Rare Pediatrics News

Disease Profile

Acute myeloid leukemia

Prevalence
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.
1-5 / 10 000

33,100 - 165,500

US Estimated

1-5 / 10 000

51,350 - 256,750

Europe Estimated

Age of onset

All ages

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ICD-10

C92.0

Inheritance

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

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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

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X-linked
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.

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

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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.

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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.

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Not applicable

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Other names (AKA)

Acute myelogenous leukemia

Summary

Acute myeloid leukemia (AML) is a cancer that affects the blood and bone marrow. Conditions are generally called "acute" when they develop quickly and have an aggressive course. The signs and symptoms of AML vary but may include easy bruising; bone pain or tenderness; fatigue; fever; frequent nosebleeds; bleeding from the gums; shortness of breath; and/or weightloss. AML is one of the most common types of leukemia among adults and is rarely diagnosed in people under age 40. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor. Treatment may include a combination of chemotherapy, radiation therapy, bone marrow transplant and/or other drug therapy.[1][2]

Symptoms

This table lists symptoms that people with this disease may have. For most diseases, symptoms will vary from person to person. People with the same disease may not have all the symptoms listed. This information comes from a database called the Human Phenotype Ontology (HPO) . The HPO collects information on symptoms that have been described in medical resources. The HPO is updated regularly. Use the HPO ID to access more in-depth information about a symptom.

Medical Terms Other Names
Learn More:
HPO ID
Percent of people who have these symptoms is not available through HPO
Acute myeloid leukemia
0004808
Autosomal dominant inheritance
0000006
Somatic mutation
0001428

Treatment

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.

  • Glasdegib(Brand name: Daurismo) Manufactured by Pfizer, Inc.
    FDA-approved indication: November 2018, glasdegib (Daurismo) was approved in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
  • Enasidenib(Brand name: Idhifa) Manufactured by Celgene Corporation
    FDA-approved indication: Treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Gemtuzumab ozogamicin(Brand name: Mylotarg) Manufactured by Wyeth Pharmaceuticals, Inc., a Pfizer Company
    FDA-approved indication: Mylotarg™ is indicated for the treatment of newly-diagnosed CD33-positive acute myeloid leukemia in adults and treatment of relapsed or refractory CD33-positive acute myeloid leukemia in adults and in pediatric patients 2 years and older.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Midostaurin(Brand name: Rydapt) Manufactured by Novartis Pharmaceuticals Corporation
    FDA-approved indication: Treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Ivosidenib(Brand name: Tibsovo) Manufactured by Agios Pharmaceuticals, Inc
    FDA-approved indication: July 20, 2018, ivosidenib (Tibsovo) was approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.
    National Library of Medicine Drug Information Portal
  • Venetoclax(Brand name: Venclexta) Manufactured by AbbVie Inc.
    FDA-approved indication: November 2018, venetoclax (Venclexta) was approved in combination with azacitidine, or decitabine, or low-dose cytarabine for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Cytarabine and daunorubicin liposome injection(Brand name: Vyxeos) Manufactured by Celator Pharmaceuticals, Inc.
    FDA-approved indication: Treatment of adults with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)
    National Library of Medicine Drug Information Portal
    Medline Plus Health Information
  • Gilteritinib(Brand name: Xospata) Manufactured by Astellas Pharma US, Inc.
    FDA-approved indication: November 2018, gilteritinib (Xospata) was approved for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test.
    National Library of Medicine Drug Information Portal

Organizations

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

    • The American Cancer Society provides information on Acute myeloid leukemia . Please click on the link to access this resource.
    • Mayo Clinic has an information page on Acute myeloid 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 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.
      • Online Mendelian Inheritance in Man (OMIM) is a catalog of human genes and genetic disorders. Each entry has a summary of related medical articles. It is meant for health care professionals and researchers. OMIM is maintained by Johns Hopkins University School of Medicine. 
      • Orphanet is a European reference portal for information on rare diseases and orphan drugs. Access to this database is free of charge.
      • PubMed is a searchable database of medical literature and lists journal articles that discuss Acute myeloid leukemia . Click on the link to view a sample search on this topic.

        References

        1. Karen Seiter, MD. Acute Myelogenous Leukemia. Medscape Reference. September 2015; https://emedicine.medscape.com/article/197802-overview#a2.
        2. Acute myeloid leukemia. MedlinePlus. May 2014; https://www.nlm.nih.gov/medlineplus/ency/article/000542.htm.