Pediatric Leukemias: Treatment

Pictured: Maura Byrnes-Casey Nurse practitioner Maura Byrnes-Casey with a young patient.

We are frequently able to make a diagnosis of leukemia and begin treatment on the same day. For each child who comes to Memorial Sloan Kettering, our doctors develop a personalized treatment strategy, based on information including the child's age, sex, complete blood count numbers, leukemia subtype, specific gene mutation, and risk of the cancer returning after remission, known as the risk group. The doctor will discuss these factors with you during the pretreatment consultation and after diagnostic test results are available.

Our multidisciplinary team of experts pioneered the use of new chemotherapy regimens that have increased long-term survival for children and young adults with leukemia.(1),(2) Memorial Sloan Kettering's leukemia team was also the first group to recognize the importance of a child's initial response to therapy as a key factor in selecting the most appropriate course of treatment.(3),(4)

Children who experience a rapid early response to treatment are usually at a lower risk of recurrence, so they can be treated with less-intensive therapy. This also results in fewer side effects. Children who do not experience a rapid early response are considered to be at a higher risk of disease recurrence, so they are treated with more-intensive therapy. With this approach, standard-risk and high-risk patients have an almost equal chance of a cure.

More than 95 percent of children and young adults with newly diagnosed leukemia treated at Memorial Sloan Kettering will experience remission, in which the treatment kills nearly all of the abnormal cells, symptoms subside, and normal blood cell production resumes.

Learn more about treatment for pediatric leukemias.

Remission Induction

In the first stage of treatment for leukemias, doctors work to put the disease into remission. This stage, known as remission induction, takes about four weeks.

Remission induction for both ALL and AML involves the use of chemotherapy medications, which kill the leukemic cells and stop abnormal white blood cells from growing. Your child may receive a combination of several chemotherapy medications. Each medication targets leukemic cells in a different way, in case the cells are resistant to a particular treatment. Our team works to treat children with the most-effective dose and combination of chemotherapy to maximize the benefit and minimize the risk of long-term side effects.

Additional medications will also be given as support for your child, to minimize nausea, constipation, and kidney damage from the breakdown of leukemic cells.

Many children can receive remission induction treatment as outpatients in our Pediatric Day Hospital. However, children are at a high risk of infection during this process. Some children remain in our inpatient unit for three to four weeks to receive intravenous antibiotics for fevers or infections because they do not have enough normal white blood cells to fight infections. Once healthy white blood cells return, these children can be discharged and receive further treatment as outpatients in the Pediatric Day Hospital.

After the first few weeks of remission induction therapy, your child's strength and stamina will gradually return, but he or she might not be able to return to school for several months due to an increased risk of infections. Subsequent inpatient hospital visits may be necessary to administer additional chemotherapy and treat infections that result from low white blood cell counts. Our doctors and nurses will provide more specific guidelines on what to expect based on your child's specific treatment and response.

Consolidation and Maintenance Therapy

After the leukemia is in remission, a child is given additional treatment at home and as an outpatient to kill any lingering cancer cells.

Consolidation Therapy

In most children, an undetectable pool of leukemic cells can “hide” in the central nervous system, which includes the brain, spinal cord, or spinal fluid. During consolidation therapy, these cells are targeted by chemotherapy drugs injected directly into the fluid surrounding the spinal column. For this procedure, known as a lumbar puncture, the child is briefly placed under anesthesia.

A small percentage of children may also receive radiation therapy to the head, called cranial irradiation, to treat any leukemic cells in the central nervous system or to prevent the disease from spreading there. This treatment is for children who are at a very high risk of cancer recurrence or have central nervous system (meningeal) leukemia when they are diagnosed.

Maintenance Therapy

Following consolidation therapy, your child will receive maintenance therapy to further reduce the chance of a recurrence. Maintenance therapy is less intensive for standard-risk children than for those who have a higher risk of the leukemia returning. It typically involves a combination of chemotherapy drugs to target cells that may be dormant, or “hiding,” anywhere in the body. Maintenance therapy can last six months for some children with AML, or two to three years for a child with ALL.

