Chronic lymphocytic leukemia (CLL) is a type of cancer that affects adults and is the most common form of Leukemia in this age group. It is typically seen in older individuals and is characterized by an abnormal increase in the number of mature B lymphocytes (a type of white blood cell) found in the blood, bone marrow and lymphoid tissues. As the numbers of CLL cells increase, they interfere with the normal functioning of the immune system. Although CLL is usually a slow growing cancer, at some stage it will require treatment as it starts to cause problems for the patient. Over the last decade, the management of CLL has changed with the introduction of targeted therapy for CLL. This treatment is designed to interfere with specific processes that support the growth and survival of cancer cells, offering additional options for patient care.

Understanding Targeted Therapy for CLL

Targeted therapies work differently than chemotherapy, targeting the particular proteins that are involved in the development of CLL and in the survival of the CLL cells. As each cancer cell has the ability to grow and divide in a unique way, targeted therapies attempt to strike that balance between destroying cancer cells and leaving most normal cells intact. For CLL, several of the most well known targeted therapies are those that interfere with the action of two particular proteins: BTK and BCL-2. These therapies are used to treat patients newly diagnosed with CLL, as well as patients with relapsed disease after prior treatment.

When Is Treatment Needed?

Just because someone has CLL does not mean that they need to start treatment right away. Many people with early stage CLL are put on active surveillance or “watch and wait” for their disease. The goal of active surveillance or “watch and wait” is to determine when a person with CLL would benefit from treatment for their disease. 

Treatment may be recommended when patients develop:

• Progressive anemia
• Low platelet counts
• Enlarging lymph nodes or spleen
• Persistent fatigue
• Frequent infections
• Night sweats
• Unexplained weight loss
• Rapidly increasing lymphocyte counts

Whether to start or to continue a treatment can be decided on the basis of the development of the disease and the symptoms of the patient and not on the basis of the diagnosis alone.

Types of Targeted Therapy Used in CLL

There are several different types of targeted therapies used to treat people with CLL.

BTK Inhibitors

BTK inhibitors are approved for use in many patients with CLL and act by targeting Bruton’s tyrosine kinase, an enzyme involved in the growth and survival of CLL cells.

BCL-2 Inhibitors

These drugs work by targeting apoptosis, a natural process of programmed cell death, which is often disrupted in cancer cells. BCL-2 inhibitors may be used alone or in combination with other treatments, depending on individual patient factors, and act by influencing pathways that help cancer cells avoid cell death.

Monoclonal Antibodies

The final class of targeted therapies are monoclonal antibodies. These medications are designed to bind to a particular protein on the surface of CLL cells, and then tag the leukemia cells for destruction by the body’s immune system. These agents can be used alone or in combination with other targeted agents.

Benefits of Targeted Therapy for CLL

Targeted therapy for CLL differs from chemotherapy and is used in various treatment approaches based on clinical factors.

Potential benefits include:

• More precise treatment of leukemia cells
• Effective disease control
• Improved progression-free survival
• Oral treatment options for many patients
• Reduced reliance on chemotherapy in appropriate patients
• Personalized treatment strategies
• Better quality of life for many individuals

Not all patients respond in the same way to the new treatments, and therapy decisions are typically made on an individual basis.

Factors That Influence Treatment Selection

There are many factors that need to be considered when choosing to use targeted therapy.

These may include:

• Patient age
• Overall health
• Kidney and liver function
• Genetic abnormalities
• TP53 mutation or deletion status
• IGHV mutation status
• Previous treatments
• Other medical conditions
• Potential drug interactions
• Patient preferences

Comprehensive evaluation is required before starting the treatment to choose the ideal treatment approach.

Monitoring During Treatment

Managing CLL includes regular follow-up to assess treatment effectiveness and early identification of potential side effects.

Routine assessments may include:

• Complete blood counts
• Physical examinations
• Kidney and liver function tests
• Assessment of lymph node size
• Monitoring for infections
• Evaluation of treatment response
• Review of medication tolerance

Even if you feel well, it is important to attend all scheduled follow-up appointments.

Possible Side Effects

Just like any cancer treatment, the targeted therapy has side effects that affect most patients, but they are usually quite tolerable.

Common side effects may include:

• Fatigue
• Diarrhea
• Nausea
• Bruising
• Mild bleeding
• Headache
• Muscle or joint pain
• Skin rash
• Increased risk of infection
• Low blood cell counts

Report any unusual bleeding, persistent fever, severe infections, or new symptoms to your healthcare provider.

Supporting Patients Throughout Treatment

Effective CLL management involves more than treatment with medications. It also requires emotional support, promoting healthy lifestyle habits, and good communication with patients’ health care providers.

Patients and caregivers can help by:

• Taking medications exactly as prescribed
• Keeping follow-up appointments
• Reporting side effects early
• Maintaining a balanced diet
• Staying physically active 
• Seeking emotional support when needed
• Discussing concerns openly with the healthcare team

Family members can also play a crucial role when supporting a CLL patient by helping with treatments, doctor’s appointments, and daily activities.

Looking Ahead

Recent developments in targeted therapy for CLL have expanded the range of available treatment options, and ongoing clinical studies continue to evaluate new therapies that focus on specific disease pathways while aiming to limit unnecessary side effects. In collaboration with an experienced healthcare provider, treatment decisions can be tailored to each patient’s individual disease characteristics, clinical condition, and personal preferences, with the goal of managing CLL effectively and supporting overall quality of life.

Stay updated, free articles. Join our Telegram channel

Join