International research networks in pediatric rheumatology: the PRINTO perspective

Abstract

Purpose of review: The purpose of this review is to highlight the problems and possible solutions for the conduct of international collaborative research for pediatric rheumatic diseases.

Recent findings: Pediatric rheumatic diseases are rare conditions associated with important sequelae on the quality of life and long-term outcome. The research aimed at studying new therapeutic approaches is difficult because of logistic, methodological, and ethical problems. To face these problems, two international networks have been founded: the Pediatric Rheumatology Collaborative Study Group (or PRCSG) and the Paediatric Rheumatology international Trials Organization (or PRINTO). The two networks have the goal to promote, facilitate, and conduct high-quality research into pediatric rheumatic diseases. In particular they have been able to standardize the evaluation of response to therapy in juvenile idiopathic arthritis, juvenile systemic lupus erythematosus, and juvenile dermatomyositis; to draft clinical remission criteria in juvenile idiopathic arthritis; and to provide cross-cultural adapted and validated quality-of-life instruments like the Childhood Health Assessment Questionnaire and the Child Health Questionnaire in 32 different languages.

Summary: The creation of large international trial networks such as PRINTO and PRCSG, the definition of internationally recognized and standardized outcome measures and definitions of improvement, the validation of quality-of-life instruments, and the adoption of adequate legislative measures (pediatric rule) have created the basic premises for the best future assessment of pediatric rheumatic diseases. This progress now affords children with pediatric rheumatic diseases the same opportunities as adults to be treated with drugs whose safety and efficacy have been assessed through legitimate scientifically valid investigations.