How Leukemia Is Treated

The treatment for leukemia depends on many factors including the type and subtype of the disease, the stage, a person's age, and general health. Since leukemia is a cancer of blood cells, that travel throughout the body, local treatments such as surgery and radiation therapy are used infrequently. Instead, options such as aggressive chemotherapy, bone marrow/stem cell transplant, targeted therapy (tyrosine kinase inhibitors), monoclonal antibodies, immunotherapy, and others may be used alone or in combination. Even a period of watchful waiting may be appropriate in some cases.

Most people with leukemia will have a team of medical professionals caring for them, with a specialist in blood disorders and cancer (a hematologist/oncologist) leading the group. The treatments for leukemia, especially acute leukemia, very often cause infertility. For this reason, people who may wish to have a child in the future should discuss fertility preservation before treatment begins.

Approaches by Disease Type

Before discussing the different types of treatments, it's helpful to understand common initial approaches to treatment for the different types of leukemia. You may find it useful to zero in on the type you have been diagnosed with, then jump ahead to the in-depth descriptions of each option.

Acute Lymphocytic Leukemia (ALL)

With acute lymphocytic leukemia (ALL), treatment of the disease can take several years. It begins with induction treatment and with the goal of remission. Consolidation chemotherapy is then given (several cycles) to address any remaining cancer cells and reduce the risk of relapse. Alternatively, some people may receive a hematopoietic stem cell transplant (though less commonly than with AML).

After consolidation therapy, maintenance chemotherapy is given (usually a lower dose) to further reduce the risk of relapse, with the goal being long-term survival. If leukemia cells are found in the central nervous system, chemotherapy is given directly into the spinal fluid (intrathecal chemotherapy). Radiation therapy may also be used if the leukemia has spread to the brain, spinal cord, or skin. For those who have Philadelphia chromosome-positive ALL, the targeted therapy asparaginase may also be used.

Unfortunately, chemotherapy drugs do not penetrate well into the brain and spinal cord due to the presence of the blood-brain barrier, a tight network of capillaries that limits the ability of toxins (such as chemotherapy) to enter the brain. For this reason, many people are given preventive treatment to prevent leukemia cells from remaining behind in the central nervous system.

Acute Myelogenous Leukemia (AML)

Similar to the treatment of ALL, treatment for acute myelogenous leukemia (AML) usually begins with induction chemotherapy. After remission is achieved, further chemotherapy may be given, or, for people with a high risk of relapse, stem cell transplantation. Among the treatments for leukemia, those for AML tend to be the most intense and suppress the immune system to the greatest degree. Those over age 60 may be treated with less intense chemotherapy or palliative care, depending on the subtype of leukemia and general health. Acute promyelocytic leukemia (APL) is treated with additional medications and has a very good prognosis.

Chronic lymphocytic leukemia

In the early stages of chronic lymphocytic leukemia, a period of no treatment referred to a watchful waiting is often the best "treatment option." This is often the best choice even if the white blood cell count is very high. If certain symptoms, physical findings, or changes in blood tests develop, treatment is often started with a combination of chemotherapy and a monoclonal antibody.

Chronic Myelogenous Leukemia

With chronic myelogenous leukemia (CML), tyrosinase kinase inhibitors (TKIs, a type of targeted therapy) have revolutionized the treatment of the disease and resulted in a dramatic improvement in survival over the past two decades. These drugs target the BCR-ABL protein that causes the cancer cells to grow. For those who develop resistance to two or more of these drugs, a newer chemotherapy drug was approved in 2012. Pegylated interferon (a type of immunotherapy) may be used for those who do not tolerate TKIs. In the past, hematopoietic stem cell transplant was the treatment of choice for CML, but is used less commonly now and primarily in younger people with the disease.

Watchful Waiting

Most leukemias are treated aggressively when diagnosed, with the exception of CLL. Many people with this type of leukemia do not require treatment in the early stages of the disease, and a period of watchful waiting or active surveillance is considered a viable standard treatment option.

Watchful waiting does not mean the same thing as foregoing treatment and does not reduce survival when used appropriately. Instead, blood counts are done every few months, and treatment is initiated if constitutional symptoms (fever, night sweats, fatigue, weight loss greater than 10 percent of body mass), progressive fatigue, progressive bone marrow failure (with a low red blood cell or platelet count), painfully enlarged lymph nodes, a significantly enlarged liver and/or spleen, or a very high white blood cell count arise.

Chemotherapy

Chemotherapy is the mainstay of treatment for acute leukemias and is often combined with a monoclonal antibody for CLL. It may also be used for CML that has become resistant to targeted therapy.

