In addition to the authors, the following investigators participated in the TRIUMPH study, the list of who is provided in the Appendix.
Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria
Version of Record online: 28 JUN 2008
© 2008 The Authors. Journal Compilation © 2008 Blackwell Publishing Ltd
British Journal of Haematology
Volume 142, Issue 2, pages 263–272, July 2008
How to Cite
Schubert, J., Hillmen, P., Röth, A., Young, N. S., Elebute, M. O., Szer, J., Gianfaldoni, G., Socié, G., Browne, P., Geller, R., Rother, R. P. and Muus, P. (2008), Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria. British Journal of Haematology, 142: 263–272. doi: 10.1111/j.1365-2141.2008.07183.x
- Issue online: 28 JUN 2008
- Version of Record online: 28 JUN 2008
- Received 3 December 2007; accepted for publication 12 February 2008
- paroxysmal nocturnal haemoglobinuria;
In paroxysmal nocturnal haemoglobinuria (PNH), chronic destruction of PNH red blood cells (RBCs) by complement leads to anaemia and other serious morbidities. Eculizumab inhibits terminal complement-mediated PNH RBC destruction by targeting C5. In the phase III, double-blind, placebo-controlled, TRIUMPH study, eculizumab reduced haemolysis, stabilized haemoglobin levels, reduced transfusion requirements and improved fatigue in patients with PNH. Herein, we explored the effects of eculizumab on measures of anaemia in patients from the TRIUMPH study and the open-label SHEPHERD study, a more heterogeneous population. Eculizumab reduced haemolysis regardless of pretreatment transfusion requirements and regardless of whether or not patients became transfusion-dependent during treatment (P < 0·001). Reduction in haemolysis was associated with increased PNH RBC counts (P < 0·001) while reticulocyte counts remained elevated. Eculizumab-treated patients demonstrated significantly higher levels of haemoglobin as compared with placebo in TRIUMPH and relative to baseline levels in SHEPHERD (P < 0·001 for each study). Eculizumab lowered transfusion requirement across multiple pretreatment transfusion strata and eliminated transfusion support in a majority of both TRIUMPH and SHEPHERD patients (P < 0·001). Patients who required some transfusion support during treatment with eculizumab showed a reduction in haemolysis and transfusion requirements and an improvement in fatigue. Eculizumab reduces haemolysis and improves anaemia and fatigue, regardless of transfusion requirements.