Table 3.

Novel agents for relapsed or refractory IP-LBCL

Study design (no. of patients)Response rateLong-term efficacyReference
BTK inhibitors     
Ibrutinib Phase 1 (N = 13) ORR = 77%, CR = 38% Median PFS = 4.6 mo 102  
 Phase 2 (N = 52) ORR = 52%, CR = 19% Median PFS = 4.8 mo 103  
Tirabrutinib Phase 2 (N = 44) ORR = 64%, CR = 34% Median PFS = 2.9 mo 105  
Immunomodulatory agents     
Lenalidomide Phase 1 (N = 7) ORR = 86%, CR = 14% Not reported 110  
Lenalidomide with rituximab Phase 2 (N = 45) ORR = 36%, CR = 29% Median PFS = 7.8 mo 111  
Pomalidomide with dexamethasone Phase 1 (N = 25) ORR = 48%, CR = 32% Median PFS = 5.3 mo 112  
Immune checkpoint inhibitors     
Nivolumab Phase 2 PCNSL (N = 47) ORR = 6% Median PFS = 1.4 mo Clinicaltrials.gov [NCT02857426] 
 Phase 2 PTL (N = 19) ORR = 26% Median PFS = 1.7 mo Clinicaltrials.gov [NCT02857426] 
Pembrolizumab Phase 2 PCNSL (N = 50) ORR = 12% Median PFS = 2.6 mo 75  
CAR T-cell therapy     
Tisangenlecleucel Phase 1/2 (N = 12) ORR = 58%, CR = 50% Not reported 113  
Axicabtagene ciloleucel Phase 1 (N = 9) ORR = 86%, CR = 86% Not reported 114  
Study design (no. of patients)Response rateLong-term efficacyReference
BTK inhibitors     
Ibrutinib Phase 1 (N = 13) ORR = 77%, CR = 38% Median PFS = 4.6 mo 102  
 Phase 2 (N = 52) ORR = 52%, CR = 19% Median PFS = 4.8 mo 103  
Tirabrutinib Phase 2 (N = 44) ORR = 64%, CR = 34% Median PFS = 2.9 mo 105  
Immunomodulatory agents     
Lenalidomide Phase 1 (N = 7) ORR = 86%, CR = 14% Not reported 110  
Lenalidomide with rituximab Phase 2 (N = 45) ORR = 36%, CR = 29% Median PFS = 7.8 mo 111  
Pomalidomide with dexamethasone Phase 1 (N = 25) ORR = 48%, CR = 32% Median PFS = 5.3 mo 112  
Immune checkpoint inhibitors     
Nivolumab Phase 2 PCNSL (N = 47) ORR = 6% Median PFS = 1.4 mo Clinicaltrials.gov [NCT02857426] 
 Phase 2 PTL (N = 19) ORR = 26% Median PFS = 1.7 mo Clinicaltrials.gov [NCT02857426] 
Pembrolizumab Phase 2 PCNSL (N = 50) ORR = 12% Median PFS = 2.6 mo 75  
CAR T-cell therapy     
Tisangenlecleucel Phase 1/2 (N = 12) ORR = 58%, CR = 50% Not reported 113  
Axicabtagene ciloleucel Phase 1 (N = 9) ORR = 86%, CR = 86% Not reported 114  

CR, complete response rate; ORR, overall response rate.

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