Chemotherapy works by eliminating rapidly dividing cells such as cancer cells, but can also affect normal cells that divide rapidly, such as those in the hair follicles. It is most often given as combination chemotherapy (two or more drugs), with different drugs working at different places in the cell cycle. The chemotherapy drugs chosen and the way in which they are used differs depending on the type of leukemia being treated.

Induction Chemotherapy

Induction chemotherapy is often the first therapy that is used when a person is diagnosed with acute leukemia. The goal of this treatment is to reduce the level of leukemia cells in the blood to undetectable levels. This does not mean that the cancer is cured, but only that it can't be detected when looking at a blood sample.

The other goal of induction therapy is to reduce the number of cancer cells in the bone marrow so that normal production of the different types of blood cells can resume. Unfortunately, further treatment is needed after induction therapy so that the cancer does not recur.

With AML, a common induction therapy is called the 3+7 protocol. This includes three days of an anthracycline, either Idamycin (idarubicin) or Cerubidine (daunorubicin), along with seven days of a continuous infusion of Cytosar U or Depocyt (cytarabine). These drugs are often given through a central venous catheter in the hospital (people are usually hospitalized for the first four to six weeks of treatment). For younger people, the majority will achieve remission.

With ALL, chemotherapy usually includes a combination of four drugs:

  • An anthracycline, usually either Cerubidine (daunorubicin) or Adriamycin (doxorubicin)
  • Oncovin (vincristine)
  • Prednisone (a corticosteroid)
  • An asparaginase: Either Elspar or L-Asnase (asparaginase) or Pegaspargase (Peg asparaginase)

People with Philadelphia chromosome-positive ALL and those over age 60 may also be treated with a tyrosine kinase inhibitor, such as Sprycel (dasatinib). After remission is achieved, preventive treatment to the central nervous system is used to prevent leukemia cells from remaining in the brain and spinal cord.

With acute promyelocytic leukemia (APL), induction therapy also includes the medication ATRA (all-trans-retinoic acid), sometimes combined with Trisenox or ATO (arsenic trioxide).

While induction therapy often achieves a complete remission, further therapy is needed so that the leukemia does not recur.

Consolidation and Intensification Chemotherapy

With acute leukemias, options after induction chemotherapy and remission include either further chemotherapy (consolidation chemotherapy) or high-dose chemotherapy plus stem cell transplantation. With AML, the most common treatment is three to five courses of further chemotherapy, though, for people with high-risk disease, a stem cell transplant is often recommended. With ALL, consolidation chemotherapy is usually followed by maintenance chemotherapy, but a stem cell transplant may also be recommended for some people.

Maintenance Chemotherapy (for ALL)

With ALL, further chemotherapy after induction and consolidation chemotherapy is often needed to reduce the risk of relapse and to improve long-term survival. Drugs used often include methotrexate or 6-MP (6-mercaptopurine).

Chemotherapy for CLL

When symptoms occur in CLL, usually a combination of the chemotherapy drug Fludara (fludarabine) with or without Cytoxan (cyclophosphamide) along with a monoclonal antibody such as Rituxan (rituximab) is recommended. As an alternative, the chemotherapy drug Treanda or Bendeka (bendamustine) may be used with a monoclonal antibody. 

Chemotherapy for CML

The mainstay of treatment for CML is monoclonal antibodies, but chemotherapy may occasionally be recommended. Drugs such as Hydrea (hydroxyurea), Ara-C (cytarabine), Cytoxan (cyclophosphamide), Oncovin (vincristine), or Myleran (busulfan) may be used to lower a very high white blood cell count or enlarged spleen.

In 2012, a new chemotherapy drug—Synribo (omacetaxine)—was approved for CML that has progressed to the accelerated phase and has become resistant to two or more tyrosine kinase inhibitors or has the T3151 mutation.

Side Effects

Common side effects of chemotherapy can vary with the different drugs used, but may include:

  • Tissue damage: Anthracyclines are vesicants and can cause tissue damage if they leak into the tissues surrounding the infusion site.
  • Bone marrow suppression: Damage to rapidly dividing cells in the bone marrow often results in low levels of red blood cells (chemotherapy-induced anemia), white blood cells such as neutrophils (chemotherapy-induced neutropenia), and platelets (chemotherapy-induced thrombocytopenia). Due to a low white blood cell count, taking precautions to reduce the risk of infections is extremely important.
  • Hair loss: Hair loss is common, not just what's on the top of the head, but the eyebrows, eyelashes, and pubic hair.
  • Nausea and vomiting: While a feared side effect, medications to both treat and prevent chemotherapy-associated vomiting have reduced this significantly.
  • Mouth sores: Mouth sores are common, though dietary changes, as well as mouth rinses, can improve comfort. Taste changes may also occur.
  • Red urine: Anthracycline medications been coined the "red devils" for this common side effect. Urine may be bright red to orange in appearance, beginning shortly after the infusion and lasting for a day or so after it's complete. Though perhaps startling, it's not dangerous.
  • Peripheral neuropathy: Numbness, tingling, and pain in a "stocking and glove" distribution (both the feet and the hands) may occur, especially with drugs such as Oncovin.
  • Tumor lysis syndrome: The rapid breakdown of leukemia cells can result in a condition known as tumor lysis syndrome. Findings include high potassium, uric acid, blood urea nitrogen (BUN), and phosphate levels in the blood. Tumor lysis syndrome is less problematic than in the past, and is treated with intravenous fluids and medications to lower the uric acid level.
  • Diarrhea

