{Reference Type}: Journal Article
{Title}: Ravulizumab in Atypical Hemolytic Uremic Syndrome: An Analysis of 2-Year Efficacy and Safety Outcomes in 2 Phase 3 Trials.
{Author}: Dixon BP;Kavanagh D;Aris ADM;Adams B;Kang HG;Wang E;Garlo K;Ogawa M;Amancha P;Chakravarty S;Heyne N;Kim SH;Cataland S;Yoon SS;Miyakawa Y;Luque Y;Muff-Luett M;Tanaka K;Greenbaum LA;
{Journal}: Kidney Med
{Volume}: 6
{Issue}: 8
{Year}: 2024 Aug
暂无{DOI}: 10.1016/j.xkme.2024.100855
{Abstract}: UNASSIGNED: 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.
UNASSIGNED: This analysis reports 2-year data from 2 phase 3, single-arm studies.
UNASSIGNED: 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).
UNASSIGNED: Patients received intravenous ravulizumab every 4-8 weeks, with the dose depending on body weight.
UNASSIGNED: 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.
UNASSIGNED: All analyses used descriptive statistics. No formal statistical comparisons were performed.
UNASSIGNED: 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.
UNASSIGNED: Limitations were the small sample of pediatric switch patients and limited availability of genetic data.
UNASSIGNED: 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.
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.