Rare Pediatrics News

Disease Profile

Childhood acute lymphoblastic leukemia

Prevalence estimates on Rare Medical Network websites are calculated based on data available from numerous sources, including US and European government statistics, the NIH, Orphanet, and published epidemiologic studies. Rare disease population data is recognized to be highly variable, and based on a wide variety of source data and methodologies, so the prevalence data on this site should be assumed to be estimated and cannot be considered to be absolutely correct.


US Estimated

Europe Estimated

Age of onset





Autosomal dominant A pathogenic variant in only one gene copy in each cell is sufficient to cause an autosomal dominant disease.


Autosomal recessive Pathogenic variants in both copies of each gene of the chromosome are needed to cause an autosomal recessive disease and observe the mutant phenotype.


dominant X-linked dominant inheritance, sometimes referred to as X-linked dominance, is a mode of genetic inheritance by which a dominant gene is carried on the X chromosome.


recessive Pathogenic variants in both copies of a gene on the X chromosome cause an X-linked recessive disorder.


Mitochondrial or multigenic Mitochondrial genetic disorders can be caused by changes (mutations) in either the mitochondrial DNA or nuclear DNA that lead to dysfunction of the mitochondria and inadequate production of energy.


Multigenic or multifactor Inheritance involving many factors, of which at least one is genetic but none is of overwhelming importance, as in the causation of a disease by multiple genetic and environmental factors.


Not applicable


Other names (AKA)

Childhood acute lymphocytic leukemia; Pediatric acute lymphoblastic leukemia; Childhood ALL


Rare Cancers


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]


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 Bcell 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


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

    Learn more

    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.


        1. General Information About Childhood Acute Lymphoblastic Leukemia. National Cancer Institute. April 13, 2015; https://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; https://emedicine.medscape.com/article/990113-overview.