Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy

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, cellular, or gene therapy for any indication and are surviving one year or more from the date of therapy.
• Related hematopoietic progenitor and stem cell donors of patients meeting the above criteria who participated or will be participating as a donor of stem cells or leukocytes, may undergo a one-time optional research blood sample collection. Donors will not remain on study for longitudinal follow-up.
• Age >= 4 years
• Recipients of HCT, cellular, or gene therapies for any indication.
• 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, cellular therapy, or gene therapy.
• 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.

• 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.

Exclusion criteria

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