TY - JOUR
T1 - Clinician-reported outcome measures in lupus trials
T2 - a problem worth solving
AU - Connelly, Kathryn
AU - Golder, Vera
AU - Kandane-Rathnayake, Rangi
AU - Morand, Eric F.
N1 - Funding Information:
KC receives a National Health and Medical Research Council postgraduate research scholarship and the Royal Australasian College of Physicians CRB Blackburn top-up scholarship. EFM receives funding from a National Health and Medical Research Council Investigator Grant. Funding sources had no specific involvement in this work.
Publisher Copyright:
© 2021 Elsevier Ltd
Copyright:
Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/8
Y1 - 2021/8
N2 - Systemic lupus erythematosus (SLE) remains a disease of high unmet clinical need. Because of substantial patient heterogeneity, the execution of clinical trials that successfully determine the efficacy of novel therapeutics compared with placebo is a continuous challenge. Clinician-reported outcome measures of treatment response used in SLE trials have evolved from the use of individual disease activity indices, including the SLE Disease Activity Index (SLEDAI) and British Isles Lupus Assessment Group (BILAG), to composite responder definitions such as the SLE Responder Index (SRI) and BILAG-Based Composite Lupus Assessment (BICLA), which are based on these indices. However, these approaches have notable drawbacks and defining the optimal clinical trial outcome measure for SLE remains a research goal. In this Viewpoint, we explore the strengths and limitations of existing indices and composite assessments, illustrating features which should be investigated in future analysis of trial data. Further, we provide a platform from which to advance new approaches to endpoint design, which is crucial to improve the interpretability and success of subsequent clinical trials in SLE.
AB - Systemic lupus erythematosus (SLE) remains a disease of high unmet clinical need. Because of substantial patient heterogeneity, the execution of clinical trials that successfully determine the efficacy of novel therapeutics compared with placebo is a continuous challenge. Clinician-reported outcome measures of treatment response used in SLE trials have evolved from the use of individual disease activity indices, including the SLE Disease Activity Index (SLEDAI) and British Isles Lupus Assessment Group (BILAG), to composite responder definitions such as the SLE Responder Index (SRI) and BILAG-Based Composite Lupus Assessment (BICLA), which are based on these indices. However, these approaches have notable drawbacks and defining the optimal clinical trial outcome measure for SLE remains a research goal. In this Viewpoint, we explore the strengths and limitations of existing indices and composite assessments, illustrating features which should be investigated in future analysis of trial data. Further, we provide a platform from which to advance new approaches to endpoint design, which is crucial to improve the interpretability and success of subsequent clinical trials in SLE.
UR - http://www.scopus.com/inward/record.url?scp=85111314408&partnerID=8YFLogxK
U2 - 10.1016/S2665-9913(21)00119-3
DO - 10.1016/S2665-9913(21)00119-3
M3 - Review Article
AN - SCOPUS:85111314408
SN - 2665-9913
VL - 3
SP - e595-e603
JO - The Lancet Rheumatology
JF - The Lancet Rheumatology
IS - 8
ER -