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Quality of life in clinical trials for children

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Abstract

Purpose

This review is aimed at: discussing the importance of measuring quality of life (QoL) in children, describing the measures actually available for measuring QoL in children, and listing methodological issues related to the inclusion of QoL outcomes in clinical trials.

Methods

A literature review was carried out. Searches were conducted in Pubmed. All articles published within the last 20 years with the objectives of assessing and reviewing use and issues related to QoL instruments in children were reviewed.

Results

Despite the numerous arguments in favour of assessing QoL, these data are not typically collected in paediatric clinical trials and research protocols. Because adult measures may fail to address the specific aspects of QoL that are important to the child, many different measures, both generic and disease-specific, have been developed to assess QoL in children; however, their quality in terms of psychometric properties often seems questionable. Methodological recommendations are provided for designing a study including QoL outcome.

Conclusions

This review confirms the importance of measuring QoL in children because of increased survival rates of children with chronic health conditions and because children account for the highest prevalence of disabling conditions. However, measuring QoL in children is still a challenge mainly because of the limitations of the currently available measures. Further research is needed to reach a consensus on the most appropriate formats for child-centred instruments.

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Acknowledgement

This contribution is part of the Task-force in Europe for Drug Development for the Young (TEDDY) Network of Excellence supported by the European Commission’s Sixth Framework Program (Contract n. 0005216 LSHBCT- 2005-005126).

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Authors and Affiliations

  1. Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy

    Annalisa Trama

  2. Milano, Italy

    Massimiliano Dieci

Authors
  1. Annalisa Trama
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  2. Massimiliano Dieci
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Correspondence to Annalisa Trama.

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Part of TEDDY Supplement

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Trama, A., Dieci, M. Quality of life in clinical trials for children. Eur J Clin Pharmacol 67 (Suppl 1), 41–47 (2011). https://doi.org/10.1007/s00228-010-0924-0

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  • DOI: https://doi.org/10.1007/s00228-010-0924-0

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