Real World Treatment Patterns and Comparative Effectiveness among Elderly Patients with Acute Myeloid Leukemia in the United States

Original Article

Ann Hematol Oncol. 2020; 7(1): 1283.

Real World Treatment Patterns and Comparative Effectiveness among Elderly Patients with Acute Myeloid Leukemia in the United States

Medeiros BC1 and Satram S2*

¹Department of Medicine, Stanford University, USA

²Department of Epidemiology, Q.D. Research, USA

*Corresponding author: Sacha Satram, Department of Epidemiology, Q.D. Research, Inc, 4120 Douglas Blvd., Ste 306436, Granite Bay, CA 95746, USA

Received: January 23, 2020; Accepted: February 13, 2020; Published: February 20, 2020


An evaluation of treatment patterns and outcomes among 11,142 first primary Acute Myeloid Leukemia (AML) patients was conducted. There were 936 (8%) patients who were treated with azacitidine and/or decitabine (HMA), 153 (1%) received a cytarabine combination regimen (Intensive), 433 (4%) received another type of agent (Other), 3250 (29%) received an unidentified agent (Unknown) and 6,370 (57%) did not receive any treatment. There were 403 (8%) patients who underwent subsequent allogeneic Hematopoietic Stem Cell Transplantation (HSCT) therapy after initial chemotherapy. Overall, treatment rates increased over the study time-period from 36% in 2000 to 55% in 2013 (P ‹0.0001). Treated patients were more likely to be younger, male, and married, and were less likely to have secondary AML, poor performance, and comorbid conditions compared to untreated patients. Receipt of all types of antileukemic therapy showed significant mortality risk reductions compared with palliative care. HSCT was associated with a 40% mortality risk reduction versus chemotherapy only, and the survival benefit was more pronounced among patients ≤75 years. These findings provide a rationale to strongly consider antileukemic therapy rather than best supportive care in older patients who do not meet criteria for more intensive regimens.

Keywords: Acute myeloid leukemia; chemotherapy; hematopoietic stem cell transplantation, treatment, survival, elderly patients


The incidence of Acute Myeloid Leukemia (AML) increases with age and over half of patients are diagnosed at age ≥65 years [1]. Although AML is a relatively rare disease accounting for just over 1% of adult cancer deaths in the United States [2], the incidence is expected to increase as the population ages. The prognosis of patients 65 years and older is very poor and worsens with advancing age as treatment efficacy and tolerability have been shown to deteriorate markedly in older adults. Without treatment, AML progresses rapidly and is fatal within a few weeks of diagnosis [3]. There is no optimal treatment strategies for older patients with AML so therapy is individualized based on medical fitness, age, cytogenetic/molecular testing, the potential benefits for short and long-term outcomes, and the potential risk of adverse events in the context of patient wishes and socio-economic support.

Conventional intensive chemotherapy remains the standard of care for younger, functionally fit patients. Older patients, however, are often not treated or given less intensive chemotherapy and have inferior clinical outcomes. In spite of these facts, the major causes of death from AML in older patients are from infection and hemorrhage related to disease-associated cytopenias [4]. Retrospective and population-based studies suggest that age is not a barrier to the beneficial effects of AML treatment in patients up to 80 years old [5,6] and others have reported high rates of response and improved survival with intensive therapy up to 90 years old [7,8]. In addition, allogeneic Hematopoietic Stem Cell Transplantation (HSCT) is often the only curative treatment modality for most AML patients older than 60 years [9-11]. Unfortunately, the majority of patients remain ineligible for HSCT.

Prospective studies may be subject to bias due to selection of younger and prognostically favorable patients receiving treatment. Randomized trials for AML patients 65 years and older are few, but have demonstrated a longer overall survival for intensively treated patients [12,13]. Prior population based analyses found that the use of chemotherapy has increased over time and was associated with a significant survival benefit compared to best supportive care [3,5]. However, there has been minimal improvement in survival as treatment strategies have not significantly changed for the past several decades ago. The goal of the study was to assess comparative effectiveness of existing therapeutic regimens, the patient characteristics associated with treatment receipt, and determine if treatment rates and survival continue to rise in a real-world population of elderly patients with AML.


Data sources

Data from the Surveillance, Epidemiology, and End Results (SEER) Medicare linked database was used for these analyses. Institutional Review Board (IRB) approval was waived because there are no personal identifiers in the SEER-Medicare database. The SEER-Medicare database is a collaborative effort of the National Cancer Institute (NCI), the SEER registries, and the Centers for Medicare & Medicaid Services and provides information on Medicare patients included in SEER, a nationally representative collection of 18 population-based registries of all incident cancers from diverse geographic areas [14]. The linked database includes all incident cancer patients reported to the SEER registries and cross-matched with a master file of enrollees in Medicare [15] with approximately 97% of persons 65 years or older eligible for Medicare. Inpatient care, skilled nursing care, home healthcare, and hospice care are covered services under Medicare Part A, while Part B reimburses for physician and outpatient care with about 95% of beneficiaries subscribing to Part B. The SEER-Medicare linkage used in this study include all Medicare eligible cancer patients reported to SEER through 2013 and their Medicare claims through 2015.

Study population

Patients were included if they were diagnosed with a first primary AML cancer from January 1, 2000 to December 31, 2013, ›66 years, and continuously enrolled in Medicare Parts A and B with no HMO coverage in the year prior to diagnosis (Supplementary Figure 1). Patients were excluded if their date of death was recorded prior to or the same month as diagnosis, if they were enrolled in a Health Maintenance Organization (HMO) at any time during the 12 months prior to diagnosis (because complete claims data were unavailable for these patients), and if they had two or more claims for chemotherapy prior to diagnosis.