Trisenox (arsenic trioxide)
An approved treatment for Acute Myeloid Leukemia.
The same compound appears under different names depending on the context. Here is how to identify Arsenic trioxide wherever you encounter it, plus the key facts at a glance.
- Generic name
- Arsenic trioxide
- Brand name
- Trisenox
- Development codes
- ATO, As2O3
- Drug class
- Arsenical (differentiating agent)
- Manufacturer
- Teva (Trisenox) and generic manufacturers
- How it's taken
- IV infusion over 1 to 2 hours, daily during induction
An IV infusion for acute promyelocytic leukemia (APL), the AML subtype defined by the PML-RARA fusion. Approved in 2000 for relapsed APL and in 2018, with tretinoin, for newly diagnosed low-risk APL, where the 2 drugs together cure most patients without conventional chemotherapy. It is not used for other types of AML.
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Where Arsenic trioxide fits
With tretinoin, the standard treatment for low-risk APL and the reason APL is now the most curable form of acute leukemia; high-risk APL adds chemotherapy. It has no role in non-APL AML.
How Arsenic trioxide works
APL cells carry a fusion protein, PML-RARA, that freezes them at the promyelocyte stage. Arsenic trioxide attaches to the PML part of that protein and marks it for destruction, so the block lifts and the cells mature and die; in the laboratory it also causes DNA fragmentation and damage to the fusion protein[1]. Tretinoin acts on the RARA half of the same protein, which is why the 2 work so well together.
Mechanism: Degrades the PML-RARA fusion protein that drives acute promyelocytic leukemia, so the leukemia cells mature and die
Side effects and safety
Arsenic trioxide carries a boxed warning for differentiation syndrome (fever, breathlessness, low oxygen, lung infiltrates, fluid around the lungs or heart, weight gain, swelling, low blood pressure and kidney, liver or multi-organ dysfunction, treated with high-dose steroids), for heart conduction problems including QT prolongation, torsades de pointes and complete heart block (ECGs and electrolytes before and during treatment), and for encephalopathy including Wernicke's, a thiamine deficiency syndrome that is treated with thiamine[1].
Other warnings are a rising white cell count, liver toxicity, harm to an unborn baby, and the carcinogenic potential of arsenic[1]. Common effects include nausea, fatigue, fever, headache, cough, rash, high blood sugar, low potassium and magnesium, and raised liver enzymes.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Arsenic trioxide
Given as an IV infusion over 1 to 2 hours in a clinic or hospital. For newly diagnosed low-risk APL with tretinoin, the label uses 0.15 mg/kg a day until bone marrow remission (up to 60 days), then 4 consolidation cycles of 0.15 mg/kg a day, 5 days a week during weeks 1 to 4 of each 8-week cycle, with tretinoin. For relapsed APL, induction is 0.15 mg/kg a day until remission (up to 60 doses) and consolidation 0.15 mg/kg a day for 25 doses over up to 5 weeks[1]. ECGs, electrolytes and blood counts are checked throughout.
Availability and cost
Generic versions may be available at a lower cost. Ask your pharmacist.
Clinical trial results
The first-line approval rests on the APL0406 trial (NCT00482833) of arsenic trioxide plus tretinoin against tretinoin plus chemotherapy in newly diagnosed low or intermediate-risk APL, described in the label, and on the earlier single-arm study of relapsed APL in which most patients reached complete remission with arsenic alone[1]. The label defines low-risk APL by a white cell count of 10 x 10^9/L or less at diagnosis.
Development history
Trisenox was approved by the FDA on September 25, 2000 for relapsed or refractory APL and on January 12, 2018 in combination with tretinoin for newly diagnosed low-risk APL; generic arsenic trioxide injection has since been approved[2].
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Common questions about Arsenic trioxide
▸What is Arsenic trioxide (Trisenox)?
An IV infusion for acute promyelocytic leukemia (APL), the AML subtype defined by the PML-RARA fusion. Approved in 2000 for relapsed APL and in 2018, with tretinoin, for newly diagnosed low-risk APL, where the 2 drugs together cure most patients without conventional chemotherapy. It is not used for other types of AML.
▸How does Arsenic trioxide work?
APL cells carry a fusion protein, PML-RARA, that freezes them at the promyelocyte stage. Arsenic trioxide attaches to the PML part of that protein and marks it for destruction, so the block lifts and the cells mature and die; in the laboratory it also causes DNA fragmentation and damage to the fusion protein[1]. Tretinoin acts on the RARA half of the same protein, which is why the 2 work so well together.
▸What are the side effects of Arsenic trioxide?
Arsenic trioxide carries a boxed warning for differentiation syndrome (fever, breathlessness, low oxygen, lung infiltrates, fluid around the lungs or heart, weight gain, swelling, low blood pressure and kidney, liver or multi-organ dysfunction, treated with high-dose steroids), for heart conduction problems including QT prolongation, torsades de pointes and complete heart block (ECGs and electrolytes before and during treatment), and for encephalopathy including Wernicke's, a thiamine deficiency syndrome that is treated with thiamine[1]. Other warnings are a rising white cell count, liver toxicity, harm to an unborn baby, and the carcinogenic potential of arsenic[1]. Common effects include nausea, fatigue, fever, headache, cough, rash, high blood sugar, low potassium and magnesium, and raised liver enzymes.
▸How is Arsenic trioxide taken?
Given as an IV infusion over 1 to 2 hours in a clinic or hospital. For newly diagnosed low-risk APL with tretinoin, the label uses 0.15 mg/kg a day until bone marrow remission (up to 60 days), then 4 consolidation cycles of 0.15 mg/kg a day, 5 days a week during weeks 1 to 4 of each 8-week cycle, with tretinoin. For relapsed APL, induction is 0.15 mg/kg a day until remission (up to 60 doses) and consolidation 0.15 mg/kg a day for 25 doses over up to 5 weeks[1]. ECGs, electrolytes and blood counts are checked throughout.
▸Is Arsenic trioxide FDA approved?
Yes, Arsenic trioxide (Trisenox) is FDA approved (2000) for the treatment of Acute Myeloid Leukemia.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- openFDA · 2025-06-30. Arsenic trioxide injection prescribing information (Eugia US). https://api.fda.gov/drug/label.json?search=set_id:%222a999752-f350-4eff-97e7-e7106dfa85b5%22
- FDA. Trisenox approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=021248