Colonisation endpoints in Streptococcus pneumoniae vaccine trials

• Published in: Vaccine Vol. 32; no. 1; pp. 153 - 158
• Main Authors: Auranen, Kari, Rinta-Kokko, Hanna, Goldblatt, David, Nohynek, Hanna, O’Brien, Katherine L., Satzke, Catherine, Simell, Birgit, Tanskanen, Antti, Käyhty, Helena
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier Ltd 17.12.2013
• Subjects: Allergy and Immunology; Carrier State - microbiology; Cross-Sectional Studies; experimental design; guidelines; Humans; Nasopharyngeal colonisation; Nasopharynx - immunology; Nasopharynx - microbiology; Outcome Assessment (Health Care); pathogens; Pneumococcal Infections - epidemiology; Pneumococcal Infections - microbiology; Pneumococcal Infections - prevention & control; Pneumococcal Vaccines - immunology; Pneumococcus; Prevalence; Streptococcus pneumoniae; Streptococcus pneumoniae - immunology; Trial endpoint; Vaccine efficacy; vaccines; PCV; OR; VET; Vaccine efficacy; Trial endpoint; VEacq; VEcol; NVT; Pneumococcus; VT; Nasopharyngeal colonisation; VE acq; vaccine efficacy against colonisation; odds ratio; VE T; vaccine efficacy against acquisition of colonisation; VE col; combined vaccine efficacy against acquisition and duration of colonisation; vaccine (sero)type(s); non-vaccine (sero)type(s); pneumococcal conjugate vaccine; VE(acq); VE(T); VE(col)
• ISSN: 0264-410X; 1873-2518; 1873-2518
• DOI: 10.1016/j.vaccine.2013.08.061

•We consider evaluation of vaccine efficacy against pneumococcal colonisation.•The need to carefully define the efficacy parameter is emphasised.•Efficacy against acquisition should be preferred over efficacy against prevalence.•We review methods to estimate efficacy against acquisition from cross-sectional data. Evaluating vaccine efficacy for protection against colonisation (VEcol) with bacterial pathogens is an area of growing interest. In this article, we consider estimation of VEcol for colonisation with Streptococcus pneumoniae (the pneumococcus). Colonisation is a common, recurrent and multi-type endpoint that requires both careful definition of the vaccine efficacy parameter and the corresponding method of estimation. We review recent developments in the area and provide practical guidelines for choosing the estimand and the estimation method in trials with a colonisation endpoint. We concentrate on methods that are based on a cross-sectional study design, in which only one nasopharyngeal sample is obtained per study subject.