{"analyses":[{"groupIds":["OG000"],"nonInferiorityComment":"testing the null hypothesis that ORR within 3 months is less than or equal to 20%","nonInferiorityType":"OTHER","pValue":"<.0001","statisticalMethod":"Clopper-Pearson"}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","lowerLimit":"72.1","upperLimit":"90.0","value":"82.3"},{"comment":"NA: Confidence Interval could not be calculated for only 1 participant.","groupId":"OG001","lowerLimit":"NA","upperLimit":"NA","value":"100.0"}]}]}],"denoms":[{"counts":[{"groupId":"OG000","value":"79"},{"groupId":"OG001","value":"1"}],"units":"Participants"}],"description":"Evaluating the efficacy of tisagenlecleucel therapy from all manufacturing facilities as measured by overall remission rate (ORR) during the 3 months after tisagenlecleucel administration. ORR included complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by an Independent Review Committee assessment. Per response criteria defined by National Comprehensive Cancer Network (NCCN), American Society of Hematology (ASH) and International Working Group (IWG) guidelines.\n\nCR is defined as:\n\nBone marrow \\<5% blasts, Peripheral blood: Neutrophils \\>1.0 x 10\\^9/L, and Platelets \\>100 x 10\\^9/L and Circulating blasts \\<1% and No evidence of extramedullary disease, at least 7 days transfusion independency.\n\nCRi is defined as all the prior criteria being met, except that the following exists: Neutrophils ≤1.0 x 10\\^9/L, and/or Platelets ≤100 x 10\\^9/L, and/or Platelet and/or neutrophil transfusions ≤7 days before the date of the peripheral blood sample for disease assessment.","dispersionType":"95% Confidence Interval","groups":[{"description":"Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).","id":"OG000","title":"Main Cohort: Single Dose of CTL019"},{"description":"Pediatric B-ALL patients who are very high risk at first relapse.","id":"OG001","title":"Cohort 1: Single Dose of CTL019"}],"paramType":"NUMBER","populationDescription":"Full Analysis Set (FAS): The Full analysis set comprised all patients who received infusion of tisagenlecleucel in the Main Cohort.","reportingStatus":"POSTED","timeFrame":"during the 3 months after tisagenlecleucel administration","title":"Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment.","type":"PRIMARY","unitOfMeasure":"Percentage of participants"}