Table 1

Eculizumab in aHUS not responding to PEX (n = 17)

Demographic and laboratory dataWeek 26Week 64
Data at presentation    
 Median age, y 28 (17-68)   
 Median platelets, ×109/L 118 (62-161)   
 Median creatinine, mg/dL 2.9 (1.4-8.9)   
 LDH (>ULN) 10/17 (59)   
Response criteria    
 Normalized platelet count  13/15 (87)* 13/15 (87)* 
 Decreased creatinine by 25%  11/17 (65) 13/17 (76) 
 Increased est. GFR ≥ 15 mL/min/1.73m2  8/17 (47) 9/17 (53) 
Demographic and laboratory dataWeek 26Week 64
Data at presentation    
 Median age, y 28 (17-68)   
 Median platelets, ×109/L 118 (62-161)   
 Median creatinine, mg/dL 2.9 (1.4-8.9)   
 LDH (>ULN) 10/17 (59)   
Response criteria    
 Normalized platelet count  13/15 (87)* 13/15 (87)* 
 Decreased creatinine by 25%  11/17 (65) 13/17 (76) 
 Increased est. GFR ≥ 15 mL/min/1.73m2  8/17 (47) 9/17 (53) 

The clinical data both at presentation and after therapy with eculizumab are presented for the 17 patients enrolled in the prospective study of eculizumab in patients with a progressive TMA after PEX therapy. Fifteen patients completed 26 weeks of therapy, with 13 patients continuing therapy beyond 26 weeks, and were available for follow-up after a median of 64 weeks of therapy.

GFR, glomerular filtration rate; ULN, upper limit of normal.

*

Two patients with normal platelet count at start of therapy not evaluable.

Four of 5 patients able to become independent of the need for dialysis after therapy.

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