Abstract
Background
Valuing patients as the experts in their experience, how they live with myelodysplastic disease, and how to reframe assessments of efficacy, patient-reported outcomes (PROs) can complement traditional measurements of response. Therefore, when used correctly, PROs serve as a valued instrument to assess a patient's Quality of Survival (QoS) throughout the course of their treatment. This study aimed to address the need for improved quality of life measurement in MDS and AML research requires identifying instruments that are appropriate for this population and ones that accurately represent the goals, needs, and concerns of these patients. Therefore research efforts should be directed to providing confirmatory data of the suitability for utilizing existing patient-reported outcome measurements (PROMs) for this population or identify the need to develop and use highly sensitive disease-specific measurements when evaluating new treatments. Identification of representative, influential, patient-reported quality of life domains first needed to be established in order to direct discovery of appropriate PROMs for this population in this two part research study (part-one: patient interviews/surveys, part two: Delphi panel/identification of appropriate PROMs [forthcoming]).
Methods
Between January and June 2021, 17 participants (AML, n = 8; HR-MDS, n = 9) were enrolled in an IRB-research study participating in concept-elicitation, semistructured interviews and surveys aimed to identify the lived experience of those being treated for HR-MDS and AML. An interpretive descriptive methodology was chosen to produce coherent conceptual descriptions aimed to expose thematic patterns and commonalities that characterize individuals with HR-MDS and AML. Using these insights, a conceptual model was derived to establish the basis for PROM content validity evaluation and to improve the accuracy of the captured quality of life measurements of this patient population.
Results
Seven (7) patient-reported themes emerged from qualitative analysis; social wellbeing, physical wellbeing, emotional wellbeing, management of side effects, treatment satisfaction, autonomy, and individual factors. Treatment Satisfaction, Autonomy, and Individual Factors (such as education, financial wellbeing, incoming treatment goal, health status, outlook, and prognosis) were found to be the most salient related to quality of life and overall wellbeing. The conceptual model highlights the main areas of impact and the relationships among concepts. Significant overlap was observed between the influential domains identified amongst those with HR-MDS and AML. Surveys, which were administered after patient interviews, were analyzed for consistency, reliability (α=0.8405), and variable correlation.
Conclusion
A clear understanding of the influential health-related quality of life domains that make up the foundation of this patient population's experience has proven fundamental in the process of identification of appropriate PROMs to measure quality of life. The patient-reported influence treatment satisfaction (confidence in treatment/provider, quality of care, compassion received, provided support, knowledge of treatment, burden of care, information of prognosis, etc) has on a patient's quality of life may lead to a more holistic approach to patient-reported outcome item-banks and instrument selection. The importance of the maintenance of autonomy during and after treatment was apparent in the reported anxiety associated with the loss of the ability of self-care, and independence. Additionally, the absence of divergent themes amongst those with HR-MDS and AML further adds to the evidence that these sub-populations share similar patient experiences.
Powers: Arrevus, Celularity, Corbus, DaVolterra, Eicos, Eli Lilly, Evofem, Eyecheck, Fuji, Gilead, GSK group of companies, Johnson & Johnson, Microbion, Mustang, OPKO, Otsuka, Romark, Shiniogi, Vir: Consultancy.
