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Types of Brain Tumors.doc.doc.doc

  1. 1. Types of Brain Tumors & their Treatments Once a diagnosis has been made, doctors will then decide on a treatment plan. It may range from observation (no need for immediate therapy at all) to a combination of surgery (in which the tumor is physically removed during a surgical procedure), radiation therapy (which uses high-energy particles or waves, such as x-rays, to destroy or damage cancer cells), and aggressive chemotherapy (which uses drugs to treat the tumor). Your child's chance of recovery depends on a number of factors, including tumor type and location, and amount of tumor spread, or metastasis, but it's important to note that there are treatments available for every type of brain tumor. Tumors that form in the tissues and cells of a child's brain are called primary brain tumors. In some cases, tumors that have originated in other parts of the body spread to the brain, but this is rare in children. The following is a list of some of the most common types of primary brain tumors found in children. Astrocytomas (also known as gliomas) Accounting for almost half of all childhood brain tumors, astrocytomas are tumors that arise in brain cells called astrocytes (astrocytes are star shaped cells in the central nervous system that support neurons and help remove debris). Astrocytomas can be further divided into the following types:  Low-grade astrocytomas, which include Grade 1 (Juvenile pilocytic astrocytomas) and Grade 2 (Fibrillary astrocytomas) astrocytomas. They may be cured with surgery alone if they can be completely removed. If not, observation only may be considered. If additional treatment is required, radiation therapy is usually used for the older children and chemotherapy is used for the younger children.  High-grade astrocytomas include Grade 3 (Anaplastic astrocytomas) and Grade 4 (Glioblastoma multiforme) astrocytomas. They are highly malignant tumors that have a much more guarded prognosis. Surgery, radiation therapy, and chemotherapy are usually recommended. Also, diffuse pontine gliomas (a type of brain stem glioma) are Find a Clinical Trial Find out about new research studies for pediatric brain tumors more
  2. 2. highly malignant astrocytomas that occur in a very delicate part of the brain. Surgery cannot be done safely, and patients are usually treated with radiation therapy and may be candidates for new investigational treatments. Prognosis is very guarded. Ependymomas Ependymomas develop within the ependymal cells lining the brain's ventricles (a series of fluid filled cavities in the brain) and are treated with surgical resection and often with radiation therapy. Ependymomas, unlike astrocytomas, typically do not spread into normal, surrounding brain tissue. Children who have ependymomas that cannot be completely resected are often also treated with chemotherapy. Though there are no treatments unique to ependymomas, radiation therapy is an extremely important part of the typical treatment plan, often including the use of intensity modulated radiation therapy (IMRT) -- a targeted treatment that delivers high-doses of radiation to tumor cells while sparing surrounding healthy tissue. Medulloblastomas and PNETs Representing approximately 15 percent of childhood brain tumors, medulloblastomas are tumors that, doctors think, arise from undeveloped stem cells in the portion of the brain that controls voluntary movement, the cerebellum. They are highly malignant, but with appropriate treatment many children can be cured. Medulloblastomas usually are accompanied by headaches and vomiting (particularly first thing in the morning). Sometimes the child will show behavioral changes and deterioration of their school performance. The diagnosis is suspected using CT (computed tomography) or MRI (magnetic resonance imaging) scan and confirmed by a pathology examination after the tumor's surgical removal. Treatment for medulloblastomas usually includes surgery, radiation therapy (except in the very young), and chemotherapy. Our investigators have developed a new treatment called radioimmunotherapy in conjunction with a decreased radiation dose (in an attempt to reduce side effects) and conventional chemotherapy. Primitive neuroectodermal tumors (PNET) are another form of rapidly growing tumor that are known as pineoblastomas when they occur in the pineal gland (a pea-sized gland at the center of the brain), and supratentorial PNETs when occurring in the cerebral hemispheres. The diagnosis is suspected via CT or How to Make an Appointment Call the Department of Pediatrics at 212-639-5954 more
  3. 3. MRI scan and confirmed by pathology after the tumor's surgical removal. PNETs can prove less responsive to therapies, but treatments are available. GermCell Tumors Germ cell tumors, as the name suggests, arise from germ cells, which during normal development of the embryo form into either egg cells or sperm cells. In the case of germ cell tumors located in the brain, the embryonic germ cells have mistakenly traveled to the brain, where they develop into tumors. Occurring most frequently in children, germ cell tumors comprise several different types of tumors (including germinomas, endodermal sinus tumors, and choriocarcinomas). Though their symptoms depend on the tumor location, germ cell tumors in the pineal region usually are accompanied by headaches and vomiting (particularly first thing in the morning). Tumors in the suprasellar region (near the pituitary gland) usually are accompanied by hormonal abnormalities, particularly increased urination, and thirst. Sometimes the child will show behavioral changes and deterioration of their school performance. Most of them are malignant tumors, but can often be cured with current treatments that may include surgery, radiation therapy, and chemotherapy. Young Children and Brain Tumors Young children (those less than three years old) with brain tumors are a special group. Regardless of the type of tumor, we often try to avoid radiation therapy due to the risk of causing learning and memory problems in this age group. Chemotherapy is more frequently recommended. Leukemias Leukemias are cancers that occur in the tissues within the body that produce the body's blood cells and bone marrow. Leukemias are the most common form of childhood cancer. About one-third of all cancers in children under the age of 15 are leukemias, as are about one-fourth of all cancers in people under 20. Stems cells are present in healthy bone marrow and naturally develop into red blood cells, white blood cells, and platelets -- all of which are found in blood. Sometimes certain types of immature white blood cells undergo a random mutation, or change, of a gene in the DNA that can Jackson's Story A hero at age ten: Jackson's battle with acute lymphoblastic leukemia more
  4. 4. cause the cell to grow and multiply uncontrollably, resulting in leukemia. These uncontrolled cells flood the body's organs and interfere with their function. The cells also inhibit the body's ability to produce red and white blood cells and platelets normally. Stem cells that produce white blood cells come in two major types: myeloid cells and lymphoid cells. When uncontrolled cell growth begins within a descendant of a lymphoid cell, it is acute lymphoblastic leukemia (ALL). This is the most common type of childhood leukemia; it accounts for about 85 percent of all childhood leukemias. When the uncontrolled growth originates within a descendant of a myeloid cell, it is acute myelogenous leukemia (AML), the second most common type of childhood leukemia.  Our Approach & Expertise Memorial Sloan-Kettering's Pediatric Hematologic Malignancy Team evaluates and treats patients up to 21 years of age who have all forms of acute and chronic leukemia, lymphoma, or other diseases that may involve the bone marrow.  Our Team of Experts Our doctors -- including pediatric oncologists, radiation oncologists, nurses, long-term care specialists, and social workers -- work as a multidisciplinary team to provide optimal care to leukemia patients.  Acute Lymphoblastic Leukemia (ALL) About 85 percent of all childhood leukemias are classified as acute lymphoblastic leukemia (ALL). In this form of leukemia, a change -- or mutation -- in the DNA of an immature white blood cell called a lymphoid cell causes the cell to grow and multiply in an uncontrolled way. About 85 percent of all childhood leukemias are classified as acute lymphoblastic leukemia (ALL). In this form of leukemia, a change -- or mutation -- in the DNA of an immature white blood cell, called a lymphoid cell, causes the cell to grow and multiply in an uncontrolled way. (Immature lymphoid cells originate in the bone marrow, thymus gland, or in the peripheral lymph nodes.) These uncontrolled cells infiltrate the body's organs and interfere with their function. The cells also interfere with the body's ability to produce other types of cells normally. There are about 2,500 new cases of ALL diagnosed in children in the United States every year. ALL is slightly more common in males. Most ALL cases (89 percent) occur in Caucasian children. Peak age at diagnosis is between two and three years of age. No one is sure exactly what causes leukemia.
