Retrospective Study of the Breadth of Donor Options for Patients With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 161
- 试验地点
- 1
- 主要终点
- To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
研究概览
简要总结
Background:
People who have certain immune system diseases often need a procedure called allo HSCT. This is short for allogeneic hematopoietic stem cell transplant. This might cure people with these diseases. Many people who need allo HSCT need donors who are relatives with similar genes. But the disease may also affect those in the donor pool. This may mean there are fewer options for people with inherited diseases. Researchers want to collect data on how transplant candidates and their donors are found.
Objective:
To find out how genetic diseases and the ways they are inherited affect the breadth of options for allo HSCT donors.
Eligibility:
Records from studies that have already been done. These will be for people ages 4 and older who were evaluated for allo HSCT or to be donors.
Design:
Participants already signed a consent form for their records to be shared.
Researchers will study the participant data.
Data will be stored in an electronic system. Researchers will use passwords to protect the data.
详细描述
This protocol is a retrospective review of donor search results for patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT).
The study will involve collecting information related to the donor search for transplant candidates and their prospective donors, using records in CRIS, Crimson, the HLA lab, and records in the transplant coordinator office (such as shipping logs of HLA typing kits). The study will not involve the use of specimens or participant contact.
The participants whose records will be reviewed will be those who were evaluated for allo HSCT or donation on an NIH primary immunodeficiency transplant protocol at a time when haplo donors were eligible (March 30, 2012 to present for GATA2, January 1, 2015 for CGD, October 6, 2015 to present for 16-C-0003, and May 21, 2014 to present for DOCK8).
The Principal Investigators on the included protocols have granted permission to conduct this study and have verified that none of the original protocols or informed consent documents preclude such a review of clinical data.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 4 Years 至 85 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
时间窗: 1 year
To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
次要结局
未报告次要终点