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. 2021 Mar 10;26(4):288–e541. doi: 10.1002/onco.13712
Disease Multiple myeloma
Stage of Disease/Treatment Metastatic/advanced
Prior Therapy More than two prior regimens
Type of Study Phase II, single arm
Primary Endpoint Overall response rate
Secondary Endpoints Complete response rate, progression‐free survival, overall survival, tolerability
Additional Details of Endpoints or Study Design The statistical design incorporated a two‐stage Simon minimax design where the first stage would enroll 13 patients, and if one response occurred, the study would proceed to the second stage for a total enrollment of 27 patients. The study would be considered a success if four total responses occurred (14.7%), with an alpha of 5% and a power of 80%.
Response Definition: The 2014 International Myeloma Working Group (IMWG) response criteria for multiple myeloma was used. In brief, response criteria are as follows:
Stringent complete response (CR): CR with normalization of serum‐free light chains
CR: No detectable monoclonal protein by serum protein electrophoresis and immunofixation
Very good partial response (PR): 90% improvement in M‐protein
PR: 50% improvement in M‐protein
Minimal response: 25% improvement in M‐protein
Stable disease (SD): Neither PR nor progressive disease (PD)
PD: 25% increase in M‐protein
In terms of ORR, a PR or better is required. In cases with light chain–only disease, the difference between involved and uninvolved light chains is used to gauge response.
Investigator's Analysis Study prematurely terminated because of the COVID‐19 pandemic, with only modest activity in a small number of patients.