  5. 5.  Symptoms Over 90 percent of patients with leukemia complain of felling tired or not feeling well. Many a lso have bone pain. Over 90 percent of patients with leukemia complain of feeling tired or not feeling well. Many also have bone pain. Any unexplained bone pain should be evaluated for possible disease of the bone marrow. Young children might not be able to express specifically what kind of pain they are feeling, so bone pain should be suspected if a child limps or refuses to walk. About two-thirds of patients with leukemia have a history of fevers that may last for various amounts of time but have no apparent cause. About half of patients have signs of bleeding, including bruises. Some patients may seem pale or have recurrent infections. Since leukemia interferes with the body's ability to produce other normal cells, some patients may also be anemic (a condition that results from low red- blood cell levels).  Diagnosis To diagnose ALL, doctors will do a physical exam and draw a sample of blood to do a complete blood count, or CBC. To diagnose ALL, doctors will perform a physical exam of the patient and draw a sample of blood to do a complete blood count or CBC. There are various components in blood that generally fall within a certain range. For example, there are normally less than 10,000 white blood cells in a microliter of blood (a microliter is one-millionth of a liter). Doctors will examine the blood to see if the cells and components are within the normal range. While an abnormal blood count is a key clue in diagnosing ALL, not every child with ALL will have an abnormal CBC. About 35 percent of ALL patients have a white blood cell count of more than 10,000 cells per microliter. Red blood cells and platelets are also counted in a CBC. The diagnosis is confirmed by looking at the bone marrow directly. The marrow is found in the center of the bone, and a sample is retrieved using a needle. Leukemia is a rare disease and may not be immediately suspected, as many other illnesses have similar symptoms. Since the best outcome depends on starting treatment as early as possible, physicians may do a bone marrow test immediately if leukemia is suspected. Cells from the marrow will be tested in a variety of ways and examined under a microscope to see if they have the distinguishing characteristics of ALL.
  6. 6.  Staging There are many factors that physicians consider when developing a treatment strategy. There are many factors that physicians consider when developing a treatment strategy. After studying large numbers of patients with ALL, it was clear to researchers that some patients did better than others, even though they received the same course of treatment. By evaluating thousands of patients, researchers were able to compile a list of factors that indicate a favorable or unfavorable prognosis. These factors include age, sex, and complete blood count numbers. The patient's initial response to therapy is also a good indicator of the long-term prognosis. Patients who are likely to have a good outcome are treated less intensively so that they experience fewer side effects. Patients who are at a high risk of disease recurrence are treated more intensively. Over the last 30 to 40 years, the outlook for children with ALL has vastly improved. Once, the disease was almost always considered fatal. Today the cure rate for ALL is close to 80 percent.  Treatment There are many factors that physicians consider when deciding on a course of treatment for a patient diagnosed with ALL. Chemotherapy ALL is usually treated with chemotherapy drugs -- drugs that are toxic to the leukemia cells and stop them from growing. Chemotherapy is given in several stages. The first stage is called induction therapy. The goal of induction therapy is to put the disease in remission -- to kill close to 99 percent of the abnormal cells while allowing normal blood- cell production to resume. Chemotherapy also kills normal cells, so patients may feel nauseous and tired, and may be prone to more infections. Several chemotherapy drugs are used in combination with other chemotherapies in order to reach any cells that are resistant to one of the drugs but may respond to the other drug. Sometimes a pool of leukemia cells may be "hiding" in the central nervous system (or, in boys, in the testes), where the chemotherapy drugs can not reach them. After the initial chemotherapy session, these hidden cells are targeted using a chemotherapy drug that is given in the fluid around the spinal column. Cranial irradiation (radiation of the head area) may also be done but only in patients at very high-risk. Maintenance Therapy
  7. 7. After the initial chemotherapy treatment, patients are given maintenance therapy as a follow up. A combination of chemotherapy drugs is given to ensure that any cells that may have been dormant initially are also targeted. The length of time that a patient undergoes maintenance therapy depends on the sex of the patient and the prognostic factors (see Staging). Maintenance therapy can last as long as three years for boys. In some cases, when there is a very high risk of disease recurrence, a bone marrow transplant may be done if a matched sibling donor is available. More about Treatment In the United States, most children with ALL are treated with protocols developed by the Children's Cancer Group (CCG) or the Pediatric Oncology Group (POG). Recently, these two groups have joined together to form the Children's Oncology Group (COG). Future treatment protocols will come through COG. Children with ALL often have low blood counts and may need blood transfusions to relieve symptoms as well as antibiotics to treat infections.  The first step in treatment for ALL is remission induction, which is aimed at achieving a state in which there is no visible evidence of disease and blood counts are normal. Hospitalization may be required for this step. A common treatment approach is a four-week course of weekly vincristine and prednisone along with L-asparaginase. Doxorubicin, daunorubicin, or cyclophosphamide may be added for patients at high risk. Patients at standard risk or low risk may only need three chemotherapy drugs. Other drugs maybe given for supportive care. For example, allopurinol may be given to reduce the uric acid that may form and trimethoprim/sulfa may be given to try to prevent Pneumocystis pneumonia.  Treatment targeting the central nervous system (CNS) -- the brain and spinal cord -- is usually given during the remission induction phase and during the first three treatment sessions, when remission is well established. The drug methotrexate is usually given intrathecally, which is by means of an injection into the cerebrospinal fluid, the liquid that bathes the central nervous system. The procedure to do this, a lumbar puncture, is repeated up to 18 times during the early and later phases of treatment. Cytarabine, hydrocortisone, or both may be added to methotrexate. Radiation therapy to the head is necessary if the disease is present in the CNS. Radiation may be used to prevent the disease from spreading there in patients at very high risk. In such cases, the treatment is called prophylactic, which means preventive.  Continuation therapy or maintenance therapy generally lasts How To Make An Appointment Call the Department of Pediatrics at 212-639-5954 more
