Acute Promyelocytic Leukemia Medication

Updated: Mar 19, 2021
  • Author: Sandy D Kotiah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication Summary

The goals of pharmacotherapy are to induce remission, prevent complications, and reduce morbidity in patients with acute promyelocytic leukemia (APL). All-trans-retinoic acid (ATRA) should be prescribed on both an inpatient and outpatient basis. Patients should also be on prophylactic antifungal and antiviral medications during treatment, as these individuals have altered lymphocyte function due to both the underlying leukemia and cytotoxic therapy.


Antineoplastic Agents

Class Summary

Antineoplastic agents inhibit cell growth and proliferation.

Arsenic trioxide (Trisenox)

May cause DNA fragmentation and damage or degrade the fusion protein PML-RAR alpha. Used in APL patients who have the presence of the t(15;17) translocation or PML/RAR-alpha gene expression. It is indicated also for induction of remission and consolidation in patients with APL who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy and in combination with tretinoin for treatment of adults with newly diagnosed low risk APL.

Idarubicin (Idamycin)

Used for induction or consolidation therapy. Topoisomerase-II inhibitor. Inhibits cell proliferation by inhibiting DNA and RNA polymerase.

Tretinoin (Vesanoid)

Induces maturation of acute promyelocytic leukemia cells in cultures. Used in both induction and maintenance phases for patients with acute promyelocytic leukemia.

Cytarabine (Cytosar-U)

Converted intracellularly to active compound cytarabine-5'-triphosphate, which inhibits DNA polymerase. Cell cycle S phase specific. Blocks the progression from G1 to S phase and in turn kills cells that undergo DNA synthesis in S phase of cell proliferation cycle.

Mitoxantrone (Novantrone)

Used for induction or consolidation therapy. Inhibits cell proliferation by intercalating DNA and inhibiting topoisomerase II.

Daunorubicin (Cerubidine)

Anthracycline antibiotic. Binds to nucleic acids by intercalation between base pairs of DNA, interfering with DNA synthesis. Causes inhibition of DNA topoisomerase II. Half-life is 14-20 h (23-40 h for active metabolite). Used in the induction phase of treatment.


Antineoplastics, Monoclonal Antibody

Gemtuzumab (Mylotarg)

Antibody-drug conjugate for newly diagnosed and relapsed or refractory CD33-positive acute myeloid leukemia. Gemtuzumab originally received accelerated approval in 2000; it was voluntarily withdrawn from the US market in 2010 after a confirmatory trial failed to show clinical benefit and reported an elevated rate of fatal toxicity, but was approved again in 2017 at a lower, fractionated dose. Gemtuzumab carries a black box warning for hepatotoxicity, including severe or fatal veno-occlusive disease.