Clinical cohort statistical characteristics of MM status, QMG scores and MG-ADL scores.
Using the criteria described in the Methods, 69 subjects were defined as responsive, 11 non-responsive and 23 were not categorized. The purpose of this approach was to establish the most rigorous definition of treatment-response to increase the likelihood of discovering treatment-responsive biomarkers. Intermediate levels (1–40% improvement in ADL or QMG score) of response were excluded from the final analysis. Based on the initial review of outcome measures, a dichotomous categorization was developed to define responsive or non-responsive sub-cohorts as described in the Methods, and were used to inform predictive biomarker discovery. No differences were identified in subjects classified into any of the responsive categories based on age, gender, receipt of thymectomy, or prednisone treatment at baseline. Therefore, we were able to perform our analysis regardless of whether patients had received a thymectomy or were receiving corticosteroids at study entry. Three patients in the non-responsive group during the first 6 months of the study received intravenous immunoglobulin, one at month 1 and two at month 4. No one in the non-responsive group received plasma exchange in the first 6 months of the study.