National Cancer Institute


Posted Date: Aug 31, 2013

Expert-reviewed information summary about the treatment of childhood acute myeloid leukemia, myelodysplastic syndromes, and other myeloproliferative disorders.

Childhood Acute Myeloid Leukemia Treatment

General Information About Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Key Points for this Section

  • Leukemia and other diseases of the blood and bone marrow may affect red blood cells, white blood cells, and platelets.
  • Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells.
  • Other myeloid diseases can affect the blood and bone marrow.
    • Chronic myelogenous leukemia
    • Juvenile myelomonocytic leukemia
    • Transient myeloproliferative disorder
    • Myelodysplastic syndromes
  • AML or MDS may occur after treatment with certain anticancer drugs and/or radiation therapy.
  • The risk factors for developing childhood AML, childhood CML, JMML, TMD, and MDS are similar.
  • Possible signs of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising.
  • Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, TMD, and MDS.
  • Certain factors affect prognosis (chance of recovery) and treatment options.

Leukemia and other diseases of the blood and bone marrow may affect red blood cells, white blood cells, and platelets.

Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

Blood cell development; drawing shows the steps a blood stem cell goes through to become a red blood cell, platelet, or white blood cell. A myeloid stem cell becomes a red blood cell, a platelet, or a myeloblast, which then becomes a granulocyte (the types of granulocytes are eosinophils, basophils, and neutrophils). A lymphoid stem cell becomes a lymphoblast and then becomes a B-lymphocyte, T-lymphocyte, or natural killer cell.
Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.

Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells.

Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly. Acute myeloid leukemia (AML) is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, or acute nonlymphocytic leukemia.

In AML, the myeloid stem cells usually become a type of immature white blood cell called myelo blasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia 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. The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a granulocytic sarcoma or chloroma.

There are subtypes of AML based on the type of blood cell that is affected. The treatment of AML is different when it is a subtype called acute promyelocytic leukemia (APL) or when the child has Down syndrome.

Other myeloid diseases can affect the blood and bone marrow.

Chronic myelogenous leukemia

In chronic myelogenous leukemia (CML), too many bone marrow stem cells become a type of white blood cell called granulocytes. Some of these bone marrow stem cells never become mature white blood cells. These are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. CML is rare in children.

Juvenile myelomonocytic leukemia

Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children around the age of 2 years. In JMML, too many bone marrow stem cells become 2 types of white blood cells called myelocytes and monocytes. Some of these bone marrow stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.

Transient myeloproliferative disorder

Transient myeloproliferative disorder (TMD) is a disorder of the bone marrow that can develop in newborns who have Down syndrome. This disorder usually goes away on its own within the first 3 weeks of life. Infants who have Down syndrome and TMD have an increased chance of developing AML before the age of 3 years.

Myelodysplastic syndromes

In myelodysplastic syndromes (MDS), the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The treatment for MDS depends on how much lower than normal the number of red blood cells, white blood cells, or platelets is. MDS may progress to AML.

This summary is about childhood AML, childhood CML, JMML, TMD, and MDS. See the following PDQ summaries for more information about other types of leukemia and diseases of the blood and bone marrow:

  • Adult Acute Myeloid Leukemia Treatment
  • Chronic Myelogenous Leukemia Treatment
  • Adult Acute Lymphoblastic Leukemia Treatment
  • Childhood Acute Lymphoblastic Leukemia Treatment
  • Chronic Lymphocytic Leukemia Treatment
  • Hairy Cell Leukemia Treatment
  • Myelodysplastic Syndromes Treatment
  • Myelodysplastic/Myeloproliferative Neoplasms Treatment

AML or MDS may occur after treatment with certain anticancer drugs and/or radiation therapy.

Cancer treatment with radiation therapy and/or certain anticancer drugs may cause therapy-related AML (t-AML) or therapy-related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the anticancer drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children.

The risk factors for developing childhood AML, childhood CML, JMML, TMD, and MDS are similar.

Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your child’s doctor if you think your child may be at risk. Possible risk factors for childhood AML, childhood CML, JMML, TMD, and MDS include the following:

  • Having a brother or sister, especially a twin, with leukemia.
  • Being Hispanic.
  • Being exposed to cigarette smoke or alcohol before birth.
  • Having a history of MDS or aplastic anemia.
  • Past treatment with chemotherapy or radiation therapy.
  • Being exposed to ionizing radiation or chemicals such as benzene.
  • Having certain genetic disorders, such as: Down syndrome.Fanconi anemia.Neurofibromatosis type 1.Noonan syndrome.Shwachman-Diamond syndrome.

Possible signs of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising.

