PDF(Pubmed)
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.
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.
摘要：
■非典型溶血性尿毒综合征（aHUS）是一种罕见的由补体失调引起的血栓性微血管病（TMA）。Ravulizumab是批准用于治疗aHUS的C5i。该分析评估了ravulizumab在成人和儿科aHUS患者中的长期结果。
■本分析报告了2个阶段3单臂研究的2年数据。
■一项研究包括C5i-Naine成年人（NCT02949128），另一个包括2组儿科患者(C5i-na-ive和从eculizumab转用ravulizumab的患者[儿科转用患者];NCT03131219).
■患者每4-8周接受静脉ravulizumab，剂量取决于体重。
■C5i初治患者研究的主要终点是完全TMA反应，包括血小板计数正常化，乳酸脱氢酶正常化，血清肌酐浓度比基线改善≥25%，在两次连续评估时，间隔≥4周。
■所有分析都使用描述性统计。没有进行正式的统计比较。
■总共，86和92例患者被纳入疗效和安全性分析，分别。在C5i初治的成人和儿科患者中，2年的TMA完全缓解率分别为61%和90%。分别。估计的肾小球滤过率从基线的中位数增加在C5i初治成人（35mL/min/1.73m2）和儿科患者（82.5mL/min/1.73m2）中维持超过2年。大多数不良事件和严重不良事件发生在前26周。未报告脑膜炎球菌感染。慢性疾病治疗的功能评估的改善-在2年内维持26周的疲劳评分。
■限制是儿科转换患者的小样本和遗传数据的有限可用性。
■对于患有aHUS的成人和儿科患者，使用ravulizumab的长期治疗具有良好的耐受性，并且与改善血液学和肾脏参数以及生活质量相关。
这项研究测试了一种名为ravulizumab的药物，用于治疗非典型溶血性尿毒综合征（aHUS）。HUS是一种罕见的疾病,导致凝块在微小的血管。这可能会损害肾脏和其他器官。我们分析了来自2项临床试验的数据，在这些临床试验中，患有aHUS的儿童和成人通过放置在静脉中的管（静脉管线）接受了ravulizumab。根据他们的体重，他们每4-8周接受一次ravulizumab。我们发现，用ravulizumab治疗患者2年与改善血液健康有关。肾功能,和生活质量，耐受性良好。这些结果支持ravulizumab作为aHUS患者的长期治疗。
■本分析报告了2个阶段3单臂研究的2年数据。
■一项研究包括C5i-Naine成年人（NCT02949128），另一个包括2组儿科患者(C5i-na-ive和从eculizumab转用ravulizumab的患者[儿科转用患者];NCT03131219).
■患者每4-8周接受静脉ravulizumab，剂量取决于体重。
■C5i初治患者研究的主要终点是完全TMA反应，包括血小板计数正常化，乳酸脱氢酶正常化，血清肌酐浓度比基线改善≥25%，在两次连续评估时，间隔≥4周。
■所有分析都使用描述性统计。没有进行正式的统计比较。
■总共，86和92例患者被纳入疗效和安全性分析，分别。在C5i初治的成人和儿科患者中，2年的TMA完全缓解率分别为61%和90%。分别。估计的肾小球滤过率从基线的中位数增加在C5i初治成人（35mL/min/1.73m2）和儿科患者（82.5mL/min/1.73m2）中维持超过2年。大多数不良事件和严重不良事件发生在前26周。未报告脑膜炎球菌感染。慢性疾病治疗的功能评估的改善-在2年内维持26周的疲劳评分。
■限制是儿科转换患者的小样本和遗传数据的有限可用性。
■对于患有aHUS的成人和儿科患者，使用ravulizumab的长期治疗具有良好的耐受性，并且与改善血液学和肾脏参数以及生活质量相关。
这项研究测试了一种名为ravulizumab的药物，用于治疗非典型溶血性尿毒综合征（aHUS）。HUS是一种罕见的疾病,导致凝块在微小的血管。这可能会损害肾脏和其他器官。我们分析了来自2项临床试验的数据，在这些临床试验中，患有aHUS的儿童和成人通过放置在静脉中的管（静脉管线）接受了ravulizumab。根据他们的体重，他们每4-8周接受一次ravulizumab。我们发现，用ravulizumab治疗患者2年与改善血液健康有关。肾功能,和生活质量，耐受性良好。这些结果支持ravulizumab作为aHUS患者的长期治疗。
