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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Acute promyelocytic leukemia


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Treatment

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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.[1]

Patients who cannot tolerate an anthracycline drug may get ATRA plus another drug called arsenic trioxide.[1] 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.[2]

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. 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.
  2. 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.


Clinical Trials & Research for this Disease

  • The National Cancer Institute has published results from a¬†clinical trial involving post-remission therapy for acute promyelocytic leukemia. Click on the link to view information related to this study.
  • ClinicalTrials.gov lists trials that are studying or have studied Acute promyelocytic leukemia. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • The Research Portfolio Online Reporting Tool (RePORT) provides access to reports, data, and analyses of research activities at the National Institutes of Health (NIH), including information on NIH expenditures and the results of NIH-supported research. Although these projects may not conduct studies on humans, you may want to contact the investigators to learn more. To search for studies, click on the link and enter the disease name in the "Terms Search" box. Then click "Submit Query".

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Arsenic trioxide
Trade Name
(Manufacturer Name)
Trisenox®
(Cephalon® Oncology)
Indication
The FDA has approved this product to be used in this manner.
For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) transloc
More Information about this product Drug Information Portal
Medline Plus Health Information

Generic Name Tretinoin
Trade Name
(Manufacturer Name)
Vesanoid®
(Roche Pharmaceuticals)
Indication
The FDA has approved this product to be used in this manner.
Induction of remission in patients with acute promyelocytic leukemia who are refractory to or unable to tolerate anthracycline based cytotoxic chemotherapeutic regimens.
More Information about this product Drug Information Portal
Medline Plus Health Information

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