Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 400
- 主要终点
- Frequencies of severe infection of children with PID
研究概览
简要总结
This study is a prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children.
详细描述
Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the research about clinical characteristics, treatment, management and prognosis of Chinese children with PID is still not perfect and there is no basic and large database. Therefore, this study is conducted, in order to set up a complete database and long term follow up of Chinese children with PID, and establish foundations for basic research and precise medicine.
This study is divided into two parts. In part one, pediatric patients with PID in the past 10 years will be collected retrospectively by review medical records from these centers, and investigate the clinical features and prognosis. In part two, all new cases with suspected PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and cellular immune functions will be detected. Gene tests would be done when necessary to confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year respectively. The trial will be completed in 60 months.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Day 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: from neonates to 18 years old
- •Fulfilling the one of the followings:
- •Severe infection after vaccination (BCG, polio arthritis pill)
- •Chronic/recurrent ear infection, sinus infections and respiratory tract infection
- •Recurrent, deep skin or deep-seated infections
- •Boys with early period and refractory thrombocytopenia
- •Infants with obviously decreased absolute number of lymphocytes
- •Severe allergy with infection
- •Infant diabetes with severe diarrhea
- •Boys with severe Epstein-Barr virus infection
- •Infants with hepatosplenomegaly and generalized lymphadenopathy
- •Infantile hemophagocytic syndrome
- •Failure of an infant to gain weight or grow normally
- •Persistent thrush in mouth or fungalinfection on skin
- •Family history of primary immunodeficiency
- •Invasive pneumococcal infection
- •Erythrodermic psoriasis
- •Gene test confirmed PID pediatric patients
排除标准
- •Subject will be excluded if she or he would not enroll into this study
结局指标
主要结局
Frequencies of severe infection of children with PID
时间窗: 60 months
Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire
次要结局
- Life quality of children with PID(60 months)
- Complications of children with PID(60 months)
- Long-term changes on lung function of children with PID(60 months)
研究者
Kunling Shen
Chief of China National Clinical Research Center for Respiratory Diseases
Beijing Children's Hospital