These and other symptoms may be caused by childhood AML, childhood CML, JMML, or MDS. Other conditions may cause the same symptoms. Check with a doctor if your child has any of the following problems:

  • Fever with or without an infection.
  • Night sweats.
  • Shortness of breath.
  • Weakness or feeling tired.
  • Easy bruising or bleeding.
  • Petechiae (flat, pinpoint spots under the skin caused by bleeding).
  • Pain in the bones or joints.
  • Pain or feeling of fullness below the ribs.
  • Painless lumps in the neck, underarm, stomach, groin, or other parts of the body. When seen in childhood AML, these lumps, called leukemia cutis, may be blue or purple.
  • Painless lumps that are sometimes around the eyes. These lumps, called chloromas, are sometimes seen in childhood AML and may be blue-green.
  • An eczema-like skin rash.

The symptoms of TMD may include the following:

  • Swelling all over the body.
  • Shortness of breath.
  • Trouble breathing.
  • Weakness or feeling tired.
  • Pain below the ribs.

Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, TMD, and MDS.

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.
  • 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.
  • Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.
  • Peripheral blood smear: A procedure in which a sample of blood is checked for blast cells, the number and kinds of white blood cells, number of platelets, and changes in the shape of the blood cells.
  • Biopsy: The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. Biopsies that may be done for childhood AML include the following: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.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.Tumor biopsy: A biopsy of a chloroma may be done.Lymph node biopsy: The removal of all or part of a lymph node.
  • Cytogenetic analysis: A laboratory 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.
  • Immunophenotyping: A process used to identify cells, based on the types of antigens or markers on the surface of the cell, that may include special staining of the blood and bone marrow cells. This process is used to diagnose the subtype of AML by comparing the cancer cells to normal cells of the immune system.
  • FISH (fluorescence in situ hybridization): A laboratory technique used to look at genes or chromosomes in cells and tissues. Pieces of DNA that contain a fluorescent dye are made in the laboratory and added to cells or tissues on a glass slide. When these pieces of DNA bind to specific genes or areas of chromosomes on the slide, they light up when viewed under a microscope with a special light.
  • Reverse transcription–polymerase chain reaction test (RT–PCR): A laboratory test in which cells in a sample of tissue are studied using chemicals to look for certain changes in the structure or function of genes.
  • Lumbar puncture: A procedure used to collect cerebrospinal fluid from the spinal column. This is done by placing a needle into the spinal column. This procedure is also called an LP or spinal tap.Lumbar puncture. A patient lies in a curled position on a table. After a small area on the lower back is numbed, a spinal needle (a long, thin needle) is inserted into the lower part of the spinal column to remove cerebrospinal fluid (CSF, shown in blue). The fluid may be sent to a laboratory for testing.

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis (chance of recovery) and treatment options for childhood AML depend on the following:

  • The age of the child at diagnosis.
  • The race or ethnic group of the child.
  • Whether the child is greatly overweight.
  • Number of white blood cells in the blood at diagnosis.
  • Whether the AML was caused by previous anticancer treatment.
  • The subtype of AML.
  • Whether there are certain chromosome or gene changes in the leukemia cells.
  • Whether the child has Down syndrome. Most children with AML and Down syndrome can be cured of their leukemia.
  • Whether the child has leukemia in the central nervous system (brain and spinal cord).
  • How quickly the leukemia responds to initial treatment.
  • Whether the AML is newly diagnosed or has recurred (come back) after being treated.
  • The length of time since treatment ended, for AML that has recurred.

The prognosis and treatment options for childhood CML depend on how long it has been since the patient was diagnosed and how many blast cells are in the blood.

The prognosis (chance of recovery) and treatment options for JMML depend on the following:

  • The age of the child at diagnosis.
  • How many red blood cells, white blood cells, or platelets are in the blood.
  • Whether the JMML is untreated or has recurred after treatment.

The prognosis (chance of recovery) and treatment options for MDS depend on the following:

  • Whether the MDS was caused by previous cancer treatment.
  • How low the numbers of red blood cells, white blood cells, or platelets are.
  • Whether the MDS is untreated or has recurred after treatment.

Stages of Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Key Points for this Section

  • Once childhood acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body.
  • There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

Once childhood acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body.

The extent or spread of cancer is usually described as stages. In childhood acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used, instead of the stage, to plan treatment. The following tests and procedures may be used to determine if the leukemia has spread:

  • Lumbar puncture: A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle into the spinal column. This procedure is also called an LP or spinal tap.
  • Biopsy of the testicles, ovaries, or skin: The removal of cells or tissues from the testicles, ovaries, or skin so they can be viewed under a microscope to check for signs of cancer. This is done only if something unusual about the testicles, ovaries, or skin is found during the physical exam.

