How it is administered

All-trans retinoic acid is administered orally in the form of capsules. Each capsule contains 10 mg of the active ingredient. The recommended dosage is 22.5 mg/m² taken by mouth twice daily, usually until complete remission is achieved or for up to 90 days, whichever comes first. Capsules should be swallowed whole with water and taken with food. Do not chew, dissolve, or open the capsules.

How it works

All-trans retinoic acid is a type of retinoid that works by promoting the maturation (differentiation) and reducing the growth (proliferation) of certain abnormal blood cells, specifically those involved in acute promyelocytic leukemia (APL). In patients with APL, this medication helps immature leukemia cells develop into normal, functioning blood cells. This process replaces the abnormal cells in the bone marrow and blood with healthy, polyclonal hematopoietic cells.

The exact mechanism by which all-trans retinoic acid works in APL is not fully understood, but it is known to target the genetic abnormality (t(15;17) translocation or PML/RARα gene expression) present in these leukemia cells. By inducing differentiation, it helps restore normal blood cell production, leading to remission in many patients.

Who Should take it

All-trans retinoic acid is indicated for adults and children (1 year and older) with acute promyelocytic leukemia (APL) that is characterized by the presence of the t(15;17) translocation or PML/RARα gene expression. It is especially used in patients who have not responded to, have relapsed from, or cannot tolerate standard anthracycline chemotherapy.

This medication is not specifically indicated for Adult T-cell Leukemia/Lymphoma, but it is used in certain types of blood cancers, particularly APL. Your doctor will determine if this medication is appropriate for your specific type of leukemia based on genetic testing and previous treatment history.

Who should not take it

You should not take all-trans retinoic acid if you have a known hypersensitivity to this medication, any of its components, or other retinoids. Signs of hypersensitivity can include rash, itching, swelling of the face, or difficulty breathing.

It should also not be used in patients who do not have the specific genetic markers (t(15;17) translocation or PML/RARα fusion) associated with APL. This medication is not recommended for use in patients without these genetic features. Additionally, it should not be used during pregnancy due to a high risk of birth defects and fetal loss. Women of childbearing potential must use effective contraception during treatment and for a period after stopping the medication.

Commonly used with

All-trans retinoic acid is often used in combination with other chemotherapy agents, especially anthracyclines, during the induction and consolidation phases of APL treatment. In cases of high white blood cell counts, cytoreductive chemotherapy (such as hydroxyurea) may be given together with all-trans retinoic acid to reduce the risk of complications.

It is important to avoid using this medication with vitamin A supplements, strong CYP3A inhibitors or inducers, and anti-fibrinolytic agents, as these can increase the risk of side effects or reduce the effectiveness of treatment.

Commonly tested with

All-trans retinoic acid has been tested in clinical studies both as a single agent and in combination with chemotherapy drugs such as anthracyclines (e.g., daunorubicin, idarubicin) in patients with APL. It is also sometimes used with cytoreductive agents like hydroxyurea to manage high white blood cell counts during treatment.

Clinical trials have evaluated its effectiveness in both previously treated and untreated patients, and it is now a standard part of APL therapy protocols.