Survival rates for acute myeloid leukemia (AML) have greatly increased over the past several decades, particularly among younger patients. Although these advances are significant, the costs associated with novel treatments continue to rise. At present, a substantial degree of uncertainty remains regarding how these changes will influence health policy, clinical outcomes, and society.

In a review article published in Expert Review of Hematology, Jan Philipp Bewersdorf, MD, of the department of internal medicine at Yale University in New Haven, Connecticut, and colleagues summarized the health care costs related to various treatment strategies used in patients with AML.

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Amer Zeidan, MBBS, MHS, who is also of the department of internal medicine at Yale University and is the corresponding author on the study told Hematology Advisor, “Although the AML community is very excited about the approval of [newer] active agents, these oral agents are very expensive.”

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Treatment Options for AML

Recently, several novel therapies have been approved by the US Food and Drug Administration (FDA) for the treatment of patients with AML. Hypomethylating agents and cytarabine- and anthracycline-based chemotherapy were the mainstay of therapy for many years, but there is growing evidence for the use of allogeneic hematopoietic stem cell transplantation (HSCT), in addition to various new oral therapies that have dramatically changed the treatment landscape.

“Starting in 2017, we witnessed a therapeutic revolution in AML with approval of 8 agents for specific subsets [of the disease] after several decades [of] no approvals,” said Dr Zeidan.

“Of these 8 newly approved agents, 5 [have] specific indications for older unfit patients, [and] the other 3 are widely used for older patients in both relapsed/refractory and frontline settings,” he explained.

Despite the multiple oral agents now available, high-intensity induction chemotherapy combined with either HSCT or consolidation chemotherapy is still the treatment of choice for younger patients. These strategies are highly dependent upon an individual’s specific genetic and cytogenetic risk profile. Currently, 5-year overall survival rates in younger patients range from 25% to 40%, while rates in older patients are typically below 10%.