Controlling childhood arthritis without serious side-effects

A new class of medications has been successful in treating childhood arthritis, yet adverse effects are raising concerns. Scientists are conducting a large international clinical study to formulate regulations for safer use.

Juvenile idiopathic arthritis (JIA) is a group of autoimmune disorders characterised by chronic joint inflammation (arthritis) that first appears in childhood and early adolescence. A class of immunomodulators called biologics have selectively inhibited molecular substrates of inflammation and tissue damage. However, recent evidence suggests that their administration can be associated with adverse and often serious side-effects.

A multidisciplinary consortium is conducting a large international study leading to the safer and more effective use of biologics in JIA. EU funding of the project PHARMACHILD is facilitating the use of extensive data from international organisations and national registries in order to evaluate adverse events, and to develop screening and detection methods. Along the way, the partners are developing standardised data collection, representation and analysis protocols leading to a database to facilitate this and future pharmacovigilance studies and regulation.

The full study protocol has been designed and the web-based instructions and database are in the final test phase. Scientists have also established standard procedures for sample collection and storage as well as biomaterial banks (BMBs) at several locations.

Over 125 European paediatric rheumatology centres demonstrated interest in participating in the study and are in various stages of approval by their medical ethics committees. The quality of PHARMACHILD is assured by its award of an ENCePP (European Network of Centres for Pharmacoepidemiology and Pharmacovigilance) study seal indicating that it adheres to the high standards set by the network.

Serum concentrations of a complex of two calcium-binding proteins (S100A8 and S100A9) have been shown to predict systemic onset of JIA (SJIA) in the presence of fever of unknown origin. In addition, concentrations of these and one more biomarker can help identify patients in unstable remission and at increased risk of relapse. These results have been published in scientific peer-reviewed journals.

The PHARMACHILD study promises to vastly improve the current state of biologics used to treat JIA. From standardising data collection on adverse events to identifying markers predicting onset, partners expect to make a significant contribution to the safe regulation of such drugs and thus the health of a vulnerable youth and adolescent population.

Related link:
http://cordis.europa.eu