The open-label study of 198 patients, who have developed the syndrome during an outbreak in Germany, demonstrated significant and persistent progression in thrombotic microangiopathy (TMA) and setback of damaged organs.
University Hospital Hamburg-Eppendorf UKE, department of nephrology chairman, trial’s lead investigator, Rolf Stahl said patients showed a rapid response to eculizumab therapy in the study.
"Patients achieved a substantial improvement in systemic TMA and vital organ involvement as early as 8 weeks, which was sustained through 28 weeks," Stahl added.
At eighth week, a complete response (CR) or a partial response (PR) in TMA and improvement in affected vital organs was observed in 94% patients, which is the primary endpoint of the study.
The key secondary endpoints such as elevated clinical and statistical significance, efficacy at 28 weeks besides progress in hematologic, renal and neurologic functions were met in the trial.
Eculizumab 28-week data was consistent with interim data that reported substantial improvement with eculizumab therapy.
Headache, hypertension and alopecia were the common adverse events associated with the multi-center trial in which eculizumab was well-tolerated.
Alexion research and development head, executive vice president Stephen Squinto said, "STEC-HUS is an unpredictable, life-threatening disease with a high rate of long-term serious clinical sequelae and no approved treatment options that address the underlying pathogenesis of the disease."