WHAT IS PRINTO

Introduction
The pediatric rheumatic diseases (PRD) are rare conditions associated with substantial morbidity, consequence on the quality of life, and monetary costs. Many studies of the impact and outcome of PRD have shown that this group of diseases is associated with greater morbidity and monetary cost than previously thought. For example, long term outcome studies of children with juvenile idiopathic arthritis (JIA) report that, after a mean follow-up of 15 years, the majority of the patients continue to experience some difficulties in daily living activities, and that moderate to severe pain is still present in 30% of the patients. There is also evidence of cumulative organ damage in patients with juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM).
Certainly childhood chronic illnesses with high levels of morbidity should be the target of intense research aimed at ameliorating and/or curing the disease. However, conducting clinical trials in PRD has proven difficult for a host of reasons.
Due to the rarity of the diseases the only possibility to gather a sufficient number of patients to obtain clinically and statistically valid results in a reasonable period of time, is to perform multi-centre studies on an international scale. The ethics of conducting any placebo-controlled trial, even in adults, has recently come under intense debate. Parents often refuse entry into studies because they are uncomfortable with the prospect of their child being assigned by chance to placebo. Securing funding for conducting clinical trials in PRD has always been difficult since the pharmaceutical industry has little interest in funding these trials due to the small potential market.
Drugs available for the treatment of PRD have been used in new dosages, new routes of administration, and new combinations. Unfortunately, data regarding the safety and effectiveness of these new treatment regimens tends to be from small, open, anecdotal, uncontrolled, non-randomized case series. Examples include the use of high dose MTX in recalcitrant JIA and of MTX usage in juvenile dermatomyositis. Many of these new approaches to management may represent improvements over existing standards, but without larger, systematic trials the data must remain suspect