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This section outlines treatments that are the standard of care (the best proven treatments available) for this specific type of cancer. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new treatment to evaluate whether it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options.
In cancer care, different types of doctors often work together to create a patient's overall treatment plan that combines different types of treatments. This is called a multidisciplinary team.
Descriptions of the most common treatment options for ALL are listed below. Treatment options and recommendations depend on several factors, including the subtype of ALL, its classification, possible side effects, and the patient's preferences and overall health.
Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells' ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time.
Patients with ALL receive several different drugs throughout their treatment, some of which are given orally (by mouth) as a pill, while others are injected into a vein (intravenously, IV). A patient may receive chemotherapy during different stages of treatment:
Remission Induction therapy. This is the first round of treatment given during the first three to four weeks after diagnosis. It is designed to destroy most of the leukemia cells, stop symptoms of the disease, and return the blood counts to normal levels.
The specific treatments used may include:
Doxorubicin (Adriamycin), cyclophosphamide (Cytoxan, Neosar),or vincristine (Oncovin, Vincasar), given by an injection into a vein
Asparaginase (Elspar), given by injection into a muscle, under the skin or into a vein
Dexamethasone (multiple brand names) or prednisone (multiple brand names) by mouth
Methotrexate (multiple brand names) or cytarabine (Cytosar-U) as an injection into the spinal fluid
The goal of induction therapy is a complete remission (CR), which means that the blood counts have returned to normal, the leukemia cannot be seen when a bone marrow sample is examined under the microscope, and the signs and symptoms of the ALL are gone. More than 95% of children and 75% to 80% of adults with ALL will have a CR. However, small amounts of leukemia can remain after treatment even if it cannot be found, and it is necessary to give additional therapy to prevent the ALL from coming back.
Remission Consolidation or Intensification therapy. This stage of therapy refers to the use of different drugs given in doses similar to or higher than the doses used to achieve remission. Depending on the subtype of the ALL, the doctor may recommend several courses of consolidation therapy.
Remission Maintenance or Continuation therapy. Treatment is given both orally and intravenously over a two-year to three-year period to keep the ALL from returning. These drugs are usually given in lower doses and have fewer side effects.
Re-induction chemotherapy. This stage is used to treat ALL if it has come back after treatment.
Central nervous system (CNS) prophylaxis (preventive treatment). This is the use of drugs, given directly in the spinal fluid by spinal tap and/or by vein, to prevent the leukemia from spreading from the blood to the brain or spinal cord. This treatment is often given in combination with radiation therapy (see below) to the head.
Side effects of chemotherapy and supportive treatment
Induction therapy usually begins in the hospital, and patients will often need to stay in the hospital for three to four weeks during treatment. However, depending on the situation, many patients can leave the hospital and are monitored closely with regular visits to the doctor. Sometimes, patients will need to stay in the hospital for consolidation therapy, but most patients are able to go home. Many patients with ALL can return to school or work while receiving maintenance therapy.
Because chemotherapy attacks rapidly dividing cells, including those in normal tissue such as the hair, lining of the mouth, intestines, and bone marrow, patients receiving chemotherapy may lose their hair, develop mouth sores, or have nausea and vomiting. Because of changes in the blood counts, most patients will need transfusions of red blood cells and platelets at some point during their treatment. Treatment with antibiotics to prevent or treat infection is usually needed as well. Chemotherapy may lower the body's resistance to infection by reducing the number of neutrophils, lead to increased bruising and bleeding because of the decrease in the number of platelets and other problems with blood clotting, and cause fatigue by lowering the number of red blood cells.
Chemotherapy may affect fertility (ability to have a child in the future) and increase the risk of developing a second cancer. Patients may want to talk with a fertility specialist before treatment begins.
The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications.
Targeted therapy is a treatment that targets the cancer's specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to normal cells.
Recent studies show that not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors involved in your leukemia. As a result, doctors can better match each patient with the most effective treatment whenever possible. In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them.
For ALL, targeted therapy is recommended in addition to standard chemotherapy for patients with Philadelphia chromosome-positive ALL (Ph+ ALL). Such drugs include imatinib (Gleevec), dasatinib (Sprycel), and nilotinib (Tasigna) for Philadelphia chromosome-positive ALL. Nelarabine (Arranon) is a new drug that targets T-cell ALL. Monoclonal antibodies such as rituximab (Rituxan) target B-cell ALL. Talk with your doctor about possible side effects for a specific medication and how they can be managed.
Radiation therapy is the use of high-energy x-rays to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. A radiation therapy regimen usually consists of a specific number of treatments given over a set period of time. For ALL, radiation therapy to the brain is sometimes used to kill cancerous cells around the brain and spinal column.
Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished.
Stem cell transplantation/bone marrow transplantation
A stem cell transplant is a medical procedure in which diseased bone marrow is destroyed and then replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. These stem cells make all of the normal cells in the blood. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.
Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and patient's age and general health.
There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). However, AUTO transplants are generally not used to treat ALL.
In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible.
