Patient-Reported Outcomes Validation of the FACT-Leu in Acute Myeloid Leukemia: A Review of Baseline Characteristics in AML2002

Introduction: Acute Myeloid Leukemia (AML) is an aggressive disease associated with poor health related quality of life (HRQoL) and short overall survival (OS), particularly for patients' ineligible for intensive chemotherapy. The HRQoL was evaluated in a cohort of patients with AML who were not considered eligible for standard chemotherapy based on the Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu) collected at baseline of AML2002 study (NCT02472145).

Methods: This analysis was based on a randomized, phase 2/3, parallel design study conducted in patients with AML. Patients' perceptions to HRQoL were evaluated using a 44-item, self-reported leukemia-specific measure, FACT-Leu. It was assessed based on 5 subscales: physical well-being (PWB), social well-being (SWB), emotional well-being (EWB), functional well-being (FWB) and leukemia-specific concerns. Additionally, FACT-Leu was also evaluated based on the trial outcome index (TOI). European Quality of Life- 5 Dimension 5-Level (EQ-5D-5L) was reported based on index values and visual analogue scale (VAS). The summary statistics from the FACT-Leu was compared to the validation paper [1]. FACT-Leu by eastern cooperative oncology group (ECOG) performance status score was also assessed based on generalized linear model and the correlation among FACT-leu subscales was assessed using the Pearsons correlation coefficient

Results: Of the 309 patients (mean age 74.9 years) enrolled, 46.3% were women, 87.3% were white (Caucasian) and 70.9% had de novo AML. For these patients, ECOG performance status distribution was 0 (18%), 1 (41.9%) and 2 (40.2%). At baseline, the mean index values for VAS of EQ-5D-5L were 0.68 and 62.5, respectively, and the mean FACT-Leu was 119.6. Except SWB, other FACT-Leu subscale and aggregated scores highly correlated with FACT-Leu (0.74-0.96; p<0.0001). Both index values (0.65) and VAS of EQ-5D-5L (0.57) showed moderate correlation with FACT-Leu. The EQ-5D-5L (0.71) and VAS (0.60) showed moderate correlation with FACT-TOI (p<0.0001). Except SWB and EWB, other FACT-Leu subscales and aggregated scores showed predicted differences in means based on the ECOG score, with higher scores associated with better ECOG status. However, compared to the results of the validation paper [1], the mean subscale scores of AML ineligible for intensive chemo therapy were lower. In addition to ECOG status, sex was also a significant predictor of FACT-Leu subscales as aggregated scores except SWB and EWB with men reporting better scores.

Conclusions: FACT-Leu scores were significantly associated with PS and sex. The lower mean subscale scores in Patients with AML ineligible for intensive chemotherapy highlight the need for new therapies to improve patient HRQoL in this group of patients and suggested that there is a need for optimized instruments for women. The FACT-Leu tool could facilitate targeted population interventions, potentially improving quality of life.

[1] Cella, Value in Health 15 (2012) 1051-1058