A total of four studies were included in this review (data taken from multiple publications) with a combined total n of 195. One study was an RCT of good quality, two studies were uncontrolled prospective open label before/after comparisons and the final study was a mixture of retrospective and prospective data that included patients from the three previous studies.
Overall eculizumab reduced transfusion requirements and anaemia, and reduced haemolysis according to an RCT (n=87; p<0.001)) and an uncontrolled study (n=97). The RCT also reported mean haemoglobin levels as significantly decreasing in the placebo group during the trial despite receiving more units of transfused red blood cells (p<0.001). Quality of life was improved following treatment with eculizumab, particularly with regard to fatigue according to a comparison of the results from the RCT and uncontrolled studies with results from an open-label study of cancer patients. The authors of the primary studies reported the findings from a pooled analysis of three published studies and suggested the rate of thromboembolism decreases up to seven-fold in patients treated with eculizumab.
The most common adverse events were headache, upper respiratory tract infection and viral infection; no patients withdrew due to side effects.