Atypical Haemolytic Uremic Syndrome (aHUS) is a rare, genetic condition, caused by uncontrolled complement activation. This is a severe and life threatening disease characterized by thrombotic microangiopathic anemia with diffuse microvascular thrombosis leading to multiorgan ischemia and damage. To increase understanding of aHUS and provide data on the long-term safety and efficacy of eculizumab, a terminal complement inhibitor, the aHUS Registry (NCT01522183) has been established. Here, we describe the baseline demographics and characteristics of patients recruited in the first 18 months.
The aHUS registry is an observational, non-interventional, global study. Patients are eligible for inclusion into the registry if they have received a clinical diagnosis of aHUS. Patients can be enrolled irrespective of their age or management, and are not required to have an identified complement mutation or anti-complement factor antibodies. On enrolment, details relating to demographic and general medical history are collected, together with information on management. Data are then collected prospectively every 6 months thereafter. The data cut-off for this analysis was 18 September, 2013.
In March 2014, 427 patients were enrolled into the registry, 54 of which came from Italy. The Table shows the baseline characteristics. Many individuals have experienced prior renal damage. Around half the patients had received eculizumab (104/211 [49%]), and the mean (SD) age on starting this treatment was 25 (21) years.
Analyses of data from the aHUS Registry will help to advance our understanding of the history and progression of aHUS, as well as increase our knowledge on safety and efficacy of the use of eculizumab in such patients compared with other management. The global nature of the registry is intended to facilitate collection of a suitable volume of data relating to a rare disease, and physicians are encouraged to enrol eligible patients.
