matched unrelated donor hematopoietic stem cells

This therapeutic approach involves the use of hematopoietic stem cells (HSCs) harvested from a matched unrelated donor (MUD) as a primary (upfront) treatment for pediatric and young adult patients with severe aplastic anemia (SAA) who lack a matched sibling donor. Traditionally, immunosuppressive therapy (IST) is the standard first-line treatment for such patients. The TransIT clinical trial (NCT02845596), led by Michael Pulsipher, is a feasibility study evaluating whether upfront MUD HSCT can provide superior event-free survival compared to standard IST. The procedure involves a conditioning regimen (typically including fludarabine, cyclophosphamide, and low-dose total body irradiation) followed by the intravenous infusion of donor stem cells, which are intended to engraft and repopulate the recipient's bone marrow to restore normal hematopoiesis.

Other names

MUD HSCTunrelated donor hematopoietic stem cell transplantupfront MUD HSCT