  8. 8. two to three years after cancer remission is attained. For patients at low-risk, this treatment is less intensive than remission induction. A common treatment approach is intermittent methotrexate and daily 6-mercaptopurine with monthly vincristine and prednisone.  About one-quarter of children with ALL will experience a cancer relapse, but most will respond to reinduction therapy. Bone marrow transplantation should be considered after a second remission is achieved, especially in patients whose cancer relapse occurs less than 36 months after diagnosis. Remission Induction Side Effects Side effects during remission induction can be significant, and patients usually remain in the hospital for a period of three to six weeks. Treatment for ALL is aimed at the leukemic blood cells, but normal blood cells are also affected. All patients will have lowered blood counts that lead to a variety of symptoms. Low white blood cell counts put patients at increased risk of infection. Treatment with antibiotics to prevent or treat infections is a likely measure oncologists will take. In some situations, your child will be in isolation, and visitors to his or her room may need to wear gowns, masks, and gloves as preventive measures. Low white blood cell counts generally also lead to inflammation of the mucous membranes in the mouth and gastrointestinal tract. This is called mucositis. The tissues become very dry and may crack or bleed. Specific oral care regimens help minimize discomfort and prevent secondary infections. Mouth sores can make eating painful. The mouth sores themselves can cause pain that requires pain medication. Nausea, vomiting, and diarrhea are side effects for which effective treatments exist. Hair may fall out, and this is can be an especially upsetting side effect. Wigs and scarves may help minimize the distress hair loss causes. Low platelet counts lead to easy bruising and bleeding. Taking care to prevent injuries is very important. Low red blood cell counts cause anemia and make patients feel tired and weak. Sometimes patients are short of breath or may experience a fast heartbeat rate. Blood transfusions will be necessary to replace red blood cells and platelets. Many patients are given stimulating factors called G-CSF or GM-CSF. These stimulate the development of new white blood cells so that cell counts are low for a shorter period of time. The initial treatment and the severity of side effects can be very difficult and wearing on a patient. While blood counts are low, your child may find it difficult to carry on normal activities. Simple exercise, such as walking, may seem overwhelming. Your doctors and nurses will do everything in their power to minimize the discomfort, sadness, and boredom that are common during this initial phase of treatment. Social workers, psychiatrists, and psychologists can help you and your child with depression and the ups
  9. 9. and downs commonly experienced during treatment. Social workers can also help with financial issues related to treatment. Finally, all hospitals have clergy who are available to speak to you and provide spiritual support and guidance. Resuming Activities After the initial hospitalization period, your child will slowly begin to resume normal activities, but work and school may have to be postponed. Strength and stamina will return gradually. The doctors and nurses will provide more specific guidelines on what to expect based on individual treatment regimens and each individual patient's responses.  Innovative ALL Treatments A selection of some of our latest innovative approaches to treating pediatric leukemia in clinical studies. Our Center is currently evaluating a new drug called clofarabine (Clofarex) in young patients with progressive acute ALL in whom the standard treatment drugs have failed. Clofarabine combines two older drugs, fludarabine and cladribine, into a new drug that may be effective in treating leukemia. In a previous dose-finding clinical trial of this drug in patients (including children) with a leukemia that has returned or has not responded to prior treatment, good responses -- including remission -- have been observed. Doctors now have determined what the safe dose of clofarabine is and can begin this new trial to clarify how effective this drug will be in more patients. We are also conducting a study to test another new two-drug combination for the treatment of recurrent ALL. The two drugs are trimetrexate and leucovorin. Trimetrexate is an investigational drug that is being evaluated as a potential anticancer therapy. Laboratory studies suggest that this drug is effective against leukemias that can't be treated effectively with methotrexate, the standard chemotherapy drug. Leucovorin is an approved drug that protects normal cells from the effects of chemotherapy, but it does not interfere with trimetrexate's action against cancer cells. This study will also evaluate the side effects of this combination therapy. After patients achieve remission, they will continue to receive consolidation chemotherapy (to kill any lingering cancer cells) or if a matched sibling donor is available, patients will receive a bone marrow (stem cell) transplant. Our investigators are currently studying the side effects of intensified therapy, which can be substantial. And it is crucial that the essential elements of intensified therapy be selectively administered to children who will benefit from these elements. The aims of this study are to determine whether stronger post-induction therapies in the treatment of children with low-risk and average-risk features will cure more
  10. 10. children without adding too many side effects. A key component of contemporary ALL treatment regimens is risk- adapted therapy. This grouping depends on how quic kly the blasts disappear during Induction therapy (rapid vs slow early response); whether or not there were very small amounts of leukemia in the bone marrow (called Minimal Residual Disease or MRD) at the end of induction; and what biologic features their leukemia blasts have. Our investigators are currently creating investigational studies, the data from which will be used to refine subsequent therapy by assignment to a specific treatment protocol for defined risk groups.  Acute Myelogenous Leukemia (AML) Acute myelogenous leukemia (AML) is responsible for nearly 15 percent of all childhood leukemias. In this form of leukemia, a change -- or mutation -- in the DNA of an immature white blood cell called a myeloid cell causes the cell to grow and multiply in an uncontrolled way. Acute myelogenous leukemia (AML) is responsible for nearly 15 percent of all childhood leukemias. In this form of leukemia, a change, or mutation, in the DNA of an immature white blood cell, called a myeloid cell, causes the cell to grow and multiply in an uncontrolled way. (Immature myeloid cells originate in the bone marrow.) These uncontrolled cells infiltrate the body's organs and interfere with their function. The cells also interfere with the body's ability to produce other types of cells normally. There are about 300 new cases of AML diagnosed in children in the United States every year. No one is sure exactly what causes leukemia.  Symptoms The symptoms of AML include fatigue and a feeling of not being well. Many patients also have bone pain. The symptoms of AML include fatigue and a feeling of not being well. Many patients also have bone pain. Young children might not be able to express specifically what kind of pain they are feeling, so bone pain might be suspected if a child limps or refuses to walk. Any unexplained bone pain should be evaluated for possible disease of the bone marrow. Since leukemia interferes with the body's ability to produce other normal cells, some patients may also be anemic and may seem pale or may have recurring infections. Other symptoms include fever and signs of bleeding, including bruises. Rarely myeloid leukemia cells can form a solid tumor and be present as a mass anywhere in the body. These tumors are called chloromas or granulocytic sarcoma.  Diagnosis
