A patient assessment tool, known as the Functional Assessment of Cancer Therapy–Leukemia (FACT-Leu), was a viable and valid outcome measure for patients with acute myeloid leukemia not eligible for intensive therapy.
A health-related quality of life assessment tool, known as Functional Assessment of Cancer Therapy—Leukemia (FACT-Leu), provided a viable and valid outcome measure for patients with acute myeloid leukemia not eligible for intensive therapy, according to data published in the journal Cancer.
The data indicate that FACT-Leu offers insight into leukemia-specific health-related quality-of-life measures in patients with AML and could play an important role in helping the field grow increasingly patient-centered.
“Although the assessment of (patient-reported outcomes) is now critical in hematologic research to more appropriately assess treatment outcomes, there are few well‐validated, disease‐specific measures in this area,” the study authors wrote. “This is particularly true for patients with AML, and in this study, we provide compelling evidence for the idea that the FACT‐Leu is reliable, valid, and responsive to change.”
Participants (317 patients) from the randomized phase 2/3 AML2002 trial completed the 44-item FACT-Leu and EuroQol 5-Dimension (EQ-5D) measures every 28 days until the end of treatment to assess quality of life. The study evaluated responsiveness to change by anchoring change in the FACT-Leu scales to a 0.10 change in the EQ-5D Health Utility Index. Of note, internal consistency reliability was estimated using Cronbach’s α.
In the study, Cronbach's α usually exceeded the threshold for good or excellent reliability. Moreover, the associations between FACT-Leu and EQ-5D scales were moderate or high.
FACT-Leu scales were found to effectively differentiate between Eastern Cooperative Oncology Group (ECOG) performance status ratings (PSR) groups with large effect sizes for an ECOG PSR of 0 versus an ECOG PSR of 2. Additionally, Functional Assessment of Cancer Therapy — General, Additional Concerns, FACT-Leu Total, and Trial Outcomes Index scales discerned differences between patients with grade 3 or lower maximum side effect profiles and those with maximum side effect profiles higher than grade 3, though effect sizes were small.
The study authors wrote that although the study sample was restricted to patients with AML, who specifically were not candidates for intensive therapy, there is belief that these results have significant implications.
“Some older patients with AML are not eligible for intensive therapy because of concerns about toxicity and a lower possibility of treatment benefit, but these patients face many burdensome disease symptoms, and optimizing their care is a challenge,” the researchers wrote. “For this reason, it is critical to understand which areas of health are the best targets for intervention in this AML subpopulation, and this in turn requires HRQOL and symptom measures that are sensitive to these patients' particular health issues.”
Of note, according to the researchers, the assessment tool showed response to changes in health among patients with AML who were not eligible for intensive therapy.
“For this reason, this article's results support the use of the FACT‐Leu in clinical trials focusing on AML,” the authors wrote. “With few exceptions, HRQOL and other types of (patient reported outcomes) have been rarely included in clinical trials, and this limits our knowledge of the impact imposed by the burden of the disease and treatment from the patient's standpoint.”
For instance, the authors note that there have been recent questions surrounding the use of survival as an endpoint in AML trials to facilitate drug approvals. The researchers mention that there has been an emphasis on including other endpoints, such as HRQOL and symptoms, when determining clinical benefit.
“There is an urgent need for methodologically sound prospective HRQOL studies in patients with AML to generate data that can facilitate the establishment of criteria for non-survival clinical benefits of AML therapies,” the authors concluded. “To this end, we propose that the implementation of the FACT‐Leu in future AML trials might help to bridge this gap and eventually facilitate integration of HRQOL as a valuable treatment endpoint.”
A version of this story originally appeared on CancerNetwork™ as, “Cancer Therapy Leukemia Instrument Shows Promise for AML Clinical Trials.”