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对多发性骨髓瘤健康状态效用进行频繁主义和贝叶斯元回归分析,包括系统评价和个体患者数据分析。

Frequentist and Bayesian meta-regression of health state utilities for multiple myeloma incorporating systematic review and analysis of individual patient data.

机构信息

Department of Statistical Science, University College London, London, UK.

Delta Hat Limited, University Nottingham University Hospital, Nottingham, UK.

出版信息

Health Econ. 2019 May;28(5):653-665. doi: 10.1002/hec.3871. Epub 2019 Feb 20.

Abstract

This analysis presents the results of a systematic review for health state utilities in multiple myeloma, as well as analysis of over 9,000 observations taken from registry and trial data. The 27 values identified from 13 papers are then synthesised in a frequentist nonparametric bootstrap model and a Bayesian meta-regression. Results were similar between the frequentist and Bayesian models with low utility on disease diagnosis (approximately 0.55), raising to approximately 0.65 on first line treatment and declining slightly with each subsequent line. Stem cell transplant was also found to be a significant predictor of health-related quality of life in both individual patient data and meta-regression, with an increased utility of approximately 0.06 across different models. The work presented demonstrates the feasibility of Bayesian methods for utility meta-regression, whilst also presenting an internally consistent set of data from the analysis of registry data. To facilitate easy updating of the data and model, data extraction tables and model code are provided as Data S1. The main limitations of the model relate to the low number of studies available, particularly in highly pretreated patients.

摘要

本分析报告呈现了一项系统性综述的结果,该综述涉及多发性骨髓瘤的健康状态效用,同时还分析了来自注册和试验数据的超过 9000 个观察结果。从 13 篇论文中确定的 27 个值随后在频率非参数自举模型和贝叶斯荟萃回归中进行了综合分析。频率论和贝叶斯模型的结果相似,疾病诊断时的效用较低(约为 0.55),一线治疗时约为 0.65,随后每增加一线治疗效用略有下降。干细胞移植在个体患者数据和荟萃回归中也被发现是健康相关生活质量的重要预测因素,不同模型中效用增加约 0.06。本研究展示了贝叶斯方法在效用荟萃回归中的可行性,同时也从注册数据的分析中呈现了一套内部一致的数据。为了便于数据和模型的更新,提供了数据提取表和模型代码作为数据 S1。该模型的主要局限性与可用研究数量较少有关,特别是在高度预处理患者中。

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