Medical Treatments for Chronic Lymphocytic Leukemia in Adults

NYU Langone doctors can often effectively manage chronic lymphocytic leukemia (CLL) using a combination of medical therapies. The result can be remission, when the signs and symptoms of a disease disappear. This means that the amount of cancerous lymphocytes is reduced so significantly that these cells are no longer detectable in the blood and bone marrow.

Doctors cannot predict how long remission may last, but treatment is sometimes not needed again for many years. Because the condition is likely to return eventually, people with chronic lymphocytic leukemia require ongoing monitoring and care.

Our doctors make decisions about the type of therapy you should receive and how often you should receive it based on the genetic features of the cancer cells, which are identified in diagnostic tests. Typically, people with chronic lymphocytic leukemia are treated with a combination of therapies.


Chemotherapy, in which drugs are used to destroy cancer cells throughout the body, is usually given through a needle into a vein in an intravenous (IV) infusion or by mouth. Treatment schedules vary, but chemotherapy is often given several days a week for three or four weeks in a row. Several of these treatment cycles are usually needed, each followed by a break or rest period.

Some of the chemotherapy drugs traditionally used to treat chronic lymphocytic leukemia include fludarabine, cyclophosphamide, and dexamethasone. Newer medications, such as bendamustine, may also be an option.

Targeted Medications

Your doctor may recommend combining chemotherapy drugs with a targeted medication called rituximab, which is in a class of medications called monoclonal antibodies. These medications consist of antibodies or immune system proteins that are designed to attack the B lymphocytes, which are the type of cells that have transformed to chronic lymphocytic leukemia. They attach to proteins found on the surface of chronic lymphocytic leukemia cells, interfering with their ability to function. Because monoclonal antibodies target only cancer cells and not healthy tissue, they may cause fewer side effects than chemotherapy.

Rituximab is usually given through an IV and often combined with chemotherapy, although it may be used alone when chronic lymphocytic leukemia does not respond to or returns after combination therapy. Recently, two other monoclonal antibodies have been shown to work even after rituximab has failed. They are ofatumimab and obinutuzimab. 

Idelalisib is another targeted therapy that your doctor may prescribe with rituximab if the cancer does not respond to or comes back after other therapy. Idelalisib blocks an enzyme found in chronic lymphocytic leukemia cells that is responsible for the cells’ division and growth. Another medication that may be used is ibrutinib, which blocks a different enzyme necessary for chronic lymphocytic cells to survive. Both of these medications are taken as pills, and while new, offer people with chronic lymphocytic leukemia additional therapeutic options.

Immunomodulating Medications

Immunomodulating medications affect how the immune system functions, making it difficult for chronic lymphocytic leukemia cells to survive, reproduce, or cause symptoms. These medicines, which include lenalidomide and thalidomide, are often taken by mouth daily for several weeks, followed by a week-long break to let the body rest.

Side Effects

Side effects of medications used to treat chronic lymphocytic leukemia may include nausea, vomiting, diarrhea, mouth sores, headache, fever, or stomach pain. If you experience these side effects, our doctors can adjust the dosing of these medications, prescribe others, or refer you to our integrative health services, where specialists can help you to manage the side effects of treatment.

Clinical Trials

Doctors at NYU Langone are evaluating new medications for the treatment of chronic lymphocytic leukemia in clinical trials—studies that test the effectiveness of new therapies. Because these studies are designed to answer specific research questions, they may not provide appropriate treatment options for every person. Your doctor can discuss with you whether you are a candidate for a trial and if so how to enroll in it.

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