Saturday, September 15, 2007

Clinical Trials

Many patients with leukemia take part in clinical trials (treatment studies). Clinical trials help doctors find out whether a new treatment is both safe and effective. They also help doctors answer questions about how the treatment works and what side effects it causes.

Patients who take part in studies may be among the first to receive treatments that have shown promise in research. In many studies, some of the patients receive the new treatment, while others receive standard treatment so that doctors can compare different treatments. Patients who take part in a trial make an important contribution to medical science. Although these patients take certain risks, they may have the first chance to benefit from improved treatment methods.

Doctors are studying new treatments for all types of leukemia. They are working on new drugs, new drug combinations, and new schedules of chemotherapy. They also are studying ways to improve bone marrow transplantation.

Many clinical trials involve various forms of biological therapy. Interleukins and colony-stimulating factors are forms of biological therapy being studied to treat leukemia. Doctors also are studying ways to use monoclonal antibodies in the treatment of leukemia. Often biological therapy is combined with chemotherapy or bone marrow transplantation.

Patients with leukemia (or their families) should talk with the doctor if they are interested in taking part in a clinical trial. They may want to read Taking Part in Clinical Trials: What Cancer Patients Need To Know, which explains some of the possible benefits and risks of treatment studies.

One way to learn about clinical trials is through PDQ, a computerized resource developed by the National Cancer Institute. PDQ contains information about cancer treatment and about clinical trials in progress throughout the country. The Cancer Information Service can provide PDQ information to doctors, patients, and the public.


Treatment for leukemia is complex. It varies with the type of leukemia and is not the same for all patients. The doctor plans the treatment to fit each patient's needs. The treatment depends not only on the type of leukemia, but also on certain features of the leukemia cells, the extent of the disease, and whether the leukemia has been treated before. It also depends on the patient's age, symptoms, and general health.

Whenever possible, patients should be treated at a medical center that has doctors who have experience in treating leukemia. If this is not possible, the patient's doctor should discuss the treatment plan with a specialist at such a center. Also, patients and their doctors can call the Cancer Information Service to request up-to-date treatment information from the National Cancer Institute's PDQ database.

Acute leukemia needs to be treated right away. The goal of treatment is to bring about a remission. Then, when there is no evidence of the disease, more therapy may be given to prevent a relapse. Many people with acute leukemia can be cured.

Chronic leukemia patients who do not have symptoms may not require immediate treatment. However, they should have frequent checkups so the doctor can see whether the disease is progressing. When treatment is needed, it can often control the disease and its symptoms. However, chronic leukemia can seldom be cured.

Many patients and their families want to learn all they can about leukemia and the treatment choices so they can take an active part in decisions about medical care. The doctor is the best person to answer these questions. When discussing treatment, the patient (or, in the case of a child, the patient's family) may want to talk with the doctor about research studies of new treatment methods. Such studies, called clinical trials, are designed to improve cancer treatment. More information about clinical trials is in the Clinical Trials section.

When a person is diagnosed with leukemia, shock and stress are natural reactions. These feelings may make it difficult to think of every question to ask the doctor. Also, patients may find it hard to remember everything the doctor says.

Often, it helps to make a list of questions to ask the doctor. Taking notes or, if the doctor agrees, using a tape recorder can make it easier to remember the answers. Some people find that it also helps to have a family member or friend with them -- to take part in the discussion, to take notes, or just to listen. Patients do not need to ask all their questions or remember all the answers at one time. They will have other chances for the doctor to explain things that are not clear and to ask for more information.

Here are some questions patients and their families may want to ask the doctor before treatment begins:

  • What type of leukemia is it?
  • What are the treatment choices? Which do you recommend? Why?
  • Would a clinical trial be appropriate?
  • What are the expected benefits of each kind of treatment?
  • What are the risks and possible side effects of each treatment?
  • If I have pain, how will you help me?
  • Will I have to change my normal activities?
  • How long will treatment last?
  • What is the treatment likely to cost? How can I find out what my insurance will cover?

Getting a Second Opinion

Sometimes it is helpful to have a second opinion about the diagnosis and treatment plan. (Many insurance companies provide coverage for a second opinion.) There are a number of ways to find a doctor who can give a second opinion:

  • The patient's doctor may be able to suggest a doctor who specializes in adult or childhood leukemia. Doctors who treat adult leukemia are oncologists and hematologists. Pediatric oncologists and hematologists treat childhood leukemia.
  • The Cancer Information Service, at 1-800-4-CANCER, can tell callers about cancer centers and other treatment facilities in their area, including programs that are supported by the National Cancer Institute.
  • Patients can get the names of specialists from their local medical society, a nearby hospital, or a medical school.

