Validation of the AML-QOL: A Quality of Life Instrument for Patients with Acute Myeloid Leukemia and Other Aggressive Myeloid Neoplasms
Authors: Buckley Sarah A , Jimenez-Sahagun Diana , Othus Megan , Walter Roland B. , Lee Stephanie J. 
Published: October 2019

Abstract
BACKGROUND: While there is increasing interest in measuring quality of life (QOL) in patients with acute myeloid leukemia (AML), few published studies have done so. Among those that have, there is considerable heterogeneity in instrument used, and interpretability is often limited by low survey compliance. Furthermore, our group has recently highlighted important limitations of non-disease-specific instruments in this population: (a) patients who face temporary logistical restrictions that have low impact on QOL cannot be easily distinguished from those who face more long-term functional restrictions that result in larger QOL impact, and (b) questionnaires may fail to ask about important concepts and (c) may instead ask about concepts that are less impactful. We therefore developed a new disease-specific quality of life instrument (the AML-QOL) based on cognitive interviews with 82 patients with AML and other aggressive myeloid neoplasms (Buckley SA et al. Cancer 2018). Here, we describe further development and validation of the AML-QOL.
METHODS: This study consists of three phases. In the first phase, iterative revisions of the AML-QOL were performed based on feedback from 16 patients, 8 medical providers, and 2 psychometricians. In the second phase, factor analysis and convergent / divergent validity testing was performed based on responses from 202 patients who completed the AML-QOL along with the PROMIS Global 10. The third phase, a prospective validation study to assess sensitivity to change and test-retest reliability, is currently underway.
RESULTS: The AML-QOL prototype underwent 4 iterative revisions followed by factor analysis, resulting in a final version containing 27 items categorized into 5 domains (physical, social, cognitive, anxiety, depression), 7 symptom questions scored individually and collectively as a symptom index, one global QOL item, and a summary score. The AML-QOL domains show high internal consistency (mean alpha: 0.82, range: 0.72-0.86). All domains demonstrated convergent validity with analogous items from the PROMIS Global 10 (Figure 1). Prospective validation testing is currently underway.
CONCLUSION: The AML-QOL measures quality of life in patients with acute myeloid leukemia and analogous aggressive myeloid neoplasms. Once fully validated, this instrument can be used for clinical and research purposes.
Figure 1. Figure 1.

Disclosures
Buckley: CTI Biopharma: Employment; TwinStrand Biosciences: Consultancy. Walter:Amphivena Therapeutics: Consultancy, Equity Ownership, Other: Clinical trial support, Research Funding; Aptevo Therapeutic: Consultancy, Other: Clinical trial support, Research Funding; Covagen AG: Consultancy, Other: Clinical trial support, Research Funding; Seattle Genetics, Inc.: Consultancy, Other: Clinical trial support, Research Funding; Boehringer Ingelheim Pharma GmbH & Co. KG: Consultancy; Pfizer: Consultancy; Amgen Inc.: Other: Clinical trial support, Research Funding; Actinium Pharmaceuticals, Inc.: Other: Clinical trial support, Research Funding. Lee:Takeda: Research Funding; Amgen: Consultancy, Research Funding; Mallinckrodt: Honoraria; Pfizer: Consultancy; Kadmon: Research Funding; Onyx: Research Funding; Incyte: Consultancy.