Patient disposition. ∗Three patients (R/R SAA, n = 2; treatment-naïve SAA, n = 1) who discontinued the treatment phase early entered the 52-week follow-up period. ^†As of data cutoff, 4 May 2023, 15 patients were receiving ongoing treatment, 9 patients discontinued (the reasons for discontinuation included AE [n = 1], physician decision [n = 3], patient/guardian decision [n = 3], and progressive disease [n = 2]), and 4 patients completed 3-year long-term follow-up.