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A New Biomarker for the Non-invasive Diagnosis of Rejection After Heart Transplantation

Completed
Conditions
Heart Transplant Rejection
Interventions
Diagnostic Test: donor-derived cell-free DNA (dd-cfDNA)
Registration Number
NCT03477383
Lead Sponsor
Vastra Gotaland Region
Brief Summary

This study is a prospective, observational multi-center cohort study. The study will consist of patients undergoing heart transplantation. The main purpose is to test a new biomarker for rejection against the gold-standard, the endomyocardial biopsy (EMB). 80 patients will be included, both children and adults.

Detailed Description

The primary objective is a comparison of an the established, invasive diagnostic test, the endomyocardial biopsy (EMB) against a new, non-invasive test. The new test is the measurement of donor-derived cell-free DNA (dd-cfDNA) circulating in the blood stream of the recipient, quantified by droplet digital PCR (polymerase-chain reaction) after targeted multiplex preamplification. Sensitivity is calculated with EMB as the gold standard. The patients will be followed during one year post-transplantation with simultaneous blood samples and EMB. The patients will further be followed clinically for 5 years. A suitable cut-off for the ratio of dd-cfDNA will be calculated using receiver operating characteristic (ROC) analysis.

Secondary objectives are description of patient outcomes with respect to organ function, quality of life and adverse events.

Secondary objectives are the calculation of the costs related to the transplantation process as well as to the follow-up with EMB. A simulation will be done with respect to possible savings achieved if the new biomarker could be implemented.

The study consists of two cohorts: all adult recipients of heart transplantation at Sahlgrenska University Hospital (one of two center conducting heart transplantation in Sweden): regional study. The other cohort consists of all children undergoing heart transplantation in Sweden:national study. Patients will undergo transplantation at Sahlgrenska University Hospital in Gothenburg as well as at Skåne University Hospital in Lund. Follow-up with blood samples and EMB of these patients will be in Gothenburg, Lund and at Karolinska University Hospital in Stockholm.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
134
Inclusion Criteria
  1. Patient on waiting list for heart transplantation
  2. Signed informed consent
Exclusion Criteria
  1. Follow-up outside Sweden

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Pediatric patientsdonor-derived cell-free DNA (dd-cfDNA)Pediatric patients (0-17 years) undergoing heart transplantation
Adult patientsdonor-derived cell-free DNA (dd-cfDNA)Adult patients (18 years or older) undergoing heart transplantation
Primary Outcome Measures
NameTimeMethod
Comparison of donor-derived cell-free DNA and endomyocardial biopsy with respect to rejection3 years

Measurement of both fraction of donor-derived cell-free DNA of all cell-free DNA (derived from recipient and donor) as well as the absolute number of copies per ml plasma derived from the donor. Comparison is made with simultaneously obtained endomyocardial biopsies that are graded with respect to rejection, according to the official guidelines provided by the International Society of Heart and Lung Transplantation.

Secondary Outcome Measures
NameTimeMethod
Immunization status5 years

Impact of pre-transplantation immunization status on primary and secondary outcomes

Prior cardiac surgery5 years

Impact of prior cardiac surgery on other outcomes

Neonatal cardiac surgery5 years

Impact of neonatal (first 4 weeks of life) cardiac surgery on other outcomes

Rejection5 years

Cumulative incidence of rejection (according to the biopsy grading system provided by the International Society of Heart and Lung Transplantation)

Graft-vasculopathy5 years

Cumulative incidence of graft-vasculopathy

Quality of life5 years

Health-related quality of life as measured by EQ5D

GFR (glomerular filtration rate)5 years

Renal function evaluated by calculated and measured GFR (Iohexol or Cr-Clearance)

Donor cardiac arrest5 years

Correlation between circulatory arrest in the donor and cardiac function

Initial immunosuppression5 years

Correlation between initial immunosuppression and the incidence of rejection

Ischemia time5 years

Correlation between ischemic graft time and cardiac function as measured by echocardiography

Re-transplantation5 years

Proportion of patients who have undergone re-transplantation

Infections5 years

Cumulative incidence of infections requiring hospital admission

Donor cardiopulmonary resuscitation (CPR) impact3 years

Impact of CPR of the donor on the incidence of assumed early rejection

Immunosuppression and adverse effects5 years

Correlation of time points with over- or under-immunosuppression (as measured by blood levels of immunosuppressive drugs and comparison with desired levels) and the incidence of severe infection, renal function decline and malignancy

Immunosuppression and rejection5 years

Correlation of time points with over- or under-immunosuppression (as measured by blood levels of immunosuppressive drugs and comparison with desired levels) with rejection events

Blood products and adverse events5 years

Correlation between the number of blood products (red blood cells, thrombocytes, plasma) given after transplantation and the incidence of adverse effects

Overweight5 years

Prevalence of overweight (BMI \>25) and obesity (BMI\>30)

Circulatory support to transplantation5 years

Proportion of patients undergoing heart transplantation from a ventricular assist device or extra-corporeal membrane oxygenation (ECMO) or from mechanical ventilation.

Malignancy5 years

Cumulative incidence of malignancy (PTLD and others)

Initial immunosuppression and malignancy5 years

Correlation between type of initial immunosuppression and incidence of malignancy

Survival3 years

Survival

Cost analysis 15 years

Costs of heart transplantation during the first year post-transplantation

Trial Locations

Locations (4)

Pediatric Heart Center, The Queen Silvia Children's Hospital, Sahlgrenska University Hospital

🇸🇪

Gothenburg, Sweden

Pediatric Heart Center, Karolinska University Hospital

🇸🇪

Stockholm, Sweden

Transplantation Center, Sahlgrenska University Hospital

🇸🇪

Gothenburg, Sweden

Pediatric Heart Center, Skåne University Hospital

🇸🇪

Lund, Sweden

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