Sustained Improvements in Transfusion Requirements, Fatigue and Thrombosis with Eculizumab Treatment in Paroxysmal Nocturnal Hemoglobinuria.
Gerard Socié, Peter Hillmen, Petra Muus, Jörg Schubert, Ulrich Dührsen, Antonio M Risitano, Russell P Rother, Robert A Brodsky, Jeffrey Szer
Blood | American Society of Hematology | Published : 2007
Abstract The long-term safety and efficacy of eculizumab (Soliris™) were examined in 187 patients with PNH initially enrolled in one of 3 parent trials (N=195) who continued to receive eculizumab in an extension trial for a median of 22 mos. All but 2 patients (99%) who enrolled in the extension trial (Phase 2 Pilot, Phase 3 TRIUMPH and SHEPHERD studies; N=187) were fully blocked at the prescribed dose (900 mg every 14±2 days); adjustment to 1200 mg every 14 days maintained complete complement blockade in the other 2 patients. Clinical benefits of eculizumab were sustained throughout the treatment period. Hemolysis (assessed by LDH) was reduced from a median of 2165 U/L at bas..View full abstract