How is acute promyelocytic leukemia (APL) treated?

Updated: May 03, 2019
  • Author: Sandy D Kotiah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Patients with acute leukemia should be treated in centers staffed by specially trained physicians and nurses. Availability of supportive care, such as platelet transfusion therapy, and a well-equipped laboratory is also crucial.

Given the frequent abrupt onset of acute promyelocytic leukemia (APL) and the risk of severe hemorrhagic events, immediate institution of all-trans-retinoic acid (ATRA) and/or arsenic trioxide (ATO) treatment and supportive therapy is indicated, to avoid early death. Current recommendations strongly suggest starting these measures upon clinical suspicion of APL and before genetic confirmation of the diagnosis. [14]

APL treatment has three phases: induction, consolidation, and maintenance. There is debate among experts about the ideal induction therapy, the best initial treatment for the elderly, the subset of patients most likely to benefit from maintenance therapy, and the most effective regimen for relapsed disease. These issues are the subject of ongoing clinical trials (see

ATRA is an important agent in all three phases of APL treatment. [20] ATRA can lead to terminal differentiation of malignant promyelocytes into mature neutrophils. However, ATRA alone cannot eradicate the malignant clone. Achievement of complete hematologic and molecular remission requires the addition of ATO or chemotherapy. [21, 22]

Resistance to ATRA has been seen with cytogenetic variants of APL, especially cases with the PLZF-RARA mutation. However, resistance may also develop as a secondary event in PML-RARA APL.

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