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Assessing Patient-Reported Outcomes in Pediatric Populations With Vaccine-Preventable Infectious Diseases: A
Michael Herdman1, Christopher K Hoyle1, Victoria Coles2
1Office of Health Economics, London, UK.
Insights
Limited research exists on patient-reported outcomes (PROs) for pediatric vaccine-preventable infectious diseases. Developing standardized PRO measures is crucial for understanding disease impact and vaccination benefits.
Area of Science:
- Pediatric infectious diseases
- Vaccinology
- Health outcomes research
Background:
- Patient-reported outcomes (PROs) are vital for assessing health status in pediatric populations.
- Vaccine-preventable infectious diseases pose a significant burden on children worldwide.
- Understanding the impact of these diseases and the benefits of vaccination requires robust outcome measures.
Purpose of the Study:
- To systematically review the utilization of PROs in pediatric populations (under 18 years) with vaccine-preventable infectious diseases.
- To identify existing PRO measures and their quality in high-income Western countries.
- To highlight gaps in research regarding PROs for these conditions.
Main Methods:
- Systematic review of studies published between 1990 and 2013 in Europe, North America, Australia, and New Zealand.
- Searches conducted in Scopus and PsycINFO, supplemented by manual reference list searches.
- Inclusion criteria focused on 17 vaccine-preventable infectious diseases and pediatric populations, adhering to PRISMA guidelines.
Main Results:
- Seventeen full-text articles were included from over 6410 titles and abstracts.
- Anogenital warts and rotavirus gastroenteritis had the most PRO studies; nine conditions had none.
- Twenty-four PRO measures were used (12 generic, 12 disease-specific), mostly of high quality, but no validated disease-specific instruments for children were found.
Conclusions:
- A significant paucity of research and standardized PRO instruments exists for pediatric vaccine-preventable infectious diseases.
- This lack of data hinders the assessment of disease impact and vaccination benefits, potentially impeding decision-making.
- Development of guidelines is recommended to direct future research in this critical area.
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.
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