Validation of the systemic lupus erythematosus responder index for use in juvenile-onset systemic lupus erythematosus.
OBJECTIVE
This study tested the concurrent validity of the systemic lupus erythematosus responder index (SRI) in assessing improvement in juvenile-onset systemic lupus erythematosus (jSLE).


METHODS
The SRI considers changes in the SELENA-SLEDAI, BILAG and a 3-cm visual analogue scale of physician-rated disease activity (PGA) to determine patient improvement. Using prospectively collected data from 760 unique follow-up visit intervals of 274 jSLE patients, we assessed the sensitivity and specificity of the SRI using these external standards: physician-rated improvement (MD-change), patient/parent-rated major improvement of wellbeing (patient-change) and decrease in prescribed systemic corticosteroids (steroid-change). Modifications of the SRI that considered different thresholds for the SELENA-SLEDAI, BILAG and 10-cm PGA were explored and agreement with the American College of Rheumatology/PRINTO provisional criteria for improvement of jSLE (PCI) was examined.


RESULTS
The sensitivity/specificity in capturing major improvement by the MD-change were 78%/76% for the SRI and 83%/78% for the PCI, respectively. There was fair agreement between the SRI and PCI (kappa=0.35, 95% CI 0.02 to 0.73) in capturing major improvement by the MD-change. Select modified versions of the SRI had improved accuracy overall. All improvement criteria tested had lower sensitivity when considering patient-change and steroid-change as external standards compared to MD-change.


CONCLUSIONS
The SRI and its modified versions based on meaningful changes in jSLE have high specificity but at most modest sensitivity for capturing jSLE improvement. When used as an endpoint of clinical trials in jSLE, the SRI will provide a conservative estimate regarding the efficacy of the therapeutic agent under investigation.
Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, , Cincinnati, Ohio, USA.
Rina
R
Mina
Marisa S
MS
Klein-Gitelman
Shannen
S
Nelson
B Anne
BA
Eberhard
Gloria
G
Higgins
Nora G
NG
Singer
Karen
K
Onel
Lori
L
Tucker
Kathleen M
KM
O'Neil
Marilynn
M
Punaro
Deborah M
DM
Levy
Kathleen
K
Haines
Alberto
A
Martini
Nicolino
N
Ruperto
Daniel
D
Lovell
Hermine I
HI
Brunner
D000293:Adolescent; D017668:Age of Onset; D002648:Child; D004334:Drug Administration Schedule; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage / Q000627:therapeutic use; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000175:diagnosis* / Q000188:drug therapy; D008297:Male; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity; D012720:Severity of Illness Index; D016896:Treatment Outcome
D005938:Glucocorticoids
Disease Activity; Epidemiology; Systemic Lupus Erythematosus