Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review

Published:September 04, 2021DOI:


      • Although the emerging curative therapies bring hope to patients with sickle cell disease (SCD), concerns about their high costs have been raised. Cost-effectiveness analysis can be used to align the costs of the curative therapies with their expected health benefits.
      • Our study is the first to comprehensively summarize and catalog health state utilities (HSUs) for SCD and its specific complications and HSUs tied to its treatments, which can inform future cost-effectiveness analyses in SCD.
      • We identify the research gaps, such as the need for more empirical studies to elicit HSUs associated with the SCD and its treatment complications from patients and HSUs from their caregivers.



      Sickle cell disease (SCD) is a complex, chronic condition that impairs health-related quality of life of affected individuals and their caregivers. As curative therapies emerge, comprehensive cost-effectiveness models will inform their value. These models will require descriptions of health states and their corresponding utility values that accurately reflect health-related quality of life over the disease trajectory. The objectives of this systematic review were to develop a catalog of health state utility (HSU) values for SCD, identify research gaps, and provide future directions for preference elicitation.


      Records were identified through searches of PubMed and Embase, Tufts Medical Center Cost-Effectiveness Analysis Registry, reference lists of relevant articles, and consultation with SCD experts (2008-2020). We removed duplicate records and excluded ineligible studies. For included studies, we summarized the study characteristics, methods used for eliciting HSUs, and HSU values.


      Five studies empirically elicited utilities using indirect methods (EQ-5D) (n = 3) and Short Form-6 Dimension (n = 2); these represent health states associated with general SCD (n = 1), SCD complications (n = 2), and SCD treatments (n = 3). Additionally, we extracted HSUs from 7 quality-adjusted life-years-based outcome research studies. The HSU among patients with general SCD without specifying complications ranged from 0.64 to 0.887. Only 36% of the HSUs used in the quality-adjusted life-year-based outcomes research studies were derived from individuals with SCD. No study estimated HSUs in caregivers.


      There is a dearth of literature of HSUs for use in SCD models. Future empirical studies should elicit a comprehensive set of HSUs from individuals with SCD and their caregivers.


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