title: Colonisation endpoints in Streptococcus pneumoniae vaccine trials.
abstract: 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.
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland. Electronic address: kari.auranen@thl.fi.
authors.forename: Kari
authors.initials: K
authors.lastname: Auranen
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland.
authors.forename: Hanna
authors.initials: H
authors.lastname: Rinta-Kokko
authors.affiliation: University College London Medical School, Institute of Child Health, London, UK.
authors.forename: David
authors.initials: D
authors.lastname: Goldblatt
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland.
authors.forename: Hanna
authors.initials: H
authors.lastname: Nohynek
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
authors.forename: Katherine L
authors.initials: KL
authors.lastname: O'Brien
authors.affiliation: Pneumococcal Research and Infectious Diseases and Microbiology, Murdoch Childrens Research Institute, Royal Children's Hospital, Parkville, VIC, Australia; Department of Microbiology and Immunology, The University of Melbourne, Parkville, VIC, Australia.
authors.forename: Catherine
authors.initials: C
authors.lastname: Satzke
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland.
authors.forename: Birgit
authors.initials: B
authors.lastname: Simell
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland.
authors.forename: Antti
authors.initials: A
authors.lastname: Tanskanen
authors.affiliation: Department of Vaccination and Immune Protection, National Institute for Health and Welfare (THL), P.O. BOX 30, FI-00271, Helsinki, Finland.
authors.forename: Helena
authors.initials: H
authors.lastname: Käyhty
mesh_terms: D002353:Carrier State / Q000382:microbiology; D003430:Cross-Sectional Studies; D006801:Humans; D009305:Nasopharynx / Q000276:immunology / Q000382:microbiology; D017063:Outcome Assessment, Health Care; D011008:Pneumococcal Infections / Q000453:epidemiology / Q000382:microbiology* / Q000517:prevention & control*; D022242:Pneumococcal Vaccines / Q000276:immunology*; D015995:Prevalence; D013296:Streptococcus pneumoniae / Q000276:immunology*
chemical_list: D022242:Pneumococcal Vaccines
keywords: NVT; Nasopharyngeal colonisation; OR; PCV; Pneumococcus; Trial endpoint; VE(T); VE(acq); VE(col); VT; Vaccine efficacy; combined vaccine efficacy against acquisition and duration of colonisation; non-vaccine (sero)type(s); odds ratio; pneumococcal conjugate vaccine; vaccine (sero)type(s); vaccine efficacy against acquisition of colonisation; vaccine efficacy against colonisation