Leukemia - B-cell Prolymphocytic Leukemia and Hairy Cell
This section outlines treatments that are the standard of care (the best proven treatments available) for this specific type of cancer. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new treatment to evaluate whether it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options.
In cancer care, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team.
The goal of treatment for leukemia is remission (absence of cancer symptoms; also called “no evidence of disease” or NED). Descriptions of the most common treatment options for B-cell leukemia are listed below. Treatment options and recommendations depend on several factors, including the type of leukemia, possible side effects, and the patient’s preferences and overall health.
Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.
Drugs that are used to treat HCL include cladribine (Leustatin) and pentostatin (Nipent). For most people with HCL, both cladribine and pentostatin can cause remissions that last for many years and are the standard initial treatment for the disease.
PLL is treated with combination chemotherapy, meaning more than one drug is used. Several regimens may be used. The regimens are chosen by the type of chemotherapy the patient may have received previously and the possible side effects.
The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.
The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications.
Immunotherapy (also called biologic therapy) is designed to boost the body's natural defenses to fight the cancer. It uses materials made either by the body or in a laboratory to bolster, target, or restore immune system function. Immunotherapy for HCL includes recombinant interferon alpha (Alferon, Intron A, Roferon-A).
Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to normal cells.
Recent studies show that not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors involved in your leukemia. As a result, doctors can better match each patient with the most effective treatment whenever possible. In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them.
Monoclonal antibodies are a type of targeted therapy that target specific proteins on the surface of the cancer cell. Rituximab (Rituxan) is an antibody directed against the surface protein CD20. BL22 immunotoxin is an antibody that is designed to attach to the surface protein CD22 and deliver a toxin to the cancer cell. Both of these have been used to treat HCL that did not go into remission after initial therapy. However, BL22 is not approved by the U.S. Food and Drug Administration (FDA) for the treatment of PLL and is only available in clinical trials. Alemtuzumab (Campath) is an antibody that targets the surface protein CD52 and has been used to treat PLL. Talk with your doctor about possible side effects for a specific medication and how they can be managed.
Surgery to remove the spleen (splenectomy), which also makes white blood cells, may be done for some patients. A surgical oncologist is a doctor who specializes in treating cancer using surgery.
Stem cell transplantation/bone marrow transplantation
A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.
Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of leukemia, results of any previous treatment, and patient’s age and general health. Transplantation is a higher-risk procedure. It is not used often for people with B-cell leukemia because it is not always an effective therapy for these diseases and because many patients with B-cell leukemia are older and the risks of the procedure are higher.
There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO transplantation is sometimes considered for younger patients with PLL when chemotherapy was not effective. However, AUTO transplantation is rarely considered for patients with PLL or HCL.
In both types, the goal of transplantation is to destroy disease cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible.
Leukemia and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the disease, an important part of care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.
Palliative care can help a person at any stage of illness. People often receive treatment for the leukemia and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.
Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and supportive care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem, so it is addressed as quickly as possible.
Recurrent B-cell leukemia
A remission is when leukemia cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED.
A remission can be temporary or permanent. This uncertainty leads to many survivors feeling worried or anxious that the disease will come back. While many remissions are permanent, it’s important to talk with your doctor about the possibility of the leukemia returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the disease does return.
If the leukemia does return after the original treatment, it is called recurrent leukemia. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the therapies described above (such as chemotherapy, immunotherapy, targeted therapy, and stem cell transplantation) but may be used in a different combination or given at a different pace. For instance, cladribine and pentostatin can work again to treat HCL if it recurs after a long period of remission. Your doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent leukemia.
People with recurrent leukemia often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope.
If the leukemia continues to worsen despite treatment, it is called refractory or resistant leukemia.
Patients with this diagnosis are encouraged to talk with doctors who are experienced in treating this type of leukemia, because there can be different opinions about the best treatment plan.
For HCL, your health care team may recommend a treatment plan that includes rituximab and/or BL22 immunotoxin. Supportive care will also be important to help relieve symptoms and side effects.
