Chronic Lymphocytic Leukemia Treatment Option Overview

There are different types of treatment for patients with chronic lymphocytic leukemia (CLL).

Different types of treatment are available for patients with CLL. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The following types of treatment are used:

Watchful waiting

Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. This is also called observation. Watchful waiting is used to treat asymptomatic and symptomatic or progressive CLL.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Different types of targeted therapy are used to treat CLL:

• Tyrosine kinase inhibitor (TKI) therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to develop into more white blood cells than the body needs. Ibrutinib, acalabrutinib, zanubrutinib, and duvelisib are TKIs used to treat symptomatic or progressive, recurrent, or refractory CLL.
• BCL2 inhibitor therapy: This treatment blocks a protein called BCL2, which is found on some leukemia cells. This may kill leukemia cells and make them more sensitive to other anticancer drugs. Venetoclax is a type of BCL2 therapy used to treat symptomatic or progressive, recurrent, or refractory CLL.
• Monoclonal antibody therapy: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells. Rituximab, ofatumumab, obinutuzumab, and alemtuzumab alone and in combination with chemotherapy are used to treat symptomatic or progressive, recurrent, or refractory CLL.

See Drugs Approved for Chronic Lymphocytic Leukemia for more information.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). Combination chemotherapy is treatment using more than one anticancer drug.

See Drugs Approved for Chronic Lymphocytic Leukemia for more information.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer, such as a group of lymph nodes or the spleen. This treatment may be used to reduce pain related to a swollen spleen or lymph nodes.

Immunotherapy

Immunotherapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer.

• Immunomodulating agent: Lenalidomide stimulates T cells to kill leukemia cells. It may be used alone or with rituximab in patients with symptomatic or progressive, recurrent, or refractory CLL.
• CAR T-cell therapy: This treatment changes the patient's T cells (a type of immune system cell) so they will attack certain proteins on the surface of cancer cells. T cells are taken from the patient and special receptors are added to their surface in the laboratory. The changed cells are called chimeric antigen receptor (CAR) T cells. The CAR T cells are grown in the laboratory and given to the patient by infusion. The CAR T cells multiply in the patient's blood and attack cancer cells. CAR T-cell therapy is being studied in the treatment of recurrent or refractory CLL.

Chemotherapy with bone marrow or peripheral blood stem cell transplant

Chemotherapy is given to kill cancer cells. Healthy cells, including blood-forming cells, are destroyed by the cancer treatment. A bone marrow or peripheral blood stem cell transplant are treatments to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI website.

Treatment for chronic lymphocytic leukemia may cause side effects.

For information about side effects caused by treatment for cancer, visit our Side Effects page.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

As you go through treatment, you will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back).