Treatments for acute promyelocytic leukemia

Acute promyelocytic leukemia (APL) is a subtype of acute myelogenous leukemia (AML) that has its own treatment options.

Induction treatment

The goal of induction treatment for APL is to treat all the leukemia cells in the blood and bone marrow and bring about a remission.

Tretinoin (all-trans retinoic acid, ATRA, Vesanoid) is used to treat APL. It is a drug derived from vitamin A. It must be followed by or given with other chemotherapy drugs to bring about a long-lasting remission. About 80%–90% of people with APL reach a long-lasting remission.

An anthracycline drug, such as one of the following, is given in combination with tretinoin:

  • daunorubicin (Cerubidine, daunomycin)
  • idarubicin (Idamycin)

Tretinoin may be given in combination with arsenic trioxide, rather than with an anthracycline drug, when the white blood cell count at diagnosis is 10,000 or less.

Consolidation treatment

Consolidation treatment is also called post-remission therapy. The goal of consolidation treatment for APL is to treat any leukemia cells that are still in the blood or bone marrow once remission is reached. It is done to maintain complete remission and prevent relapse.

During consolidation treatment for APL, tretinoin is continued along with idarubicin or daunorubicin. Other drugs that may be added include:

  • cytarabine (Cytosar)
  • mercaptopurine (Purinethol, 6-MP)
  • methotrexate
  • arsenic trioxide

Maintenance treatment

The goal of maintenance treatment for APL is to prevent leukemia cells from coming back (called relapse).

Maintenance treatment includes continuing tretinoin for 1–2 years. Tretinoin may be used alone or in combination with:

  • methotrexate
  • mercaptopurine

Treatments for relapsed or refractory APL

Relapsed, or recurrent, APL means the leukemia has come back after treatment and reaching remission. Refractory APL means the leukemia did not respond to treatment. Complete remission has not been reached because the chemotherapy did not kill enough leukemia cells.

The most common treatment for relapsed or refractory APL includes:

  • tretinoin and a chemotherapy drug such as cytarabine and daunorubicin
  • arsenic trioxide
  • allogeneic or autologous stem cell transplant (offered for relapsed APL if a second remission is reached)

Supportive therapy

Supportive therapy is important during every phase of APL treatment. It is used to treat the complications that usually happen with treatments for APL and the disease itself.

Supportive therapies given during treatment for relapsed or refractory APL may include:

  • antibiotics and antifungals to treat infections
  • growth factors to help the bone marrow recover from chemotherapy (chemotherapy can affect the bone marrow so it doesn’t make enough healthy blood cells, which can increase the risk for infection)
  • transfusions of red blood cells, platelets, fresh frozen plasma and cryoprecipitate (a product that replaces clotting factors) as needed

Treatments for differentiation syndrome

Differentiation syndrome used to be called retinoic acid syndrome. It is a complication caused by tretinoin, and it may also happen with arsenic trioxide. It is most often seen during the first cycle of treatment with these drugs.

The symptoms of differentiation syndrome include:

  • fever
  • weight gain
  • swelling caused by a buildup of fluid in the body (called edema)
  • buildup of fluid around the lungs (called pleural effusion)
  • buildup of fluid around the heart (called pericardial effusion)
  • shortness of breath
  • difficulty breathing
  • low blood pressure

If the symptoms of differentiation syndrome are mild, you can usually keep taking tretinoin or arsenic trioxide. Doctors will also prescribe a steroid, such as dexamethasone (Decadron, Dexasone).

If differentiation syndrome is severe, doctors will stop giving tretinoin or arsenic trioxide and give steroids until the symptoms completely go away. Once the symptoms go away, you can start taking the drug again, usually along with dexamethasone.

Clinical trials

You may be asked if you want to join a clinical trial for APL. Find out more about clinical trials.

Expert review and references

  • American Cancer Society. Leukemia - Acute Myeloid (Myelogenous). Atlanta, GA: American Cancer Society; 2013:
  • American Society of Clinical Oncology (ASCO). Leukemia - Acute Myeloid - AML: Treatment Options. Alexandria, VA.: American Society of Clinical Oncology (ASCO); 2013:
  • Kebriaei P, Champlin R, de Lima M, et al . Management of acute leukemias. DeVita VT Jr, Lawrence TS, & Rosenberg SA. Cancer: Principles & Practice of Oncology. 9th ed. Philadelphia: Wolters Kluwer Health/Lippincott Williams & Wilkins; 2014: 131: pp. 1928-1954.
  • Kurtin SE . Leukemia and myelodysplastic syndromes. Yarbro, CH, Wujcki D, & Holmes Gobel B. (eds.). Cancer Nursing: Principles and Practice. 7th ed. Sudbury, MA: Jones and Bartlett; 2011: 57: pp. 1369-1398.
  • National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ®) Health Professional Version. Bethesda, MD: National Cancer Institute; 2014:
  • Seiter K . Acute myelogenous leukemia treatment & management. WebMD LLC; 2014.
  • Wiernic PH, Gallagher RE, Tallman MS . Acute promyelocytic leukemia. Wiernik PH, Goldman JM, Dutcher JP & Kyle RA (eds.). Neoplastic Diseases of the Blood. 5th ed. Springer; 2013: 23: pp. 403-453.