For many patients, a diagnosis of refractory leukemia can be very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.
Doctors and scientists are always looking for better ways to treat patients with B-cell leukemia. To make scientific advances, doctors create research studies involving people, called clinical trials.
Many clinical trials are focused on new treatments, evaluating whether a new treatment is safe, effective, and possibly better than the current (standard) treatment. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment. Patients who participate in clinical trials are often among the first to receive new treatments before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.
There are also clinical trials that study new ways to ease symptoms and side effects during treatment and manage the late effects that may occur after treatment. Talk with your doctor about clinical trials regarding side effects. In addition, there are ongoing studies about ways to prevent the disease.
Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating B-cell leukemia. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with B-cell leukemia.
Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials is rare. When a placebo is used in a study, it is done with the full knowledge of the participants.
To join a clinical trial, patients must participate in a process known as informed consent. During informed consent, the doctor should list all of the patient’s options so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.
Patients who participate in a clinical trial may stop participating at any time for any personal or medical reason. This may include that the new treatment is not working or there are serious side effects. Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that patients participating in a clinical trial talk with their doctor and researchers about who will be providing their treatment and care during the clinical trial, after the clinical trial ends, and/or if the patient chooses to leave the clinical trials before it ends.
Fear of treatment side effects is common after a diagnosis of leukemia, but it may help to know that preventing and controlling side effects is a major focus of your health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.
Common side effects from each treatment option for leukemia are described in detail within the Treatment section. Side effects depend on a variety of factors, including the length and dosage of treatment(s), and your overall health.
Before treatment begins, talk with your doctor about possible side effects of each type of treatment you will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving you may need during treatment and recovery, as family members and friends often play an important role in the care of a person with leukemia.
In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies.
During and after treatment, be sure to tell the health care team about the side effects you experience, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care.
After treatment for B-cell leukemia ends, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations, blood tests, bone marrow biopsies, and possibly scans or other imaging tests to monitor your recovery for the coming months and years.
Several long-term side effects from leukemia treatment may occur:
Lung damage from certain drugs or radiation therapy to the lungs.
A weakened heart muscle from higher doses of doxorubicin (Adriamycin) or radiation therapy to the chest.
Infertility (inability to have a child) or premature menopause from high-dose cyclophosphamide (Cytoxan, Neosar) or other chemotherapy.
A secondary leukemia or a secondary cancer (a cancer that develops because of the treatment for another type of cancer) is more common after treatment with chemotherapy and radiation therapy. Specifically, people with CLL are at a higher risk for developing skin cancer and should protect their skin from the sun. They also have a higher risk of lung cancer, and smoking is strongly discouraged.
People recovering from B-cell leukemia are encouraged to follow established guidelines for good health, such as, maintaining a healthy weight, not smoking, eating a balanced diet, and having recommended cancer screening tests. People experiencing a long-term remission are encouraged to follow cancer screening recommendations for the general population to watch for other types of cancer. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level.
Doctors are working to learn more about B-cell leukemia, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the diagnostic and treatment options best for you.
Targeted therapy. New treatments are being tested in clinical trials, including those that use angiogenesis inhibitors and monoclonal antibodies. Angiogenesis inhibitors are focused on stopping angiogenesis, which is the process of making new blood vessels. As discussed in the Treatment section, BL22 and LMB-2 are monoclonal antibodies linked to toxins that are designed to attach to the surface proteins of leukemia cells. Doctors are using these and other monoclonal antibodies in clinical trials for people with HCL when other treatments no longer work.
Recent research has shown that most of the abnormal cells involved in HCL have a mutation in a gene called BRAF. In the future, finding out whether this gene is mutated may help diagnose HCL. In addition, drugs that keep a mutated BRAF gene from working have helped slow or stop the growth of other cancers and are likely to be studied for HCL when other treatments do not work.
Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current B-cell leukemia treatments in order to improve patients’ comfort and quality of life.