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. Author manuscript; available in PMC: 2018 May 1.
Published in final edited form as: Arthritis Rheumatol. 2017 Apr 6;69(5):898–910. doi: 10.1002/art.40064

Table 2. Detailed performance characteristics of patient profiles for the top five candidate definition presented at the consensus conference.

Improvement category Candidate definition for response criteria Candidate definition type based on final consensus rank order Core set measures Sensitivity (%) Specificity (%) Threshold AUC‡ Total Improvement Score AUC§ PRINTO trial* RIM trial† Rank
Tx (%) Ctrl (%) P value Response criteria, improved‖ Response criteria, not improved‖ P value
Minimal Improvement Score ≥ 30 Conjoint analysis, absolute percent change Model 3 (Table 3) IMACS 89 91 0.90 0.98 75 53 0.009 2.0 3.0 <0.001 1
PRINTO 89 98 0.93 0.99 73 55 0.038
Moderate Improvement Score ≥ 45 IMACS 92 99 0.95 0.99 70 53 0.057 2.0 3.0 <0.001
PRINTO 94 94 0.94 0.99 71 51 0.023
Major Improvement Score ≥ 70 IMACS 91 86 0.89 0.96 51 43 0.341 2.0 3.0 0.006
PRINTO 98 85 0.91 0.98 58 49 0.331
Minimal Improvement Score ≥ 33 Conjoint analysis, relative percent change# Model 1 (Supplementary Table 3) IMACS 99 87 0.93 0.98 75 55 0.018 2.0 4.0 <0.001 2
PRINTO 96 98 0.97 1.00 74 55 0.027
Moderate Improvement Score ≥ 60 IMACS 97 93 0.95 0.99 73 51 0.011 2.0 3.0 <0.001
PRINTO 97 96 0.96 1.00 70 51 0.032
Major Improvement Score ≥ 80 IMACS 91 87 0.89 0.96 57 49 0.396 1.5 3.0 <0.001
PRINTO 98 86 0.92 0.97 61 49 0.179
Minimal Improvement Score ≥ 33 Conjoint analysis, relative percent change# Model 2 (Supplementary Table 3) IMACS 95 94 0.94 0.98 75 53 0.009 2.0 4.0 <0.001 3
PRINTO 94 98 0.96 0.99 74 55 0.027
Moderate Improvement Score ≥ 55 IMACS 95 95 0.95 1.00 70 51 0.032 2.0 3.0 <0.001
PRINTO 97 98 0.98 1.00 70 51 0.032
Major Improvement Score ≥ 77 IMACS 93 86 0.90 0.97 49 47 0.814 1.0 2.0 0.011
PRINTO 96 90 0.93 0.99 59 49 0.273
Minimal Improvement Points given when CSM≥20; Worsening Points given when CSM worse by > 30 Weighted definition, relative percent change** IMACS 95 100 0.97 NA 70 51 0.032 2.0 3.0 <0.001 4
PRINTO 92 98 0.95 NA 73 53 0.021
Moderate Improvement Points given when CSM ≥ 50%; Worsening Points given when CSM worse by >30% IMACS 95 91 0.93 NA 68 51 0.045 2.0 3.0 <0.001
PRINTO 95 92 0.94 NA 71 51 0.023
Major Improvement Points given when CSM≥75%; Worsening Points given when CSM worse by >30% IMACS 100 81 0.91 NA 64 47 0.050 1.5 3.0 <0.001
PRINTO 98 85 0.91 NA 62 49 0.142
Minimal 3 of any 6 improved by ≥20%; no more than 1 worse by > 30%; which cannot be MMT/CMAS (8) Previously published definition (8;9), relative percent change IMACS 93 100 0.97 NA 70 51 0.032 2.0 3.0 <0.001 5
PRINTO 88 100 0.94 NA 71 51 0.023
Moderate 3 of any 6 improved by ≥ 50%; no more than 1 worse by > 30%; which cannot be MMT/CMAS (9) IMACS 90 95 0.93 NA 66 51 0.081 2.0 3.0 <0.001
PRINTO 90 96 0.93 NA 68 51 0.045
Major 3 of any 6 improved by ≥ 70%; no more than 1 worse by > 30%; which cannot be MMT/CMAS (9) IMACS 99 83 0.91 NA 63 49 0.111 2.0 3.0 <0.001
PRINTO 99 89 0.94 NA 60 49 0.223

The performance characteristics of patient profiles for definitions ranked 6-14 are presented in Supplementary Table 1.

Abbreviations: AUC, area under the curve; Minimal, minimal improvement; Moderate, moderate improvement; Major, major improvement; IMACS, International Myositis Assessment and Clinical Studies Group; PRINTO, Paediatric Rheumatology International Trials Organization; CSM, core set measure; NA, not applicable; MMT, manual muscle testing; CMAS, Childhood Myositis Assessment Scale.

Note that either IMACS or PRINTO CSM may be used in these candidate definitions of response; the candidate definitions were developed in parallel with IMACS or PRINTO CSM.

*

PRINTO juvenile dermatomyositis trial of prednisone alone versus prednisone with methotrexate or cyclosporine (n = 139) (11).

†

Rituximab in Myositis (RIM) Trial, juvenile dermatomyositis arm (n =48). Comparison of the treating physician's rating of improvement if the improvement criteria are met versus not at week 24 (19). A 1-point difference in physician rating of improvement from no improvement to minimal improvement was considered not just statistically significant, but also was clinically significant.

‡

Threshold AUC, area under the curve, calculated as the AUC from the receiver operating characteristic curve for the Total Improvement Score and the threshold for minimal, moderate, and major improvement.

§

Total Improvement Score AUC, calculated as the AUC from the receiver operator characteristic curve, using the Total Improvement Score and the threshold cutoffs for minimal, moderate, and major improvement, which applies only to continuous definitions.

‖

Median Physician Improvement Score.

Conjoint analysis–based continuous candidate response criteria using absolute percent change in core set measures (absolute percent change model) is presented in Table 3. These criteria are also the top response criteria for adult dermatomyositis/polymyositis, but with different thresholds in the Total Improvement Score for minimal, moderate, and major improvement (22).

#

Conjoint analysis–based continuous candidate definitions using relative percent change in core set measures are presented in Supplementary Table 3. These criteria are also the second and third choice criteria for adult dermatomyositis/polymyositis, but with different thresholds in the Total Improvement Score for minimal, moderate, and major improvement (22).

**

Improvement = at least 3.5 Improvement Points out of 10 Total Improvement Points, and no more than 1.5 Worsening Points, where Physician Global Activity = 2 points; Parent Global Activity = 1 point; MMT or CMAS = 3 points; CHAQ = 1.5 points, Extramuscular Global Activity or Disease Activity Score = 1.5 points, Enzyme or Physical Summary Score of the Child Health Questionnaire-Parent Form 50 = 1 point.