Table 2

Table summarizing the response, OS, relapse, and clonal evolution in refractory AA patients treated with alemtuzumab-based IST

ReferenceStudy designIndicationNAlemtuzumab regimenCSAResponseOSRelapseClonal evolution
Scheinberg et al18  Prospective randomized Refractory to hATG + CSA 27 10 mg/kg/d × 10 IV None 37% 83% at 3 y 1 (−7) 
Risitano et al19  Prospective phase 2 Untreated (n = 6); refractory (n = 5) 11 3, 10, 30, 30 and 30 mg/d SC (total dose 103 mg) 1 mg/kg/d 8/11 (73%) (4/6 untreated) 73% 
       
Retrospective Refractory   3/8 (37%)    
Kim et al20  Prospective dose escalating Untreated (n = 13) Refractory to ATG + CSA/danazol (n = 4) 17 Cohort I: 10, 20, 30 mg IV Cohort II: 30 mg/d × 3 IV 3 mg/kg/d 4/13 (31%) 2/4 (50%) 81.6% at 2 y 1 (MDS) 
ReferenceStudy designIndicationNAlemtuzumab regimenCSAResponseOSRelapseClonal evolution
Scheinberg et al18  Prospective randomized Refractory to hATG + CSA 27 10 mg/kg/d × 10 IV None 37% 83% at 3 y 1 (−7) 
Risitano et al19  Prospective phase 2 Untreated (n = 6); refractory (n = 5) 11 3, 10, 30, 30 and 30 mg/d SC (total dose 103 mg) 1 mg/kg/d 8/11 (73%) (4/6 untreated) 73% 
       
Retrospective Refractory   3/8 (37%)    
Kim et al20  Prospective dose escalating Untreated (n = 13) Refractory to ATG + CSA/danazol (n = 4) 17 Cohort I: 10, 20, 30 mg IV Cohort II: 30 mg/d × 3 IV 3 mg/kg/d 4/13 (31%) 2/4 (50%) 81.6% at 2 y 1 (MDS) 

hATG indicates horse ATG.

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