Drug

Oral tretinoin (ATRA)

A capsule treatment for acute promyelocytic leukaemia (APL); it must be started at the first suspicion

Updated: 28 October 20261 min read3 references
Generic name
Tretinoin
Brand names
Vesanoid, ATRA
Drug class
Retinoid (differentiation inducer)
Route
Oral (capsule)
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Key points

  • Tretinoin makes APL leukaemia cells mature, and together with arsenic it cures most patients [2].
  • It must be started at the first suspicion of APL, before the genetic test is confirmed; most deaths in the first days are from bleeding [3].
  • Differentiation syndrome (fever, shortness of breath, swelling) is a dangerous but treatable side effect [1].

What is this drug and how does it work?

For patients

In APL, a faulty gene stops white blood cells from maturing. Tretinoin (a form of vitamin A) removes this block so that the cells mature and die off [1].

For physiciansSpecialist notes
For physicians

Binds to retinoic acid receptors and degrades the PML-RARA fusion protein; induces differentiation of promyelocytes [1].

Which cancers is it used for?

For patients
What is this drug for?
For physiciansApproved uses and evidence
For physicians
Approved uses and evidence
DiseaseUseKey evidence
Low-‎ and intermediate-risk APLWith arsenicAPL0406: 2-year event-free survival 97% vs 86% with chemotherapy [2]
High-risk APLWith chemotherapy, or with arsenic and idarubicin/gemtuzumabELN [3]

How it is taken and dose

Show detailsHide

Capsules twice a day with food [1]. If the patient cannot swallow, the doctor will suggest another way. Do not stop treatment on your own.

For physiciansStandard dose (FDA label), 1 regimen
For physicians

Standard dose (FDA label)

Tretinoin

APL

APL

DrugDoseRouteSchedule
Tretinoin22.5 mg/m² twice a day (45 mg/m² a day)Oral, with foodDaily until complete remission
Cycle length
Induction until remission; consolidation 2 weeks on, 2 weeks off
Duration
According to protocol

Dose as in the label [1]; the schedule with arsenic is in the ATRA and arsenic regimen [2].

Evidence: APL0406 (2013)

Doses follow the trial protocol, guideline or official drug label (source given in the notes of each regimen). Before prescribing, check blood counts, renal and hepatic function, drug interactions and the patient’s condition, and adjust the dose according to the drug label.

Side effects

For patients

The most common side effects: headache, fever, dry skin and lips, bone pain, nausea and raised blood fats [1].

For physiciansSpecialist notes
For physicians

Headache (sometimes with raised intracranial pressure), fever, dry skin and mucous membranes, bone pain, hypertriglyceridaemia, raised liver enzymes [1].

Serious side effects

For patients

Serious side effects: differentiation syndrome (fever, shortness of breath, weight gain, swelling, low blood pressure), a rapid rise in white blood cells, blood clots and raised pressure inside the skull (severe headache with blurred vision) [1].

For physiciansSpecialist notes
For physicians
  • Differentiation syndrome: at the first sign, dexamethasone 10 mg IV twice a day [1, 3].
  • Leucocytosis, thrombosis, pseudotumour cerebri, hepatotoxicity [1].
  • Highly teratogenic [1].

When should I call my doctor?

  • Fever, shortness of breath or cough
  • Rapid weight gain or swelling
  • Severe headache or blurred vision
  • Bleeding

Interactions, pregnancy and precautions

  • Pregnancy: tretinoin causes severe harm to an unborn baby; a pregnancy test before starting, and reliable contraception during treatment and for one month afterwards [1].
  • Other medicines: do not take extra vitamin A or tetracyclines [1].
For patients

Frequently asked questions

Why not wait for the genetic test result?

In APL, bleeding can be fatal within days; starting tretinoin early lowers this risk [3].

Related cancer types

Treatment regimens (physicians)

References

3 references
Show all references, 1 with a study cardClose the reference list

Numbers in the text, such as [1], refer to these sources. Sources with a “Study card” button are summarised in the site’s evidence library.

  1. 1
    TRETINOIN — Prescribing InformationPar Health Usa, LLC · FDA label · 2026
    Source Back to text: 1 2 3 4 5 6 7 8 9 10 11 12 13 14
  2. 2
    Retinoic acid and arsenic trioxide for acute promyelocytic leukemiaLo-Coco F, Avvisati G, Vignetti M, et al · N Engl J Med · 2013;369:111-21
    Study cardDOIPubMed Back to text: 1 2 3 4
  3. 3
    Management of acute promyelocytic leukemia: updated recommendations from an expert panel of the European LeukemiaNetSanz MA, Fenaux P, Tallman MS, et al · Blood · 2019;133:1630-1643
    DOIPubMed Back to text: 1 2 3 4 5

This page is based on reputable scientific sources and was last updated on 28 October 2026. The content of this website is for educational and informational purposes only and does not replace consultation, diagnosis or treatment by a specialist physician. Consult your treating physician about any treatment decision.