Congenital Heart Disease (CHD)
Conditions
Keywords
pediatric congenital heart disease, congenital heart disease, Autologous thymus transplantation
Brief summary
This pilot study will look at whether putting back a child's own thymus tissue during heart surgery is safe and may help support the immune system in children with congenital heart disease (CHD). During some heart surgeries, the thymus gland is removed to allow surgeons access to the heart. The thymus is important for helping the body develop infection-fighting immune cells. Researchers will compare children who have their thymus tissue reimplanted with children who have the thymus removed but not reimplanted. The study will evaluate the safety of the procedure, whether the transplanted tissue survives and functions, and whether it helps maintain or improve immune system health. Researchers will also monitor infections, recovery after surgery, hospital stays, quality of life, and overall health outcomes. Blood samples will be collected over time to better understand how the immune system changes after surgery and whether thymus reimplantation provides long-term benefits. Participants will be followed for up to 24 months after surgery.
Interventions
PROCEDUREAutologous Thymus Transplant
Autologous thymus transplantation (ATT) is an emerging therapeutic approach by the Cardiothoracic Surgery team at CCHMC that involves using the patient's own thymic tissue to restore immune function. For children with congenital heart disease (CHD), especially those undergoing cardiac surgery, the thymus is often removed or damaged, leading to potential immunodeficiencies. ATT aims to mitigate these issues.
Sponsors
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Months to 2 Years
Healthy volunteers
No
Inclusion criteria
* Infants diagnosed with CHD requiring their first cardiac surgery through a sternotomy. * Age: 2 months to 2 years old at time of cardiac surgery. * Parents or legal guardians have provided informed consent.
Exclusion criteria
* Patients with previous cardiac surgery through a sternotomy. * Patients with a planned partial thymectomy. * Patients with congenital athymia or thymic atrophy. * Known severe immunodeficiency unrelated to thymus function. * Presence of severe non-cardiac congenital anomalies. * Parent or guardians who refuse to consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of Complications from Autologous Thymus Reimplantation | Within 30 days after surgery | Proportion of participants experiencing one or more thymus reimplantation-related postoperative complications (e.g., surgical site infection, bleeding requiring transfusion, thromboembolic events, or unplanned return to the operating room). |
| Rate of Success of Thymus Reimplantation | Baseline, 6 months, and 12 months after surgery | Evidence of immune reconstitution based on T-cell counts, recent thymic emigrants (TREC assays), T-cell repertoire diversity, and T-cell functional assessments. |
| Number of Enrolled Participants | From enrollment to the end of treatment (approximately 24 months). | Percentage of eligible patients who enroll in the study compared with those approached and eligible. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Postoperative Infections | Within 12 months after surgery | Number and type of postoperative infections documented in the medical record. |
| Length of Hospital Stay | Total number of days spent in the hospital from surgery to discharge assessed up to 30 days post-surgery. | Total number of days from surgery to hospital discharge. |
| Number of Participants with Postoperative Complications | Within 12 months after surgery. | Incidence and severity of postoperative complications, including wound infections, respiratory infections, systemic infections, bleeding, and graft-related adverse events. |
| Change from Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Score | 12 months after surgery. | Parent/guardian-reported quality of life will be assessed using the Pediatric Quality of Life Inventory (PedsQL), a validated pediatric quality of life questionnaire. The outcome measure will be the change in PedsQL Total Score from baseline to 12 months following surgery. PedsQL scores range from 0 to 100, with higher scores indicating better quality of life. |
| Rate of Overall Survival | 24 months after surgery | Survival status of study participants |
| Change From Baseline in T-cell, B-cell, and NK-cell Subset Frequencies | Baseline and 1, 3, 6, 12, and 24 months after surgery | Changes in T-cell, B-cell, and natural killer (NK) cell subsets measured by flow cytometry. Results will be reported as the percentage and/or absolute count of each immune cell subset among peripheral blood mononuclear cells. |
| Change From Baseline in Immune Cell Functional Assay Results | Baseline and 1, 3, 6, 12, and 24 months after surgery. | Changes in immune cell function assessed by validated functional assays, including measures of immune cell activity and responsiveness. |
| Change From Baseline in Immune-Related Gene Expression Levels | Baseline and 1, 3, 6, 12, and 24 months after surgery. | Changes in expression levels of immune-related genes measured using gene expression analyses of peripheral blood samples. |
Contacts
CONTACTHannah Cope
PRINCIPAL_INVESTIGATORCarolina Moore, PhD, MBA
Children's Hospital Medical Center, Cincinnati
Outcome results
None listed