Medication for Acute Lymphoblastic Leukemia

NYU Langone oncologists and hematologists at Perlmutter Cancer Center, who specialize in managing blood cancer, often use chemotherapy, a group of drugs that destroy cancer cells throughout the body, to treat acute lymphoblastic leukemia.

If the cancer has certain genetic features, doctors may also prescribe targeted drugs, which are designed to eliminate cancer cells while sparing healthy tissue.

Chemotherapy for acute lymphoblastic leukemia almost always includes a combination of medications. Conventional treatment involves giving chemotherapy in three phases—induction, consolidation, and maintenance. This treatment regimen often involves high doses of drugs.

This regimen may be too strong for you because of other health conditions. In that case, doctors can modify the treatment. Some people find certain combinations of chemotherapy more tolerable than others.

For example, doctors may recommend using a combination of drugs called hyper-CVAD in older adults. “Hyper” refers to giving more than one treatment with the same medication during a single day, and “CVAD” stands for the different drugs used: cyclophosphamide, vincristine sulfate, Adriamycin®, and dexamethasone. Also, NYU Langone doctors may use pediatric-inspired treatment—a combination of drugs successfully used in children—in younger adults.

For these approaches, doctors usually combine several chemotherapy drugs during the first few days of a treatment cycle—a three- or four-week period—with additional chemotherapy drugs given during the in-between days. Treatment is given in an outpatient setting and can last for six months or longer.

Often, doctors perform screening lumbar punctures during treatment because some of the chemotherapy drugs are injected in the lower spine. This approach, called intrathecal chemotherapy, is used to prevent acute lymphoblastic leukemia from spreading to the central nervous system—the brain and spinal cord.

Induction Therapy

Induction chemotherapy is the first phase of conventional chemotherapy treatment for acute lymphoblastic leukemia. People usually receive the treatment while in the hospital, through a vein with intravenous (IV) infusion.

Chemotherapy is given for a few hours a day, sometimes several days a week, over the course of several weeks. During this initial phase, people are often hospitalized for several weeks to manage complications from the leukemia or side effects of the treatment.

Common chemotherapy drugs used during this phase include vincristine, cyclophosphamide, and doxorubicin. Corticosteroids, powerful anti-inflammatory medications, are usually part of the chemotherapy regimen. Doctors also use medications that reach the spinal canal and the brain, such as methotrexate or cytarabine, to treat any cancer in these parts of the body or to prevent it from spreading there.

Doctors may add a targeted medication called dasatinib to chemotherapy if genetic testing shows the lymphoblasts have a BCR–ABL fusion gene. Dasatinib and other medications called tyrosine kinase inhibitors target BCR–ABL proteins on the surface of lymphoblasts. These proteins “tell” cells to grow rapidly and divide. These medications act to block these signals, destroying leukemia cells.

The goal of the induction phase is to put the disease into remission, meaning acute lymphoblastic leukemia is no longer found in the blood or bone marrow and the signs and symptoms disappear. If the condition does not go into remission, our doctors may recommend other types of chemotherapy or participation in a clinical trial.

Even if the condition goes into remission, lymphoblasts can still be present in microscopic amounts in the body. This is called minimal residual disease. Even when doctors cannot detect leukemia cells, people still require further therapy to remove potential minimal residual disease. This treatment is called consolidation therapy, which may be followed by maintenance therapy to prevent recurrence.

Our doctors use sensitive tests to detect any remaining lymphoblasts. Results of these tests may change recommendations for further treatment. People with a very low level of minimal residual disease tend to have a better chance of avoiding a recurrence.

Consolidation Therapy

During the consolidation phase of treatment, people with acute lymphoblastic leukemia receive additional chemotherapy to maintain a remission. A doctor may prescribe some of the same medications that were given during the induction phase. Consolidation may last for several months and is performed on an outpatient basis. The goal of consolidation is to destroy any minimal residual disease still in the body.

During this phase, doctors may recommend an allogeneic stem cell transplant. A stem cell transplant involves the use of high intensity chemotherapy, with or without radiation therapy, to destroy the cancer. It can also support the immune system in mounting an attack against leukemia cells. It may especially benefit people with high levels of minimal residual disease or those with genetic changes that increase the risk of the cancer coming back after treatment with standard chemotherapy doses.

Maintenance Therapy

The consolidation treatment phase may be followed by maintenance therapy. During this phase, NYU Langone doctors often prescribe low intensity medications such as prednisone or methotrexate. These are taken by mouth or given as an injection on a frequent basis for up to two years. Blood tests are performed every few months to ensure that leukemia has not returned and medication is given at the proper doses.

Managing Side Effects

Side effects of the medications used to treat acute lymphoblastic leukemia may include nausea, vomiting, diarrhea, and fatigue. Other complications include infections and bleeding. To ease side effects, our doctors can adjust the dose of a medication, prescribe other medications, or refer you to our Integrative Health Services, which offer therapies that may alleviate side effects.

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