Clinical Research Directory

Expanded Donor Stem Cells for Poor Graft Function After Stem Cell Transplantation in Children

Poor graft function (PGF) is a serious complication after allogeneic hematopoietic stem cell transplantation (HSCT) and may cause persistent low blood cell counts despite donor-cell engraftment. This single-center, open-label, Phase I pilot study will enroll three children aged 6-18 years with PGF after allogeneic HSCT who have not responded adequately to conventional hematopoietic supportive treatments. CD34-positive hematopoietic stem cells will be collected from each participant's original related transplant donor and expanded outside the body for \~10 days using the small-molecule compound HEB-001 together with the cytokines stem cell factor, thrombopoietin, and FLT3 ligand. The expanded cells will then be administered as a single intravenous infusion. The main objectives are to assess acute infusion-related adverse events during the first 30 days and to examine genetic stability through 1 year. Preliminary hematopoietic recovery will be assessed using neutrophil and platelet recovery and donor chimerism. All participants will receive the investigational cell infusion; there is no comparator group.