什么是相对生存?它在血液学中的作用是什么?

IF 2.2 4区 医学 Q3 HEMATOLOGY Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI:10.1016/j.beha.2023.101474
Maja Pohar Perme , Liesbeth C. de Wreede , Damjan Manevski
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引用次数: 0

摘要

在许多血液病中,生存概率是关键的结果。然而,当患者年龄较大且随访时间较长时,很大一部分死亡不能归因于所研究的疾病。这降低了常见生存率分析指标(如总生存率)的重要性,并表明需要其他需要更复杂方法的结果指标。当对疾病特异性信息感兴趣,但数据中没有死亡原因时,相对生存方法就变得至关重要。相对生存率的概念是将观察到的数据集与普通人群的死亡率数据合并,从而可以间接估计疾病的负担。在这项工作中,概述了血液学领域可能感兴趣的不同措施。我们介绍了报告由于感兴趣的疾病而死亡的概率的粗略死亡率;净生存率仅关注超额风险,是比较不同总体人群死亡率人群患者疾病负担的关键指标;以及相对生存率,它给出了患者和一般人群生存率的简单比较。我们解释了每个测度的性质,并给出了一些关于估计的简要说明。此外,我们还描述了如何研究与协变量的关联。所有方法及其估计量都在一个老年患者的子队列中进行了说明,这些患者因骨髓增生异常综合征或继发性急性髓系白血病接受了第一次异基因造血干细胞移植,以展示不同的方法如何为数据提供不同的见解。
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What is relative survival and what is its role in haematology?

In many haematological diseases, the survival probability is the key outcome. However, when the population of patients is rather old and the follow-up long, a significant proportion of deaths cannot be attributed to the studied disease. This lessens the importance of common survival analysis measures like overall survival and shows the need for other outcome measures requiring more complex methodology. When disease-specific information is of interest but the cause of death is not available in the data, relative survival methodology becomes crucial. The idea of relative survival is to merge the observed data set with the mortality data in the general population and thus allow for an indirect estimation of the burden of the disease.

In this work, an overview of different measures that can be of interest in the field of haematology is given. We introduce the crude mortality that reports the probability of dying due to the disease of interest; the net survival that focuses on excess hazard alone and presents the key measure in comparing the disease burden of patients from populations with different general population mortality; and the relative survival ratio which gives a simple comparison of the patients' and the general population survival. We explain the properties of each measure, and some brief notes are given on estimation. Furthermore, we describe how association with covariates can be studied. All the methods and their estimators are illustrated on a sub-cohort of older patients who received a first allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes or secondary acute myeloid leukemia, to show how different methods can provide different insights into the data.

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来源期刊
CiteScore
4.20
自引率
0.00%
发文量
42
审稿时长
35 days
期刊介绍: Best Practice & Research Clinical Haematology publishes review articles integrating the results from the latest original research articles into practical, evidence-based review articles. These articles seek to address the key clinical issues of diagnosis, treatment and patient management. Each issue follows a problem-orientated approach which focuses on the key questions to be addressed, clearly defining what is known and not known, covering the spectrum of clinical and laboratory haematological practice and research. Although most reviews are invited, the Editor welcomes suggestions from potential authors.
期刊最新文献
Erratum to “Special issue 37.2 and 37.3 Genetics and Function of HLA and immune-related genes in transplantation and cellular immunotherapy” [Best Pract Res Clin Haematol (2024) 101588] Editorial Board From clones to immunopeptidomes: New developments in the characterization of permissive HLA-DP mismatches in hematopoietic cell transplantation Relevance of donor-specific HLA antibodies in hematopoietic cell transplantation HLA structure and function in hematopoietic-cell transplantation
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