Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Time to Macrophage Activation Syndrome (MAS) laboratory remission
研究概览
简要总结
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
详细描述
This is an observational, retrospective cohort study on the treatment utilization and outcomes of Macrophage Activation Syndrome (MAS) refractory to glucocorticoids (GC) in patients with Still's disease. The study will be conducted entirely through medical chart abstraction; all data will be taken from the patient's medical record, with no additional assessments.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 6 Months 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age >6 months and ≤80 years at the beginning of the index MAS episode.
- •Diagnosis of Still's disease (sJIA (systemic Juvenile Idiopathic Arthritis) or AOSD (Adult Onset Stills Disease) diagnosis).
- •Diagnosis of MAS according to treating physician in the medical record.
- •Patients who have received at least 3 consecutive days of GC after diagnosis of MAS and/or are judged by the Investigator to be refractory to GC due to clinical worsening of patient's condition .
- •The onset of the index MAS episode occurred between 01 January 2012 and 30 September 2022.
排除标准
- •A diagnosis of primary Hemophagocytic Lymphohistiocytosis (HLH) prior to the beginning of the index MAS episode.
- •Confirmed malignancy prior to the beginning of the index MAS episode.
- •Patient treated with any investigational product as a part of clinical trial during the index MAS episode.
结局指标
主要结局
Time to Macrophage Activation Syndrome (MAS) laboratory remission
时间窗: From Index date up to 26 weeks
Time to normalization for all key laboratory values
Time to partial MAS laboratory remission
时间窗: From Index date up to 26 weeks
Time to normalization for at least 3 key laboratory values
Clinical signs
时间窗: At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
No of MAS symptoms (Fever, Skin rash, Hemorrhagic manifestations, Evidence of CNS involvement) present
Number of recurrent MAS episodes
时间窗: through study completion
Occurring any time after the end of the data collection period for the index MAS episode
Overall survival
时间窗: 1 year
The proportion of patients alive after 1-year of follow-up
Administration of organ support care
时间窗: from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
Proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs
Time to laboratory value normalization
时间窗: From Index date up to 26 weeks
Time to normalization for the key laboratory values
Time to tapering of Glucocorticoids (GCs)
时间窗: from index date to the last of 7 consecutive days
From index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN).
Characteristics of MAS treatment
时间窗: for MAS episodes occurring during the data collection period
description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes.
次要结局
- Duration of Clinical Response(Until week 26)
- Time to intensive care unit (ICU) discharge(Up to 26 weeks)
- Time to hospital discharge(Up to 26 weeks)