Table 3.

Treatment with C5i

Reason for commencing C5iNumber (%)
Intravascular hemolysis 452 (88.8%) 
Other PNH complication 13 (2.6%) 
Allogeneic bone marrow transplant 13 (2.6%) 
Pregnancy 19 (3.7%) 
Exceptional case 12 (2.4%) 
Treatment on C5i  
Median PNH granulocyte clone size at initiation of C5i 85.5% (range 12%-100%) 
Median PNH erythrocyte clone size at initiation of C5i 28.% (range 0%-100%) 
Death 91 (17.9%) 
Thromboses 23 (4.5%) 
Neisseria meningitidis infection 11 (2.2%) 
Clonal evolution to MDS or AML 14 (2.8%) 
Mean hemoglobin level 12 months after starting C5i  107.7g/l 
Mean hemoglobin level 24 months after starting C5i§  108.6g/l 
Requiring transfusions in the most recent 12 months on C5i  123 (26.7%) 
Normalization of hemoglobin level at 12 months  86 (20.4%) 
Normalization of hemoglobin level at 24 months§  76 (20.3%) 
Discontinuation of C5i because of disease remission 20 (3.9%) 
Reason for commencing C5iNumber (%)
Intravascular hemolysis 452 (88.8%) 
Other PNH complication 13 (2.6%) 
Allogeneic bone marrow transplant 13 (2.6%) 
Pregnancy 19 (3.7%) 
Exceptional case 12 (2.4%) 
Treatment on C5i  
Median PNH granulocyte clone size at initiation of C5i 85.5% (range 12%-100%) 
Median PNH erythrocyte clone size at initiation of C5i 28.% (range 0%-100%) 
Death 91 (17.9%) 
Thromboses 23 (4.5%) 
Neisseria meningitidis infection 11 (2.2%) 
Clonal evolution to MDS or AML 14 (2.8%) 
Mean hemoglobin level 12 months after starting C5i  107.7g/l 
Mean hemoglobin level 24 months after starting C5i§  108.6g/l 
Requiring transfusions in the most recent 12 months on C5i  123 (26.7%) 
Normalization of hemoglobin level at 12 months  86 (20.4%) 
Normalization of hemoglobin level at 24 months§  76 (20.3%) 
Discontinuation of C5i because of disease remission 20 (3.9%) 

Interquartile range, 67% to 95%; available for 494 cases.

Interquartile range, 15% to 46%; available for 488 cases.

Available for 421 patients.

§

Available for 374 patients.

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