Pediatric Liver Transplantation Clinical Trial
Official title:
Safety and Tolerance of Immunomodulating Therapy With Donor-specific Mesenchymal Stem Cells in Pediatric Living-Donor Liver Transplantation, a 24-month, Non-randomized, Open-label, Prospective, Single-center Pilot Trial
Since the introduction of calcineurin-based immunosuppression, patient and graft survival in
pediatric liver transplantation (LT) improved significantly. However, in contrast,
calcineurin inhibitor (CNI) toxicity leads to significant morbidity and impairs quality of
life for recipients. Moreover, CNI cannot prevent long-term allograft inflammation and
fibrosis.
Mesenchymal stem (stromal) cells (MSC) have potent immunomodulatory properties potentially
promoting allograft tolerance and ameliorating toxicity of exposure to high dose CNI.
Previous trials for non-solid organ transplant indications have shown an excellent safety
profile of intravenous MSC application. The MYSTEP1 trial aims to investigate safety and
benefits portal and intravenous MSC infusion in pediatric LT.
Background: Calcineurin inhibitors (CNI) have significantly improved patient and graft
survival in pediatric liver transplantation (pLT). However, CNI toxicity leads to significant
morbidity. Moreover, CNIs cannot prevent long-term allograft injury.
Mesenchymal stem (stromal) cells (MSC) have potent immunomodulatory properties, which may
promote allograft tolerance and ameliorate toxicity of high-dose CNI. The MYSTEP1 trial aims
to investigate safety and feasibility of donor-derived MSCs in pLT.
Methods/Design: 7 to 10 children undergoing living-donor pLT will be included in this
open-label, prospective pilot trial. A dose of 1 × 106 MSCs/kg body weight will be given at
two time points: first by intraportal infusion intraoperatively and second by intravenous
infusion on postoperative day 2. In addition, participants will receive standard
immunosuppressive treatment. Our primary objective is to assess the safety of intraportal and
intravenous MSC infusion in pLT recipients. Our secondary objective is to evaluate efficacy
of MSC treatment as measured by the individual need for immunosuppression and the incidence
of biopsy-proven acute rejection. We will perform detailed immune monitoring to investigate
immunomodulatory effects.
Discussion: Our study will provide information on the safety of donor-derived MSCs in
pediatric living-donor liver transplantation and their effect on immunomodulation and graft
survival.
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