Validated Instruments of Quality of Life (QOL) in Patients With Acute Myeloid Leukemia (AML) and Other Cancers

Maribel Salas; Mackenzie Henderson; Angelika Wientzek-Fleischmann; Zahidul Islam; Nora Tu; Aikaterini Bilitou; Maggie Elsharkawy; Ulf Stellmacher

doi:10.3389/fphar.2020.01109

Validated Instruments of Quality of Life (QOL) in Patients With Acute Myeloid Leukemia (AML) and Other Cancers

Front Pharmacol. 2020 Jul 24:11:1109. doi: 10.3389/fphar.2020.01109. eCollection 2020.

Authors

Maribel Salas^{1

2}, Mackenzie Henderson^{1

3}, Angelika Wientzek-Fleischmann⁴, Zahidul Islam¹, Nora Tu¹, Aikaterini Bilitou⁵, Maggie Elsharkawy¹, Ulf Stellmacher⁴

Affiliations

¹ Epidemiology and Clinical Safety and Pharmacovigilance, Daiichi Sankyo, Inc., Basking Ridge, NJ, USA.
² Centers for Clinical Epidemiology and Biostatistics and Center for Pharmacoepidemiology Research and Training, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, United States.
³ Rutgers Institute for Pharmaceutical Industry Fellowships, Rutgers University, Piscataway, NJ, United States.
⁴ Daiichi Sankyo Europe GmbH, Munich, Germany.
⁵ HEOR/RWE: Health Economics and Outcomes Research (HEOR)/Real-World Evidence (RWE), Market Access, DSE, Munich, Germany.

Abstract

Introduction: Acute myeloid leukemia (AML) can negatively impact quality of life (QOL). Few QOL instruments are specific to and have been validated in AML. This review aims to identify QOL instruments that have been validated in patients with AML and other cancers and summarize their psychometric properties reported in published literature. A literature review search was performed using PubMed and OVID (Biosis, Embase, MEDLINE) databases through June 25, 2020. Search terms included: QOL, health-related QOL, patient-reported outcomes and validity, reliability, validated, tools, instruments, test-retest, and leukemia myeloid acute, leukemia, myeloid, acute, acute myeloid leukemia. Articles were included if they focused on cancer and reported psychometric properties that could be extracted. Abstracts and their references were reviewed for inclusion.

Results: Twelve evaluating ten instruments were included. Functional Assessment of Cancer Therapy Leukemia (FACT-Leu) showed internal consistency (IC) of α = 0.86 to >0.9, correlation with EQ-5D-3L of r > 0.50, correlation with European Organisation for Research and Treatment of Cancer (EORTC) QLQ-Leu of ρ = 0.29-0.63, test-retest reliability of κ = 0.861. FACT-F showed correlations with EORTC QLQ-C30 of r = 0.40-0.83. Hematological Malignancy Patient-Reported Outcome (HM-PRO) showed intraclass correlation coefficient (ICC) of 0.94-0.98. EORTC-8D and EQ-5D-3L showed ICC = 0.595, correlations with each other of ρ = 0.137-0.634 and with EORTC QLQ-C30 of r = 0.651-0.917. EORTC QLQ-C30 showed person separation reliability of 0.47 to 0.90 and patient-observer agreement of 0.85. Life Ingredient Profile (LIP) showed IC of α = 0.29-0.77 and test-retest reliability of κ = 0.42-1.0. QOL-E showed correlation with FACT-general of R = 0.71, internal validity of α = 0.7, and test-retest reliability of standardized Cronbach's α = 0.7-0.92. EORTC QLQ-Leu showed IC of α = 0.6-0.79. The Acute Myeloid Leukemia-Quality of Life (AML-QOL) instrument showed IC of α = 0.72, correlations with EORTC QLQ-30 of magnitudes ρ = 0.59-0.72, and test-retest reliability of ICC = 0.52-0.91.

Conclusion: Although several QOL instruments have been validated, more research is needed to determine the most clinically useful instruments in patients with AML.

Keywords: acute myeloid leukemia; instrument validation; patient-reported outcomes (PRO); quality of life; review (article); validation.

Publication types

Review