Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 2,000
- 试验地点
- 1
- 主要终点
- Basic characteristics of the patient population
研究概览
简要总结
Background:
People who have had an allogeneic hematopoietic stem cell transplant (HCT), cellular therapy (CT), or gene therapy (GT) have bone marrow or an immune system that is damaged. They get stem cells from a donor who is a relative. Researchers want to study stem cell donors and recipients to learn about the long-term effects of HCT, CT, or GT. They want to learn how the stem cells change and how to improve their ability to fight cancer.
Objective:
To provide long-term follow-up care for people who underwent or will undergo HCT, CT, or GT. To collect data, blood, and tissue samples to learn about late complications after HCT, CT, or GT.
Eligibility:
Adults age 18 and older who will undergo HCT or underwent HCT, Cellular Therapy (CT), or Gene Therapy (GT) and are surviving one year or more from the date of therapy. The stem cell donors for these recipients are also needed.
Design:
Recipients will have 1 visit each year. They will have a physical exam. They will answer questions about their medical history and health. They will receive screening and surveillance testing. They will complete brief questionnaires.
Recipients will have blood tests. They may have tissue biopsies or specimens (such as tissue in their cheek or skin or bone marrow biopsy).
Recipients will give their current address and phone number, and the same data for one or two other people, who can get in contact with them.
After the first visit at the clinic, some recipients may see a doctor close to home to get the necessary information and send it to NIH.
Donors will come to the clinic for 1 visit. They will answer questions about their medical history. Blood samples will be taken.
详细描述
Background:
- Patients who survive more than 3 years after allogeneic hematopoietic stem cell transplantation (HCT) have a high probability of being cured from their underlying disease; however, mortality rates remain 4 to 9-fold higher than the general population for at least 30 years after HCT.
- The most common causes of late mortality in HCT survivors include second malignancies, relapsed/recurrent disease, infections, chronic graft-versus-host disease (GVHD), respiratory diseases, and cardiovascular diseases.
- Guidelines for recommended screening and preventive practices for long-term survivors after HCT have been developed, and can be implemented to detect or prevent late complications after HCT.
- Understanding the pattern of immune reconstitution and immune dysregulation after HCT may help to elucidate the root mechanisms of late complications.
Objective:
-To collect clinical data on patients and late complications after HCT
Eligibility:
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
- 4 Years 至 120 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •INCLUSION CRITERIA FOR PATIENT SUBJECTS:
- •Individuals who underwent HCT or received cellular or gene therapy for any indication (malignant or non-malignant) and are surviving one year or more from the date of therapy
- •Age >= 4 years
- •Ability of individual or individual s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
- •Individuals will need to have a primary physician within the US (primary care, oncologist, hematologist, etc.) that will provide continued comprehensive care for the duration of participation in the study. PI may allow for established medical providers to be located outside of the US.
排除标准
- •FOR PATIENT SUBJECTS:
- •Individuals with active disease relapse or new hematologic malignancy including post-transplant lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.
- •INCLUSION CRITERIA FOR DONOR SUBJECTS:
- •Related stem cell donors of patients meeting the above criteria as a donor of hematopoietic progenitor and stem cells or leukocytes
- •Age >= 4years
- •Ability of patient or patient s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
- •INCLUSION CRITERIA FOR PARENTS COMPLETING SURVEYS:
- •Parents/guardians of minors enrolled on the study who have undergone HCT
- •Willingness to complete surveys about the minor that underwent HCT
研究组 & 干预措施
2/Donors
Related stem cell donors of those in Patients cohort.
3/Parents of patients
Parents/guardians of minors enrolled in cohort 1
1/Patients
Patients who underwent hematopoietic stem cell transplant or received cellular therapy or gene therapy for any indication (malignant or non-malignant).
结局指标
主要结局
Basic characteristics of the patient population
时间窗: at death
Survival
次要结局
- Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup(Ongoing throughout study)
- Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant(Ongoing throughout study)
- Monitoring of late effects after stem cell transplant for patients of ETIB protocols(Ongoing throughout study)
- Obtain blood samples from stem cell transplant donors to study how biological factors related to the donor are associated with late complications(Ongoing throughout study)
- Monitoring of late effects after stem cell transplant, cellular therapy, or gene therapy for patients of NIH transplantation and cellular therapy protocols(Ongoing throughout study)