Abstract
BackgroundPreference valuations of health status are essential in health technology and economic appraisal. This study estimated utilities for treatment-related health states of acute myeloid leukemia (AML) and disutilities of severe adverse events (SAEs) using a representative sample of adults from the general population in the United States (US).MethodsTreatment-related AML health states, defined based on literature and interviews with clinicians, included complete remission (CR), no CR, relapse, stem cell transplant (SCT), and post SCT short-term recovery. Six attributes with varying levels, including fever, lack of energy, problems with daily function, anxiety/depression, blood transfusions, and hospitalization, were used to define health states. An online survey using discrete choice experiment methodology was designed to capture preferences for health status scenarios including the identified attributes and key grade 3/4 chemotherapy-related SAEs. Health state utilities and SAE disutilities were generated from a conditional logistic regression with generalized estimating equations.ResultsOf the 300 survey participants, the demographic distributions were within a 3% margin of those in the 2010 US Census. CR had the highest utility value (0.875), followed by post-SCT short-term recovery (0.398), relapse (0.355), no CR (0.262), and SCT (0.158). Of the SAEs, serious infection had the highest decline in utility (0.218), followed by severe diarrhea (0.176), abnormally low blood cell counts (0.100), and severe redness/skin peeling (0.060).ConclusionsAML and treatments can result in reduced quality of life and impaired ability to perform daily activities. Findings of this study underline the value that society places on treatment-related AML health states and SAEs.
Highlights
Preference valuations of health status are essential in health technology and economic appraisal
Background a type of rare disease, acute myeloid leukemia (AML) is the most common form of acute leukemia in adults, which accounts for approximately 25% of all leukemia in adults in the Western world [1]
The qualitative phase intended to identify the key attributes to define treatment-related AML health states and inform the important Severe adverse event (SAE) associated with chemotherapies that were impactful in the health-related quality of life (HRQL) of these patients
Summary
Preference valuations of health status are essential in health technology and economic appraisal. This study estimated utilities for treatment-related health states of acute myeloid leukemia (AML) and disutilities of severe adverse events (SAEs) using a representative sample of adults from the general population in the United States (US). A type of rare disease, acute myeloid leukemia (AML) is the most common form of acute leukemia in adults, which accounts for approximately 25% of all leukemia in adults in the Western world [1]. The median diagnosis age of AML is 68 years [2]. The incidence of AML is expected to rise. AML progresses rapidly if left untreated; even with treatment, the survivorship remains poor, among a few subtypes of AML. The 5-year survival rate is low at approximately 27% [2].
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