Clinical trial NCT03000244: Long-Term Follow-up of People Undergoing Hematopoietic Stem Cell Transplantation
Official title: Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hematopoietic Stem Cell Transplantation
Tissue Donors
Keywords: Natural History
HCT
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
First posted: 2016-12-22
Enrollment: 2000
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who have received an allogeneic stem cell transplant as well as donors of stem cells who are related to the patients who are study participants. Parents of minor recipients may also be enrolled to complete surveys. Participants can be from any community or co-enrolled on another NIH protocol.

Brief summary: Background:

People who have had an allogeneic hematopoietic stem cell transplant (HCT) 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. 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. To collect data, blood, and tissue samples to learn about late complications after HCT.

Eligibility:

Adults age 18 and older who will undergo HCT or underwent HCT and are surviving one year or more from the date of HCT. 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....

Detailed description: 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:

* Patients who underwent HCT for any indication and are surviving one year or more from the date of HCT
* Related stem cell donors of patients meeting the above criteria who participated or will be participating as a donor of stem cells or leukocytes.
* Age \>= 4 years
* Any active disease relapse or new hematologic malignancy including post-transplant lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.

Design:

* Prospective, longitudinal study of the natural history of long-term survivors after HCT.
* Clinical data on post-transplant complications and peripheral blood samples will be collected at yearly evaluations by the Principal Investigator, LAI, AI or representative investigator of the original HCT protocol.
* If patient develops complications such as new cancers, relapsed disease, new hematologic malignancy or PTLD they are exempt from required evaluations, and followed for survival and causes of death (management of complications as per primary team).
* As this is a longitudinal registry study without any planned intervention, there is no formal statistical analysis plan or accrual goal; descriptive data will be reported.

Eligibility criteria: * INCLUSION CRITERIA FOR PATIENT SUBJECTS:
* Patients who underwent HCT for any indication (malignant or non-malignant) and are surviving one year or more from the date of HCT (patients may be at any time point after HCT as long as it is \>=1 year).
* Age \>= 4 years
* Ability of patient or patient's Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
* Patients will need to have a primary physician within the US (primary care, oncologist, hematologist, etc.) that will provide continued comprehensive care throughout the patient's participation in the study

EXCLUSION CRITERIA FOR PATIENT SUBJECTS:

-Patients 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 stem cells or leukocytes
* Age \>= 4 years
* 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

Primary outcomes:
measure: Basic characteristics of the patient population
timeFrame: at death
Secondary outcomes:
measure: Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup
timeFrame: Ongoing throughout study
measure: Obtain blood samples from stem cell transplant donors to study how biological factors related to the donor are associated with late complications
timeFrame: Ongoing throughout study
measure: Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant
timeFrame: Ongoing throughout study
measure: Monitoring of late effects after stem cell transplant for patients of ETIB protocols
timeFrame: Ongoing throughout study
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center
state: Maryland
Study officials:
affiliation: National Cancer Institute (NCI)
name: Najla El Jurdi, M.D.
role: PRINCIPAL_INVESTIGATOR
References: