Stem Cell Transplant Using Selected CD34 Cells and Reduced Donor T-Cells to Treat Severe Aplastic Anemia and Bone Marrow Failure Syndromes

What this Trial Is About

Researchers are studying a new approach to allogeneic hematopoietic stem cell transplantation aHSCT for patients aged 4 to 80 years with severe aplastic anemia, myelodysplastic syndrome, or other bone marrow failure syndromes. This Phase 2 trial aims to reduce serious complications such as chronic graft versus host disease cGVHD and graft rejection by modifying the stem cell transplant process. The study evaluates whether co-infusing selected CD34 stem cells with a reduced dose of donor T-cells can lower cGVHD incidence to levels seen with traditional bone marrow transplants while maintaining successful engraftment. Participants include donors related or unrelated to recipients who provide white blood cells and stem cells through procedures involving filgrastim injections and apheresis. Recipients undergo chemotherapy to prepare their immune systems before receiving a single infusion of the donor cells. After transplant, recipients receive medications to prevent rejection and remain hospitalized for up to one month. Donors provide initial white blood cells before mobilization and stem cell collection. During the study, participants have regular medical evaluations, including physical exams, medical history reviews, and laboratory testing. Recipients are followed with periodic visits for up to three years and beyond to monitor transplant success, side effects, and quality of life. The trials primary outcome is the rate of chronic graft versus host disease after one year, with secondary measures including transplant-related mortality, engraftment success, donor-recipient cell compatibility, and overall survival.

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells