Callegaro Andrea, Curran Desmond, Matthews Sean
GSK Vaccines, Rue de l'Institut, Rixensart, Belgium.
GSK Vaccines, Avenue Fleming, Wavre, Belgium.
Pharm Stat. 2020 Sep;19(5):636-645. doi: 10.1002/pst.2020. Epub 2020 Mar 27.
In recent years, many vaccines have been developed for the prevention of a variety of diseases. Although the primary objective of vaccination is to prevent disease, vaccination can also reduce the severity of disease in those individuals who develop breakthrough disease. Observations of apparent mitigation of breakthrough disease in vaccine recipients have been reported for a number of vaccine-preventable diseases such as Herpes Zoster, Influenza, Rotavirus, and Pertussis. The burden-of-illness (BOI) score was developed to incorporate the incidence of disease as well as the severity and duration of disease. A severity-of-illness score S > 0 is assigned to individuals who develop disease and a score of 0 is assigned to uninfected individuals. In this article, we derive the vaccine efficacy statistic (which is the standard statistic for presenting efficacy outcomes in vaccine clinical trials) based on BOI scores, and we extend the method to adjust for baseline covariates. Also, we illustrate it with data from a clinical trial in which the efficacy of a Herpes Zoster vaccine was evaluated.
近年来，已研发出许多用于预防多种疾病的疫苗。尽管接种疫苗的主要目的是预防疾病，但接种疫苗也可以降低那些发生突破性疾病的个体的疾病严重程度。对于一些疫苗可预防的疾病，如带状疱疹、流感、轮状病毒和百日咳，已有报告称在疫苗接种者中观察到突破性疾病明显减轻。疾病负担（BOI）评分旨在综合考虑疾病的发病率以及疾病的严重程度和持续时间。对于发生疾病的个体，赋予疾病严重程度评分S>0，而对于未感染个体，赋予评分0。在本文中，我们基于BOI评分推导出疫苗效力统计量（这是在疫苗临床试验中呈现效力结果的标准统计量），并扩展该方法以对基线协变量进行调整。此外，我们用一项评估带状疱疹疫苗效力的临床试验数据对其进行说明。