  11. 11. To diagnose AML, doctors will do a physical exam and draw a sample of blood to do a complete blood count, or CBC. To diagnose AML, doctors first perform a physical exam of the patient and draw a sample of blood to do a complete blood count or CBC. There are various components in blood that generally fall within a certain range. For example, there are normally less than 10,000 white blood cells in a microliter of blood (a microliter is one-millionth of a liter). Doctors will examine the patient's blood to see if the cells and components are within the normal range. While an abnormal blood count is a key clue in diagnosing AML, not every child with AML will have an abnormal CBC. About 10 percent of AML patients have a white blood cell count of more than 100,000 cells per microliter. Red blood cells and platelets are also counted in a CBC. The doctor's diagnosis is confirmed by looking at the bone marrow directly to see if abnormal cells are present. The marrow is found in the center of the bone and a sample is retrieved using a needle. Leukemia is a rare disease and may not be immediately suspected, as many other illnesses have similar symptoms. Since the best outcome depends on starting treatment as early as possible, physicians may do a bone marrow test immediately if leukemia is suspected. Cells from the marrow will then be tested in a variety of ways and examined under a microscope to see if they have the distinguishing characteristics of AML.  Staging There are many factors that physicians consider when developing a treatment strategy. There are many factors that physicians consider when developing a treatment strategy. These factors include a patient's age, sex, and complete blood count numbers, among other things. Before choosing which treatment to use, a doctor will study all available test results to determine the extent that the disease has spread, a process called staging. Over the past 30 to 40 years the outlook for children with AML has vastly improved. Once, the disease was almost always considered fatal. Today the cure rate for AML is close to 50 percent with chemotherapy treatment, and the cure rate is 70 percent with a matched sibling donor bone marrow (stem cell) transplant.  Treatment AML is usually treated with chemotherapy drugs -- drugs that are toxic to the leukemia cells and stop them from growing. AML is usually treated with chemotherapy drugs -- drugs that are toxic
  12. 12. to the leukemia cells and stop them from growing. Chemotherapy is given in several stages. The first stage is called induction therapy. The goal of induction therapy is to put the disease in remission -- to kill close to 99 percent of the abnormal cells and allow normal blood cell production to resume. Chemotherapy also kills normal cells. As a result, patients may feel nauseous and tired, and be prone to more infections. Several chemotherapy drugs are used together, so if any cells are resistant to one of the drugs, those cells will be targeted by the other drug. AML Treatment Facts  The first treatment phase is called remission induction. Chemotherapy drugs are used to achieve cancer remission, the state in which there is no clinical evidence of disease and blood counts are normal. The remission induction therapy for children is generally daunorubicin and cytarabine given over the course of seven to ten days. Other drugs such as thioguanine or etoposide may be added.  Even if the central nervous system (CNS) shows no clinical evidence of disease, treatment of that area with cytarabine is also recommended. This drug is usually administered directly to the cerebrospinal fluid. Preventive chemotherapy for the CNS is commonly recommended for children. Irradiation is used to treat disease that has entered the CNS, and irradiation may also be used to prevent CNS involvement.  Treatment following remission induction may be bone marrow transplantation using an HLA-matched sibling. To learn more about stem cell transplants, you may wish to order the Memorial Sloan-Kettering Cancer Center CD-ROM program entitled Bone Marrow and Stem Cell Transplantation. Transplantation may not be recommended for the rare child who generally does well with standard treatment. In this case, the child may receive intensive-dose cytarabine followed by two to six months of combination chemotherapy. Treatment Decisions Most patients, adults and children, will be treated according to cooperative group protocols. Often these will be clinical trials seeking to improve outcomes. Some drugs are more effective against specific subtypes of leukemia, and treatments are often based on the patient's specific gene mutation. Your doctor will discuss this during the treatment consultation following immunophenotyping and other diagnostic tests. During Treatment How To Make An Appointment Call the Department of Pediatrics at 212-639-5954 more
  13. 13. Side effects during remission induction can be significant, and patients usually remain in the hospital for a period of four to six weeks. Treatment for AML is aimed at the leukemic blood cells, but normal blood cells are also affected by chemotherapy treatment. All patients undergoing treatment will experience lowered blood counts that lead to a variety of symptoms. Low white blood cell counts put patients at increased risk of infection. Treatment with antibiotics to prevent or treat infection is likely. In some situations, the patient will be in isolation, and visitors to the patient's room may need to wear gowns, masks, and gloves to reduce the risk of infection. Low white blood cell counts generally also lead to inflammation of the mucous membranes in the mouth and gastrointestinal tract. This is called mucositis. The tissues become very dry and may crack or bleed. Specific oral care regimens help minimize discomfort and prevent secondary infections. Mouth sores can make eating painful. The mouth sores themselves can cause pain that requires pain medication. Nausea, vomiting, and diarrhea are side effects for which effective treatments exist. Hair may fall out, and this is can be an especially upsetting side effect. Wigs and scarves may help minimize the distress hair loss causes. Low platelet counts lead to easy bruising and bleeding. Taking care to prevent injuries is very important. Low red blood cell counts causing anemia make patients feel tired and weak. Sometimes, patients may be short of breath or experience a fast heartbeat rate. Transfusions will be necessary to replace red blood cells and platelets. Many patients are given stimulating factors called G-CSF or GM-CSF. These stimulate the development of new white blood cells so that cell counts are low for a shorter period of time. The initial course of treatment and the severity of side effects can be very difficult and wearing on patients. While blood counts are low, your child may find it difficult to carry on normal activities. *Simple exercise, such as walking, may seem overwhelming. Your doctors and nurses will do everything in their power to minimize the discomfort, sadness, and boredom that are common during this initial phase of treatment. Social workers, psychiatrists, and psychologists can help you and your child with depression and the ups and downs you will experience during treatment. Social workers can also help with financial issues related to treatment. Finally, all hospitals have clergy who are available to speak to you and provide spiritual support and guidance. Resuming Activities After the initial hospitalization, patients will slowly begin to resume their normal activities, but school may have to be postponed for many months. Subsequent shorter hospital stays, ranging from one to two
