TY - JOUR
T1 - Conceptual framework for defining disease modification in systemic lupus erythematosus
T2 - A call for formal criteria
AU - van Vollenhoven, Ronald
AU - Askanase, Anca D.
AU - Bomback, Andrew S.
AU - Bruce, Ian N.
AU - Carroll, Angela
AU - Dall'Era, Maria
AU - Daniels, Mark
AU - Levy, Roger A.
AU - Schwarting, Andreas
AU - Quasny, Holly A.
AU - Urowitz, Murray B.
AU - Zhao, Ming-Hui
AU - Furie, Richard
PY - 2021
Y1 - 2021
N2 - Disease modification has become a well-established concept in several therapeutic areas; however, no widely accepted definition of disease modification exists for SLE. We reviewed established definitions of disease modification in other conditions and identified a meaningful effect on ‘disease manifestations’ (ie, signs, symptoms and patient-reported outcomes) and on ‘disease outcomes’ (eg, long-term remission or progression of damage) as the key principles of disease modification, indicating a positive effect on the natural course of the disease. Based on these findings and the treatment goals and outcome measures for SLE, including lupus nephritis, we suggest a definition of disease modification based on disease activity indices and organ damage outcomes, with the latter as a key anchor. A set of evaluation criteria is also suggested. Establishing a definition of disease modification in SLE will clarify which treatments can be considered disease modifying, provide an opportunity to harmonise future clinical trial outcomes and enable comparison between therapies, all of which could ultimately help to improve patient outcomes. This publication seeks to catalyse further discussion and provide a framework to develop an accepted definition of disease modification in SLE.
AB - Disease modification has become a well-established concept in several therapeutic areas; however, no widely accepted definition of disease modification exists for SLE. We reviewed established definitions of disease modification in other conditions and identified a meaningful effect on ‘disease manifestations’ (ie, signs, symptoms and patient-reported outcomes) and on ‘disease outcomes’ (eg, long-term remission or progression of damage) as the key principles of disease modification, indicating a positive effect on the natural course of the disease. Based on these findings and the treatment goals and outcome measures for SLE, including lupus nephritis, we suggest a definition of disease modification based on disease activity indices and organ damage outcomes, with the latter as a key anchor. A set of evaluation criteria is also suggested. Establishing a definition of disease modification in SLE will clarify which treatments can be considered disease modifying, provide an opportunity to harmonise future clinical trial outcomes and enable comparison between therapies, all of which could ultimately help to improve patient outcomes. This publication seeks to catalyse further discussion and provide a framework to develop an accepted definition of disease modification in SLE.
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85131264890&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/35346982
U2 - https://doi.org/10.1136/lupus-2021-000634
DO - https://doi.org/10.1136/lupus-2021-000634
M3 - Review article
C2 - 35346982
SN - 2053-8790
VL - 9
JO - Lupus science & medicine
JF - Lupus science & medicine
IS - 1
M1 - e000634
ER -