The Dallas Hereditary Spherocytosis Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 55
- 主要终点
- Health related quality of life
研究概览
简要总结
The purpose of this study is to
- better characterize the short term and long term natural history of hereditary spherocytosis (HS) including diagnosis, complications, and indications for and response to splenectomy
- evaluate and describe the health-related quality of life in children with HS.
详细描述
Patients with a new or established diagnosis of HS seen at Children's Medical Center will be asked to enroll in the study. Previous and current medical records will be reviewed to systematically catalogue their history of HS, including diagnosis, complications, hospitalizations, medications and laboratory data. Health-related quality of life questionnaires will be given to the patients and their parents at enrollment and periodically during the follow-up. Those who agree will have up to three small samples of blood collected and frozen for future laboratory studies of complications associated with HS and/or splenectomy.
We anticipate enrolling approximately 200 children and young adults with HS in this study and following them until adulthood (age 18-21 years).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of HS with or without prior splenectomy
- •Age 0 - 21 years
- •Spanish-speaking subjects are eligible to participate
排除标准
- •Unable to provide contact information for follow-up
结局指标
主要结局
Health related quality of life
时间窗: Approximately at 5 years
PedsQL measurement
次要结局
- Complications of HS(Approximately at 5 years and at 10 years)
- Diagnosis of HS(At enrollment)
- Primary indications for splenectomy(Approximately at 5 years)
- Complications of splenectomy(Approximately at 5 years)