Optimizing treatment in paediatric rheumatology--lessons from oncology.

Abstract

Treatment of children with cancer, in particular with acute lymphoblastic leukaemia (ALL), has been highly successful in the past two decades owing to the implementation of treatment optimization studies. Study centres appointed by scientific societies design treatment optimization study protocols (TOSPs) that address an investigator-initiated research question and detail treatment procedures according to these aims. Nearly all children with malignant diseases are treated within TOSPs, whereas children with juvenile idiopathic arthritis (JIA) and other common paediatric rheumatic diseases are mostly treated outside TOSPs and clinical trials. Despite the differences in natural course and prognosis between malignant and inflammatory diseases, aiming for the recruitment of all children with defined rheumatic diseases into TOSPs or similar protocols would enable the longitudinal collection of crucial clinical data and improve evidence-based approaches. Successful research networks already exist in paediatric rheumatology that could facilitate the implementation of this approach. Paediatric rheumatic diseases have a considerable impact on patients and their families; thus, I propose that research networks in paediatric rheumatology should recruit most--if not all--children with rheumatic diseases into study protocols with standardized treatment and outcome measures.