Table 2. Studies demonstrating a... | American Society of Hematology

Table 2.

Studies demonstrating a reduction in thrombotic events in PNH and aHUS

First author/ reference	Disease	Rate of TE under best supportive therapy (number of patients)	Rate of TE under complement inhibition by eculizumab (number of patients)	Comment
Hillman³⁸	PNH prevalence: ∼13 per million¹⁴⁹	7.37 events/ 100 patient-years (195) 10.61 events/100 patient-years on antithrombotic therapy (103)	1.07 events/100 patient-years (195) 0.62 events/100 patient-years on antithrombotic therapy (103)	• Although TEs under eculizumab therapy are significantly reduced they may still occur • Overall, a similar positive response is expected for other C5 (or other appropriate complement) inhibitors in clinical trials • Percentage of patients with thrombosis correlates with PNH clone size or LDH levels as marker for intravascular hemolysis^33,150
Kelly⁴⁰		5.6 events per 100 patient-years (79)	0.8 events per 100 patient-years (eculizumab)
Loschi¹⁵¹		27% of patients (191)	4% of patients (123)
Legendre¹⁵²	aHUS prevalence: ∼5 per million¹⁵³	100% since diagnosis of TMA is per se a thrombotic event (37 of 37 patients)	19% (7 of 37) did not reach complete TMA-free statues during 62-64 wk of treatment	• Similar response is expected for other C5 inhibitors in clinical trials

First author/ reference	Disease	Rate of TE under best supportive therapy (number of patients)	Rate of TE under complement inhibition by eculizumab (number of patients)	Comment
Hillman³⁸	PNH prevalence: ∼13 per million¹⁴⁹	7.37 events/ 100 patient-years (195) 10.61 events/100 patient-years on antithrombotic therapy (103)	1.07 events/100 patient-years (195) 0.62 events/100 patient-years on antithrombotic therapy (103)	• Although TEs under eculizumab therapy are significantly reduced they may still occur • Overall, a similar positive response is expected for other C5 (or other appropriate complement) inhibitors in clinical trials • Percentage of patients with thrombosis correlates with PNH clone size or LDH levels as marker for intravascular hemolysis^33,150
Kelly⁴⁰		5.6 events per 100 patient-years (79)	0.8 events per 100 patient-years (eculizumab)
Loschi¹⁵¹		27% of patients (191)	4% of patients (123)
Legendre¹⁵²	aHUS prevalence: ∼5 per million¹⁵³	100% since diagnosis of TMA is per se a thrombotic event (37 of 37 patients)	19% (7 of 37) did not reach complete TMA-free statues during 62-64 wk of treatment	• Similar response is expected for other C5 inhibitors in clinical trials

TE, thromboembolism.