The goal of maintenance therapy for acute myeloid leukemia (AML) is to keep the disease in remission, or at a low leukemic cell count. Therapy management and strategies for care disparities, side effects, and adherence challenges were discussed in an oral presentation at the 2023 ONA Summit Live Virtual Meeting.

“We are in an era where both patients and clinicians have more treatment options available for specific subsets of AML, and this increases the complexity of the decision making process,” stated Ashley Leak Bryant, PhD, RN, OCN, FAAN, associate professor for the School of Nursing, The University of North Carolina at Chapel Hill, and assistant director of the Cancer Research Training and Education Coordination at UNC Lineberger Comprehensive Cancer Center in Chapel Hill, North Carolina, in her presentation.

Treatment of AML consists of induction, consolidation, and maintenance. Induction involves cytarabine-based therapy (7 days continuous IV) and an anthracycline (idarubicin, doxorubicin; 3 days of IV push), with the goal of achieving remission (defined as <5% leukemia cells). Consolidation therapy is used to control leukemia cells left in the body via subsequent cycles of chemotherapy to decrease the risk of relapse.

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Maintenance therapy is used to keep the AML in remission via low-dose intensity therapies (azacitidine alone or azacitidine with venetoclax). This therapy is offered to patients who are older than 60 years and who are not candidates for intensive induction therapy.

Early clinical trials of AML maintenance resulted in excessive toxicity, lack of a clear clinical benefit, inconsistent improvements in recurrence-free and disease-free survival, and no significant improvements in overall survival (OS). However, oral azacitidine demonstrated both efficacy and safety as maintenance therapy, and was granted US FDA approval in this setting in September 2020.

Patients eligible for oral azacitidine therapy include those in first complete remission and those in complete remission/complete remission with incomplete count recovery who do not proceed to allogeneic hematopoietic cell transplantation.

Oral azacitidine is approved for post induction or consolidation maintenance in patients who are in remission and are not eligible for transplant. The active drug is the same in the oral formulation as in the injectable or subcutaneous formulations, but is not bioequivalent.

However, oral azacitidine has significant clinical benefits and improved OS compared with the injectable form. Additionally, prolonged drug exposure with oral azacitidine results in sustained therapeutic activity.

Oral azacitidine is given at a dose of 300 mg for days 1 to 14 of each 28-day cycle for at least the first 2 cycles. This agent is moderately emetogenic, and so premedication with ondansetron 8 mg for at least the first 2 cycles is recommended. Complete blood counts to check for myelosuppression should be obtained every 2 weeks for the first 2 cycles.

Advantages of oral anticancer therapy are convenience for patients and caregivers, decreased need to travel to the infusion clinic, reduced burden on patients and caregivers, and reduced risk of relapse. Despite these advantages, access to specialty pharmacies — especially in hard-to-reach areas — and high cost (financial toxicity) are challenges to successful oral therapy. Other challenges are side effect management and patient-related potential for missed doses.

Other disparities that impact care that multidisciplinary oncology care teams may need to address include the impact of structural racism and inequities, low literacy and inequitable access to technology, healthcare inequities, and drug and nursing shortages.


Leak Bryant A. Supporting patients during maintenance therapy for AML. Oral presentation at: 2023 ONA Summit Live Virtual Meeting; March 17-19, 2023.

This article originally appeared on Oncology Nurse Advisor