  14. 14. weeks, may be necessary to administer chemotherapy and treat infections that result from low white blood cell counts. Strength and stamina will return gradually. The doctors and nurses will provide more specific guidelines on what is to expect based on individual treatment regimes and individual responses to treatment.  Our Clinical Trials A continually updated listing of Memorial Sloan-Kettering's current clinical trials for pediatric leukemias. Pediatric Cancer Care > Pediatric Cancers > Types of Cancer > Pediatric Leukemias Our Clinical Trials Leukemia TOP  A Clinical Trial for Patients with Standard Risk B-Precursor Acute Lymphoblastic Leukemia Shukla, Neerav, MD [Protocol 06-039]  A Non-Myeloablative Conditioning Regimen with Peri-Transplant Rituximab and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients with B cell Lymphoid Malignancies Barker, Juliet, MBBS [Protocol 06-066]  A Phase I Study of Beta-Glucan and Rituximab in Children and Adolescents with Relapsed CD20-Positive Lymphoma or Leukemia or Post-Transplant Lymphoproliferative Disease Modak, Shakeel, MD [Protocol 03-095]  A Phase I Study of Clofarabine Plus Topotecan, Vinorelbine, Thiotepa, and Dexamethasone in Pediatric Patients with Relapsed or Refractory Acute Leukemia Steinherz, Peter, MD [Protocol 07-012]  A Phase I Study of WT1-Sensitized T cells for Residual or Relapsed
  15. 15. Leukemia after Allogeneic Hematopoietic Progenitor Cell Transplantation O'Reilly, Richard, MD [Protocol 07-055]  A Phase I/II Study of the Toxic and Therapeutic Effects of T Cells in the Treatment of Epstein Barr Virus-Associated Lymphoproliferative Disorders O'Reilly, Richard, MD [Protocol 95-024]  A Phase I/II Trial of Clofarabine, Melphalan, and Thiotepa Followed by an Allogeneic Stem Cell Transplant for the Treatment of High-Risk and/or Advanced Hematologic Malignancies Boulad, Farid, MD [Protocol 06-125]  A Phase II Study of Peri-transplant Palifermin followed by an Allogeneic T-Cell-Depleted Stem Cell Transplant in Patients with Advanced Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Evolved from MDS Castro-Malaspina, Hugo, MD [Protocol 08-008]  A Phase II Trial of Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplants after a Myeloablative Preparative Regimen for Adult Patients with Lymphohematopoietic Disorders Jakubowski, Ann, MD, PhD [Protocol 01-070]  A Phase II Trial of Busulfan, Melphalan & Fludarabine Followed by Stem Cell Transplantation for the Treatment of Lymphohematopoietic Disorders Boulad, Farid, MD [Protocol 01-055]  A Phase II Trial of HLA Haploidentical Natural Killer Cell Infusion for Treatment of Relapsed or Persistent Leukemia Following Allogeneic
  16. 16. Hematopoietic Stem Cell Transplant Hsu, Katharine, MD, PhD [Protocol 07-035]  Cyclophosphamide, Fludarabine, and Total Body Irradiation Followed by the Transplantation of Unrelated Double-Unit Donor Umbilical Cord Blood Grafts for Patients with Hematological Malignancy Barker, Juliet, MBBS [Protocol 06-014]  Participation in the National Marrow Donor Program Registry Kernan, Nancy, MD [Protocol 90-091]  More About Pediatric Leukemias The information here will help parents to understand what is going on in their children's bodies, how this disease is diagnosed and treated, and how to work with a doctor to plan a course of treatment. Children in the Hospital Overview Being hospitalized can be a frightening experience for a child. Most children are used to a predictable and safe world of family, home, and school. When they are faced with a world full of new people and strange machines and their parents seem scared and upset, many feel a loss of control and order. In the following sections are some tips that can help you and your child to cope with hospitalization, including how to prepare a child for procedures and what medical information is appropriate for various age groups. If your child has just been diagnosed with leukemia, you are likely to be feeling a wide range of emotions, including shock, anger, hope, fear, guilt, and even grief. These are all normal and expected as you adjust to the news of a serious illness. You will probably find that over time you accept the diagnosis and focus more on treatment. Your emotions may become less intense as time goes by. Don't hesitate to seek counseling - talking with a trained counselor or participating in a support group can be very helpful for parents, as well as for siblings and other relatives. Be Your Child's Advocate You know what is likely to scare your child and what might comfort him. While you can't protect him from everything that might hurt in the hospital, you can do much to minimize the fear and discomfort. Just being with your child will make a big difference. If the hospital says you can't stay with him overnight, ask why not. Enlist grandparents and friends to cover shifts when you can't be there. Insist that procedures be done in the most painfree
  17. 17. way possible, even if that requires a little more preparation time. Double-check that all procedures were ordered by the doctor and are necessary. In other words, stand up for what you and your child need, and be an informed medical consumer. What to Tell Children In years past, parents often didn't tell children when they were seriously ill. Most do so these days, keeping the information appropriate for the child's age. Knowing what is going on seems to help children cope with procedures better. And even if you try to hide it from your child, he is likely to figure it out anyway - and to perhaps try to hide that knowledge to spare you any further pain. Keeping such secrets only makes it even harder for children. It often helps to have trained social workers or nurses who can help you shape an age- appropriate message. You may also find books and videos that can help you to explain at some of the organizations listed in the Resources section of this program. Encourage your child to ask questions. When telling your child that he is seriously ill, keep in mind the fears and anxieties that are typical for his age.  Children under the age of 4 or 5 mostly fear separation from their loved ones.  Children aged 4 or 5 to 12 can understand quite a bit of what is going on. The fear of separation is less acute and may be replaced with a fear of pain and injury. Make sure your child understands that nothing he did - such as falling off a bicycle - caused the illness. Also make sure he understands it is not contagious.  Teenagers are usually well aware of how serious their disease is and may fear illness and death more than anything else. They also are likely to be quite concerned about possible changes to their bodies and how their peers will see them. Teenagers also may find it hard to suddenly be so dependent on others, just when they were becoming more independent. Helping Children Cope with Procedures Medical procedures such as blood draws and bone marrow aspirations can be frightening for children. A child may have no idea what is going to happen and may think he is being punished. He may feel a severe loss of control over his life, and may react with anger and frustration.  Make sure you understand the procedure fully so you can explain it to your child. Be honest, but don't overwhelm him with information.  Use dolls, drawings, books, and videos to help explain what will happen. Get a toy medical kit so you and your child can act out the procedure on stuffed animals or dolls.  Ask a doctor, nurse, or other health care worker for help in explaining procedures to your child. You know your child well, of course, but they have lots of experience in what medical information children are comfortable hearing and common misperceptions.  Try to have procedures done by health care professionals who have experience with children. If possible, have the same people do procedures in the future - familiarity can be comforting.  Find out how much advance notice of procedures your child wants. Some like to know several days ahead of time, some don't. Encourage questions and expression of feelings. Don't dismiss their fears as nothing. Remember that children can imagine some very wild ideas of what is going to happen.  If possible, stay with your child during the procedure or be there when he wakes up.
