The evolving landscape of rheumatic drugs and their indications in paediatric and adolescent care

Paediatric rheumatic diseases (RMDs) are characterized by dysregulation of the immune system functions due to a combination of genetic, epigenetic and environmental factors. In many cases, children and young people (CYP) with RMDs require long-term pharmacological interventions to control their symptoms, minimize the risk of disease relapse and organ damage and ultimately preserve their quality of life. The past decades have seen significant progress in the expansion of therapeutic options licensed or available off-license for CYP with RMDs, and an unprecedented number of paediatric interventional clinical trials testing new therapies. This review aims to appraise the paediatric rheumatology community on available pharmacological therapies for use in childhood-onset RMDs, including conventional, biologic and targeted synthetic disease modifying anti-rheumatic drugs, immunoglobulins and cell-based therapies, highlighting their known indications, as well as current guidelines and consensus recommendations supporting their use off-license. We review the paediatric dosing regimens available for the treatment of RMDs and other autoimmune conditions, the toxicity profile of available therapeutic agents and provide a comprehensive evaluation of emerging therapies for childhood RMDs, currently tested in clinical trials.