Acute Promyelocytic Leukemia Treatment

Acute promyelocytic leukemia (APL) is an aggressive form of acute myeloid leukemia (AML) characterized by a rapid buildup of immature white blood cells (promyelocytes) in the bone marrow and blood. Usually, the condition is caused by a genetic mutation involving a translocation between chromosomes 15 and 17, which disrupts normal blood cell development.

APL can affect people of all ages, but it most frequently occurs in adults aged 20 to 50. Symptoms can include easy bruising, fatigue, frequent infections, and, in some cases, a life-threatening bleeding disorder. Usually, this type of blood cancer is diagnosed through blood work, bone marrow biopsy and genetic testing.

Acute promyelocytic leukemia is relatively rare, accounting for approximately 15% of AML cases. Although the condition is considered a medical emergency, it can often be managed with immediate, specialized care.

What does acute promyelocytic leukemia treatment involve?

In many cases, APL is treated with a targeted approach that addresses both its genetic cause and its potentially life-threatening complications, such as excessive bleeding and blood clots. The cornerstone of treatment is a combination of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), which work together to promote the maturation of abnormal promyelocytes into healthy white blood cells. This regimen can significantly improve the outcome and may eliminate the need for traditional chemotherapy in a low-risk case.

In a high-risk case involving elevated white blood cell counts, ATRA and ATO may be combined with chemotherapy agents, such as anthracyclines, for heightened effectiveness. Beginning in the initial phase of treatment, many patients benefit from supportive care to help manage bleeding risks and correct coagulation abnormalities, which are common in acute promyelocytic leukemia.

The phases of APL treatment are:

Induction therapy

The initial phase of APL treatment aims to achieve complete remission. During induction therapy, the patient will be carefully monitored for differentiation syndrome, a potentially serious complication caused by the rapid maturation of leukemic cells. If it occurs, the symptoms can typically be managed with corticosteroids and other supportive interventions.

Consolidation therapy

Once cancer remission is achieved, consolidation therapy will begin. This phase of APL treatment typically involves additional cycles of all-trans retinoic acid and arsenic trioxide to eliminate any remaining leukemic cells and help prevent relapse. Consolidation therapy may include chemotherapy or other targeted agents as appropriate.

Maintenance therapy

Following consolidation therapy, some patients receive low doses of all-trans retinoic acid, methotrexate or mercaptopurine administered over several months or years, depending on the specifics of the treatment plan. However, with current ATRA and ATO treatment protocols, maintenance therapy is not always necessary.

Throughout all phases of APL treatment, the patient will undergo periodic blood tests and bone marrow evaluations to help the physician assess the response to therapy and detect any signs of recurrence.

What are the potential benefits of acute promyelocytic leukemia treatment?

The primary benefit of APL treatment is the potential for long-term cancer remission and cure. When detected early and managed with targeted therapy, acute promyelocytic leukemia is one of the most treatable hematologic cancers. Most patients who complete treatment with all-trans retinoic acid and arsenic trioxide achieve remission, and many remain cancer-free for years.

In addition to high remission rates, APL treatment can help prevent serious complications, such as life-threatening bleeding and organ damage caused by the buildup of immature white blood cells. Effective therapy can restore normal blood cell development, potentially improving the patient’s energy levels, immune function and overall well-being.

With a timely diagnosis and close adherence to a physician-prescribed treatment plan, most patients can achieve a significantly improved quality of life and a strong chance of returning to their normal daily activities after recovery.

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What are the possible side effects of acute promyelocytic leukemia treatment?

Although APL treatment can be highly effective, it carries certain risks. The side effects can vary based on the medications used and the patient’s response and overall health. The hallmark treatment regimen—all-trans retinoic acid combined with arsenic trioxide—can sometimes lead to:

Differentiation syndrome

One of the most serious potential side effects of APL treatment is differentiation syndrome, a life-threatening complication that occurs when large numbers of leukemic cells mature rapidly and release inflammatory substances into the bloodstream. This can trigger a variety of symptoms, such as fever, sudden weight gain, shortness of breath, low blood pressure and fluid accumulation around the lungs or heart. Early recognition and prompt treatment with corticosteroids are essential to managing this complication effectively.

Cardiac effects

Arsenic trioxide can affect the heart’s rhythms and may lead to QT prolongation, which occurs when the heart’s electrical system takes longer than normal to recharge between beats, increasing the risk of irregular heartbeats. Electrolyte imbalances may worsen this side effect, so careful monitoring is essential during APL treatment.

Changes in liver and kidney function

Both ATRA and ATO can elevate liver enzymes, such as alanine aminotransferase (ALT) and aspartate aminotransferase (AST), which may stress and inflame the liver. In rare cases, these medications may also contribute to more serious liver toxicity. Possible signs include yellowing of the skin or eyes (jaundice), dark urine and abdominal discomfort. If significant liver enzyme elevations are observed, the APL treatment plan may need to be adjusted or paused.

ATO can also affect kidney function by altering the levels of electrolytes, such as potassium, magnesium and creatinine, in the bloodstream. In some cases, this can impair the ability of the kidneys to filter waste effectively. Fluid retention or dehydration may also contribute to reduced kidney performance. During APL treatment, the patient will be closely monitored for signs of kidney stress, and supportive measures such as hydration and electrolyte supplementation may be used to protect kidney function.

Fatigue

As the body recovers and healthy blood cell development resumes, the patient may experience periods of weakness and tiredness. Fatigue is especially common during the induction phase of APL treatment.

Other side effects

Other possible side effects of APL treatment include headache, nausea, dry skin, rash, bone pain and increased blood lipid levels. Most can be managed with supportive care and adjustments to the treatment plan if necessary. Close monitoring by a specialized healthcare team can help minimize the risks of APL therapy and ensure the patient’s safety throughout treatment.

What is the recovery process like?

Recovering from APL treatment is an ongoing process that generally involves regular follow-up care, physical healing and emotional adjustment. Although many patients achieve cancer remission, continued monitoring will be essential to ensure the leukemia does not return and manage any lingering side effects.

Medical follow-up

Typically, the patient will undergo frequent blood tests and bone marrow evaluations in the months following APL treatment to confirm continued remission. During these visits, the physician will monitor the patient’s blood cell counts and organ function and check for signs of relapse. Over time, the frequency of the follow-up appointments may decrease if the patient remains in remission.

Physical recovery

Treatment-related side effects, such as fatigue, weakness and reduced immune function, may persist for up to several weeks or months after APL therapy ends. Many patients gradually regain their strength, but the recovery timeline can vary based on individual health factors and the intensity of treatment. A balanced diet, regular exercise and adequate rest can support this process.

Emotional and psychological adjustment

The experience of facing a potentially life-threatening illness may lead to emotional challenges, such as anxiety, depression or fear of recurrence. Support groups, counseling services and survivorship programs can provide valuable guidance and reassurance as the patient transitions back to everyday life.

Long-term considerations

Some patients may experience late-onset side effects of treatment, including changes in heart, liver or kidney function. With ongoing care from an oncologist or hematologist, these risks can be addressed. Most patients who complete APL treatment and remain in remission for several years are considered cured.

Overall, recovery from APL treatment is highly individualized. With consistent medical oversight and supportive care, most patients can return to their normal activities and enjoy a good quality of life.

Benefit from world-class care at Moffitt Cancer Center

If you would like to learn more about acute promyelocytic leukemia treatment, you can request an appointment with a specialist in the renowned Malignant Hematology Program at Moffitt by calling 1-888-663-3488 or submitting a new patient registration form online. We do not require referrals.