Performance of the 2016 ACR-EULAR myositis response criteria in juvenile dermatomyositis therapeutic trials and consensus profiles

Hanna Kim, Didem Saygin, Christian Douglas, Jesse Wilkerson, Brian Erman, Angela Pistorio, John A. McGrath, Ann M. Reed, Chester V. Oddis, Claudia Bracaglia, Annet Van Royen-Kerkhof, Blanca Bica, Pavla Dolezalova, Virginia P.L. Ferriani, Berit Flato, Ana G. Bernard-Medina, Troels Herlin, Frederick W. Miller, Jiri Vencovsky, Nicolino RupertoRohit Aggarwal, Lisa G. Rider*, Iago Pinal-Fernandez, Susan Kim, Dana Ascherman, Adam Schiffenbauer

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)3680-3689
Number of pages10
JournalRheumatology (United Kingdom)
Volume62
Issue number11
DOIs
Publication statusPublished - 1 Nov 2023

Keywords

  • clinical trial
  • JDM
  • myositis
  • outcome assessment
  • outcome measure
  • response criteria

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