leukemia (KRAH-nik fayz KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)
* A phase of chronic myelogenous leukemia in which fewer than 10% of the cells in the blood and bone marrow are blast cells (immature blood cells). This phase may last from several months to several years, and there may be no symptoms of leukemia.
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.
donor (DOH-ner)
* In medicine, a person who gives blood, cells, tissue, or an organ for use in another person, such as in a blood transfusion or an organ transplant.
donor lymphocyte infusion (DOH-ner LIM-foh-site in-FYOO-zhun)
* A type of therapy in which lymphocytes from the blood of a donor are given to a patient who has already received a stem cell transplant from the same donor. The donor lymphocytes may kill remaining cancer cells. Donor lymphocyte infusion is used to treat chronic myelogenous leukemia (CML) that has come back and myeloma. It is being studied in the treatment of other types of cancer.
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.
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.
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.
palliative therapy (PA-lee-uh-tiv THAYR-uh-pee)
* Treatment given to relieve the symptoms and reduce the suffering caused by cancer and other life-threatening diseases. Palliative cancer therapies are given together with other cancer treatments, from the time of diagnosis, through treatment, survivorship, recurrent or advanced disease, and at the end of life.
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.
quality of life (KWAH-lih-tee ... life)
* The overall enjoyment of life. Many clinical trials assess the effects of cancer and its treatment on the quality of life. These studies measure aspects of an individual’s sense of well-being and ability to carry out various activities.
red blood cell (red blud sel)
* A cell that carries oxygen to all parts of the body. Also called erythrocyte and RBC.
relapse (REE-laps)
* The return of a disease or the signs and symptoms of a disease after a period of improvement.
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.
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).
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.
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.
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.
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.
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.
Table of Links
1 http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=39069&tt=1&format=1&cn=1
2 http://www.cancer.gov/clinicaltrials
3 http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=39070&tt=1&format=1&cn=1
4 http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=39071&tt=1&format=1&cn=1
5 http://www.cancer.gov/Search/ClinicalTrialsLink.aspx?Diagnosis=38720&tt=1&format=1&cn=1
* * * * * * * * * * * *
Chapter 2A: Chronic Myelogenous Leukemia Health Professional Information
Chronic Myelogenous Leukemia Treatment
General Information About Chronic Myelogenous Leukemia
Incidence and Mortality
Note: Estimated new cases and deaths from chronic myelogenous leukemia (CML) in the United States in 2010:[1]
• New cases: 4,870.
• Deaths: 440.
CML is one of a group of diseases called the myeloproliferative disorders. Other related entities include the following:
• Polycythemia vera.
• Myelofibrosis.
• Essential thrombocythemia.
CML is a clonal disorder that is usually easily diagnosed because the leukemic cells of more than 95% of patients have a distinctive cytogenetic abnormality, the Philadelphia chromosome (Ph1).[2,3] The Ph1 results from a reciprocal translocation between the long arms of chromosomes 9 and 22 and is demonstrable in all hematopoietic precursors.[4] This translocation results in the transfer of the Abelson (ABL) on chromosome 9 oncogene to an area of chromosome 22 termed the breakpoint cluster region (BCR).[4] This in turn results in a fused BCR/ABL gene and in the production of an abnormal tyrosine kinase protein that causes the disordered myelopoiesis found in CML. Furthermore, these molecular techniques can now be used to supplement cytogenetic studies to detect the presence of the 9;22 translocation in patients without a visible Ph1 (Ph1-negative).
Ph1-negative CML is a poorly defined entity that is less clearly distinguished from other myeloproliferative syndromes. Patients with Ph1-negative CML generally have a poorer response to treatment and shorter survival than Ph1-positive patients.[5] Ph1-negative patients who have BCR/ABL gene rearrangement detectable by Southern blot analysis, however, have prognoses equivalent to Ph1-positive patients.[6,7] A small subset of patients have BCR/ABL detectable only by reverse transcriptase-polymerase chain reaction (RT-PCR), which is the most sensitive technique currently available. Patients with RT-PCR evidence of the BCR/ABL fusion gene appear clinically and prognostically identical to patients with a classic Ph1; however, patients who are BCR/ABL-negative by RT-PCR have a clinical course more consistent with chronic myelomonocytic leukemia, which is a distinct clinical entity related to myelodysplastic syndrome.[6,8,9] Fluorescent in situ hybridization of the BCR/ABL translocation can be performed on the bone marrow aspirate or on the peripheral blood of patients with CML.[10]
At the time of diagnosis of patients with CML, splenomegaly is the most common finding on physical examination.[10] The spleen may be enormous, filling most of the abdomen and presenting a significant clinical problem, or the spleen may be only minimally enlarged. In about 10% of patients, the spleen is neither palpable nor enlarged on splenic scan.
The median age of patients with Ph1-positive CML is 67 years.[1] The median survival is 4 to 6 years, with a range of less than 1 year to more than 10 years. Survival after development of an accelerated phase is usually less than 1 year and after blastic transformation is only a few months.[10]
References
1. American Cancer Society.: Cancer Facts and Figures 2010. Atlanta, Ga: American Cancer Society, 2010. Also available online. Last accessed June 16, 2011.
2. Kurzrock R, Kantarjian HM, Druker BJ, et al.: Philadelphia chromosome-positive leukemias: from basic mechanisms to molecular therapeutics. Ann Intern Med 138 (10): 819-30, 2003.
3. Goldman JM, Melo JV: Chronic myeloid leukemia-advances in biology and new approaches to treatment. N Engl J Med 349 (15): 1451-64, 2003.
4. Deininger MW, Goldman JM, Melo JV: The molecular biology of chronic myeloid leukemia. Blood 96 (10): 3343-56, 2000.
5. Onida F, Ball G, Kantarjian HM, et al.: Characteristics and outcome of patients with Philadelphia chromosome negative, bcr/abl negative chronic myelogenous leukemia. Cancer 95 (8): 1673-84, 2002.
6. Martiat P, Michaux JL, Rodhain J: Philadelphia-negative (Ph-) chronic myeloid leukemia (CML): comparison with Ph+ CML and chronic myelomonocytic leukemia. The Groupe Francais de Cytogenetique Hematologique. Blood 78 (1): 205-11, 1991.
7. Cortes JE, Talpaz M, Beran M, et al.: Philadelphia chromosome-negative chronic myelogenous leukemia with rearrangement of the breakpoint cluster region. Long-term follow-up results. Cancer 75 (2): 464-70, 1995.
8. Oscier DG: Atypical chronic myeloid leukaemia, a distinct clinical entity related to the myelodysplastic syndrome? Br J Haematol 92 (3): 582-6, 1996.
9. Kurzrock R, Bueso-Ramos CE, Kantarjian H, et al.: BCR rearrangement-negative chronic myelogenous leukemia revisited. J Clin Oncol 19 (11): 2915-26, 2001.
10. Sawyers CL: Chronic myeloid leukemia. N Engl J Med 340 (17): 1330-40, 1999.