Table 2.

Pharmacologic treatment options for relapsed or refractory cold agglutinin disease from case series

TreatmentInitial doseOR, %Median time To response (range)Median response duration (range)Relapse rate (at 1-2 y), %CommentsReference
Chlorambucil 4-20 mg orally once a day 16-46 NA 11 mo NA Included newly diagnosed and previously treated patients (N = 19-37) 64, 65 
Cyclophosphamide 50-150 mg orally once a day 
Rituximab 375 mg/m2 IV every week × 4 wk 45-54 1.5 mo (1-2) 10 mo (8-27) 50-83 Included newly diagnosed and previously treated patients (N = 20-32) 64, 67, 68 
Rituximab + prednisone Rituximab: 100 mg IV every week × 4 wk; Prednisone: 1 mg/kg per day orally × 30 d, then taper 56 2 wk NA 33 Included newly diagnosed and previously treated patients (N = 19) 35 
Rituximab + fludarabine Rituximab: 375 mg/m2 IV every 4 wk; Fludarabine: 40 mg/m2 orally on days 1-5 every 4 wk; both × 4 cycles 76 4 mo >66 mo (3-66) 23 Grade 3-4 hematologic toxicities in 41% (N = 29) 73 
TreatmentInitial doseOR, %Median time To response (range)Median response duration (range)Relapse rate (at 1-2 y), %CommentsReference
Chlorambucil 4-20 mg orally once a day 16-46 NA 11 mo NA Included newly diagnosed and previously treated patients (N = 19-37) 64, 65 
Cyclophosphamide 50-150 mg orally once a day 
Rituximab 375 mg/m2 IV every week × 4 wk 45-54 1.5 mo (1-2) 10 mo (8-27) 50-83 Included newly diagnosed and previously treated patients (N = 20-32) 64, 67, 68 
Rituximab + prednisone Rituximab: 100 mg IV every week × 4 wk; Prednisone: 1 mg/kg per day orally × 30 d, then taper 56 2 wk NA 33 Included newly diagnosed and previously treated patients (N = 19) 35 
Rituximab + fludarabine Rituximab: 375 mg/m2 IV every 4 wk; Fludarabine: 40 mg/m2 orally on days 1-5 every 4 wk; both × 4 cycles 76 4 mo >66 mo (3-66) 23 Grade 3-4 hematologic toxicities in 41% (N = 29) 73 

NA, not available.

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