Table of ContentsView All
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Table of Contents
Watchful Waiting
Chemotherapy
Targeted Therapy
Monoclonal Antibodies
Immunotherapy
Bone Marrow/Stem Cell Transplants
Complementary Medicine
Clinical Trials
Frequently Asked Questions
The treatment forleukemiadepends on many factors, including the type, subtype, and stage of the disease, and a person’s age and general health. Treatment typically involves powerful chemotherapy, bone marrow/stem cell transplant, targeted therapy (tyrosinekinase inhibitors), monoclonal antibodies, and immunotherapy—used alone or in combination.
In some cases, a period of watchful waiting may be appropriate. Leukemia is a cancer of blood cells that travels throughout the body, and local treatments such as surgery and radiation therapy are not used frequently.
Most people with leukemia will have a team of medical professionals caring for them, with ahematologist/oncologist(a doctor specializing in blood disorders and cancer) leading the group.
The treatments for leukemia often cause infertility. If you wish to have a child in the future, you should discuss fertility preservation before treatment begins.
Before discussing the different types of treatments, it’s helpful to understand common approaches to treatment for thedifferent 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)
Treatment ofacute lymphocytic leukemia(ALL) can take several years. It begins with induction treatment, with the goal of remission. Then, consolidation chemotherapy in several cycles is used to treat 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).
Chemotherapy drugs do not penetrate well into the brain and spinal cord due to theblood-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 treatment toprevent leukemiacells from remaining behind in the central nervous system.
Acute Myelogenous Leukemia (AML)
Treatment for acute myelogenous leukemia (AML) usually begins with induction chemotherapy. After remission is achieved, you might have further chemotherapy. People who have a high risk of relapse might have 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. If you are over age 60, you may be treated with less intense chemotherapy or palliative care, depending on the subtype of your leukemia and your general health.
Acute promyelocytic leukemia (APL) is treated with additional medications and has a very good prognosis.
Chronic Lymphocytic Leukemia
In the early stages ofchronic lymphocytic leukemia(CLL), a period of watchful waiting without treatment is often recommended as 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 BTK inhibitor, such as Imbruvica (ibrutinib) or Calquence (acalabrutinib), or a BCL-2 inhibitor, such as Venclexta (venetoclax).
Chronic Myelogenous Leukemia
Tyrosine kinase inhibitors (TKIs, a type of targeted therapy) have revolutionized the treatment ofchronic myelogenous leukemia(CML) 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 or can’t tolerate two or more of these drugs. Pegylated interferon (a type of immunotherapy) may be used.
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.
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 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. 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 develop.
Chemotherapyis 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.
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 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. Further treatment is needed after induction therapy so that cancer does not recur.
With AML, the 7+3 protocol is used.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 acentral venous catheterin the hospital (people are usually hospitalized for the first four to six weeks of treatment). For younger people, the majority will achieve remission.
Chemotherapy DrugsWith ALL, chemotherapy usually includes a combination of four drugs:An anthracycline, usually either daunorubicin or Adriamycin (doxorubicin)VincristinePrednisone (a corticosteroid)An asparaginase: Either L-asparaginase or Oncaspar (pegaspargase)
Chemotherapy Drugs
With ALL, chemotherapy usually includes a combination of four drugs:An anthracycline, usually either daunorubicin or Adriamycin (doxorubicin)VincristinePrednisone (a corticosteroid)An asparaginase: Either L-asparaginase or Oncaspar (pegaspargase)
With ALL, chemotherapy usually includes a combination of four drugs:
People with Philadelphia chromosome-positive ALL may also be treated with a tyrosine kinase inhibitor, such as Sprycel (dasatinib). After remission is achieved, treatment is used to prevent leukemia cells from remaining in the brain and spinal cord.
Withacute promyelocytic leukemia(APL), induction therapy also includes the medication ATRA (all-trans retinoic acid) combined with Trisenox (arsenic trioxide).
While induction therapy often achieves a complete remission, further therapy is needed so that 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.
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, a BTK inhibitor or a BCL-2 inhibitor may be used.
Chemotherapy for CML
The mainstay of treatment for CML is tyrosine kinase inhibitors, but chemotherapy may occasionally be recommended. Drugs such as Hydrea (hydroxyurea), cytarabine, cyclophosphamide, vincristine, or Myleran (busulfan) may be used to lower a very high white blood cell count or enlarged spleen.
In 2021, a new chemotherapy drug, Scemblix (asciminib), was approved for Philadelphia chromosome-positive CML in the chronic phase that has been previously treated with two or more tyrosine kinase inhibitors or that has the T3151 mutation.
Side Effects
Common side effects of chemotherapy can vary with the different drugs used but may include:
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.
Potentiallong-term side effects of chemotherapymay include an increased risk of heart disease, secondary cancers, and infertility, among others.
