Variability in management and outcomes of therapy with eculizumab in atypical hemolytic uremic syndrome

ABSTRACT Objectives: Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy caused by complement dysregulation. The aim of this study was to establish the efficacy and safety of eculizumab in patients with aHUS in clinical practice and to describe different individualization strategies. Methods: Authors performed an observational, longitudinal, and ambispective study at a tertiary care center. Clinical histories of patients in treatment with eculizumab were reviewed. Effectiveness and safety were assessed with the evolution of analytical parameters, symptoms and concomitant therapies required. Results: Authors included five patients (two children). The patients were followed up from diagnosis and first administration of eculizumab. Four patients discontinued eculizumab: one because he had anti-factor H autoantibodies that could be managed with immunosuppressive therapy, another because of non-response, and the other two because of clinical stabilization, resolution of TMA, and no findings of high-risk mutations in complement factors. Therapy was tapered in the remaining patients in aHUS remission. No adverse events were identified during or after treatment. Conclusion: Eculizumab is an effective and safe treatment for patients diagnosed with primary or secondary aHUS. Personalized treatment, tapering or discontinuation should be taken on an individual basis by a multidisciplinary team in order to increase the cost-effectiveness of this therapy.