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What is relative survival and what is its role in haematology?
Best Practice & Research Clinical Haematology ( IF 2.1 ) Pub Date : 2023-05-08 , DOI: 10.1016/j.beha.2023.101474
Maja Pohar Perme 1 , Liesbeth C de Wreede 2 , Damjan Manevski 1
Affiliation  

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.



中文翻译:

什么是相对生存率以及它在血液学中的作用是什么?

在许多血液疾病中,生存概率是关键结果。然而,当患者群体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。这降低了总体生存等常见生存分析指标的重要性,并表明需要其他需要更复杂方法的结果指标。当特定疾病的信息令人感兴趣但数据中无法提供死亡原因时,相对生存方法就变得至关重要。相对生存的想法是将观察到的数据集与一般人群的死亡率数据合并,从而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。我们引入了粗死亡率,它报告了因感兴趣的疾病而死亡的概率;净生存率仅关注过度危险,是比较具有不同总人口死亡率的人群的患者疾病负担的关键指标;相对生存率,对患者和一般人群的生存进行简单比较。我们解释了每个度量的属性,并给出了一些关于估计的简短注释。此外,我们描述了如何研究与协变量的关联。所有方法及其估计量均在因骨髓增生异常综合征或继发性急性髓性白血病接受首次同种异体造血干细胞移植的老年患者亚队列中进行说明,以显示不同的方法如何提供对数据的不同见解。

更新日期:2023-05-12
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