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      The long-acting C5 inhibitor, Ravulizumab, is effective and safe in adult patients with atypical hemolytic uremic syndrome naïve to complement inhibitor treatment.

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          Ravulizumab is a long-acting C5 inhibitor engineered from eculizumab with increased elimination half-life, allowing an extended dosing interval from two to eight weeks. Here we evaluate the efficacy and safety of ravulizumab in adults with atypical hemolytic uremic syndrome presenting with thrombotic microangiopathy. In this global, phase 3, single arm study in complement inhibitor-naïve adults (18 years and older) who fulfilled diagnostic criteria for atypical hemolytic uremic syndrome, enrolled patients received ravulizumab through a 26-week initial evaluation period. The primary endpoint was complete thrombotic microangiopathy response defined as normalization of platelet count and lactate dehydrogenase and 25% or more improvement in serum creatinine. Secondary endpoints included changes in hematologic variables and renal function. Safety was also evaluated. Ravulizumab treatment resulted in an immediate, complete, and sustained C5 inhibition in all patients. Complete thrombotic microangiopathy response was achieved in 53.6% of patients. Normalization of platelet count, lactate dehydrogenase and 25% or more improvement in serum creatinine was achieved in 83.9%, 76.8% and 58.9% of patients, respectively. Improvement in estimated glomerular filtration rate by one or more stage was achieved in 68.1% of patients by day 183. No unexpected adverse events were reported across a safety analysis set of 58 patients. Four deaths occurred (three within one month of study initiation, including one in a patient excluded based on eligibility criteria after the first dose) with none considered treatment-related by the study investigator. Thus, treatment with ravulizumab once every eight weeks resulted in rapidly improved hematologic and renal endpoints with no unexpected adverse events in adults with atypical hemolytic uremic syndrome.

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          Author and article information

          Kidney Int.
          Kidney international
          Elsevier BV
          Jun 2020
          : 97
          : 6
          [1 ] Intensive Care Nephrology and Transplantation Department, Assistance Publique, Hôpitaux de Paris 6, Sorbonne Université, Paris, France. Electronic address: eric.rondeau@aphp.fr.
          [2 ] Department of Haematology, University College London Hospitals (UCLH) and Cardio-metabolic Programme, National Institute for Health Research UCLH/UC Biomedical Research Centre, London, UK.
          [3 ] Paediatric Nephrology Department, University Hospital Vall d'Hebron, Barcelona, Spain.
          [4 ] Kidney Care, Royal Melbourne Hospital, Melbourne, Australia.
          [5 ] Division of Hematology, The Ohio State University Medical Center, Columbus, Ohio, USA.
          [6 ] Department of Kidney and Hypertension Diseases, University Hospital Tübingen, Tübingen, Germany.
          [7 ] Department of General Internal Medicine, Saitama Medical University, Iruma, Japan.
          [8 ] Clinical Pharmacology, Alexion Pharmaceutical, Inc., Boston, Massachusetts, USA.
          [9 ] Clinical Development, Alexion Pharmaceutical, Inc., Boston, Massachusetts, USA.
          [10 ] Biostatistics, Alexion Pharmaceutical, Inc., Boston, Massachusetts, USA.
          [11 ] Department of Internal Medicine, Seoul National University Hospital, Seoul, Republic of Korea.
          [12 ] National Renal Complement Therapeutics Centre, Royal Victoria Hospital, Newcastle University, Newcastle upon Tyne, UK.
          [13 ] Department of Nephrology and Hypertension, Hannover Medical School, Hannover, Germany.


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