Efficacy results of prospective studies C08-002 and C08-003 by week 26
| Outcomes | C08-002 (n=17) | C08-003 (n=20) |
|---|---|---|
| Change in platelet count from baseline through | 73 (40 to 105) | 5 (−17.5 to 28) |
| Week 26 (×109/L), point estimate (95% CI) | p=0.0001 | p=0.64 |
| Normalisation of platelet count | ||
| All patients, n (%) (95% CI) | 14 (82) (57 to 96) | 18 (90) (68 to 99) |
| Patients with abnormal baseline, n (%) | 13/15 (87%) | 3/20 (15%) |
| TMA event-free status*, n (%) (95% CI) | 15 (88) (64 to 99) | 16 (80) (56 to 94) |
| TMA intervention rate† | ||
| Daily pre-eculizumab rate, median (minimum, maximum) | 0.88 (0.04, 1.59) | 0.23 (0.05, 1.09) |
| Daily posteculizumab rate, median (minimum, maximum) | 0 (0, 0.31) | 0 |
| p Value | p<0.0001 | p<0.0001 |
| CKD improvement by ≥1 stage, n (%) (95% CI) | 10 (59) (33 to 82) | 7 (35) (15 to 59) |
| eGFR change mL/min/1.73 m2, median (range) at 26 weeks | 20 (−1 to 98) | 5 (−1 to 20) |
| eGFR improvement ≥15 mL/min/1.73 m2, n (%) (95% CI) | 9 (53) (28 to 77) | 1 (5) (0 to 25) |
| Change in Hb>20 g/L, n (%) (95% CI) | 11 (65) (38 to 86) | 9 (45) (23 to 68) |
| Haematological normalisation‡, n (%) (95% CI) | 13 (76) (50 to 93) | 18 (90) (68 to 99) |
| Complete TMA response§, n (%) (95% CI) | 11 (65) (38 to 86) | 5 (25) (9 to 49) |
| Death | 0 | 0 |
*TMA event-free status is ≥12 weeks of stable platelet count, no PE/PI and no new dialysis.
†TMA intervention rate is the number of PE or PI interventions and number of new dialyses required per patient per day.
‡Haematological normalisation is the normal platelet and lactate dehydrogenase levels, ≥2 consecutive measurements and ≥4 weeks apart.
§Complete TMA response is haematological normalisation plus at least a 25% reduction in serum creatinine for a minimum of 4 weeks.
Source: Electronic Medicines Compendium.31
CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; Hb, haemoglobin; PE, plasma exchange; PI, plasma infusion; TMA, thrombotic microangiopathy.