Common brand names

• Vesanoid

How it is administered

All-trans retinoic acid is administered orally in the form of capsules. Each capsule contains 10 mg of the medication. The recommended dosage for blood cancer (specifically acute promyelocytic leukemia, or APL) is 22.5 mg/m² taken twice daily, with the capsules swallowed whole and taken with a meal. Treatment continues until complete remission is documented, or for a maximum of 90 days.

How it works

All-trans retinoic acid is a retinoid that works by inducing the maturation (differentiation) and reducing the proliferation of abnormal promyelocytes in the bone marrow and blood. In patients with acute promyelocytic leukemia (APL), this medication causes the immature, cancerous cells to mature into normal blood cells. This process helps to restore normal blood cell production and reduces the number of abnormal cells, leading to remission.

The exact mechanism is not fully understood, but it is known that all-trans retinoic acid targets the genetic abnormality (t(15;17) translocation or PML/RARα gene fusion) found in APL. By promoting the differentiation of these abnormal cells, the medication helps repopulate the bone marrow and peripheral blood with healthy, functioning cells.

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 t(15;17) translocation or PML/RARα gene expression. It is especially used for patients who have not responded to, have relapsed from, or cannot tolerate anthracycline-based chemotherapy.

This medication is specifically for the induction of remission in APL, a subtype of acute myeloid leukemia. It is not used for other types of blood cancers or leukemias.

Who should not take it

All-trans retinoic acid should not be taken by patients who have a known hypersensitivity to this medication, any of its components, or other retinoids. Symptoms of hypersensitivity can include rash, itching, facial swelling, or difficulty breathing.

It should also not be used in pregnant women due to the 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. It is not recommended for patients without the specific genetic markers (t(15;17) translocation or PML/RARα fusion) associated with APL.

Commonly used with

All-trans retinoic acid is often used in combination with cytoreductive chemotherapy, such as anthracyclines or hydroxyurea, especially in cases of high white blood cell counts (leukocytosis) or to reduce the risk of complications. Most patients receive additional chemotherapy during the remission phase after initial treatment with all-trans retinoic acid.

Commonly tested with

In clinical studies, all-trans retinoic acid has been tested both as a single agent and in combination with cytotoxic chemotherapy agents, particularly anthracyclines. It is also tested with hydroxyurea for managing leukocytosis during induction therapy for APL.