  18. 18.  Help your child to maintain some sense of control. Let him make as many choices as possible: choosing a toy to hold during a procedure, choosing which arm gets a needle stick, or choosing which pills to swallow first.  Consider the promise of a special treat or toy after the procedure.  Allow your child's preferences to change over time. Treatment for leukemia can take months or years. Your child's needs will change during that time; teenagers, for example, might no longer want their parents to accompany them during exams and procedures. Respect their decisions. Pain Control Some hospitals routinely use pain medications or anesthesia during procedures such as bone marrow biopsies. Others do not. Find out before the procedure what your doctor's policy is, and if no pain control is offered, ask why not. Ask to consult with an anesthesiologist or other pain control expert if necessary. There are several options for pain control.  EMLA cream is used to anesthetize the skin before a blood draw or other procedure.  Valium and versed are sedatives that relax the patient.  Morphine and fentanyl are powerful pain relievers.  Propofol is a type of general anesthesia. It can be given at doses that do not leave the patient unconscious and do not relieve all pain, but do block any later memory of it. Some people also find hypnosis, guided imagery, or distraction to be helpful in maintaining calm and coping with discomfort. To find someone who can help you use these tools, ask your doctor or social worker for a referral or consult one of the organizations in the Resources section of this program. Making the Hospital Livable There are many things you can do to make a hospital experience more tolerable.  Find the playroom. It may be staffed by professionals who can help your child deal with his feelings through play and art. Many hospitals have play specialists who will visit the child's bedside if he can't get to the playroom.  Bring lots of toys, books, CDs, drawing paper, coloring books, loose clothing, and photos of home.  Bring audio and video tapes of family members, friends, and classmates. Encourage those who can't visit often to send new tapes periodically. For small children, if one parent cannot be there often due to distance or work responsibilities, have him or her tape record a special bedtime story.  Bring a laptop computer so the child can communicate with friends and family by e- mail. He may also want to participate in an age-appropriate online support group.  Give a teen-ager a pre-paid phone card so he can call his friends when he wants to, without relying on change from mom and dad or running up huge bills on the hospital phone.  Make use of the skills and knowledge of the social workers and other staff; they can help everyone in the family cope. Explore counseling, support groups, and other programs.  Let friends and family help you cope. If someone asks what he can do to help, give him a job - even something as simple as mowing your lawn, driving your other
  19. 19. children to activities, or feeding your pets. It lets him help you and frees up your time.  If you want to keep a group of friends and family informed about the child's progress, consider setting up a standard e-mail distribution list that you can use to update everyone at once. Or ask a friend to set up a phone tree or to be the designated information source. Some families even set up a web site complete with pictures and daily updates to keep everyone informed.  It's natural to want to indulge your sick child's every whim, but it's important to continue to set rules and boundaries. It helps to maintain some sense of normalcy. Some children say that being overindulged was actually scary - they knew something was really wrong. Siblings Don't forget to tell your other children what is going on. Siblings tend to be shunted aside when one child is seriously ill. The disruption to the family might cause them to feel guilt, jealousy, fear, or a sense of abandonment. Some will act out these feelings in school or regress behaviorally. Talk to them honestly about what is happening. Encourage questions. Try to get siblings involved in the hospital routine in some way - for example, ask them to record a fun videotape or put together a box of toys that will help entertain the sick child. As much as possible, spend time with them apart from the hospital scene. Also explore some resources just for them - many hospitals have programs and support groups for siblings that can help them to understand what is going on and to feel like part of the team. School Many children continue to attend school while being treated for leukemia, but your doctor will give you specific instructions. School can help to provide the child with a sense of normalcy - a place to just be a kid, rather than a patient, and to enjoy learning and social contact with peers. Most adjust well to being in school during treatment, even if issues such as hair loss or weight gain provoke some initial reluctance. Talk with your child's teachers and the school nurse about any limitations or precautions to be followed. However, while the staff may want to be protective of the child, they should be encouraged to treat the child as just another student, not a patient. If the child is expected to be absent frequently, talk to the teachers about how he can keep pace with the class or make up work later. Your child's classmates will have questions and concerns. You might want to have a social worker or nurse give a presentation to the class on leukemia. If your child's appearance has changed you might want to send a few photos along with the social worker or nurse so she can explain the changes. Even when your child is not in school, encourage ongoing contact with classmates through phone calls, letters, or drawings. Camp During the summer months camp can help to provide children with a sense of normalcy. Investigate the possibilities - some camps are just for children with cancer, while other "mainstream" camps are also well prepared to accept children with serious illnesses. Some camps have programs for the siblings of cancer patients. The American Camping Association and other organizations listed in the Resources section of this program can help you find a suitable camp.  Acute Lymphocytic Leukemia Overview In the United States, most children with ALL are treated with protocols developed by the Children's Cancer Group (CCG) or the Pediatric Oncology Group (POG). Recently, these two
  20. 20. groups have joined to form the Children's Oncology Group (COG). Future portocols will come through COG. Children with ALL often have low blood counts and may need transfusions to relieve symptoms and antibiotics to treat infections.  The first step in treatment is remission induction, which is aimed at achieving a state in which there is no visible evidence of disease and blood counts are normal. Hospitalization may be required for this step. A common approach is a four-week course of weekly vincristine and prednisone along with L-asparaginase. Doxorubicin, daunorubicin, or cyclophosphamide may be added for high-risk patients. Patients at standard or low risk may need only three drugs. Other drugs maybe given for supportive care. For example, allopurinol may be given to reduce the uric acid that may be formed and trimethoprim/sulfa to try to prevent Pneumocystis pneumonia.  Treatment targeting the central nervous system - the brain and spinal cord (CNS) - is usually given during the remission induction phase and during the first three of treatment when remission is well established. The drug methotrexate is usually given intrathecally, which is aninjection into the cerebrospinal fluid, the liquid that bathes the central nervous system. The procedure to do this, a lumbar puncture, is repeated up to 18 times during the early and later phases of treatment. Cytarabine, hydrocortisone, or both may be added to methotrexate. Radiation therapy to the head is necessary if the disease is present in the CNS. Radiation may be used to prevent the disease from spreading there in very high-risk patients. In this case, the treatment is called prophylactic, which means preventive.  Continuation or maintenance therapy generally lasts two to three years after remission is attained. For low-risk patients, this treatment is less intensive than remission induction. A common approach is intermittent methotrexate and daily 6- mercaptopurine with monthly vincristine and prednisone.  About one quarter of children with ALL will relapse, but most will respond to reinduction therapy. Bone marrow transplantation should be considered after a second remission is achieved, especially in patients who relapse less than 36 months after diagnosis. Drugs that might be used to treat ALL are listed at the bottom of the page. Click on the drug(s) you will be receiving to get more information. Side effects during remission induction can be significant and patients usually remain in the hospital for three to six weeks. Treatment for ALL is aimed at the leukemic blood cells, but normal blood cells are also affected. All patients will have lowered blood counts that lead to a variety of symptoms. Low white blood cell counts put patients at increased risk of infection. Treatment with antibiotics to prevent or treat infections is likely. In some situations, your child will be in isolation and visitors to the room may need to wear gowns, masks, and gloves. Low white blood cell counts generally also lead to inflammation of the mucous membranes in the mouth and gastrointestinal tract. This is called mucositis. The tissues become very dry and may crack or bleed. Specific oral care regimens help minimize discomfort and prevent secondary infections. Mouth sores can make eating painful. The mouth sores themselves can cause pain that requires pain medication. Nausea and vomiting and diarrhea are side effects for which effective treatments exist. Hair may fall out and this is can be an especially upsetting side effect. Wigs and scarves may help minimize the distress hair loss causes. Low platelet counts lead to easy bruising and bleeding. Care to prevent injuries is very important. Low red blood cell counts causing anemia make patients feel tired and weak. Sometimes, patients are short of breath or have a fast heart rate.
