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评估美国急性髓细胞白血病相关治疗健康状况的效用值。

Assessing utility values for treatment-related health states of acute myeloid leukemia in the United States.

机构信息

Memorial Sloan Kettering Cancer Center, New York, NY, USA.

Analysis Group, Inc, Boston, MA, USA.

出版信息

Health Qual Life Outcomes. 2018 Sep 21;16(1):193. doi: 10.1186/s12955-018-1013-9.

Abstract

BACKGROUND

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).

METHODS

Treatment-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.

RESULTS

Of 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).

CONCLUSIONS

AML 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.

摘要

背景

健康状况偏好评估对于健康技术和经济评估至关重要。本研究使用美国普通人群的代表性样本,估计了与急性髓系白血病(AML)治疗相关的健康状态的效用和严重不良事件(SAE)的不效用。

方法

根据文献和与临床医生的访谈,定义了与治疗相关的 AML 健康状态,包括完全缓解(CR)、无 CR、复发、干细胞移植(SCT)和 SCT 后短期恢复。使用六个具有不同水平的属性,包括发热、缺乏能量、日常功能问题、焦虑/抑郁、输血和住院,来定义健康状态。使用离散选择实验方法设计了在线调查,以捕获包括所确定属性和关键 3/4 级化疗相关 SAE 的健康状态场景的偏好。使用广义估计方程的条件逻辑回归生成健康状态效用和 SAE 不效用。

结果

在 300 名调查参与者中,人口统计学分布与 2010 年美国人口普查的分布相差在 3%以内。CR 的效用值最高(0.875),其次是 SCT 后短期恢复(0.398)、复发(0.355)、无 CR(0.262)和 SCT(0.158)。在 SAE 中,严重感染导致的效用下降最高(0.218),其次是严重腹泻(0.176)、异常低血细胞计数(0.100)和严重发红/皮肤剥落(0.060)。

结论

AML 和治疗可能导致生活质量下降和日常活动能力受损。本研究的结果强调了社会对与治疗相关的 AML 健康状态和 SAE 的重视。

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