Treatment Options for Chronic Lymphocytic Leukemia

Indications for Treatment of Chronic Lymphocytic Leukemia

Because CLL is a slowly progressive type of leukemia, many patients do not need treatment for years until the number of lymphocytes begins to increase, the lymph nodes begin to enlarge, or other clinical symptoms such as splenomegaly (enlargement of the spleen) occur.

Many patients with chronic lymphocytic leukemia are diagnosed in the early stages of the disease with only evidence of lymphocytosis but without clinical involvement of other organ systems such as lymph nodes, spleen, and liver or other hematological abnormalities such as anemia and/or thrombocytopenia. Patients in the early stages of CLL can remain stable for years without treatment before the disease progresses to the next stage. This approach to treatment is called "watchful waiting" and is appropriate for many patients with early-stage CLL who are asymptomatic (have no symptoms of the disease). The "watchful waiting" strategy is based upon the findings from previous studies that early treatment of asymptomatic CLL patients does not confer any survival advantage compared to no treatment.

In 1996, the National Cancer Institute (NCI) Working Group for Chronic Lymphocytic Leukemia published revised guidelines for the diagnosis and treatment of CLL. These guidelines still serve as a basis for the clinical management of patients with CLL. One of the issues addressed by the NCI Working Group is when to initiate treatment for patients with CLL. According to the NCI Working Group, indications for initiation of treatment for CLL include:

  • Patients with progressive bone marrow failure who develop anemia (low red blood cell counts) and/or thrombocytopenia (low platelet counts). These patients correspond to Rai Stages III and IV and Binet Stage C.

  • Patients with rapidly progressive lymphocytosis:

    • 50% or greater increase in lymphocytes over a period of 2 months
    • lymphocyte doubling time in less than 6 months

  • Patients with progressive enlargement of either:

    • spleen (splenomegaly)
    • liver (hepatomegaly)
    • lymph nodes (lymphadenopathy)

  • Patients who develop autoimmune hemolytic anemia

  • Patients who develop CLL-related symptoms, known as B symptoms, such as:

    • fatigue and generalized weakness
    • unintentional weight loss of 10% or more of body weight within a 6-month period
    • profuse night sweats
    • fever lasting longer than 2 weeks