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 cancer in check for years or even decades, as is often the case with CML, they are not acurefor 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:
Kinase Inhibitors for CLL
In addition to monoclonal antibodies, which are the mainstay of treatment, kinase inhibitors may be used for CLL.
Drugs include:
Copiktra comes with a warning regarding a possible increased risk of death and serious side effects. If you are taking Copiktra, talk with your healthcare provider about the risks and benefits of treatment with Copiktra. In addition, discuss any questions or concerns you may have, including possible alternative treatments.
Monoclonal antibodies are similar to the natural antibodies that your body makes to fight infections. These are synthetic versions that are 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:
These drugs can be very effective, though they do not work as well for people who have 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.
CAR T-Cell Therapy
CAR T-cell therapy (chimeric antigen receptor T-cell therapy) uses a person’s own (T cells) to fight cancer. It starts with a procedure in which T cells are collected from the body and modified in a laboratory 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 2024, the FDA issued Safety Labeling Changes for CAR-T therapies. The warnings inform providers and patients of the potential risk of these therapies causing secondary cancer.
Interferon
Types
While bone marrow transplants (cells harvested from the bone marrow and injected) were once more common, peripheral blood stem cell transplants are now used. 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:
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. Stem cell transplants were once the treatment of choice for CLL, 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 leukemia recurs after a previous stem cell transplant.
Phases of Stem Cell Transplants
Stem cell transplants have three distinct phases:
Side Effects and Complications
Stem cell transplants are major procedures that can sometimes bring about a cure. But they can have significant mortality. This is primarily due to the absence of infection-fighting cells between conditioning and the time it takes the donated cells to develop in the marrow, during which there are few white blood cells left to fight infections.
A few possible complications include:
Finding a Stem Cell DonorIf you’re considering a stem cell transplant, your 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.
Finding a Stem Cell Donor
If you’re considering a stem cell transplant, your 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.
There are currently no alternative treatments that are effective in treating leukemia, though some integrative cancer treatments such as meditation, prayer, yoga, andmassagemay helpyou copewith the symptoms of leukemia and its treatments.
It’s important to note that somevitamins may interfere with cancer treatments.
Some research suggests that vitamin C may be helpful when combined with a class of medications called PARP inhibitors (which are not currently approved for leukemia), but there have also been studies that suggestvitamin C supplementationmakes 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.
There are many differentclinical trialsin 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.
Frequently Asked QuestionsThe five-year relative survival for all types of leukemia combined is 65%.People who have certain types of leukemia can achieve long-term remission. For example, ALL has a long-term remission rate of 90% in children and 40% in adults.The term “cure” is rarely used, however, as cancer can come back.
The five-year relative survival for all types of leukemia combined is 65%.
People who have certain types of leukemia can achieve long-term remission. For example, ALL has a long-term remission rate of 90% in children and 40% in adults.The term “cure” is rarely used, however, as cancer can come back.
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Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.National Cancer Institute.Adult lymphoblastic leukemia treatment (PDQ): health professional version.National Cancer Institute.Adult acute myeloid leukemia treatment (PDQ): health professional version.National Cancer Institute.Chronic lymphocytic leukemia treatment (PDQ): health professional version.National Institutes of Health: Genetic and Rare Diseases Information Center.Chronic myeloid leukemia.American Cancer Society.Treatment response rates for acute myeloid leukemia (AML).National Library of Medicine: DailyMed.Scemblix—asciminib tablet, film coated[drug label].Chopade P, Akard LP.Improving outcomes in chronic myeloid leukemia over time in the era of tyrosine kinase inhibitors.Clin Lymphoma Myeloma Leuk. 2018;18(11):710-723. doi:10.1016/j.clml.2018.06.029Food and Drug Administration.FDA warns about possible increased risk of death and serious side effects with cancer drug Copiktra (duvelisib).National Cancer Institute.Chronic myelogenous leukemia treatment (PDQ): health professional version.Wesa KM, Cassileth BR.Is there a role for complementary therapy in the management of leukemia?.Expert Rev Anticancer Ther. 2009;9(9):1241-9. doi:10.1586/era.09.100National Cancer Institute.Cancer stat facts: leukemia.Cleveland Clinic.Leukemia.
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
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Food and Drug Administration.FDA warns about possible increased risk of death and serious side effects with cancer drug Copiktra (duvelisib).
National Cancer Institute.Chronic myelogenous leukemia treatment (PDQ): health professional version.
Wesa KM, Cassileth BR.Is there a role for complementary therapy in the management of leukemia?.Expert Rev Anticancer Ther. 2009;9(9):1241-9. doi:10.1586/era.09.100
National Cancer Institute.Cancer stat facts: leukemia.
Cleveland Clinic.Leukemia.
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