Quality of life from the perspective of the patient with acute myeloid leukemia.

BACKGROUND: Although outcomes for acute myeloid leukemia (AML) have improved over time, they remain poor overall, and toxicity from both the disease and its treatment can affect quality of life (QOL). One barrier to including QOL endpoints in clinical trials is the lack of a disease-specific QOL instrument that can efficiently capture the major QOL deficits in this population. METHODS: A cross-sectional study was performed to elicit concepts for inclusion in a new AML-specific QOL instrument called the AML-QOL. Eighty-two patients at various stages of disease were interviewed about sources of support (positive concepts) and problems and symptoms (negative concepts) experienced over the past week, and they were asked to grade how much each affected their QOL. In addition, patients were asked to complete 2 validated instruments: the Functional Assessment of Cancer Therapy with the leukemia and transplant modules and the 29-item Patient-Reported Outcomes Measurement Information System questionnaire. RESULTS: With data from the open-ended and questionnaire-based portions of the interview, 7 positive concepts and 64 negative concepts were elicited. From these, 5 positive concepts and 21 negative concepts were selected for inclusion in the preliminary AML-QOL on the basis of concept prevalence and the impact on QOL. CONCLUSIONS: These concepts will form the basis of a new QOL instrument specific to AML. Cancer 2018;124:145-52. © 2017 American Cancer Society.

Development and validation of the AML-QOL: a quality of life instrument for patients with acute myeloid leukemia.

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.