TY - JOUR
T1 - Performance of the 2016 ACR-EULAR myositis response criteria in juvenile dermatomyositis therapeutic trials and consensus profiles
AU - Kim, Hanna
AU - Saygin, Didem
AU - Douglas, Christian
AU - Wilkerson, Jesse
AU - Erman, Brian
AU - Pistorio, Angela
AU - McGrath, John A.
AU - Reed, Ann M.
AU - Oddis, Chester V.
AU - Bracaglia, Claudia
AU - Van Royen-Kerkhof, Annet
AU - Bica, Blanca
AU - Dolezalova, Pavla
AU - Ferriani, Virginia P.L.
AU - Flato, Berit
AU - Bernard-Medina, Ana G.
AU - Herlin, Troels
AU - Miller, Frederick W.
AU - Vencovsky, Jiri
AU - Ruperto, Nicolino
AU - Aggarwal, Rohit
AU - Rider, Lisa G.
AU - Pinal-Fernandez, Iago
AU - Kim, Susan
AU - Ascherman, Dana
AU - Schiffenbauer, Adam
N1 - Publisher Copyright:
© 2023 Published by Oxford University Press on behalf of the British Society for Rheumatology.
PY - 2023/11/1
Y1 - 2023/11/1
N2 - Objectives: The 2016 ACR-EULAR Response Criteria for JDM was developed as a composite measure with differential weights of six core set measures (CSMs) to calculate a Total Improvement Score (TIS). We assessed the contribution of each CSM, representation of muscle-related and patient-reported CSMs towards improvement, and frequency of CSM worsening across myositis response criteria (MRC) categories in validation of MRC. Methods: Data from JDM patients in the Rituximab in Myositis trial (n = 48), PRINTO JDM trial (n = 139), and consensus patient profiles (n = 273) were included. Observed vs expected CSM contributions were compared using Sign test. Characteristics of MRC categories were compared by Wilcoxon tests with Bonferroni adjustment. Spearman correlation of changes in TIS and individual CSMs were examined. Agreement between physician-Assessed change and MRC categories was evaluated by weighted Cohen's kappa. Results: Of 457 JDM patients with IMACS CSMs and 380 with PRINTO CSMs, 9-13% had minimal, 19-23% had moderate and 41-50% had major improvement. The number of improved and absolute percentage change of CSMs increased by MRC improvement level. Patients with minimal improvement by MRC had a median of 0-1 CSM worsened, and those with moderate/major improvement had a median of zero worsening CSMs. Of patients improved by MRC, 94-95% had improvement in muscle strength and 93-95% had improvement in ≥1 patient-reported CSM. IMACS and PRINTO CSMs performed similarly. Physician-rated change and MRC improvement categories had moderate-To-substantial agreement (Kappa 0.5-0.7). Conclusion: The ACR-EULAR MRC perform consistently across multiple studies, supporting its further use as an efficacy end point in JDM trials.
AB - Objectives: The 2016 ACR-EULAR Response Criteria for JDM was developed as a composite measure with differential weights of six core set measures (CSMs) to calculate a Total Improvement Score (TIS). We assessed the contribution of each CSM, representation of muscle-related and patient-reported CSMs towards improvement, and frequency of CSM worsening across myositis response criteria (MRC) categories in validation of MRC. Methods: Data from JDM patients in the Rituximab in Myositis trial (n = 48), PRINTO JDM trial (n = 139), and consensus patient profiles (n = 273) were included. Observed vs expected CSM contributions were compared using Sign test. Characteristics of MRC categories were compared by Wilcoxon tests with Bonferroni adjustment. Spearman correlation of changes in TIS and individual CSMs were examined. Agreement between physician-Assessed change and MRC categories was evaluated by weighted Cohen's kappa. Results: Of 457 JDM patients with IMACS CSMs and 380 with PRINTO CSMs, 9-13% had minimal, 19-23% had moderate and 41-50% had major improvement. The number of improved and absolute percentage change of CSMs increased by MRC improvement level. Patients with minimal improvement by MRC had a median of 0-1 CSM worsened, and those with moderate/major improvement had a median of zero worsening CSMs. Of patients improved by MRC, 94-95% had improvement in muscle strength and 93-95% had improvement in ≥1 patient-reported CSM. IMACS and PRINTO CSMs performed similarly. Physician-rated change and MRC improvement categories had moderate-To-substantial agreement (Kappa 0.5-0.7). Conclusion: The ACR-EULAR MRC perform consistently across multiple studies, supporting its further use as an efficacy end point in JDM trials.
KW - clinical trial
KW - JDM
KW - myositis
KW - outcome assessment
KW - outcome measure
KW - response criteria
UR - http://www.scopus.com/inward/record.url?scp=85176495178&partnerID=8YFLogxK
U2 - 10.1093/rheumatology/kead111
DO - 10.1093/rheumatology/kead111
M3 - Article
C2 - 36929918
AN - SCOPUS:85176495178
SN - 1462-0324
VL - 62
SP - 3680
EP - 3689
JO - Rheumatology (United Kingdom)
JF - Rheumatology (United Kingdom)
IS - 11
ER -