Response rates. (A) ORR. (B) Best ORR. Full analysis set. Data cutoff was 4 May 2023. Response was assessed per NAPAAC (North American Pediatric Aplastic Anemia Consortium) criteria. Values displayed above the bars are the ORRs, along with 95% confidence interval (CI). ORR is defined as the proportion of patients achieving CR or PR. Best overall response is the proportion of patients with CR or PR at any time point up to data cutoff; 95% CI values were computed with the exact method of Clopper-Pearson. NR, nonresponder.