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. 2019 Feb 26;39(Suppl 1):1–151. doi: 10.1007/s10875-019-00597-5

All Patients

(N=34)

Patients Failing Conventional Therapy (n=27)
ORR, n/N (%) 22/34 (65)* 17/27 (63)**
Time to response, days (median [95% CI]) 8.0 [5.0-10.0] 8.0 [7.0-14.0]
Cumulative duration of response, % days of treatment (median [Q1, Q3]) 75.7 [33.3-91.2] 75.4 [30.8-91.2]
Proceeded to HSCT, n/N (%) 22/34 (65) 19/27 (70)
Overall Survival
 Alive at last observations, n/N (%) 24/34 (70.6) 20/27 (74.1)
 12-month pOS estimate (% [95% CI]) 69 [50-82] 73 [52-86]
 Survival to HSCT, n/N (%)*** 27/34 (79.4) 22/27 (81.5)
 Survival post HSCT, n/N (%) 20/22 (90.9) 17/19 (89.5)