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Genetic and Rare Diseases Information Center (GARD)

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Acute promyelocytic leukemia


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Overview

What is acute promyelocytic leukemia?

What causes acute promyelocytic leukemia?

Is acute promyelocytic leukemia inherited?

How might acute promyelocytic leukemia be treated?

What is acute promyelocytic leukemia?

Acute promyelocytic leukemia is an aggressive type of acute myeloid leukemia in which there are too many immature blood-forming cells in the blood and bone marrow.[1][2] It is usually marked by a translocation of chromosomes 15 and 17.[2] Acute promyelocytic leukemia usually occurs in middle-aged adults. Symptoms may include both bleeding and forming blood clots.[1]
Last updated: 6/23/2011

What causes acute promyelocytic leukemia?

Acute promyelocytic leukemia (APL) is caused by a chromosomal translocation (rearrangement of material) that occurs in some of the body's cells during a person's lifetime (a somatic mutation). The translocation involves the fusion of two genes: the PML gene on chromosome 15 and the RARA gene on chromosome 17.  The protein produced by this fusion is referred to as PML-RARα. The PML-RARα protein functions differently than what is typically produced by the normal PML and RARA genes. As a result of the abnormal function, blood cells become "stuck" at the promyelocyte stage and they proliferate (reproduce) abnormally. Excess promyelocytes then accumulate in the bone marrow, disrupting the formation of normal white blood cells and leading to APL. Translocations involving the RARA gene and other genes have been identified in only a few cases of APL.[3]
Last updated: 2/6/2012

Is acute promyelocytic leukemia inherited?

Acute promyelocytic leukemia is not inherited. The condition arises from a translocation in some of the body's cells (somatic cells) that occurs after conception.[3] This is referred to as a somatic mutation. Somatic mutations may affect the individual by causing cancers or other diseases, but they are not passed on to offspring.[3]
Last updated: 2/6/2012

How might acute promyelocytic leukemia be treated?

Most cases of acute promyelocytic leukemia (APL) are treated with an anthracycline chemotherapy drug (daunorubicin or idarubicin) plus the non-chemotherapy drug, all-trans-retinoic acid (ATRA), which is a relative of vitamin A. This treatment leads to remission in 80% to 90% of patients.[4]

Patients who cannot tolerate an anthracycline drug may get ATRA plus another drug called arsenic trioxide.[4] Arsenic trioxide has also proven to be an effective alternative for the 20% to 30% of patients with APL who don't respond to initial treatment or who relapse. If treatment with arsenic trioxide achieves a remission, further courses of this drug may be given. An autologous stem cell transplant may also be an option. If a second remission is not achieved, treatment options may include an allogeneic stem cell transplant or taking part in a clinical trial.[5]

Additional information related to treatment of acute promyelocytic leukemia can be accessed through eMedicine. This includes detailed information related to the use of arsenic trioxide.

Last updated: 2/3/2012

References
  1. General Information About Adult Myeloid Leukemia. National Cancer Institute (NCI). 2009; http://www.cancer.gov/cancertopics/pdq/treatment/adultAML/Patient#Keypoint2. Accessed 6/23/2011.
  2. Acute promyelocytic leukemia. Genetics Home Reference (GHR). April 2011; http://ghr.nlm.nih.gov/condition/acute-promyelocytic-leukemia. Accessed 6/23/2011.
  3. Acute promyelocytic leukemia. Genetics Home Reference. April 2011; http://ghr.nlm.nih.gov/condition/acute-promyelocytic-leukemia. Accessed 2/6/2012.
  4. Treatment of acute promyelocytic (M3) leukemia. American Cancer Society. December 2010; http://www.cancer.org/Cancer/Leukemia-AcuteMyeloidAML/DetailedGuide/leukemia-acute-myeloid-myelogenous-treating-m3-leukemia. Accessed 6/23/2011.
  5. What if the leukemia doesn`t respond or comes back after treatment?. American Cancer Society. December 2010; http://www.cancer.org/Cancer/Leukemia-AcuteMyeloidAML/DetailedGuide/leukemia-acute-myeloid-myelogenous-treating-recurrence. Accessed 6/23/2011.


See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.