Most children with ALL are cured with maintenance therapy. Fewer than 20 percent of high-risk patients and fewer than 10 percent of standard-risk patients will experience a cancer relapse. Most who relapse will return to a second remission with another intensive remission induction therapy.

Bone Marrow Transplantation

Children with AML or ALL who will not be cured with chemotherapy alone may benefit from a bone marrow transplant, also called a stem cell transplant. At Memorial Sloan Kettering, we look to identify these characteristics at the time the child is diagnosed. If we determine that your child needs a transplant, we will work with the bone marrow transplant team to identify potential donors for him or her, both in the family and in the general population through the National Marrow Donor Program.

Innovative Treatments

If your child has relapsed more than once, his or her leukemic cells are likely resistant to previously used treatments. However, these cells might still respond to other treatments. Experts at Memorial Sloan Kettering have evaluated many new combinations of drugs for children who relapse.

One promising treatment we have identified includes combinations of chemotherapy with drugs that have been used extensively to treat other types of cancer.(5) With this treatment, 30 to 40 percent of children who have resistant leukemia have achieved another remission. For children with resistant disease, we are also studying a new medication — called clofarabine — alone and in combination with other chemotherapies. This treatment has successfully returned many children into remission.(6),(7),(8)

For children who are not responding to therapies that are known to be effective, our team can often offer new treatments through clinical trials.

  1. Steinherz PG, Gaynon P,Miller DR, Reaman G, Bleyer A, Finklestein J, Evans RG, Meyers P,Steinherz LJ, Sather H, Hammond D. Improved disease-freesurvival of children with acute lymphoblastic leukemia at high riskfor early relapse with the “New York” regimen ‑ a new intensivetherapy protocol. J Clin Oncol 1986;4:744‑752.
  2. Steinherz PG, Redner A, Steinherz L, Meyers P, Tan C, HellerG. Development of a new intensive therapy for the treatment ofacute lymphoblastic leukemia in children at increased risk of earlyrelapse - The MSK - New York - II protocol. Cancer1993;72:3120-30.
  3. Gaynon PS, Bleyer WA,Steinherz PG, Finklestein JZ, Littman PS, Miller DR, Reaman GH,Sather HN, Hammond GD. Day-7 marrow response and outcome forchildren with acute lymphoblastic leukemia and unfavorableprognostic features. Med Ped Oncol 1990;18:273-279.
  4. Steinherz PG, Gaynon PS, Breneman JC, Cherlow JM, Grossman NJ,Kersey JH, Johnstone HS, Sather HN, Trigg, ME, Hammond D, BleyerWA. Cytoreduction and prognosis in acute lymphoblasticleukemia: The importance of rapid early response. Report fromthe Childrens Cancer Group. J Clin Oncol1996;14:389-398.
  5. Kolb A, Steinherz P. A new multidrug re-induction protocol with topotecan, vinorelbine, thiotepa, dexamethasone, and gemcitabine (TVTG) for relapsed or refractory acute leukemia. Leukemia 2003;17:1967-1972.
  6. Steinherz PG, Shukla N, Kobos R, Steinherz L. Remission re-induction chemotherapy with clofarabine, topotecan, thiotepa, and vinorelbine for patients with relapsed or refractory leukemia. Ped Blood & Cancer 2009;54:687-693.
  7. Hijiya N, Gaynon P, Barry E, Silverman L, Thomson B, Chu R, Cooper T, Kadota R, Rytting M, Steinherz P, Shen V, Jeha S, Abichandani R, Carroll WL. A multi-center phase I study of Clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute leukemia. Leukemia 2009;23:2259-64, Epub 2009 Sept10.
  8. Jeha S, S Razzouk B, Rytting M, Rheingold S, Albano E, Kadota R, Luchtman-Jones L, Bomgaars L, Gaynor P, Goldman S, Ritchey K, Arceci R, Altman A, Stine K, Steinherz L, Steinherz P. The Phase II Study of Clofarabine for Pediatric Patients with Refractory or Relapsed Acute Myeloid Leukemia. J Clin Onc 2009;27:4392-97.