Methods of Treatment

Most patients with leukemia are treated with chemotherapy. Some also may have radiation therapy and/or bone marrow transplantation (BMT) or biological therapy. In some cases, surgery to remove the spleen (an operation called a splenectomy) may be part of the treatment plan.

Chemotherapy is the use of drugs to kill cancer cells. Depending on the type of leukemia, patients may receive a single drug or a combination of two or more drugs.

Some anticancer drugs can be taken by mouth. Most are given by IV injection (injected into a vein). Often, patients who need to have many IV treatments receive the drugs through a catheter.

One end of this thin, flexible tube is placed in a large vein, often in the upper chest. Drugs are injected into the catheter, rather than directly into a vein, to avoid the discomfort of repeated injections and injury to the skin.

Anticancer drugs given by IV injection or taken by mouth enter the bloodstream and affect leukemia cells in most parts of the body. However, the drugs often do not reach cells in the central nervous system because they are stopped by the blood-brain barrier. This protective barrier is formed by a network of blood vessels that filter blood going to the brain and spinal cord. To reach leukemia cells in the central nervous system, doctors use intrathecal chemotherapy. In this type of treatment, anticancer drugs are injected directly into the cerebrospinal fluid.

Intrathecal chemotherapy can be given in two ways. Some patients receive the drugs by injection into the lower part of the spinal column. Others, especially children, receive intrathecal chemotherapy through a special type of catheter called an Ommaya reservoir. This device is placed under the scalp, where it provides a pathway to the cerebrospinal fluid. Injecting anticancer drugs into the reservoir instead of into the spinal column can make intrathecal chemotherapy easier and more comfortable for the patient.

Chemotherapy is given in cycles: a treatment period followed by a recovery period, then another treatment period, and so on. In some cases, the patient has chemotherapy as an outpatient at the hospital, at the doctor's office, or at home. However, depending on which drugs are given and the patient's general health, a hospital stay may be necessary.

Here are some questions patients and their families may want to ask the doctor before starting chemotherapy:

  • What drugs will be used?
  • When will the treatments begin? How often will they be given? When will they end?
  • Will I have to stay in the hospital?
  • How will we know whether the drugs are working?
  • What side effects occur during treatment? How long do the side effects last? What can be done to manage them?
  • Can these drugs cause side effects later on?

Radiation therapy is used along with chemotherapy for some kinds of leukemia. Radiation therapy (also called Radiotherapy) uses high-energy rays to damage cancer cells and stop them from growing. The radiation comes from a large machine.

Radiation therapy for leukemia may be given in two ways. For some patients, the doctor may direct the radiation to one specific area of the body where there is a collection of leukemia cells, such as the spleen or testicles. Other patients may receive radiation that is directed to the whole body. This type of radiation therapy, called total-body irradiation, usually is given before a bone marrow transplant.

Here are some questions patients and their families may want to ask the doctor before having radiation therapy:

  • When will the treatments begin? How often are they given? When will they end?
  • Can normal activities be continued?
  • How will we know if the treatment is working?
  • What side effects can be expected? How long will they last? What can be done about them?
  • Can radiation therapy cause side effects later on?

Bone marrow transplantation also may be used for some patients. The patient's leukemia-producing bone marrow is destroyed by high doses of drugs and radiation and is then replaced by healthy bone marrow. The healthy bone marrow may come from a donor, or it may be marrow that has been removed from the patient and stored before the high-dose treatment. If the patient's own bone marrow is used, it may first be treated outside the body to remove leukemia cells. Patients who have a bone marrow transplant usually stay in the hospital for several weeks. Until the transplanted bone marrow begins to produce enough white blood cells, patients have to be carefully protected from infection. Research Report: Bone Marrow Transplantation provides more information about this complex treatment.

Here are some questions patients and their families may want to ask the doctor about bone marrow transplantation:

  • What are the benefits of this treatment?
  • What are the risks and side effects? What can be done about them?
  • How long will I be in the hospital? What care will be needed after I leave the hospital?
  • What changes in normal activities will be necessary?
  • How will we know if the treatment is working?

Biological therapy involves treatment with substances that affect the immune system's response to cancer. Interferon is a form of biological therapy that is used against some types of leukemia.

Here are some questions patients and their families may want to ask the doctor before starting biological therapy:

  • What kind of treatment will be used?
  • What side effects can be expected? How long do the side effects last? What can be done to manage them?
  • How will we know whether the treatment is working?