There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

Childhood AML is described as newly diagnosed, in remission, or recurrent.

Newly diagnosed childhood AML

Newly diagnosed childhood AML has not been treated except to relieve symptoms such as fever, bleeding, or pain, and one of the following is true:

Childhood AML in remission

In childhood AML in remission, the disease has been treated and the following are true:

Recurrent Childhood Acute Myeloid Leukemia

Recurrent childhood acute myeloid leukemia (AML) has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord).

Treatment Option Overview

Key Points for this Section

  • There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).
  • Children with AML, CML, JMML, TMD, or MDS should have their treatment planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood.
  • Some cancer treatments cause side effects months or years after treatment has ended.
  • The treatment of childhood AML usually has two phases.
  • Seven types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or MDS.
    • Chemotherapy
    • Radiation therapy
    • Stem cell transplantation
    • Targeted therapy with a tyrosine kinase inhibitor
    • Other drug therapy
    • Watchful waiting
    • Supportive care
  • 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 children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

Different types of treatment are available for children with AML, CML, JMML, TMD, or MDS. 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.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with AML, CML, JMML, TMD, or MDS should have their treatment planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood.

Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists:

Some cancer treatments cause side effects months or years after treatment has ended.

Regular follow-up exams are very important. Some cancer treatments cause side effects that continue or appear months or years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include:

  • Physical problems.
  • Changes in mood, feelings, thinking, learning, or memory.
  • Second cancers (new types of cancer).

Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information).

The treatment of childhood AML usually has two phases.

The treatment of childhood AML is done in phases:

Treatment called central nervous system ( CNS) sanctuary therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the cells are able to "find sanctuary" (hide) in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and keep the cancer from recurring (coming back). CNS sanctuary therapy is also called CNS prophylaxis.

Seven types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or MDS.

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 (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas ( regional chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug.

The way the chemotherapy is given depends on the type of cancer being treated.

In AML, the leukemia cells may spread to the brain and/or spinal cord. Anticancer drugs given by mouth or vein to treat AML cannot cross the blood-brain barrier and enter the fluid that surrounds the brain and spinal cord. Instead, an anticancer drug is injected into the fluid-filled space to kill leukemia cells that may have spread there. This is called intrathecal chemotherapy.Intrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed.

See Drugs Approved for Acute Myeloid Leukemia for more information.

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. External radiation therapy may be used to treat childhood AML that has spread, or may spread, to the brain and spinal cord. When used this way, it is called central nervous system (CNS) sanctuary therapy or CNS prophylaxis.

Stem cell transplantation

Stem cell transplant is a way of giving chemotherapy and replacing blood-forming cells that are abnormal or 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.

Targeted therapy with a tyrosine kinase inhibitor

Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitor (TKI) therapy is a type of targeted therapy that blocks signals needed for tumors to grow. TKIs blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells ( granulocytes or blasts) than the body needs. Imatinib (Gleevec) is one of the TKIs used to treat childhood CML.

TKIs may be used in combination with other anticancer drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).

See Drugs Approved for Myeloproliferative Disorders for more information.

Other drug therapy

Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change.

Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia (APL).

See Drugs Approved for Acute Myeloid Leukemia for more information.

Watchful waiting

Watchful waiting is closely monitoring a patient’s condition without giving any treatment until symptoms appear or change. It is sometimes used to treat MDS or TMD.

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

  • Transfusion therapy: A way of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. The blood may be donated from another person or it may have been taken from the person earlier and stored until needed.
  • Drug therapy, such as antibiotics.
  • Leukapheresis: A procedure in which a special machine is used to remove white blood cells from the blood. Blood is taken from the patient and put through a blood cell separator where the white blood cells are removed. The rest of the blood is then returned to the patient's bloodstream.

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.

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. Monoclonal antibodies, proteasome inhibitors, tyrosine kinase inhibitors, and natural killer (NK) cells are types of targeted therapies being studied in the treatment of childhood AML.

Monoclonal antibody therapy uses antibodies made in the laboratory, from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Monoclonal antibodies may be used in combination with chemotherapy as adjuvant therapy.

Proteasome inhibitors break down proteins in cancer cells and kill them. Bortezomib is a proteasome inhibitor used to treat childhood acute promyelocytic leukemia.

Sorafenib is a tyrosine kinase inhibitor being studied in the treatment of childhood AML.

Natural killer (NK) cells are white blood cells that can kill tumor cells. These may be taken from a donor and given to the patient by infusion to help kill leukemia cells.

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


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