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.