%0 Journal Article
%T Ravulizumab in Atypical Hemolytic Uremic Syndrome: An Analysis of 2-Year Efficacy and Safety Outcomes in 2 Phase 3 Trials.
%A Dixon BP
%A Kavanagh D
%A Aris ADM
%A Adams B
%A Kang HG
%A Wang E
%A Garlo K
%A Ogawa M
%A Amancha P
%A Chakravarty S
%A Heyne N
%A Kim SH
%A Cataland S
%A Yoon SS
%A Miyakawa Y
%A Luque Y
%A Muff-Luett M
%A Tanaka K
%A Greenbaum LA
%J Kidney Med
%V 6
%N 8
%D 2024 Aug
%M 39105067
暂无%R 10.1016/j.xkme.2024.100855
%X <B>UNASSIGNED: </B>Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) caused by complement dysregulation. Ravulizumab is a C5i approved for the treatment of aHUS. This analysis assessed long-term outcomes of ravulizumab in adults and pediatric patients with aHUS.<BR><B>UNASSIGNED: </B>This analysis reports 2-year data from 2 phase 3, single-arm studies.<BR><B>UNASSIGNED: </B>One study included C5i-naïve adults (NCT02949128), and the other included 2 cohorts of pediatric patients (C5i-naïve and those who switched to ravulizumab from eculizumab [pediatric switch patients]; NCT03131219).<BR><B>UNASSIGNED: </B>Patients received intravenous ravulizumab every 4-8 weeks, with the dose depending on body weight.<BR><B>UNASSIGNED: </B>The primary endpoint in the studies of C5i-naïve patients was complete TMA response, which consisted of platelet count normalization, lactate dehydrogenase normalization, and ≥25% improvement in serum creatinine concentrations from baseline, at 2 consecutive assessments ≥4 weeks apart.<BR><B>UNASSIGNED: </B>All analyses used descriptive statistics. No formal statistical comparisons were performed.<BR><B>UNASSIGNED: </B>In total, 86 and 92 patients were included in efficacy and safety analyses, respectively. Complete TMA response rates over 2 years were 61% and 90% in C5i-naïve adults and pediatric patients, respectively. The median increase in estimated glomerular filtration rate from baseline was maintained over 2 years in C5i-naïve adults (35 mL/min/1.73 m2) and pediatric patients (82.5 mL/min/1.73 m2). Most adverse events and serious adverse events occurred during the first 26 weeks. No meningococcal infections were reported. Improvement in the Functional Assessment of Chronic Illness Therapy - Fatigue score achieved by 26 weeks was maintained over 2 years.<BR><B>UNASSIGNED: </B>Limitations were the small sample of pediatric switch patients and limited availability of genetic data.<BR><B>UNASSIGNED: </B>Long-term treatment with ravulizumab is well tolerated and associated with improved hematologic and renal parameters and quality of life in adults and pediatric patients with aHUS.<BR>This research tested a drug called ravulizumab for the treatment of atypical hemolytic uremic syndrome (aHUS). aHUS is a rare disease that causes clots in tiny blood vessels. This can damage the kidneys and other organs. We analyzed data from 2 clinical trials in which children and adults with aHUS received ravulizumab through a tube placed in a vein (intravenous line). They received ravulizumab every 4-8 weeks depending on their weight. We found that treating patients for 2 years with ravulizumab was associated with improved blood health, kidney function, and quality of life and was well tolerated. These results support ravulizumab as a long-term treatment for people with aHUS.