Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents

Status: Recruiting
DFCI Protocol ID: 16-001

Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow (along with other blood cells) and help fight infections. In ALL, the cancerous lymphocytes are called lymphoblasts. They do not help fight infection and crowd out the normal blood cells in the bone marrow so that the body cannot make enough normal blood cells. ALL is always fatal if it is not treated. With current treatments, most children and adolescents with this disease will be cured. The standard treatment for ALL involves about 2 years of chemotherapy. The drugs that are used, and the doses of the drugs, are similar but not identical for all children and adolescents with ALL. Some children and adolescents receive stronger treatment, especially during the first several months. A number of factors are used to decide how strong the treatment should be to give the best chance for cure. These factors are called "risk factors". This trial is studying the use of a new, updated set of risk factors to decide how strong the treatment will be. The study also will test a new way of dosing a chemotherapy drug called pegaspargase (which is part of the standard treatment for ALL) based on checking levels of the drug in the blood and adjusting the dose based on the levels.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Lewis Silverman, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

Inclusion Criteria:

  1. Confirmed diagnosis of acute lymphoblastic leukemia. Diagnosis should be made by bone
     marrow aspirate or biopsy demonstrating ≥ 25% involvement by lymphoblasts, with flow
     cytometry or immunohistochemistry confirming B-precursor or T-ALL phenotype.

     -- For patients with circulating blasts in the peripheral blood, flow cytometry
     confirmation of B-ALL or T-ALL phenotype is sufficient for registration onto the
     study. Bone marrow aspirate and/or biopsy should be performed as soon as feasible,
     preferably prior to the initiation of any therapy.

  2. Prior Therapy: No prior therapy is allowed except for the following:

       -  Corticosteroids: Short courses of corticosteroid (defined as ≤ 7 days of
  corticosteroids within the 4-weeks preceding registration) are allowed prior to

  --- Participants who have been on corticosteroids chronically (defined as more
  than 7 days of corticosteroids within the 4-weeks preceding registration or more
  than 28 days of corticosteroids over the preceding 6 months) are not eligible.

       -  IT cytarabine: A single dose of intrathecal cytarabine (at the time of the
  diagnostic lumbar puncture) is allowed prior to registration. If patient has
  received IT cytarabine prior to registration, Day 1 IT cytarabine should not be

       -  Emergent Radiation Therapy: Emergent radiation to the mediastinum or other
  life-threatening masses is allowed prior to registration.

  3. Age: 365 days to < 22 years

  4. Direct bilirubin < 1.4 mg/dL (23.9 micromoles/L).

  5. Ability of parent or guardian to understand and the willingness to sign a written
     informed consent document.

Exclusion Criteria:

  1. Mature B-cell (Burkitt's) ALL (defined by the presence of surface immunoglobulin
     and/or the t(8;14)(q24;q32), t(8;22), or t(2;8) translocation and/or c-myc-gene

  2. World Health Organization diagnostic criteria of mixed phenotype acute leukemia
     (MPAL) or leukemia of ambiguous lineage

  3. Any chemotherapy or radiotherapy for previous malignancy are not eligible.

  4. Treatment in past with any anti-neoplastic agent, including methotrexate,
     6-mercaptopurine, 6-thioguanine, vincristine cyclophosphamide, cytarabine (except for
     IT cytarabine), or any anthracycline, for any reason (eg, rheumatologic or autoimmune

  5. Currently receiving any investigational agents.

  6. Known HIV-positivity

  7. Uncontrolled intercurrent illness including, but not limited to ongoing infection
     with vital sign instability (hypotension, respiratory insufficiency),
     life-threatening acute tumor lysis syndrome (eg, with renal failure), symptomatic
     congestive heart failure, cardiac arrhythmia, intracranial or other uncontrolled
     bleeding, or psychiatric illness/social situations that would limit compliance with
     study requirements.

  8. Pregnant women are excluded from this study because many of the agents used on this
     protocol have potential for teratogenic and/or abortifacient effects. Because there
     is an unknown but potential risk of adverse events in nursing infants secondary to
     treatment of the mother with these chemotherapy agents, breastfeeding should be
     discontinued if the mother is enrolled.

  9. History of a previous malignancy. Exception: Individuals with a previous malignancy
     treated with surgery only (no chemotherapy or radiotherapy) more than 5 years prior
     to registration may be enrolled.

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