  21. 21. Transfusions will be necessary to replace red blood cells and platelets. Many patients are given stimulating factors called G-CSF or GM-CSF. These stimulate the development of new white blood cells so that cell counts are low for a shorter period of time. The initial treatment and the severity of side effects can be very difficult and wearing. While blood counts are low, your child may find it difficult to carry on normal activities. Simple exercise, such as walking, may seem overwhelming. Your doctors and nurses will do everything in their power to minimize the discomfort, sadness, and boredom that are common during this initial phase of treatment. Social workers, psychiatrists, and psychologists can help you and your child with depression and the ups and downs commonly experienced during treatment. Social workers can also help with financial issues related to treatment. Finally, all hospitals have clergy who are available to speak to you and provide spiritual guidance. Resuming Activities After the initial hospitalization, your child will slowly begin to resume your normal activities, but work and school may have to be postponed. Strength and stamina will return gradually. The doctors and nurses will provide more specific guidelines on what to expect based on individual treatment regimens and responses. Clinical Trials Many leukemia patients are asked to participate in a clinical trial. A clinical trial is a research program that attempts to find better treatments. A phase I clinical trial aims to find the safest way to give a new treatment. Patients are watched closely for side effects. Once the phase I trial is completed, the new treatment may be evaluated in a phase II clinical trial. At this stage the doctors are looking for anti-cancer activity, as well as side effects. After that, a phase III clinical trial will compare the new treatment to a standard treatment, randomly assigning patients to either group. Patients may be offered the chance to take part in any of the three types of trials. Some people worry that they will receive only a placebo, such as an ineffective sugar pill, in a clinical trial, but that is very rarely done in cancer treatment clinical trials.Patients are not required to take part in clinical trials. Those who choose to, however, may gain satisfaction from helping to evaluate new treatments, and may also have access to therapies that are not yet available to the general public. Patients may leave a clinical trial at any time, for any reason. Your doctor will explain why you may or may not be a candidate for a clinical trial. For a listing of current clinical trials, see the following website: http://www.nci.nih.gov/clinical_trials/  Asparaginase  Cyclophosphamide - IV  Cyclophosphamide - Oral  Cytarabine  Daunorubicin  Doxorubicin  Doxorubicin Liposomal  Etoposide - IV  Etoposide - Oral  Mercaptopurine  Acute Myelogenous Leukemia Overview  The first phase is remission induction. Chemotherapy drugs are used to achieve a remission, the state in which there is no clinical evidence of disease and blood counts are normal. The remission induction therapy for children is generally daunorubicin
  22. 22. and cytarabine given over seven to ten days. Other drugs such as thioguanine or etoposide may be added.  Even if the central nervous system (CNS) shows no clinical evidence of disease, treatment of that area with cytarabine is also recommended. This drug is usually administered directly to the cerebrospinal fluid. Preventive chemotherapy for the CNS is commonly recommended for children. Irradiation is used to treat disease that has entered the CNS and may also be used to prevent CNS involvement.  Treatment following remission induction may be bone marrow transplantation using an HLA-matched sibling. To learn more about stem cell transplants, you may wish to order the Memorial Sloan-Kettering Cancer Center CD-ROM program Bone Marrow and Stem Cell Transplantation. Transplantation may not be recommended for the rare children who generally do well with standard treatment. In this case, they may receive intensive-dose cytarabine followed by two to six months of combination chemotherapy. Drugs that might be used to treat AML are listed at the bottom of the page. Click on the drug(s) you will be receiving to get more information. Treatment Decisions Most patients, adults and children, will be treated according to cooperative group protocols. Often, these will be clinical trials seeking to improve outcomes. Some drugs are more effective against specific subtypes of leukemia and treatments are often based on the gene mutation. The doctor will discuss this during the treatment consultation following immunophenotyping and other diagnostic tests. During Treatment Side effects during remission induction can be significant and patients usually remain in the hospital for four to six weeks. Treatment for AML is aimed at the leukemic blood cells, but normal blood cells are also affected. All patients will have lowered blood counts that lead to a variety of symptoms. Low white blood cell counts put patients at increased risk of infection. Treatment with antibiotics to prevent or treat infections is likely. In some situations, you will be in isolation and visitors to the room may need to wear gowns, masks, and gloves. Low white blood cell counts generally also lead to inflammation of the mucous membranes in the mouth and gastrointestinal tract. This is called mucositis. The tissues become very dry and may crack or bleed. Specific oral care regimens help minimize discomfort and prevent secondary infections. Mouth sores can make eating painful. The mouth sores themselves can cause pain that requires pain medication. Nausea and vomiting and diarrhea are side effects for which effective treatments exist. Hair may fall out and this is can be an especially upsetting side effect. Wigs and scarves may help minimize the distress hair loss causes. Low platelet counts lead to easy bruising and bleeding. Care to prevent injuries is very important. Low red blood cell counts causing anemia make patients feel tired and weak. Sometimes, patients are short of breath or have a fast heart rate. Transfusions will be necessary to replace red blood cells and platelets. Many patients are given stimulating factors called G-CSF or GM-CSF. These stimulate the development of new white blood cells so that cell counts are low for a shorter period of time. The initial treatment and the severity of side effects can be very difficult and wearing. While blood counts are low, your child may find it difficult to carry on normal activities. Simple exercise, such as walking, may seem overwhelming. Your doctors and nurses will do everything in their power to minimize the discomfort, sadness, and boredom that are common during this initial phase of treatment. Social workers, psychiatrists, and psychologists can help you and your child with depression and the ups and downs you will experience during treatment.
