Long-Term Follow-up of People Undergoing Hematopoietic Stem Cell Transplantation

Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation

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.

Study Overview

• Status: Recruiting
• Conditions: Hematopoietic Stem Cell Transplantation; Tissue Donors

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 OR patients who will be undergoing HCT on an ETIB protocol.
• 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 greater than or equal to 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.

• Study Type: Observational
• Enrollment (Estimated): 2000

Contacts and Locations

• Study Contact: Name: Steven Z Pavletic, M.D.; Phone Number: (240) 760-6174; Email: sp326h@nih.gov
• Study Contact Backup: Name: Ashley E Carpenter; Phone Number: (240) 760-6009; Email: carpentera@mail.nih.gov

Study Locations

• United States
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • Recruiting
      • National Institutes of Health Clinical Center
      • Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office; Phone Number: 888-624-1937

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• 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.

Description

• 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 greater than or equal to 1 year) OR patients who will be undergoing HCT for any indication (malignant or non-malignant) on an ETIB protocol. Patients do not need to have been transplanted under an ETIB protocol.
• Age greater than or equal to 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 care physician 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 greater than or equal to 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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
1/Patients; Patients who underwent hematopoietic stem cell transplant for any indication (malignant or non-malignant).
2/Donors; Related stem cell donors of those in Patients cohort.
3/Parents of patients; Parents/guardians of minors enrolled in cohort 1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Basic characteristics of the patient population	Survival	at death

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup	Observation of adherence and effects of standard of care recommendations	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	Studying donor samples for potential correlations to transplant recipient complications.	Ongoing throughout study
Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant	Immune monitoring for analysis	Ongoing throughout study
Monitoring of late effects after stem cell transplant for patients of ETIB protocols	Survival and observation of long-term outcomes.	Ongoing throughout study

Collaborators and Investigators

• Sponsor: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Steven Z Pavletic, M.D., National Cancer Institute (NCI)

Publications and helpful links

Helpful Links

• NIH Clinical Center Detailed Web Page

Study record dates

Study Major Dates

• Study Start (Actual): April 26, 2017
• Primary Completion (Estimated): August 12, 2050
• Study Completion (Estimated): August 12, 2050

Study Registration Dates

• First Submitted: December 21, 2016
• First Submitted That Met QC Criteria: December 21, 2016
• First Posted (Estimated): December 22, 2016

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 14, 2024
• Last Verified: November 2, 2023

More Information

Terms related to this study

• Keywords: Natural History; HCT
• Other Study ID Numbers: 170027; 17-C-0027

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: .All collected IPD will be shared.@@@@@@All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
• IPD Sharing Time Frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
• IPD Sharing Access Criteria: Data from this study may be requested by contacting the PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No