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Abstract：
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.
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.
摘要：
镰状细胞病（SCD）是一个复杂的，损害受影响个体及其照顾者健康相关生活质量的慢性疾病。随着治愈疗法的出现，全面的成本效益模型将为其提供价值。这些模型将需要描述健康状况及其相应的效用值，以准确反映疾病轨迹上与健康相关的生活质量。本系统评价的目的是开发SCD的健康状态效用(HSU)值目录，确定研究差距，并为偏好启发提供未来的方向。
通过搜索PubMed和Embase识别记录，塔夫茨医疗中心成本-效果分析登记处，相关文章的参考列表，并咨询SCD专家（2008-2020年）。我们删除了重复的记录，并排除了不合格的研究。对于纳入的研究，我们总结了研究特点，用于引发HSU的方法，和HSU值。
五项研究使用间接方法（EQ-5D）（n=3）和ShortForm-6Dimension（n=2）凭经验得出的实用程序；这些代表与一般SCD（n=1）相关的健康状况，SCD并发症（n=2），和SCD治疗(n=3)。此外,我们从7项基于质量调整生命年的结局研究中提取了HSUs.无特定并发症的一般SCD患者的HSU范围为0.64至0.887。在质量调整的基于生命年的结果研究中，只有36％的HSU来自SCD患者。没有研究估计护理人员的HSU。
在SCD模型中使用HSU的文献缺乏。未来的实证研究应该从患有SCD的个体及其照顾者那里获得一套全面的HSU。
通过搜索PubMed和Embase识别记录，塔夫茨医疗中心成本-效果分析登记处，相关文章的参考列表，并咨询SCD专家（2008-2020年）。我们删除了重复的记录，并排除了不合格的研究。对于纳入的研究，我们总结了研究特点，用于引发HSU的方法，和HSU值。
五项研究使用间接方法（EQ-5D）（n=3）和ShortForm-6Dimension（n=2）凭经验得出的实用程序；这些代表与一般SCD（n=1）相关的健康状况，SCD并发症（n=2），和SCD治疗(n=3)。此外,我们从7项基于质量调整生命年的结局研究中提取了HSUs.无特定并发症的一般SCD患者的HSU范围为0.64至0.887。在质量调整的基于生命年的结果研究中，只有36％的HSU来自SCD患者。没有研究估计护理人员的HSU。
在SCD模型中使用HSU的文献缺乏。未来的实证研究应该从患有SCD的个体及其照顾者那里获得一套全面的HSU。
