Deriving a Preference-Based Measure for Myelofibrosis from the EORTC QLQ-C30 and the MF-SAF

Deriving a Preference-Based Measure for Myelofibrosis from the EORTC QLQ-C30 and the MF-SAF
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DOI:
10.1016/j.jval.2015.07.004
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发表时间:
2015-09-01
期刊:
影响因子:
4.5
通讯作者:
Nafees, Beenish
Nafees, Beenish
中科院分区:
医学2区
文献类型:
--
作者:
Mukuria, Clara;Rowen, Donna;Nafees, Beenish

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背景:使用成本效用分析进行经济评价需要效用值。通常,使用EuroQol五维问卷等通用指标,但这可能无法适当反映癌症(包括骨髓纤维化)患者的健康相关生活质量。目的:使用适当的现有指标、骨髓纤维化-症状评估表和欧洲癌症研究与治疗组织30项生活质量问卷,推导出基于病情偏好的骨髓纤维化指标。研究方法:来自口服JAK抑制剂治疗的对照骨髓纤维化研究试验(n = 309)的数据用于推导健康状态分类系统。心理测量和因素分析被用来确定的分类系统的尺寸。然后使用心理测量和Rasch分析来选择一个项目来代表每个维度,项目选择由专家进行验证。选择的健康状态的一般人群的成员使用时间权衡的价值。最后,使用回归分析对健康状态值进行建模,以产生每个状态的效用值。结果如下:骨髓纤维化8维度有8个维度:身体功能,情绪功能,疲劳,瘙痒,左侧肋骨下疼痛,腹部不适,骨骼或肌肉疼痛和盗汗。回归模型估计使用的时间权衡数据,从246名成员的一般人口的价值,共33个国家。表现最好的模型是随机效应最大似然模型,其效用值范围为0.089至1。结论:骨髓纤维化8维是一种基于条件特异性偏好的骨髓纤维化指标。该测量可用于生成包含骨髓纤维化-症状评估表和欧洲癌症研究和治疗组织30生活质量问卷数据的任何数据集的骨髓纤维化的效用值
Background: Utility values are required for economic evaluation using cost-utility analyses. Often, generic measures such as the EuroQol five-dimensional questionnaire are used, but this may not appropriately reflect the health-related quality of life of patients with cancer including myelofibrosis. Objective: To derive a condition-specific preference based measure for myelofibrosis using appropriate existing measures, the Myelofibrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire. Methods: Data from the Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment trial (n = 309) were used to derive the health state classification system. Psychometric and factor analyses were used to determine the dimensions of the classification system. Psychometric and Rasch analyses were then used to select an item to represent each dimension item selection was validated with experts. A selection of health states was valued by members of the general population using time trade-off. Finally, health state values were modeled using regression analysis to produce utility values for every state. Results: The Myelofibrosis 8 dimensions has eight dimensions: physical functioning, emotional functioning, fatigue, itchiness, pain under ribs on the left side, abdominal discomfort, bone or muscle pain, and night sweats. Regression models were estimated using time trade-off data from 246 members of the general population valuing a total of 33 states. The best performing model was a random effects maximum likelihood model producing utility values ranging from 0.089 to 1. Conclusions: The Myelofibrosis 8 dimensions is a condition-specific preference based measure for myelofibrosis. This measure can be used to generate utility values for myelofibrosis for any data set containing the Myelonbrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire data