TY - JOUR
T1 - Personalised medicine in juvenile dermatomyositis
T2 - From novel insights in disease mechanisms to changes in clinical practice
AU - Veldkamp, Saskia R.
AU - van Wijk, Femke
AU - van Royen-Kerkhof, Annet
AU - Jansen, Marc HA
N1 - Publisher Copyright:
© 2024
PY - 2024/9
Y1 - 2024/9
N2 - Juvenile dermatomyositis is characterized by childhood-onset chronic inflammation of the muscles and skin, with potential involvement of other organs. Patients are at risk for long-term morbidity due to insufficient disease control and steroid-related toxicity. Personalised treatment is challenged by a lack of validated tools that can reliably predict treatment response and monitor ongoing (subclinical) inflammation, and by a lack of evidence regarding the best choice of medication for individual patients. A better understanding of the involved disease mechanisms could reveal potential biomarkers and novel therapeutic targets. In this review, we highlight the most relevant immune and non-immune mechanisms, elucidating the effects of interferon overexpression on tissue alongside the interplay between the interferon signature, mitochondrial function, and immune cells. We review mechanism-based biomarkers that are promising for clinical implementation, and the latest advances in targeted therapy development. Finally, we discuss key steps needed for translating these discoveries into clinical practice.
AB - Juvenile dermatomyositis is characterized by childhood-onset chronic inflammation of the muscles and skin, with potential involvement of other organs. Patients are at risk for long-term morbidity due to insufficient disease control and steroid-related toxicity. Personalised treatment is challenged by a lack of validated tools that can reliably predict treatment response and monitor ongoing (subclinical) inflammation, and by a lack of evidence regarding the best choice of medication for individual patients. A better understanding of the involved disease mechanisms could reveal potential biomarkers and novel therapeutic targets. In this review, we highlight the most relevant immune and non-immune mechanisms, elucidating the effects of interferon overexpression on tissue alongside the interplay between the interferon signature, mitochondrial function, and immune cells. We review mechanism-based biomarkers that are promising for clinical implementation, and the latest advances in targeted therapy development. Finally, we discuss key steps needed for translating these discoveries into clinical practice.
KW - Biomarkers
KW - Interferon
KW - Juvenile dermatomyositis
KW - Personalised medicine
UR - http://www.scopus.com/inward/record.url?scp=85202020907&partnerID=8YFLogxK
U2 - 10.1016/j.berh.2024.101976
DO - 10.1016/j.berh.2024.101976
M3 - Review article
C2 - 39174374
AN - SCOPUS:85202020907
SN - 1521-6942
VL - 38
JO - Best Practice and Research: Clinical Rheumatology
JF - Best Practice and Research: Clinical Rheumatology
IS - 3
M1 - 101976
ER -