Since many people who develop leukemia are young and are expected to survive treatment, the late effects of treatment that may occur years or decades after treatment are of particular concern. Potential long-term side effects of chemotherapy may include an increased risk of heart disease, secondary cancers, and infertility among others.

Targeted Therapy

Targeted therapies are medications that work by specifically targeting cancer cells or pathways involved in the growth and division of cancer cells. Unlike chemotherapy drugs, which can affect both cancer cells and normal cells in the body, targeted therapies focus on mechanisms that support the growth of a cancer specifically. For this reason, they may have fewer side effects than chemotherapy (but not always).

Unlike chemotherapy drugs that are cytotoxic (cause the death of cells), targeted therapies control the growth of cancer but do not kill cancer cells. While they may hold a cancer in check for years or even decades, as is often the case with CML, they are not a cure for cancer.

In addition to the targeted therapies mentioned below, there are a number of drugs that may be used for leukemia that has relapsed or leukemias that harbor specific genetic mutations.

Tyrosine Kinase Inhibitors (TKIs) for CML

Tyrosine inhibitors (TKIs) are medications that target enzymes called tyrosine kinases to interrupt the growth of cancer cells.

With CML, TKIs have revolutionized treatment and have vastly improved survival over the past two decades. Continued use of the drugs can often result in long-term remission and survival with CML. Medications currently available include:

  • Gleevec (imatinib)
  • Bosulif (bosutinib)
  • Sprycel (dasatinib)
  • Tasigna (nilotinib)
  • Iclusig (ponatinib)

Kinase Inhibitors for ALL

With high-risk ALL, the TKIs Sprycel or Jakafi (ruxolitinib) may be used.

Kinase Inhibitors for CLL

In addition to monoclonal antibodies which are the mainstay of treatment, kinase inhibitors may be used for CLL. Drugs include:

  • Imbruvica (ibrutinib): This drug that inhibits Bruton's tyrosine kinase may be effective for difficult-to-treat CLL.
  • Zydelig (idelalisib): This drug blocks a protein (P13K) and may be used when other treatments are not working.
  • Venclextra (venetoclax): This drug blocks a protein (BCL-2) and may be used second line to treat CLL.

Monoclonal Antibodies

Monoclonal antibodies are similar to the antibodies many people are familiar with that attack viruses and bacteria, but instead are man-made and designed to attack cancer cells.

For CLL, monoclonal antibodies are a mainstay of treatment, often combined with chemotherapy. These drugs target a protein (CD20) found on the surface of B cells. Drugs currently approved include:

  • Rituxan (rituximab)
  • Gazyva (obinutuzumab)
  • Arzerra (ofatumumab)

These drugs can be very effective, though they do not work as well for people with a mutation or deletion in chromosome 17.

For refractory B cell ALL, the monoclonal antibodies Blincyto (blinatumomab) or Besponsa (inotuzumab) may be used.

Proteasome Inhibitors

For refractory ALL in children, the proteasome inhibitor Velcade (bortezomib) may be used.

Immunotherapy

There is a wide range of treatments that fall under the general category of immunotherapy. These drugs work by using the immune system or principles of the immune system to fight cancer.

CAR T-Cell Therapy

CAR T-cell therapy (chimeric antigen receptor T-cell therapy) or gene therapy uses a person's own cancer-fighting cells (T cells). In this procedure, T cells are harvested from the body and modified to target a protein on the surface of leukemia cells. They are then allowed to multiply before being injected back into the body, where they often eliminate leukemia cells within a few weeks.

In 2017, the drug Kymriah (tisagenlecleucel) received U.S. Food and Drug Administration (FDA) approval for children and young adults with B cell ALL or other types of ALL that have recurred.

Interferon

Interferons are substances made by the human body that function to control the growth and division of cancer cells, among other immune functions. In contrast to the CAR T-cell therapy, which is designed to attack particular markers on leukemia cells, interferons are non-specific and have been used in many settings from cancer to chronic infections. Interferon alpha, a man-made interferon, was once commonly used for CML, but is now used more often for people with CML who are intolerant of other treatments. It can be given by injection (either subcutaneously or intramuscularly) or intravenously, and is given for a long period of time.