Leukemia and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the disease, an important part of care is relieving a person's symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.
Palliative care can help a person at any stage of illness. People often receive treatment for the leukemia and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.
Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and supportive care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem, so it is addressed as quickly as possible.
A remission is when the leukemia cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED.
A remission can be temporary or permanent. This uncertainty leads to many survivors feeling worried or anxious that the disease will come back. While many remissions are permanent, it's important to talk with your doctor about the possibility of the leukemia returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the disease does return.
If the leukemia does return after the original treatment, it is called recurrent or relapsed leukemia. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the therapies described above (such as chemotherapy, targeted therapy, and radiation therapy) but may be used in a different combination or given at a different pace. Your doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent leukemia.
Treatment for recurrent ALL depends on the length of the remission and is usually given in cycles for two to three years. If a recurrence occurs after a long remission, the leukemia may respond again to the original treatment. If the remission was short, then other drugs are used, often in the form of new drugs being tested in clinical trials. An ALLO stem cell transplant is generally recommended to patients whose leukemia has come back. Supportive care will also be important to help relieve symptoms and side effects.
People with recurrent leukemia often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope.
Refractory ALL occurs when a complete remission is not achieved because the drugs did not kill enough leukemia cells. These patients often continue to have low blood counts, need transfusions, and have a risk of bleeding or infection.
Patients with this diagnosis are encouraged to talk with doctors who are experienced in treating this type of leukemia, because there can be different opinions about the best treatment plan. Learn more about seeking a second opinion before starting treatment, so you are comfortable with the treatment plan chosen. This discussion may include clinical trials.
Your health care team may recommend a treatment plan that includes new drugs being tested in clinical trials or ALLO stem cell transplantation. Supportive care will also be important to help relieve symptoms and side effects.
For many patients, a diagnosis of refractory leukemia can be very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.
If treatment fails
Recovery from leukemia is not always possible. If treatment is not successful, the disease may be called advanced or terminal leukemia.
This diagnosis is stressful because the disease is not curable, and it may be difficult to discuss. However, it is important to have open and honest conversations with your doctor and health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.
Palliative care given toward the end of a person's life is called hospice care. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families.
Doctors and scientists are always looking for better ways to treat patients with ALL. To make scientific advances, doctors create research studies involving people, called clinical trials.
Many clinical trials are focused on new treatments, evaluating whether a new treatment is safe, effective, and possibly better than the current (standard) treatment. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment. Patients who participate in clinical trials are often among the first to receive new treatments, such as new chemotherapy before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.
There are also clinical trials that study new ways to ease symptoms and side effects during treatment and manage the late effects that may occur after treatment. Talk with your doctor about clinical trials regarding side effects. In addition, there are ongoing studies about ways to prevent the disease.
Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating ALL. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with ALL.
Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials is rare. When a placebo is used in a study, it is done with the full knowledge of the participants.
To join a clinical trial, patients must participate in a process known as informed consent. During informed consent, the doctor should list all of the patient's options, so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.
Coping with Side Effects
Fear of treatment side effects is common after a diagnosis of leukemia, but it may help to know that preventing and controlling side effects is a major focus of your health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.
Common side effects from each treatment option for ALL are described in detail within the Treatment section. Side effects depend on a variety of factors, including the subtype and classification, the length and dosage of treatment(s), and your overall health.
Before treatment begins, talk with your doctor about possible side effects of each type of treatment you will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of care giving you may need during treatment and recovery, as family members and friends often play an important role in the care of a person with ALL.
In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies. During and after treatment, be sure to tell the health care team about the side effects you experience, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care.
After treatment for ALL ends, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your recovery for the coming months and years.
It's very important for people in remission to receive regular follow-up examinations for several years to watch for early signs of recurrence or late effects of chemotherapy.
People recovering from ALL are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, not smoking, eating a balanced diet, and having recommended cancer screening tests. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level.
Doctors are working to learn more about ALL, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Most cancer centers are actively involved in clinical trials aimed at increasing the rate of cure from ALL. Always talk with your doctor about the diagnostic and treatment options best for you.
New drugs and treatment regimens. Researchers are looking at the use of new types of chemotherapy, as well as different schedules and doses of current drugs. The drug clofarabine (Clolar) has been approved for children with recurrent ALL and is being researched for adults with ALL. In addition, several studies have shown that adolescents and younger to middle-aged adults benefit from the more intensive treatments most often used for younger children with ALL.
Immunotherapy. Immunotherapy (also called biologic therapy) is designed to boost the body's natural defenses to fight the cancer. It uses materials either made by the body of in a laboratory to bolster, target, or restore immune system function. Researchers are studying the use of different antibodies directed against ALL cells.
Stem cell/bone marrow transplantation. Different ways to make stem cell transplantation safer and easier are also being studied.
Tests to detect small amounts of ALL after treatment. Researchers are studying other molecular or immunologic tests that can help find small amounts of ALL in patients in remission. Then, a change in treatment may prevent the ALL from coming back.
Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current ALL treatments in order to improve patients' comfort and quality of life.