Ciurtin, Coziana;
Sparchez, Mihaela;
Elephtheriou, Despina;
Brogan, Paul;
(2025)
The evolving landscape of rheumatic drugs and their indications in paediatric and adolescent care.
Rheumatology
, Article keaf455. 10.1093/rheumatology/keaf455.
(In press).
Preview |
Text
Brogan_keaf455.pdf Download (469kB) | Preview |
Abstract
Paediatric rheumatic diseases (RMDs) are characterised 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, minimise the risk of disease relapse and organ damage, and ultimately preserve their quality of life. The last 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.
| Type: | Article |
|---|---|
| Title: | The evolving landscape of rheumatic drugs and their indications in paediatric and adolescent care |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1093/rheumatology/keaf455 |
| Publisher version: | https://doi.org/10.1093/rheumatology/keaf455 |
| Language: | English |
| Additional information: | Copyright © The Author(s) 2025. Published by Oxford University Press on behalf of the British Society for Rheumatology. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited. |
| Keywords: | Paediatric rheumatic drugs, targeted biologic treatments, small molecules, cell therapies, licensed therapies, toxicity |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10212802 |
Archive Staff Only
 |
View Item |
