Umbilical Cord Blood Transplant for Severe Aplastic Anemia and Myelodysplastic Syndrome Using Expanded Stem Cells to Improve Recovery

What this Trial Is About

Severe aplastic anemia SAA and myelodysplastic syndrome MDS are serious bone marrow diseases often requiring bone marrow transplants. Researchers are studying a new approach using a combination of blood stem cells from a family donor and cord blood stem cells from an unrelated donor to see if this method is safe and effective. The study focuses on improving transplant outcomes, including faster engraftment and reducing graft rejection, by using a special expanded cord blood product called CordInTM. Participants who qualify will undergo detailed screening including blood tests, lung and heart function tests, bone marrow biopsy, and CT scans. Before transplant, recipients receive chemotherapy infusions and a radiation dose. The CordInTM product, which includes ex vivo expanded stemprogenitor cells, is infused through an IV line. Donors receive injections to stimulate stem cell production followed by stem cell collection. Hospital stays last about 3-4 weeks post-transplant, with follow-up visits weekly for the first few months and then every six months for five years. During the study, recipients will have regular assessments including blood counts and monitoring for transplant-related complications such as graft-versus-host disease. Researchers will evaluate engraftment success by day 100 and monitor treatment-related mortality and other outcomes up to 200 days. Quality of life will also be assessed. Donors undergo physical exams and blood testing to ensure suitability. Total study participation can last several years with ongoing monitoring after discharge.

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome