Abstract

BackgroundPatient engagement in end-stage renal disease (ESRD) is expected to result in a more patient-centered approach to care that aligns with patients’ values, preferences, and goals for treatment. Nevertheless, no previous studies of which we are aware have evaluated patients’ benefit-risk preferences for the management of anemia associated with ESRD. The primary objective of this study was to quantify the tradeoffs patients are willing to make between cardiovascular risks associated with some anemia medicines and red blood cell (RBC) transfusions. A secondary objective was to quantify the importance of avoiding transfusion-related risks.MethodsA survey instrument was developed from the clinical literature, clinician input, patient-education resources, and a patient focus group. The survey instrument was qualitatively pretested before its administration to a broader sample of patients. The National Kidney Foundation invited individuals in the United States to participate in the survey. In a discrete-choice experiment (DCE), respondents chose between two hypothetical anemia medications in a series of questions. Each medication was defined by symptom relief, frequency of transfusions, cardiovascular risk, mode of administration, and out-of-pocket cost. The survey also included a best-worst scaling (BWS) exercise to quantify the importance of avoiding attributes of blood transfusions. Results from the DCE were used to estimate relative importance and marginal willingness to pay. Results from the BWS were converted to relative importance weights.ResultsA total of 200 individuals completed the survey. Patients were willing to accept a 6% medication-related risk of heart attack to avoid having two RBC transfusions per month. Symptom relief and mode of administration were of moderate importance. The most important transfusion-related risk to avoid was transfusion-related lung injury.ConclusionsPatients with ESRD and anemia have measurable treatment preferences and are willing to accept risks associated with anemia medications to avoid transfusions.

Highlights

  • Patient engagement in end-stage renal disease (ESRD) is expected to result in a more patient-centered approach to care that aligns with patients’ values, preferences, and goals for treatment

  • Severe cases of anemia in patients with ESRD undergoing hemodialysis may be treated with both Erythropoietin-stimulating agent (ESA) and red blood cell (RBC) transfusions to manage hemoglobin levels

  • To elicit patients’ preferences for anemia-management options, we developed and administered a discretechoice experiment (DCE) survey in which patients chose between hypothetical treatment options defined by attributes to which different pre-specified levels were applied

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Summary

Introduction

Patient engagement in end-stage renal disease (ESRD) is expected to result in a more patient-centered approach to care that aligns with patients’ values, preferences, and goals for treatment. The primary objective of this study was to quantify the tradeoffs patients are willing to make between cardiovascular risks associated with some anemia medicines and red blood cell (RBC) transfusions. Anemia is prevalent among patients with end-stage renal disease (ESRD) and, left untreated, has a negative impact on quality of life [1,2,3]. Severe cases of anemia in patients with ESRD undergoing hemodialysis may be treated with both ESAs and red blood cell (RBC) transfusions to manage hemoglobin levels. ESAs decrease the need for RBC transfusions, thereby improving patients’ health-related quality of life, but carry risks. A tenet of patientcentered care is shared decision making, whereby treatment

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