Assessing Patient-Reported Outcomes in Pediatric Populations With Vaccine-Preventable Infectious Diseases: A Systematic Review of the Literature (the PROCHID Study).

OBJECTIVE: To investigate the use of patient-reported outcomes (PROs) in pediatric populations with vaccine-preventable infectious diseases in high-income Western countries.

METHODS: Systematic review of PRO use in populations younger than 18 years with any of 17 infectious diseases for which vaccines are available or in development. The search was limited to studies performed in Europe, North America, Australia, and New Zealand and published between January 1, 1990, and July 31, 2013. Searches were conducted in Scopus and PsycINFO, and reference lists were manually searched. Results are reported using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines.

RESULTS: Of 6410 titles and abstracts and 174 full-text articles reviewed, 17 full-text articles were included for data extraction. The largest number of PRO studies was carried out in patients with anogenital warts and rotavirus gastroenteritis. No PRO studies were identified for nine conditions. A total of 24 PRO measures (12 generic and 12 disease-specific) were used in the studies reviewed. Most of the instruments used were of high quality. Proxy responses were occasionally obtained when self-report would have been feasible. No validated disease-specific instruments for children with any of the conditions studied were found.

CONCLUSIONS: The paucity of studies and PRO instruments to assess pediatric health status in vaccine-preventable infectious diseases, and the lack of a standardized approach to measurement, makes it difficult to capture the impact of disease and the benefit of vaccination and could potentially hinder decision making. Guidelines from relevant bodies to steer research in this area would be useful.