Abstract
There is currently no validated quality of life (QOL) instrument specific to patients with acute myeloid leukemia (AML). A previous cross-sectional interview-based study elicited concepts for inclusion in a novel QOL instrument. Here, we further develop and validate this new instrument, the AML-QOL. Iterative revisions of the draft AML-QOL were refined based on feedback from 16 patients, 8 medical providers, and 3 psychometricians. The instrument underwent factor analysis based on responses from 202 patients with AML and analogous aggressive myeloid neoplasms receiving AML-like therapy. A prospective validation study was then undertaken in 50 patients who completed the AML-QOL at multiple time points while undergoing a cycle of intensive chemotherapy to establish test-retest reliability and sensitivity to change. The final AML-QOL contains 27 items and is categorized into 5 domains (Physical, Social, Cognitive, Anxiety, Depression), one Symptom Index, a single item assessing overall quality of life, and a Summary Score. The AML-QOL domains show high internal consistency (median alpha: 0.85), good test-retest reliability (median interclass correlation: 0.82), and had convergent and divergent validity when compared to a non-disease-specific instrument (the EORTC QLQ-C30). The Summary Score demonstrated good sensitivity to change when anchored to patient perception of QOL change. The AML-QOL is a reliable and valid measure of QOL in patients with AML and analogous aggressive myeloid neoplasms. A clinically meaningful difference is 8-10 points out of 100 on the Summary Score.
Disclosure statement
None directly with this work; others with AML therapeutics: S.A.B: CTI Biopharma (employment), TwinStrand Biosciences (consultancy); M.O.: Celgene (independent DSMB), Glycomimmetics (independent DSMB); R.B.W: Amphivena Therapeutics (Consultancy, Equity Ownership, clinical trial support, research funding), Aptevo Therapeutics (Consultancy, clinical trial support, research funding), Covagen AG (consultancy, clinical trial support, research funding), Seattle Genetics (Consultancy, clinical trial support, research funding), Boehringer Ingelheim Pharma GmbH & Co (consultancy), Pfizer (consultancy), Amgen (clinical trial support, research funding), Actinium Pharmaceuticals (clinical trials support, research funding) S.J.L.: Pfizer (consultancy).