Pediatric drug safety signal detection: a new drug-event reference set for performance testing of data-mining methods and systems.

Background: Better evidence regarding drug safety in the pediatric population might be generated from existing data sources such as spontaneous reporting systems and electronic healthcare records. The Global Research in Paediatrics (GRiP)-Network of Excellence aims to develop pediatric-specific methods that can be applied to these data sources. A reference set of positive and negative drug-event associations is required.

Defining off-label and unlicensed use of medicines for children: results of a Delphi survey.

The aim of this Delphi survey is to develop common definitions for unlicensed and off-label drug use in children to be used for research and regulatory purposes. After a literature review on the current status of unlicensed/off-label definitions, a two-stage, web-based Delphi survey was conducted among experts in Europe. Their opinion on concerns, rules and scenarios regarding the unlicensed and off-label use of medicines were obtained.

Challenges in prescribing drugs for children with cancer.

Paediatric oncology has achieved high cure rates despite the limited availability of drugs that have been specifically studied for use in children with cancer. Efficacy of these drugs has received more attention than their safety, but permanent side-effects in growing children need to be considered. An absence of pharmacokinetic data, dose-defining studies, schedules defined by age, and appropriate formulations can lead to underdosing or overdosing in specific age groups, resulting in a potential lack of benefit, development of resistance, and increased adverse drug reactions.

Risk factors for death in patients with beta-thalassemia major: results of a case-control study.

This retrospective one to one matched case-control study was aimed at evaluating risk factors for death in beta-thalassemic patients followed in Italian centers between 1997 and 2001. The mortality risk was lower in patients with good compliance to iron chelation therapy and in those treated with deferiprone.