Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Proportion of subjects with recurrent disease
研究概览
简要总结
The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.
详细描述
Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US).
Data collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first .
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Retrospective
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient's aged ≤18 years at start of caplacizumab treatment initiation
- •Patient has a diagnosis of iTTP documented in the medical records
- •Patient was treated with caplacizumab within the eligibility period
排除标准
- •Patient declined use of data for study (where local regulations require patient notification of planned study)
- •Patient's medical chart is missing or not retrievable
结局指标
主要结局
Proportion of subjects with recurrent disease
时间窗: From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)
Total duration of hospitalization stays
时间窗: From index date up to 12 weeks after last dose of caplacizumab
Proportion of patients achieving clinical response
时间窗: From index date up to 12 weeks after last dose of caplacizumab
defined as a normal platelet countand LDH \< 2 ULN for at least 48 hours following initial normalization or response of platelet count
Platelet count response
时间窗: From index date up to 12 weeks after last dose of caplacizumab
defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days
Proportion of subjects with refractory iTTP
时间窗: From index date up to 12 weeks after last dose of caplacizumab
defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range
Number of participants with Adverse event
时间窗: From index date up to 12 weeks after last dose of caplacizumab
including serious adverse events
Time to normalization of organ damage marker levels
时间窗: From index date up to 12 weeks after last dose of caplacizumab
Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN
Duration of intensive care unit (ICU) stay
时间窗: From index date up to 12 weeks after last dose of caplacizumab
Duration of therapeutic PE
时间窗: From index date up to 12 weeks after last dose of caplacizumab
Time to ADAMTS13 activity ≥ 20%
时间窗: From index date up to 12 weeks after last dose of caplacizumab
where available and feasible
次要结局
- Treatment pattern of caplacizumab therapy(From index date up to 12 weeks after last dose of caplacizumab)
- Types and duration of concomitant medications(From index date up to 12 weeks after last dose of caplacizumab)