Bone Marrow/Stem Cell Transplants

Hematopoietic cell transplants, or bone marrow and stem cell transplants, work by replacing the hematopoietic cells in the bone marrow that develop into the different types of blood cells. In these transplants, a person's bone marrow cells are destroyed. They are then replaced with donated cells that restock the bone marrow and eventually produce healthy white blood cells, red blood cells, and platelets.

Types

While bone marrow transplants (cells harvested from the bone marrow and injected) were once more common, peripheral blood stem cell transplants are now more so. Stem cells are harvested from the blood of a donor (in a procedure similar to dialysis) and collected. Medications are given to the donor prior to this procedure to increase the number of stem cells in the peripheral blood.

Types of hematopoietic cell transplants include:

  • Autologous transplants: Transplants in which a person's own stem cells are used
  • Allogeneic transplants: Transplants in which stem cells are derived from a donor, such as a sibling or unknown but matched donor
  • Transplants from umbilical cord blood
  • Non-ablative stem cell transplant: These transplants are less invasive "mini-transplants" that do not require obliterating the bone marrow prior to the transplant. Mini-transplants work by something called "graft versus malignancy" in which the donor cells help fight off the cancer cells, rather than by replacing the cells in the bone marrow.

Uses

A hematopoietic cell transplant may be used after induction chemotherapy with both AML and ALL, especially for high-risk disease. The goal of treatment with acute leukemia is long-term remission and survival. With CLL, stem cell transplantation may be used when other treatments do not control the disease. With CML, stem cell transplants were once the treatment of choice, but are now used much less often.

Non-ablative transplants may be used for people who would not tolerate the high-dose chemotherapy required for a traditional stem cell transplant (for example, people over the age of 50). They may also be used when a leukemia recurs after a previous stem cell transplant.

Phases of Stem Cell Transplants

Stem cell transplants have three distinct phases:

  • Induction: The induction phase is similar to that noted under chemotherapy for acute leukemias above and consists of using chemotherapy to reduce the white blood cell count and, if possible, induce a remission.
  • Conditioning: During this phase, high-dose chemotherapy and/or radiation therapy is used to destroy the bone marrow. In this phase, chemotherapy is used to essentially sterilize/obliterate the bone marrow so that no hematopoietic stem cells remain.
  • Transplantation: In the transplantation phase, the donated stem cells are given. Following transplantation, it usually takes from two to six weeks for the donated cells to grow in the bone marrow and produce functioning blood cells, what's known as engraftment.

Side Effects and Complications

Stem cell transplants are major procedures and, though they can sometimes bring about a cure, have significant mortality (primarily due to the absence of infection-fighting cells between conditioning and the time it takes the donated cells to grow up in the marrow, when people have essentially no white blood cells left to fight infections). A few possible complications include:

  • Immunosuppression: As noted, a severely suppressed immune system is responsible for the relatively high mortality rate of this procedure.
  • Graft-versus-host disease: Graft-versus-host disease occurs when the donated cells attack a person's own cells and can be both acute and chronic.

Finding a Stem Cell Donor

For those considering as stem cell transplant, the oncologist will first want to check your siblings for a potential match. There are a number of resources available on how to find a donor, if needed.

Complementary Medicine

There are currently no alternative treatments that are effective in successfully treating leukemia, though some integrative cancer treatments such as meditation, prayer, yoga, and massage may help people cope with the symptoms of leukemia and its treatments.

While we often think of vitamins, minerals, and dietary supplements as relatively harmless, it's important to note that some vitamins may interfere with cancer treatments. This is easier to understand if you think about how cancer treatments work. Chemotherapy, for example, works by creating oxidative stress and damaging DNA in cells. While taking antioxidant preparations may be a healthful dietary practice for someone without cancer, there's a risk that using these same preparations may help "protect" cancer cells from the treatments designed to eliminate them.

While there has been some research that suggests that vitamin C may be helpful when combined with a class of medications called PARP inhibitors (which are not currently approved for leukemia), there have also been studies that suggest vitamin C supplementation makes chemotherapy less effective with leukemia. The general uncertainty in this area is a good reminder to talk to your oncologist about any vitamins, dietary supplements, or over-the-counter medications you consider taking.

Clinical Trials

There are many different clinical trials in progress looking at more effective ways to treat leukemia or methods that have fewer side effects. With treatments for cancer rapidly improving, the National Cancer Institute recommends that people talk with their oncologist about the option of a clinical trial.

Some of the treatments being tested combine therapies mentioned above, whereas others are looking at unique ways to treat leukemia, including many next-generation drugs. The science is changing rapidly. For example, the first monoclonal antibody was only approved in 2002, and since then, second- and third-generation drugs have become available. Similar progress is being made with other types of targeted therapies and immunotherapy.

Living Your Best Life With Leukemia
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