Chemotherapy for Leukemia in Children

Experts at the Stephen D. Hassenfeld Children’s Center for Cancer and Blood Disorders, part of Hassenfeld Children’s Hospital at NYU Langone, helped pioneer chemotherapy treatments for childhood leukemia. These chemotherapy drugs, which are given to your child by mouth or through a vein with intravenous (IV) infusion, kill leukemia cells. They are the first treatments our doctors offer when a child is diagnosed with leukemia. 

Our doctors recommend specific chemotherapy drugs based on the type of leukemia and how aggressive it is, the chromosomal or genetic make-up of the cancer cells, and whether it is found in the central nervous system.

Chemotherapy for Childhood Acute Lymphoblastic Leukemia

There are usually four general phases of chemotherapy for children with acute lymphoblastic leukemia. Treatment typically takes two to three years to complete. Our oncologists, nurses, radiologists, psychologists, and wellness experts provide support for your family—from medical care to emotional and logistical assistance—during and after treatment.


During the induction phase of chemotherapy, the aim is to quickly eliminate as many leukemia cells as possible. The goal is that remission—when the signs and symptoms of cancer have disappeared—can be achieved after this first phase of treatment.

Induction generally begins during an initial hospital stay, when chemotherapy drugs are administered intravenously through a vein and by mouth. Most children are hospitalized for the first week of induction but may stay longer if infections or other complications occur. Your child then continues to take the medication at home with frequent visits to our outpatient center at the Stephen D. Hassenfeld Children’s Center for Cancer and Blood Disorders.

Children with acute lymphoblastic leukemia typically go into remission after the four-week induction stage. During this stage, your child’s response to the treatment is measured by how fast the cancer cells disappear. Tests such as flow cytometry, which identifies white blood cell types in blood and bone marrow, can help your child’s doctor detect the presence of “minimal residual disease”—often called simply MRD—when small amounts of leukemia can be detected among normal blood cells. The result of these tests can lead to further tailoring of treatment to your child’s needs.

Consolidation or Intensification

In the consolidation or intensification phase, your child’s doctor administers another course of chemotherapy several times a week for a period of up to eight weeks. This phase is designed to kill cancer cells that did not respond to the chemotherapy drugs used during induction. It usually doesn’t require a hospital stay, but children are sometimes admitted for complications such as fever or infection.

The more intense the chemotherapy schedule used during consolidation, the greater the odds of neutropenia, which is an unusually low number of normal cells called neutrophils that help the immune system fight infection.

Interim Maintenance and Delayed Intensification

After consolidation is complete, a combination of chemotherapy drugs is given to your child in specific doses and sequences based on his or her risk group defined at diagnosis. During these phases of treatment, occasional hospitalization may be needed to manage side effects, which can include fever, lethargy, low blood cell levels, nausea, or vomiting. 

Interim maintenance lasts about eight weeks and can require periodic hospitalization for chemotherapy. The next phase, delayed intensification, includes a shortened version of the induction and consolidation cycles to prevent resistant cells from appearing and growing.

Maintenance Therapy

After these initial, more intensive chemotherapy phases, maintenance chemotherapy is given by mouth and IV to prevent leukemia from returning. Maintenance therapy is given as an outpatient treatment periodically for about two years in girls and three years in boys. Because many of the side effects typically experienced in earlier stages of treatment are less common during maintenance, children can usually return to school if they have not already done so.

Chemotherapy for Childhood Acute Myeloid Leukemia

Chemotherapy for acute myeloid leukemia is more intense than chemotherapy for acute lymphoblastic leukemia; it involves multiple phases and requires an extended hospital stay. Extra time is required to address complications such as fever and infections or for treatments such as blood transfusions, as needed. Usually completed in five to six months, the treatment is determined by the subtype of acute myeloid leukemia, the presence of certain genetic abnormalities, and the response of leukemia cells to treatment.


At Hassenfeld Children’s Hospital, childhood acute myeloid leukemia is treated with a more intense IV chemotherapy during the induction phase, requiring weeks in the hospital for each cycle. The goal is to destroy quickly as many leukemia cells as possible to achieve remission. Usually, children with acute myeloid leukemia are treated with two induction cycles of IV medication. About 90 percent of children with this condition go into remission after induction.

Consolidation or Intensification

Consolidation or intensification is a second cycle of intensive chemotherapy given after induction to eliminate remaining leukemia cells that go undetected in laboratory tests. Usually, there are three to four cycles of re-induction and consolidation or intensification chemotherapy. During these cycles, chemotherapy drugs are given for several days at a time, requiring that your child stay in the hospital during this treatment. Children with acute myeloid leukemia who are classified as high risk because of genetic subtype or slow response to treatment are candidates for stem cell transplantation.

Most children with acute myeloid leukemia do not need maintenance chemotherapy, because studies have shown maintenance therapy to be ineffective. Sometimes, children with certain subtypes of acute myeloid leukemia may receive maintenance chemotherapy drugs during treatment to ensure that the leukemia stays in remission.

Targeted Chemotherapy Drugs for Childhood Chronic Myelogenous Leukemia

At Hassenfeld Children’s Hospital, childhood chronic myelogenous leukemia is usually treated with targeted medications given by mouth that block the protein known as BCR-ABL tyrosine kinase without damaging healthy cells.

Children with chronic myelogenous leukemia have an abnormal gene called BCR-ABL that produces an excessive amount of tyrosine kinase. This leads to the uncontrolled growth of cancerous cells. The medications block the signals sent by the protein, preventing the cancer cells from growing. 

For most children with chronic myelogenous leukemia, the only necessary treatment is a daily pill taken for life. Most children with this condition receive one of the following medications, depending on the type of gene mutation and how well your child tolerates the medication:

  • Gleevec® (imatinib)
  • Sprycel® (dasatinib)
  • Tasigna® (nilotinib)

Childhood chronic myelogenous leukemia is a lifelong cancer. Although there is no cure for this condition, daily medications taken by mouth can keep this type of leukemia under control for a lifetime. Rarely, children may be slowly transitioned off therapy.

In rare instances, a stem cell transplant may be required to prevent childhood chronic myelogenous leukemia from worsening.

Managing the Side Effects of Chemotherapy

Chemotherapy is designed to attack cells that divide quickly. This means that chemotherapy drugs affect not just cancer cells but also healthy cells in the bone marrow, the hair follicles, the intestines, and the mouth. This can lead to certain side effects that may affect your child throughout treatment, including hair loss, mouth sores, nausea, vomiting, numbness in the hands and feet, fatigue, and stomach pain. 

Chemotherapy also increases a child’s risk of infection because it reduces the number of normal white blood cells circulating in the blood during treatment. These side effects usually go away after chemotherapy is completed. 

The side effects your child may experience depend on the type of chemotherapy drugs he or she receives and the duration of treatment. Our doctors, nurses, and wellness experts provide medical, emotional, and psychological support to your child throughout treatment to ensure that side effects are managed as quickly and effectively as possible.

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