Pediatric Rheumatology Online Journal

Angelo Ravelli, MD, and Alberto Martini, MD
Dipartimento di Pediatria, Università di Genova,

Divisone di Pediatria II, IRCCS Istituto G. Gaslini, L.go G.Gaslini 5, 16147 Genova, Italy
Tel.: +39-010-5636386; Fax: +39-010-5636211
E-mail: angeloravelli@ospedale-gaslini.ge.it

We’re in this together

        Most pediatric rheumatic diseases (RD) are rare, heterogeneous, and have unknown etiology. This makes cooperation among different groups fundamental to hasten progress in research, diagnosis and treatment.
        Since its birth, the flowering of pediatric rheumatology as a clinical discipline has been significantly fostered by the distinct propensity of its scientific community to work together. The first collaborative efforts took place separately in USA and Europe in the mid seventies, and led to the development of the classification criteria for juvenile rheumatoid arthritis (JRA) [1] and juvenile chronic arthritis (JCA) [2], respectively. Subsequent attempts to set up an internationally accepted nosography have resulted recently in the proposed [3] and revised [4] criteria for the classification of juvenile idiopathic arthritis (JIA). In the late seventies, a pediatric section was created within the American College of Rheumatology (formerly American Rheumatism Association) with the aim of improving clinical and scientific knowledge and of stimulating cooperation among clinical and basic research scientists. In 1999, the Pediatric Rheumatology European Society (PRES) was founded; it gathers European healthcare professionals in the field of pediatric rheumatology and has the main missions of promoting knowledge and research and of providing guidelines and standards for good clinical practice.
        Starting in the eighties, the Pediatric Rheumatology Collaborative Study Group (PRCSG) in the US has pioneered the application of evidence-based medicine to pediatric rheumatology and has carried out seminal randomized controlled trials, which represent the guide to the present therapy of children with JIA [5-8]. The “Pediatric Rheumatology International Trial Organization” (PRINTO), founded in 1996, has grown rapidly and now includes 43 countries, not only from Europe but also from Latin America, Africa, and Asia. In 1996 a combined effort, under the direction of Ed Giannini, between PRCSG and PRINTO led to the development of the preliminary definition of improvement in children with JIA [9]. PRINTO has performed the translation and cross-cultural validation in 32 languages of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) [10] and has recently completed a randomized clinical trial aimed to compare the use of medium vs. higher dose of methotrexate in children with JIA who failed to respond to the standard doses of the drug. [11].
        Several controlled trials with new drugs are now being performed in pediatric RD. This is due not only to the availability of the large international pediatric rheumatology networks, but also to the implementation of a new adequate legislation. Indeed, in 1999 the FDA approved the so-called “pediatric rule”. (Editor's Note: Since suspended by court order but under appeal and bill introduced by Congress) Thanks to this rule, a pharmaceutical company who wish to register a new medication for use in adults has to provide data on safety and efficacy also in children if, in children, a disease exists similar to that for which the registration is requested in adults. It is hoped that the pediatric rule is reinstated by court order or Congress. It is likely that a similar legislation will be adopted in the future by the EMEA, the European equivalent of the FDA.
        If international collaboration has already greatly contributed to current achievement, it is even more needed for the many problems that remain to be solved. The etiology of most pediatric RD diseases is still unknown. We will be able to provide the best cure only when we will understand the underlying causes and mechanisms. This is, however, a formidable task that requires a close collaboration between clinicians and laboratories all over the world. Common research programs and centralization of investigations according to different expertise would be of great help.
        Looking worldwide, many regions lack adequately trained pediatric rheumatologists or sufficient numbers to deal with the burden of childhood RD. This means that many children still do not have proper diagnosis and adequate therapy. The international pediatric rheumatology community has to make a big effort in order to disseminate information on diagnosis and treatment of pediatric RD. The facilities provided by information technology represent a very powerful tool to promote teaching all over the world.
        Many childhood RD are rare and can only be studied through a large collaborative effort. Moreover, all of us are often challenged with odd diseases or disease manifestations that do not fit our current knowledge; only the sharing of information about these odd entities will allow us to gain new insights. Major differences exist in the epidemiology of childhood RD: for example, acute rheumatic fever is still a problem in developing countries, whereas HIV and tuberculosis related musculoskeletal problems are mostly prevalent in Africa and part of Asia. An effort has to be done so that developing countries can adequately take profit of the technological and research advances achieved in the Western World, in order to improve the care of the RD that are more prevalent in their setting. Another source of concern is that the availability of new medications for pediatric RD may widen the gap between rich and poor countries, because they are expensive and not universally available. Strategies must be developed to ensure that those patients who are in greatest need or may benefit most are selected for treatment and not only those who can afford these treatments.
        Taking advantage of the existence of large pediatric rheumatology networks and of an adequate legislation (pediatric rule), much effort has to be done to assess efficacy and safety of new drugs as well as of “old” drugs that are used “off-label”.
        Thus, any further progress in our specialty heavily relies on international collaboration. We are leaving in the globalization era, with the spectrum of contacts between countries and cultures growing rapidly. Countries, regions, and entire continents are becoming interdependent and interactive. Through the Internet, knowledge is increasingly accessible at any place at any time. By this way, knowledge can be applied and generated in countries where there is no long tradition of an education system and where there are only a few pediatric rheumatologists. This new on-line pediatric rheumatology journal would like to represent a contribution to disseminate worldwide the knowledge of pediatric rheumatology and to foster international collaboration among clinicians and researchers.

REFERENCES
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9. Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Felson DT, Martini A. Preliminary definition of improvement in juvenile arthritis, Arthritis Rheum 1997; 40: 1202-9.
10. Ruperto N, Ravelli A, Pistorio A, et al. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology, Clin Exp Rheumatol. 2001; 19(Suppl. 23): S1-9.
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Vol. 1, No. 1 (3-7) 2003