TY - JOUR
T1 - American College of Rheumatology Provisional Criteria for Clinically Relevant Improvement in Children and Adolescents With Childhood-Onset Systemic Lupus Erythematosus
AU - Paediatric Rheumatology International Trial Organisation and Pediatric Rheumatology Collaborative Study Group
AU - Brunner, Hermine I
AU - Holland, Michael J
AU - Beresford, Michael W
AU - Ardoin, Stacy P
AU - Appenzeller, Simone
AU - Silva, Clovis A
AU - Flores, Francisco
AU - Goilav, Beatrice
AU - Avar Aydin, Pinar Ozge
AU - Wenderfer, Scott E
AU - Levy, Deborah M
AU - Ravelli, Angelo
AU - Khubchandani, Raju
AU - Avcin, Tadej
AU - Klein-Gitelman, Marisa S
AU - Ruperto, Nicolino
AU - Feldman, Brian M
AU - Ying, Jun
AU - Study group members AMC, null
AU - Schonenberg-Meinema, Dieneke
N1 - © 2019, American College of Rheumatology.
PY - 2019/5
Y1 - 2019/5
N2 - OBJECTIVE: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRIcSLE ).METHODS: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRIcSLE and rate a total of 433 unique patient profiles for the presence/absence of CRIcSLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0-1).RESULTS: During an international consensus conference, unanimous agreement on a definition of CRIcSLE was achieved; cSLE experts (n = 13) concurred (100%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0-100, a CHILI score of ≥54 had outstanding accuracy for identifying CRIcSLE (AUC 0.93, sensitivity 81.1%, and specificity 84.2%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1%, and specificity ≥73.4%).CONCLUSION: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.
AB - OBJECTIVE: To develop a Childhood Lupus Improvement Index (CHILI) as a tool to measure response to therapy in childhood-onset systemic lupus erythematosus (cSLE), with a focus on clinically relevant improvement (CRIcSLE ).METHODS: Pediatric nephrology and rheumatology subspecialists (n = 213) experienced in cSLE management were invited to define CRIcSLE and rate a total of 433 unique patient profiles for the presence/absence of CRIcSLE . Patient profiles included the following cSLE core response variables (CRVs): global assessment of patient well-being (patient-global), physician assessment of cSLE activity (MD-global), disease activity index score (here, we used the Systemic Lupus Erythematosus Disease Activity Index), urine protein-to-creatinine ratio, and Child Health Questionnaire physical summary score. Percentage and absolute changes in these cSLE-CRVs (baseline versus follow-up) were considered in order to develop candidate algorithms and validate their performance (sensitivity, specificity, area under the receiver operating characteristic curve [AUC]; range 0-1).RESULTS: During an international consensus conference, unanimous agreement on a definition of CRIcSLE was achieved; cSLE experts (n = 13) concurred (100%) that the preferred CHILI algorithm considers absolute changes in the cSLE-CRVs. After transformation to a range of 0-100, a CHILI score of ≥54 had outstanding accuracy for identifying CRIcSLE (AUC 0.93, sensitivity 81.1%, and specificity 84.2%). CHILI scores also reflect minor, moderate, and major improvement for values exceeding 15, 68, and 92, respectively (all AUC ≥0.92, sensitivity ≥93.1%, and specificity ≥73.4%).CONCLUSION: The CHILI is a new, seemingly highly accurate index for measuring CRI in cSLE over time. This index is useful to categorize the degree of response to therapy in children and adolescents with cSLE.
KW - Adolescent
KW - Algorithms
KW - Antirheumatic Agents/therapeutic use
KW - Child
KW - Delphi Technique
KW - Humans
KW - Lupus Erythematosus, Systemic/drug therapy
KW - Outcome Assessment, Health Care/methods
KW - Severity of Illness Index
U2 - https://doi.org/10.1002/acr.23834
DO - https://doi.org/10.1002/acr.23834
M3 - Article
C2 - 30680946
SN - 2151-464X
VL - 71
SP - 579
EP - 590
JO - Arthritis care & research
JF - Arthritis care & research
IS - 5
ER -