  23. 23. Social workers can also help with financial issues related to treatment. Finally, all hospitals have clergy who are available to speak to you and provide spiritual guidance. Resuming Activities After the initial hospitalization, patients will slowly begin to resume their normal activities, but school may have to be postponed for many months. Subsequent shorter hospital stays, ranging from one to two weeks, may be necessary to administer chemotherapy and treat infections that result from low white blood cell counts. Strength and stamina will return gradually. The doctors and nurses will provide more specific guidelines on what to expect based on individual treatment regimes and responses. Clinical Trials Many leukemia patients are asked to participate in a clinical trial. A clinical trial is a research program that attempts to find better treatments. A phase I clinical trial aims to find the safest way to give a new treatment. Patients are watched closely for side effects. Once the phase I trial is completed, the new treatment may be evaluated in a phase II clinical trial. At this stage the doctors are looking for anti-cancer activity, as well as side effects. After that, a phase III clinical trial will compare the new treatment to a standard treatment, randomly assigning patients to either group. Patients may be offered the chance to take part in any of the three types of trials. Some people worry that they will receive only a placebo, such as an ineffective sugar pill, in a clinical trial, but that is very rarely done in cancer treatment clinical trials. Patients are not required to take part in clinical trials. Those who choose to, however, may gain satisfaction from helping to evaluate new treatments, and may also have access to therapies that are not yet available to the general public. Patients may leave a clinical trial at any time, for any reason. Your doctor will explain why you may or may not be a candidate for a clinical trial. For a listing of current clinical trials, see the following website: http://www.nci.nih.gov/clinical_trials/  Cyclophosphamide - IV  Cyclophosphamide - Oral  Cytarabine  Daunorubicin  Etoposide - IV  Etoposide - Oral  Idarubicin  Mitoxantrone  Topotecan  Vincristine  Chronic Myelogenous Leukemia Overview Treatment for chronic myelogenous leukemia (CML) depends on which stage of disease the patient is in: the chronic phase, accelerated phase, or blast phase. Options include chemotherapy, immunotherapy, clinical trials, and bone marrow transplantation. Chronic Phase Gleevac is a drug that was licensed by the Food and Drug Administration (FDA) in June 2001. It may be recommended at the time of diagnosis. It works by decreasing the number of cells that contain the Philadelphia chromosome. Gleevac is a pill that is taken by mouth. Because it is new and it is not yet clear what the long-term benefits may be, the role of
  24. 24. bone marrow transplant has become less clear. For example, some doctors will recommend staying on Gleevac for a period of time, even if the patient has a bone marrow donor. Interferon is also considered an important drug in this disease. It too, can decrease the number of cells with the Philadelphia chromosome. However, interferon typically has more side effects. It is given by injection under the skin. Bone marrow transplantation may be recommended in the chronic phase for patients who have an appropriate donor. Accelerated Phase This is the phase of CML when the leukemic cells begin to grow more rapidly and drug treatments fail. The goal of treatment is to reduce the white blood cell count. Blast Phase Treatment during this phase is aimed at getting the patient back into the chronic phase. Options for patients in the blast stage are a combination of hydroxyurea, 6-mercaptopurine, and prednisone; daunorubicin or doxorubicin and cytosine arabinoside; anagrelide (Agrylin®); and the interferons: interferon alpha 2a and interferon alpha 2b. Drugs that might be used to treat CML are listed at the bottom of the page. Click on the drug(s) you will be receiving to get more information. Treatment Decisions The decision to attempt stem cell or bone marrow transplant will be guided by the patient's age and general health and the availability of a donor. Treatment with interferon often impacts quality-of-life and some patients may not tolerate it. However, interferon can prolong life in some patients. In the chronic phase, symptoms may be few and treatment with hydroxyurea improves these symptoms rather than extending survival. Clinical Trials Many leukemia patients are asked to participate in a clinical trial. A clinical trial is a research program that attempts to find better treatments. A phase I clinical trial aims to find the safest way to give a new treatment. Patients are watched closely for side effects. Once the phase I trial is completed, the new treatment may be evaluated in a phase II clinical trial. At this stage the doctors are looking for anti-cancer activity, as well as side effects. After that, a phase III clinical trial will compare the new treatment to a standard treatment, randomly assigning patients to either group. Patients may be offered the chance to take part in any of the three types of trials. Some people worry that they will receive only a placebo, such as an ineffective sugar pill, in a clinical trial, but that is very rarely done in cancer treatment clinical trials. Patients are not required to take part in clinical trials. Those who choose to, however, may gain satisfaction from helping to evaluate new treatments, and may also have access to therapies that are not yet available to the general public. Patients may leave a clinical trial at any time, for any reason. Your doctor will explain why you may or may not be a candidate for a clinical trial. For a listing of current clinical trials, see the following website: http://www.nci.nih.gov/clinical_trials During Treatment Treatment for CML usually does not require hospitalization, unless there are complications. The drugs are taken by mouth at home and patients are followed as outpatients.
  25. 25. Activities Patients are not generally limited in their activities during the chronic phase, but interferon immunotherapy may slow some down. The chronic phase often lasts for several years or more and patients may not have to make any changes in their lifestyle.  Cytarabine  Daunorubicin  Doxorubicin  Doxorubicin Liposomal  Hydroxyurea  Imatinib  Interferon Alpha-2A  Mercaptopurine  Vincristine Detection & Diagnosis
  26. 26. The Diagnostic Process During a physical exam the doctor will look for the outward signs of leukemia, such as swollen lymph nodes or an enlarged spleen. The doctor might suspect leukemia based on such signs, but to properly diagnose the disease and to determine the exact type of leukemia present, further tests are necessary. In most cases, the doctor will need to take blood and bone marrow samples and perform several laboratory tests on the samples. These tests can uncover important characteristics of the patient's condition, such as:  Blood counts, or the number of blood cells of each type that are found in a sample.  Morphology of the cells, or what they look like under a microscope. For example, it might be possible to determine the level of maturity and whether or not certain structures are present, which can sometimes indicate the type of leukemia present.  Immunologic properties of the cells - characteristic markers that can indicate what type of leukemia is present.  Histochemical properties of the cells - certain characteristics that can be revealed through the use of stains.  Cytogenetic properties of the cells, or chromosomal and genetic abnormalities. Results for some tests are available very quickly, while others take a few days or more. Some of these tests, because they reveal specific characteristics of the disease, are also helpful in determining the best course of treatment. Some are also used throughout the course of treatment to monitor a patient's response to therapy and susceptibility to infection. Some questions to ask the doctor about procedures:  What will this procedure reveal about my condition?  Do I need to do anything to prepare?  What can I expect during the procedure? Will it be painful or uncomfortable? Will I need any pain medication?  How much recovery time is needed? Are there any known side effects?

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