National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Childhood acute lymphoblastic leukemia


Other Names:
Childhood acute lymphocytic leukemia; Pediatric acute lymphoblastic leukemia; Childhood ALL
Categories:
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Childhood acute lymphoblastic leukemia (ALL) is a type of cancer of the blood and bone marrow, and the most common type of cancer in children. In children with this condition, too many stem cells made by the bone marrow become lymphoblasts, B lymphocytes, or T lymphocytes. These cells do not function normally and have trouble fighting off infections. Signs and symptoms may include fever; easy bruising or bleeding; bone or joint pain; painless lumps in the neck, underarm, stomach, or groin; weakness; fatigue; and/or loss of appetite. Treatment depends on several factors and may include combination chemotherapy, targeted therapy, and/or stem cell transplant.[1]

On August 30 2017, Kymriah became the first gene therapy approved by the FDA. Kymriah is now an option for children and young adults up to 25 years old with B-Cell precursor ALL that has proven resistant to other treatments or has relapsed two or more times.[2]
Last updated: 9/18/2017

FDA-Approved Treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.

  • Tisagenlecleucel-T (Brand name: Kymriah) - Manufactured by Novartis Pharmaceuticals Corporation
    FDA-approved indication: August 2017 approved for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Dasatinib (Brand name: Sprycel) - Manufactured by Bristol-Myers Squibb Company
    FDA-approved indication: December 2018, dasatinib (Sprycel) was approved for the treatment of pediatric patients 1 year of age and older with Ph+ CML in chronic phase and newly diagnosed Ph+ ALL in combination with chemotherapy. Previously in June 2013, it was approved for treatment of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia with resistance or intolerance to prior therapy.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Teniposide (Brand name: Vumon) - Manufactured by Bristol-Myers Squibb Co
    FDA-approved indication: July 1992, teniposide (Vumon) was approved for Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Methotrexate oral solution (Brand name: Xatmep) - Manufactured by Silvergate Pharmaceuticals, Inc.
    FDA-approved indication: April 2017, methotrexate oral solution (Xatmep) was approved for the treatment of pediatric patients with acute lymphoblastic leukemia (ALL) as a component of a combination chemotherapy maintenance regimen.
    National Library of Medicine Drug Information Portal
The long-term outlook (prognosis) for people with childhood acute lymphoblastic leukemia (ALL), including the potential for long-term cure, depends on the severity of the condition, laboratory features, and treatment. Factors that may affect prognosis include the age and white blood cell count at the time of diagnosis; biologic characteristics of the leukemia cells; response to chemotherapy; and the amount of cancer cells that remain during treatment or remission. This information can be used to assess the risk for each affected person.[3]

Overall, the cure rate for childhood ALL is more than 80%. Five-year survival rates for children rose to 90% from 2000-2005. Improvement in survival has been seen for all age groups of children, except for infants younger than 1 year (who have a poor outcome). In low-income countries, treatment results have been less encouraging.[3]

Potential acute complications can include tumor lysis syndrome, renal failure, sepsis, bleeding, thrombosis (development of a blood clot), inflammation of the cecum (typhlitis), neuropathy, encephalopathy, or seizures.[3]

Lifelong follow-up is necessary because survivors may have late complications from the treatment, such as secondary malignancy, short stature, growth hormone deficiency, learning disabilities, or cognitive defects.[3]
Last updated: 4/20/2015

Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.

Clinical Research Resources

  • ClinicalTrials.gov lists trials that are related to Childhood acute lymphoblastic leukemia. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

    Please note: Studies listed on the ClinicalTrials.gov website are listed for informational purposes only; being listed does not reflect an endorsement by GARD or the NIH. We strongly recommend that you talk with a trusted healthcare provider before choosing to participate in any clinical study.

Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.

Organizations Supporting this Disease

Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.

Financial Resources

  • Good Days provides help to patients with life-altering conditions. Assistance includes help with the cost of medications and travel.
  • Patient Access Network Foundation (PAN Foundation) has Assistance Programs for those with health insurance who reside in the United States. The disease fund status can change over time, so you may need to check back if funds are not currently available. 

These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.

Where to Start

  • Cancer.net provides oncologist-approved cancer information from the American Society of Clinical Oncology and has information about Childhood acute lymphoblastic leukemia.
  • The Mayo Clinic Web site provides further information on Childhood acute lymphoblastic leukemia.
  • MedlinePlus was designed by the National Library of Medicine to help you research your health questions, and it provides more information about this topic.
  • The Merck Manual provides information on this condition for patients and caregivers. 
  • The National Cancer Institute provides the most current information on cancer for patients, health professionals, and the general public.

In-Depth Information

  • Medscape Reference provides information on this topic. You may need to register to view the medical textbook, but registration is free.
  • The Monarch Initiative brings together data about this condition from humans and other species to help physicians and biomedical researchers. Monarch’s tools are designed to make it easier to compare the signs and symptoms (phenotypes) of different diseases and discover common features. This initiative is a collaboration between several academic institutions across the world and is funded by the National Institutes of Health. Visit the website to explore the biology of this condition.
  • Orphanet is a European reference portal for information on rare diseases and orphan drugs. Access to this database is free of charge.

Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question

  • Is this considered a rare disease? What makes this disease rare? What are the outcomes for children diagnosed with this disease? What are some long term effects that can be expected? See answer


  1. General Information About Childhood Acute Lymphoblastic Leukemia. National Cancer Institute. April 13, 2015; http://www.cancer.gov/cancertopics/pdq/treatment/childALL/Patient/page1.
  2. FDA OKs 1st Gene Therapy for Use in U.S.. MedlinePlus Health Day. August 30, 2017; https://medlineplus.gov/news/fullstory_168110.html.
  3. Vikramjit S Kanwar. Pediatric Acute Lymphoblastic Leukemia. Medscape. December 5, 2014; http://emedicine.medscape.com/article/990113-overview.