An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea (ESR-25-22871)
This multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. By analyzing electronic medical records (EMR) from participating institutions, the study investigates intravascular hemolysis control, transfusion avoidance, breakthrough hemolysis (BTH) characteristics, and overall clinical outcomes in an Asian population.
研究概览
研究类型
注册 (估计的)
联系人和位置
学习地点
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Seoul、韩国
- Yonsei University College of Medicine
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
Korean patients with Paroxysmal Nocturnal Hemoglobinuria whose retrospective clinical data are available in the electronic medical records (EMR) of the participating hospitals.
描述
Inclusion Criteria:
- Adult patients (18 years of age or older) with a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) documented by flow cytometry.
- Patients who initiated ravulizumab treatment on or before February 28, 2025, and have at least 6 months of follow-up data available.
- To reflect real-world clinical practice, meningococcal (Neisseria meningitidis) vaccination status will be collected if available, but is not a mandatory requirement for study inclusion.
Exclusion Criteria:
- Less than 18 years of age at the time of ravulizumab treatment initiation.
- Known hypersensitivity to ravulizumab or any of its excipients.
- Untreated Neisseria meningitidis infection at the time of ravulizumab treatment initiation.
- Prior or concomitant use of other complement inhibitors for PNH (with the exception of prior eculizumab use).
学习计划
研究是如何设计的？
设计细节
- 观测模型：COHORT
- 时间观点：RETROSPECTIVE
队列和干预
团体/队列 |
|---|
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PNH patients treated with Ravulizumab
Patients diagnosed with PNH and prescribed Ravulizumab at multiple centers in South Korea, whose electronic medical records (EMR) are available for retrospective review. |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN)
大体时间：Up to 4 years after ravulizumab initiation
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Assessment of intravascular hemolysis control, defined as achieving and maintaining serum LDH levels at or below 1.5 times the ULN during the 4-year post-marketing observation period. |
Up to 4 years after ravulizumab initiation
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Incidence and clinical characteristics of breakthrough hemolysis (BTH)
大体时间：hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
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BTH is defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin less than 10 g/dL], major adverse vascular events [MAVE, including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) levels of at least 2 times the upper limit of normal (ULN), after prior reduction of LDH to less than 1.5 times the ULN during therapy. Associated contextual factors, including complement-amplifying conditions, hospitalizations, transfusions, and the use of rescue medication, will also be recorded and characterized at each event. |
hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
|
合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- 4-2026-0786
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
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