Background. Calcineurin inhibitors (CNI) have significantly improved patient and graft survival in pediatric liver transplantation\n(pLT). However, CNI toxicity leads to significant morbidity. Moreover, CNIs cannot prevent long-term allograft injury.\nMesenchymal stem (stromal) cells (MSC) have potent immunomodulatory properties, which may promote allograft tolerance\nand ameliorate toxicity of high-dose CNI. The MYSTEP1 trial aims to investigate safety and feasibility of donor-derived\nMSCs in pLT. Methods/Design. 7 to 10 children undergoing living-donor pLT will be included in this open-label,\nprospective pilot trial. A dose of 1 Ã?â?? 106 MSCs/kg body weight will be given at two time points: first by intraportal\ninfusion intraoperatively and second by intravenous infusion on postoperative day 2. In addition, participants will receive\nstandard immunosuppressive treatment. Our primary objective is to assess the safety of intraportal and intravenous MSC\ninfusion in pLT recipients. Our secondary objective is to evaluate efficacy of MSC treatment as measured by the\nindividual need for immunosuppression and the incidence of biopsy-proven acute rejection. We will perform detailed\nimmune monitoring to investigate immunomodulatory effects. Discussion. Our study will provide information on the\nsafety of donor-derived MSCs in pediatric living-donor liver transplantation and their effect on immunomodulation and\ngraft survival.
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